Leber's Hereditary Optic Neuropathy
Conditions
Keywords
LHON, Ocuvia™, elamipretide, MTP-131
Brief summary
This is a Phase 2, prospective, randomized, double-masked, vehicle controlled, single-center study in approximately 12 subjects with LHON to evaluate safety, tolerability and efficacy of elamipretide (MTP-131) topical ophthalmic solution in this patient population. At the conclusion of 52 weeks of treatment, subjects will be offered the opportunity to enter an Open Label Extension for up to 48 additional weeks of treatment.
Detailed description
This was a prospective, randomized, double-masked (DM), vehicle-controlled, single-center study plus open label extension period (OLE) in which approximately 12 subjects with LHON having the genetic mtDNA mutation m.11778G\>A were randomized in a masked manner into 1 of 3 groups in a 1:1:1 ratio: one drop of elamipretide 1.0% topical ophthalmic solution twice daily (BID) in the: left eye, right eye, or both eyes. After completion of the 52-week treatment period or the Week 56 follow-up period, subjects were invited to participate in the OLE period for up to 108 weeks. During the OLE participants received 1% topical opthalmic elamipretide in both eyes (OU) daily. If a subject did not consent to the OLE, he/she completed the study at the Week 56 (±7 days) visit. There were 4 periods in this study: (1) screening period (up to 6 weeks); (2) double-masked treatment period (52 weeks); (3) OLE period (up to 108 weeks), and (4) follow-up period (4 weeks) after completion of End-of-Treatment (EOT) visit.
Interventions
DRUGelamipretide (MTP-131) 1% topical ophthalmic solution
DRUGVehicle topical ophthalmic solution
Sponsors
Stealth BioTherapeutics Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 50 Years
Healthy volunteers
No
Inclusion criteria
* Adults ≥18 and ≤ 50 years old at the time of loss of vision in the second eye. * Able to provide informed consent and willing to comply with all study visits and examinations * Diagnosis of LHON based on clinical and ophthalmic functional/anatomic test findings, and satisfactory documentation of the mitochondrial DNA mutation m.11778G\>A * Loss of vision in both eyes of ≥1 year and ≤10 years at the time of the Screening Visit and current clinically stable visual function (as assessed by the Investigator) * Able to self-administer eye drops as demonstrated at screening or having a care provider who can do so * Documentation of having satisfactorily completed at least two previous Humphrey automated visual field tests prior to screening. * Women of childbearing potential must agree to use birth control as specified in the protocol from the date they sign the informed consent form
Exclusion criteria
* Any other ocular pathology requiring treatment with prescription topical ophthalmic drops (e.g., glaucoma, dry eye) * Cup to disc ratio of \> 0.8 in either eye * Media opacity, suboptimal pupillary dilatation, or refractive error that interferes with adequate retinal imaging * Known to be immunocompromised or receiving systemic immunosuppression * Any disease or medical condition that in the opinion of the Investigator would prevent the subject from participating in the study or might confound study results * Participation in other investigational drug or device clinical trials within 30 days prior to enrollment, or planning to participate in any other investigational drug or device clinical trials within 30 days of study completion * Women who are pregnant or lactating Other protocol-defined inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Double Masked Period: Incidence of Ocular TEAEs | Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit) | The incidence of ocular treatment emergent adverse events (TEAEs). |
| Open Label Extension: Incidence of Ocular TEAEs | Assessed at Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit) | Open Label Extension: The incidence of ocular treatment emergent adverse events (TEAEs). |
| Double Masked Period: Severity of Ocular TEAEs | Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit) | The severity of ocular treatment emergent adverse events (TEAEs). |
| OLE: Severity of Ocular TEAEs | Assessed at Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit) | The severity of ocular treatment emergent adverse events (TEAEs). |
| Double Masked Period: Best Corrected Visual Acuity (BCVA) | Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit) | Double Masked Period: Best corrected visual acuity (BCVA) using the using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale by visit. ETDRS charts present a series of five letters of equal difficulty on each row, with standardized spacing between letters and rows; there is a total of 14 lines (70 letters), with letter size increasing further geometrically and equivalently in every line by a factor of 1.2589 (or 0.1 log unit), moving up the chart. Minimum score of zero, maximum score of 100. Change from baseline: a more negative score is worse outcome, a more positive score is better outcome. A lower score means less letters were read correctly (worse outcome) and a higher score means more letters were read correctly (better outcome). |
| Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Assessed at each visit from Baseline from Week 68 to Week 160 (follow-up visit). | Open Label Extension Period: Best corrected visual acuity (BCVA) using the using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale by visit. ETDRS charts present a series of five letters of equal difficulty on each row, with standardized spacing between letters and rows; there is a total of 14 lines (70 letters), with letter size increasing further geometrically and equivalently in every line by a factor of 1.2589 (or 0.1 log unit), moving up the chart. Minimum score of zero, maximum score of 100. Change from baseline: a more negative score is worse outcome, a more positive score is better outcome. A lower score means less letters were read correctly (worse outcome) and a higher score means more letters were read correctly (better outcome). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Double Masked Period: PhNR/B-wave Amplitude Ratio | Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit. | Double Masked Period: Change from baseline in PhNR/b-wave amplitude ratio by Visit. Photopic negative response electroretinography (PhNR assesses retinal cell function. A decrease in PhNR amplitude means worse outcome. |
| Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Assessed at each visit from Baseline from Week 68 to Week 160 | Open Label Extension Period: Change from baseline by visit PhNR/b Wave Amplitude Ratio by visit. A reduced amplitude ratio equals worse outcome. |
| Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit. | Double Masked Period: Change from baseline by visit in PhNR Peak to Trough Adjusted for a-wave Amplitude by Visit. Calculated as (b-wave amplitude - PhNR amplitude)/a-wave amplitude. A reduced amplitude ratio equals worse outcome. |
| Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Assessed at each visit from Baseline from Week 68 to Week 160 | Open Label Extension Period: Change from baseline by visit in PhNR Peak to Trough Adjusted for a-wave Amplitude by Visit. \[Calculated by b-wave amplitude - PhNR amplitude)/a-wave amplitude.\] Photopic negative response electroretinography (PhNR-ERG) assesses retinal cell function. A decrease in the amplitude of the a-wave is associated with worse outcomes. A reduced amplitude ratio equals worse outcome. |
| Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit. | Double Masked Period: Change from baseline in PhNR Peak to Trough Amplitude (Unadjusted) by Visit. Calculated as b-wave amplitude - PhNR amplitude. Photopic negative response electroretinography (PhNR-ERG) assesses retinal cell function. A decrease in amplitude is associated with worse outcomes. |
| Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Assessed at each visit from Baseline from Week 68 to Week 160 | Open Label Extension Period: Change from baseline in PhNR Peak to Trough Amplitude (Unadjusted) by Visit. b-wave amplitude - PhNR amplitude. Photopic negative response electroretinography (PhNR-ERG) assesses retinal cell function. A decrease in the amplitude of the a-wave is associated with worse outcomes. |
| Double Masked: VFQ-39 Composite | Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit) | Double Masked: Change from baseline in Visual Function Questionnaire (VFQ-39) Composite score by visit. National Eye Institute VFQ-39 measures health-related quality of life in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, peripheral vision, and composite score. For each domain: the lowest and highest possible scores are 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Open Label Extension Period: VFQ-39 Composite Score | Assessed at Baseline, Week 160 (End of OLE Period) | Change from baseline in Visual Function Questionnaire (VFQ-39) Composite score.National Eye Institute VFQ-39 measures health-related quality of life in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, peripheral vision, and composite score. For each domain: the lowest and highest possible scores are 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Open Label Extension Period: VFQ-39 General Health Score | Assessed at Baseline, Week 160 (End of OLE Period) | Change from baseline in Visual Function Questionnaire (VFQ-39) General Health score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Double Masked: VFQ-39 General Vision Score | Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit) | Change from baseline in VFQ-39 General Vision Score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculate |
| Open Label Extension Period: VFQ-39 General Vision Score | Assessed at Baseline, Week 160 (End of OLE Period) | Change from baseline in VFQ-39 General Vision score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculate |
| Double Masked: VFQ-39 Ocular Pain Score | Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit) | Change from baseline in VFQ-39 Ocular Pain score. National Eye Institute VFQ-39 measures health-related quality of life in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, peripheral vision, and composite score. For each domain: the lowest and highest possible scores are 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Open Label Extension Period: VFQ-39 Ocular Pain Score | Assessed at Baseline, Week 160 (End of OLE Period) | Change from baseline in VFQ-39 Ocular Pain score by visit. National Eye Institute VFQ-39 measures health-related quality of life in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, peripheral vision, and composite score. For each domain: the lowest and highest possible scores are 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Double Masked: VFQ-39 Near Activities Score | Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit) | Change from Baseline in VFQ-39 Near Activities Score from Baseline. National Eye Institute VFQ-39 measures health-related quality of life in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, peripheral vision, and composite score. For each domain: the lowest and highest possible scores are 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Open Label Extension Period: VFQ-39 Near Activities | Assessed at Baseline, Week 160 (End of OLE Period) | Change from Baseline in Visual Function Questionnaire (VFQ-39) Near Activities score.. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; score ranges from 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Double Masked Period: Mean Retinal Nerve Fiber (RNFL) Layer Thickness | Assessed at Baseline and Week 52 (end-of-treatment visit) | Change from baseline in retinal nerve fiber layer thickness by spectral domain optical coherence tomography (SD-OCT). RNFL measures the loss of retinal ganglion cell axons. RNFL thickness decreases as disease progresses. A positive number, or, absence of change from baseline reflects a good clinical outcome, a negative number reflects loss of thickness, or a bad outcome. |
| Open Label Extension Period: Retinal Nerve Fiber Layer Thickness | Assessed at Baseline, Week 148 | Open Label Extension Period: Change from baseline in Retinal Nerve Fiber Layer Thickness by SD-OCT by visit. Change from baseline in retinal nerve fiber layer thickness by spectral domain optical coherence tomography (SD-OCT). RNFL measures the loss of retinal ganglion cell axons. RNFL thickness decreases as disease progresses. A positive number, or, absence of change from baseline reflects a good clinical outcome, a negative number reflects loss of thickness, or a bad outcome. |
| Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit. | Change from Baseline in visual field Mean Deviation(MD) as measured by Humphrey automated visual field testing stimulus III by visit. The Humphrey visual field test measures the entire area of peripheral vision that can be seen while the eye is focused on a central point. Mean deviation (MD) is the mean deviation in the patient's results compared to those expected from the age-matched normative database. Lower/More negative scores mean worse outcome, higher/more positive score means better outcome. |
| Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Assessed at each visit from Baseline from Week 68 to Week 160 | Change from Baseline in visual field Mean Deviation(MD) as measured by Humphrey automated visual field testing stimulus III by visit. The Humphrey visual field test measures the entire area of peripheral vision that can be seen while the eye is focused on a central point. Mean deviation (MD) is the mean deviation in the patient's results compared to those expected from the age-matched normative database. Lower/More negative scores mean worse outcome, higher/more positive score means better outcome. |
| Double Masked Period: Color Discrimination - Number of Plates | Assessed at each visit from Baseline to Week 56 (follow-up visit) | Double Masked Period: Change from Baseline in Color Discrimination - Number of Plates from Baseline by Visit by Ishihara Test. Score range: 0-38. Higher number equals better color discrimination equals better outcome. Lower number equals worse color discrimination equals worse outcome. |
| Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Assessed at each visit from Baseline from Week 68 to Week 160 | Open Label Extension Period: Change from Baseline in Color Discrimination - Number of Plates from Baseline by Visit by Ishihara Test. Score range: 0-38. Higher number equals better color discrimination equals better outcome. Lower number equals worse color discrimination equals worse outcome. |
| Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Assessed at each visit from Baseline to Week 56 (follow-up visit) | Letters are arranged on a 60 x 85 cm chart in sets of triplets which contain the same contrast, decreasing in log10 contrast from top to bottom and left to right as participant progresses from one triplet to the other. Each group of three letters is decreased in contrast by a factor 0.71 (1/√2) (log contrast 0.15) of the proceeding set. Participant reads letters, starting with highest contrast, until unable to read letters in a single group. Score is based on the contrast of the last group read correctly.The size of the letters on the chart subtend 0.5 degrees at 3m, The test is scored in LogCS units, where each set of triplets advances in steps of 0.15 log units ranging from LogCS of 0.00 (approx. 100% contrast) to LogCS 2.25 (approx. 0.56% contrast). Change from baseline: the more negative the number means poor outcome, the more positive the number means better outcome. |
| Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Assessed at each visit from Baseline from Week 68 to Week 160 | Letters are arranged on a 60 x 85 cm chart in sets of triplets which contain the same contrast, decreasing in log10 contrast from top to bottom and left to right as participant progresses from one triplet to the other. Each group of three letters is decreased in contrast by a factor 0.71 (1/√2) (log contrast 0.15) of the proceeding set. Participant reads letters, starting with highest contrast, until unable to read letters in a single group. Score is based on the contrast of the last group read correctly. The size of the letters on the chart subtend 0.5 degrees at 3m, The test is scored in LogCS units, where each set of triplets advances in steps of 0.15 log units ranging from LogCS of 0.00 (approx. 100% contrast) to LogCS 2.25 (approx. 0.56% contrast). Change from baseline: the more negative the number means poor outcome, the more positive the number means better outcome. |
| Double Masked Change From Baseline in Retinal Ganglion Cell Layer Thickness | Assessed at Baseline and Week 52 (end-of-treatment visit) | Change from baseline in Retinal Ganglion Cell Layer Thickness by SD-OCT From Baseline by Visit |
| Open Label Extension Period: Change From Baseline in Retinal Ganglion Cell Layer Thickness | Assessed at Baseline, Week 148, | Open Label Extension Period: Change from baseline in Retinal Ganglion Cell Layer Thickness by SD-OCT by SD-OCT by visit |
| Double Masked: VFQ-39 Distance Activities Score | Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit) | Change from baseline in Visual Function Questionnaire (VFQ-39) Distance Activities score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Open Label Extension Period: VFQ-39 Distance Activities Score | Assessed at Baseline, Week 160 (End of OLE Period) | Change in Visual Function Questionnaire (VFQ-39) Distance Activities Score.National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Open Label Extension Period: VFQ-39 Social Functioning Score | Assessed at Baseline, Week 160 (End of OLE Period) | Change from baseline in Visual Function Questionnaire (VFQ-39) Social Functioning Score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Double Masked: VFQ-39 Social Functioning Score. | Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit) | Change from baseline in Visual Function Questionnaire (VFQ-39) Social Functioning score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Double Masked: VFQ-39 Mental Health Score | Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit) | Change from baseline in Visual Function Questionnaire (VFQ-39) Mental Health score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Open Label Extension Period: VFQ-39 Mental Health Score | Assessed at Baseline, Week 160 (End of OLE Period) | Change from baseline in Visual Function Questionnaire (VFQ-39) Mental Health Score . National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Double Masked: VFQ-39 Role Difficulties Score | Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit) | Change from baseline in Visual Function Questionnaire (VFQ-39) Role Difficulties score National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Open Label Extension Period: VFQ-39 Role Difficulties Score | Assessed at Baseline, Week 160 (End of OLE Period) | Change from baseline in Visual Function Questionnaire (VFQ-39) Role Difficulties Score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Open Label Extension Period:VFQ-39 Dependency Score | Assessed at Baseline, Week 160 (End of OLE Period) | Change from baseline in Visual Function Questionnaire (VFQ-39 ) Dependency Score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Double Masked: VFQ-39 Dependency Score | Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit) | Change from baseline in Visual Function Questionnaire (VFQ-39) Dependency score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Double Masked: VFQ-39 Color Vision Score | Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit) | Change from baseline in Visual Function Questionnaire (VFQ-39) Color Vision score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Open Label Extension Period: VFQ-39 Color Vision Score | Assessed at Baseline, Week 160 (End of OLE Period) | Change from baseline in Visual Function Questionnaire (VFQ-39) Color Vision score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Double Masked: VFQ-39 Peripheral Score | Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit) | Change from baseline in Visual Function Questionnaire (VFQ-39) Peripheral Score . National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Open Label Extension Period: Visual Function Questionnaire (VFQ-39) Peripheral Score | Assessed at Baseline, Week 160 (End of OLE Period) | Change from baseline in Visual Function Questionnaire (VFQ-39) Peripheral Score . National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Double Masked Period: Change in Intraocular Pressure (IOP) | Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit) | Double Masked Period: Change in intraocular Pressure (IOP) in mmHg from Baseline by Visit |
| Open Label Extension Period: Intraocular Pressure (IOP) | Assessed at Baseline, Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit) | Change from baseline in Intraocular Pressure (IOP) in mmHg by Visit. |
| OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Assessed at Baseline, Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit) | Open Label Extension: Shift in Slit Lamp Examination from Baseline by Visit: number of eyes that changed from normal or abnormal not clinically significant, to abnormal clinically significant for Vehicle Eyes |
| OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Assessed at Baseline, Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit) | Open Label Extension: Shift in Slit Lamp Examination from Baseline by Visit: number of eyes that changed from normal or abnormal not clinically significant, to abnormal clinically significant for Elamipretide Eyes |
| Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit) | Incidence of Change from normal or Abnormal, clinically insignificant to normal clinically significant |
| Double Masked: Dilated Fundus Changes From Baseline: Vehicle Eye and Elamipretide Eyes | Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit) | Double Masked: Dilated Fundus Changes from Baseline: Vehicle Eye and Elamipretide Eyes Incidence of Change from normal or Abnormal-clinically insignificant, to normal clinically significant |
| Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit. | Change from baseline in photopic negative response electroretinography (PhNR-ERG) a-wave amplitude by Visit. Photopic negative response electroretinography (PhNR-ERG) assesses retinal cell function. A decrease in the amplitude of the a-wave is associated with worse outcomes. Change from baseline in a-wave amplitude: a more positive number equals a decrease in amplitude, and therefore a worse outcome, a more negative number mean a better outcome. |
| Double Masked: VFQ-39 General Health Score | Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit) | Change from baseline in Visual Function Questionnaire (VFQ-39) General Health score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated. |
| Open Label Extension Period: Dilated Fundus Changes From Baseline: Vehicle Eye and Elamipretide Eyes | Assessed at Baseline, Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit) | Incidence of Change from normal or Abnormal-clinically insignificant, to normal clinically significant |
| Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Assessed at each visit from Baseline from Week 68 to Week 160 | Change from baseline in photopic negative response electroretinography (PhNR-ERG) a-wave Amplitude by Visit. Photopic negative response electroretinography (PhNR-ERG) assesses retinal cell function. A decrease in the amplitude of the a-wave is associated with worse outcomes. Change from baseline in a-wave amplitude: a more positive number equals a decrease in amplitude, and therefore a worse outcome, a more negative number mean a better outcome. |
| Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit. | Change from baseline in photopic negative response electroretinography (PhNR-ERG) b-wave amplitude by visit. Photopic negative response electroretinography (PhNR -ERG) assesses retinal cell function. A decrease in the amplitude of the b-wave is associated with worse outcomes. Change from baseline in b-wave amplitude: a more negative number equals a decrease in amplitude, and therefore a worse outcome, a more positive number mean a better outcome. |
| Open Label Extension Period: PhNR-ERG B-wave Amplitude | Assessed at each visit from Baseline from Week 68 to Week 160 | Change from baseline in photopic negative response electroretinography (PhNR-ERG) b-wave Amplitude by Visit. Photopic negative response electroretinography (PhNR -ERG) assesses retinal cell function. A decrease in the amplitude of the b-wave is associated with worse outcomes. Change from baseline in b-wave amplitude: a more negative number equals a decrease in amplitude, and therefore a worse outcome, a more positive number means a better outcome. |
| Double Masked Period: PhNR Amplitude | Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit. | Double Masked Period: Change from baseline in PhNR Amplitude by Visit. Photopic negative response electroretinography (PhNR -ERG) assesses retinal cell function. A decrease in PhNR amplitude means worse outcome. Change from baseline: A more negative number means a better outcome, a more positive number means a worse outcome. |
| Open Label Extension Period: PhNR Amplitude | Assessed at each visit from Baseline from Week 68 to Week 160 | Open Label Extension Period: Change from baseline in PhNR Amplitude by Visit. Photopic negative response electroretinography (PhNR -ERG) assesses retinal cell function. A decrease in PhNR amplitude means worse outcome. Change from baseline: A more negative number means a better outcome, a more positive number means a worse outcome. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Group 2 One drop elamipretide (MTP-131) 1% topical ophthalmic solution BID applied to a single eye and one drop of vehicle topical ophthalmic solution BID in the fellow eye. | 8 |
| Group 3 One drop elamipretide (MTP-131) 1% topical ophthalmic solution BID applied to both eyes (OU). | 4 |
| Total | 12 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Open-Label Period | Withdrawal by Subject | 0 | 1 |
Baseline characteristics
| Characteristic | Group 2 | Group 3 | Total |
|---|---|---|---|
| Age, Continuous | 35.4 years STANDARD_DEVIATION 14.47 | 30.0 years STANDARD_DEVIATION 4.83 | 33.6 years STANDARD_DEVIATION 12.11 |
| BMI | 25.70 kg/m^2 STANDARD_DEVIATION 3.937 | 29.43 kg/m^2 STANDARD_DEVIATION 2.081 | 26.94 kg/m^2 STANDARD_DEVIATION 3.796 |
| Elamipretide eye both eyes | 0 Participants | 4 Participants | 4 Participants |
| Elamipretide eye left eye only | 4 Participants | 0 Participants | 4 Participants |
| Elamipretide eye right eye only | 4 Participants | 0 Participants | 4 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 2 Participants | 3 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 6 Participants | 2 Participants | 8 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 4 Participants | 4 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) White | 7 Participants | 0 Participants | 7 Participants |
| Sex: Female, Male Female | 1 Participants | 1 Participants | 2 Participants |
| Sex: Female, Male Male | 7 Participants | 3 Participants | 10 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 8 | 0 / 4 | 0 / 8 | 0 / 4 |
| other Total, other adverse events | 6 / 8 | 3 / 4 | 7 / 8 | 4 / 4 |
| serious Total, serious adverse events | 0 / 8 | 0 / 4 | 0 / 8 | 0 / 4 |
Outcome results
Primary
Double Masked Period: Best Corrected Visual Acuity (BCVA)
Double Masked Period: Best corrected visual acuity (BCVA) using the using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale by visit. ETDRS charts present a series of five letters of equal difficulty on each row, with standardized spacing between letters and rows; there is a total of 14 lines (70 letters), with letter size increasing further geometrically and equivalently in every line by a factor of 1.2589 (or 0.1 log unit), moving up the chart. Minimum score of zero, maximum score of 100. Change from baseline: a more negative score is worse outcome, a more positive score is better outcome. A lower score means less letters were read correctly (worse outcome) and a higher score means more letters were read correctly (better outcome).
Time frame: Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit)
Population: All participants for whom BCVA was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 16 | 3.0 letters | Standard Deviation 3.38 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 32 | 4.8 letters | Standard Deviation 15.54 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 4 | 0.3 letters | Standard Deviation 3.33 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 36 | 6.6 letters | Standard Deviation 11.73 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 12 | 2.8 letters | Standard Deviation 5.15 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 40 | 2.6 letters | Standard Deviation 11.56 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 24 | 4.1 letters | Standard Deviation 9.98 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 48 | 5.1 letters | Standard Deviation 16.58 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 8 | 2.0 letters | Standard Deviation 3.02 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 52 | 4.6 letters | Standard Deviation 16.27 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 44 | 6.1 letters | Standard Deviation 11.85 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 56 (follow up Visit) | 1.7 letters | Standard Deviation 2.89 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 28 | 5.5 letters | Standard Deviation 11.99 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Day 5 | -3.1 letters | Standard Deviation 9.34 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 20 | 4.0 letters | Standard Deviation 10.42 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Day 5 | -0.5 letters | Standard Deviation 4.5 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 20 | 1.7 letters | Standard Deviation 4.63 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 4 | 1.0 letters | Standard Deviation 3.54 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 8 | 0.5 letters | Standard Deviation 5.29 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 12 | 0.4 letters | Standard Deviation 6.47 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 16 | 0.6 letters | Standard Deviation 4.06 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 44 | 3.5 letters | Standard Deviation 5.97 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 24 | 2.1 letters | Standard Deviation 3.79 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 28 | 1.4 letters | Standard Deviation 4.99 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 32 | 2.7 letters | Standard Deviation 7.67 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 36 | 1.9 letters | Standard Deviation 4.53 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 40 | 3.2 letters | Standard Deviation 4.4 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 48 | 4.8 letters | Standard Deviation 4.89 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 52 | 2.9 letters | Standard Deviation 5.45 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Best Corrected Visual Acuity (BCVA) | Week 56 (follow up Visit) | 5.3 letters | Standard Deviation 4.16 |
Primary
Double Masked Period: Incidence of Ocular TEAEs
The incidence of ocular treatment emergent adverse events (TEAEs).
Time frame: Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit)
Population: All participants for whom ocular AEs were measured.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Conjunctival hyperaemia | 3 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Punctate keratitis | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Dry eye | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Conjunctival oedema | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Eyelid oedema | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Eye irritation | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Eyelid irritation | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Eyelid exfoliation | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Lacrimation increased | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Choroidal neovascularisation | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Noninfective conjunctivitis | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Immune system disorders: Hypersensitivity | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Infections and infestations Conjunctivitis | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Injury, poisoning and procedural complications: Foreign body in eye | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Injury, poisoning and procedural complications: Skin abrasion | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Investigations: Intraocular pressure increased | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Incidence of Ocular TEAEs | Nervous system disorders: Parosmia | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Infections and infestations Conjunctivitis | 1 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Conjunctival hyperaemia | 8 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Lacrimation increased | 2 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Punctate keratitis | 3 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Nervous system disorders: Parosmia | 2 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Choroidal neovascularisation | 1 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Injury, poisoning and procedural complications: Foreign body in eye | 1 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Conjunctival oedema | 1 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Noninfective conjunctivitis | 1 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Eyelid oedema | 1 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Dry eye | 3 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Investigations: Intraocular pressure increased | 1 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Eye irritation | 2 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Immune system disorders: Hypersensitivity | 1 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Eyelid irritation | 2 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Injury, poisoning and procedural complications: Skin abrasion | 1 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Incidence of Ocular TEAEs | Eyelid exfoliation | 1 eyes |
Primary
Double Masked Period: Severity of Ocular TEAEs
The severity of ocular treatment emergent adverse events (TEAEs).
Time frame: Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit)
Population: All participants for whom ocular AEs were measured.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Eyelid exfoliation | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Eye irritation | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Foreign body in eye | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Eyelid oedema | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Investigations: Intraocular pressure increased | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Immune system disorders: Hypersensitivity | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Punctate keratitis | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Infections and infestations Conjunctivitis | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Conjunctival oedema | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Noninfective conjunctivitis | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Dry eye | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Choroidal neovascularisation | 2 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Skin abrasion | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Eyelid irritation | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Lacrimation increased | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Conjunctival hyperaemia | 2 eyes |
| Double Masked Group Vehicle Eyes | Double Masked Period: Severity of Ocular TEAEs | Nervous system disorders: Parosmia | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Dry eye | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Foreign body in eye | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Eyelid exfoliation | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Eyelid oedema | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Conjunctival oedema | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Lacrimation increased | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Immune system disorders: Hypersensitivity | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Infections and infestations Conjunctivitis | 1 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Choroidal neovascularisation | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Investigations: Intraocular pressure increased | 1 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Conjunctival hyperaemia | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Nervous system disorders: Parosmia | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Eye irritation | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Punctate keratitis | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Skin abrasion | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Eyelid irritation | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Severity of Ocular TEAEs | Noninfective conjunctivitis | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Choroidal neovascularisation | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Immune system disorders: Hypersensitivity | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Conjunctival oedema | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Noninfective conjunctivitis | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Eyelid oedema | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Investigations: Intraocular pressure increased | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Conjunctival hyperaemia | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Skin abrasion | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Punctate keratitis | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Eye irritation | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Eyelid irritation | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Foreign body in eye | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Eyelid exfoliation | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Dry eye | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Infections and infestations Conjunctivitis | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Lacrimation increased | 0 eyes |
| Vehicle Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Nervous system disorders: Parosmia | 0 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Noninfective conjunctivitis | 0 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Dry eye | 3 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Eyelid oedema | 1 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Conjunctival hyperaemia | 1 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Punctate keratitis | 3 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Eye irritation | 2 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Eyelid irritation | 2 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Eyelid exfoliation | 1 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Lacrimation increased | 2 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Choroidal neovascularisation | 0 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Conjunctival oedema | 1 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Immune system disorders: Hypersensitivity | 1 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Infections and infestations Conjunctivitis | 0 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Foreign body in eye | 1 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Skin abrasion | 1 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Investigations: Intraocular pressure increased | 0 eyes |
| Elamipretide Eyes: Mild | Double Masked Period: Severity of Ocular TEAEs | Nervous system disorders: Parosmia | 2 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Choroidal neovascularisation | 0 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Immune system disorders: Hypersensitivity | 0 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Lacrimation increased | 0 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Punctate keratitis | 0 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Infections and infestations Conjunctivitis | 1 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Eyelid exfoliation | 0 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Eyelid irritation | 0 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Foreign body in eye | 0 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Eye irritation | 0 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Skin abrasion | 0 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Conjunctival hyperaemia | 1 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Eyelid oedema | 0 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Nervous system disorders: Parosmia | 0 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Investigations: Intraocular pressure increased | 1 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Dry eye | 0 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Noninfective conjunctivitis | 1 eyes |
| Elamipretide Eyes: Moderate | Double Masked Period: Severity of Ocular TEAEs | Conjunctival oedema | 0 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Noninfective conjunctivitis | 0 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Punctate keratitis | 0 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Conjunctival hyperaemia | 0 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Immune system disorders: Hypersensitivity | 0 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Lacrimation increased | 0 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Nervous system disorders: Parosmia | 0 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Eyelid exfoliation | 0 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Choroidal neovascularisation | 1 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Skin abrasion | 0 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Infections and infestations Conjunctivitis | 0 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Investigations: Intraocular pressure increased | 0 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Eyelid irritation | 0 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Eyelid oedema | 0 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Dry eye | 0 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Conjunctival oedema | 0 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Foreign body in eye | 0 eyes |
| Elamipretide Eyes: Severe | Double Masked Period: Severity of Ocular TEAEs | Eye irritation | 0 eyes |
Primary
OLE: Severity of Ocular TEAEs
The severity of ocular treatment emergent adverse events (TEAEs).
Time frame: Assessed at Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit)
Population: All participants for whom ocular AEs were measured.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications Eye contusion | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Fall | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Severity of Ocular TEAEs | Eyelid irritation | 1 eyes |
| Double Masked Group Vehicle Eyes | OLE: Severity of Ocular TEAEs | Eye disorders Cataract nuclear | 5 eyes |
| Double Masked Group Vehicle Eyes | OLE: Severity of Ocular TEAEs | Visual acuity reduced | 1 eyes |
| Double Masked Group Vehicle Eyes | OLE: Severity of Ocular TEAEs | Eye irritation | 2 eyes |
| Double Masked Group Vehicle Eyes | OLE: Severity of Ocular TEAEs | Immune system disorders Seasonal allergy | 1 eyes |
| Double Masked Group Vehicle Eyes | OLE: Severity of Ocular TEAEs | Dry eye | 1 eyes |
| Double Masked Group Vehicle Eyes | OLE: Severity of Ocular TEAEs | General disorders and administration site conditions Application site pain | 3 eyes |
| Double Masked Group Vehicle Eyes | OLE: Severity of Ocular TEAEs | Ocular hyperaemia | 1 eyes |
| Double Masked Group Vehicle Eyes | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications Corneal abrasian | 1 eyes |
| Double Masked Group Vehicle Eyes | OLE: Severity of Ocular TEAEs | Visual impairment | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Severity of Ocular TEAEs | Punctate keratitis | 1 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications Eye contusion | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Severity of Ocular TEAEs | Eyelid irritation | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Severity of Ocular TEAEs | General disorders and administration site conditions Application site pain | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Severity of Ocular TEAEs | Ocular hyperaemia | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Fall | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Severity of Ocular TEAEs | Punctate keratitis | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Severity of Ocular TEAEs | Eye disorders Cataract nuclear | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Severity of Ocular TEAEs | Visual acuity reduced | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Severity of Ocular TEAEs | Dry eye | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Severity of Ocular TEAEs | Visual impairment | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications Corneal abrasian | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Severity of Ocular TEAEs | Eye irritation | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Severity of Ocular TEAEs | Immune system disorders Seasonal allergy | 0 eyes |
| Vehicle Eyes: Severe | OLE: Severity of Ocular TEAEs | Punctate keratitis | 0 eyes |
| Vehicle Eyes: Severe | OLE: Severity of Ocular TEAEs | Ocular hyperaemia | 0 eyes |
| Vehicle Eyes: Severe | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications Corneal abrasian | 0 eyes |
| Vehicle Eyes: Severe | OLE: Severity of Ocular TEAEs | Immune system disorders Seasonal allergy | 0 eyes |
| Vehicle Eyes: Severe | OLE: Severity of Ocular TEAEs | Eye irritation | 0 eyes |
| Vehicle Eyes: Severe | OLE: Severity of Ocular TEAEs | Dry eye | 0 eyes |
| Vehicle Eyes: Severe | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Fall | 0 eyes |
| Vehicle Eyes: Severe | OLE: Severity of Ocular TEAEs | Eyelid irritation | 0 eyes |
| Vehicle Eyes: Severe | OLE: Severity of Ocular TEAEs | Eye disorders Cataract nuclear | 0 eyes |
| Vehicle Eyes: Severe | OLE: Severity of Ocular TEAEs | Visual acuity reduced | 0 eyes |
| Vehicle Eyes: Severe | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications Eye contusion | 0 eyes |
| Vehicle Eyes: Severe | OLE: Severity of Ocular TEAEs | Visual impairment | 0 eyes |
| Vehicle Eyes: Severe | OLE: Severity of Ocular TEAEs | General disorders and administration site conditions Application site pain | 0 eyes |
| Elamipretide Eyes: Mild | OLE: Severity of Ocular TEAEs | Ocular hyperaemia | 1 eyes |
| Elamipretide Eyes: Mild | OLE: Severity of Ocular TEAEs | Visual impairment | 2 eyes |
| Elamipretide Eyes: Mild | OLE: Severity of Ocular TEAEs | Visual acuity reduced | 1 eyes |
| Elamipretide Eyes: Mild | OLE: Severity of Ocular TEAEs | General disorders and administration site conditions Application site pain | 5 eyes |
| Elamipretide Eyes: Mild | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications Eye contusion | 1 eyes |
| Elamipretide Eyes: Mild | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Fall | 1 eyes |
| Elamipretide Eyes: Mild | OLE: Severity of Ocular TEAEs | Eye disorders Cataract nuclear | 6 eyes |
| Elamipretide Eyes: Mild | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications Corneal abrasian | 0 eyes |
| Elamipretide Eyes: Mild | OLE: Severity of Ocular TEAEs | Punctate keratitis | 0 eyes |
| Elamipretide Eyes: Mild | OLE: Severity of Ocular TEAEs | Dry eye | 1 eyes |
| Elamipretide Eyes: Mild | OLE: Severity of Ocular TEAEs | Eye irritation | 1 eyes |
| Elamipretide Eyes: Mild | OLE: Severity of Ocular TEAEs | Eyelid irritation | 1 eyes |
| Elamipretide Eyes: Mild | OLE: Severity of Ocular TEAEs | Immune system disorders Seasonal allergy | 1 eyes |
| Elamipretide Eyes: Moderate | OLE: Severity of Ocular TEAEs | Immune system disorders Seasonal allergy | 0 eyes |
| Elamipretide Eyes: Moderate | OLE: Severity of Ocular TEAEs | General disorders and administration site conditions Application site pain | 0 eyes |
| Elamipretide Eyes: Moderate | OLE: Severity of Ocular TEAEs | Punctate keratitis | 0 eyes |
| Elamipretide Eyes: Moderate | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications Eye contusion | 0 eyes |
| Elamipretide Eyes: Moderate | OLE: Severity of Ocular TEAEs | Eye disorders Cataract nuclear | 0 eyes |
| Elamipretide Eyes: Moderate | OLE: Severity of Ocular TEAEs | Dry eye | 0 eyes |
| Elamipretide Eyes: Moderate | OLE: Severity of Ocular TEAEs | Eye irritation | 0 eyes |
| Elamipretide Eyes: Moderate | OLE: Severity of Ocular TEAEs | Eyelid irritation | 0 eyes |
| Elamipretide Eyes: Moderate | OLE: Severity of Ocular TEAEs | Ocular hyperaemia | 0 eyes |
| Elamipretide Eyes: Moderate | OLE: Severity of Ocular TEAEs | Visual acuity reduced | 0 eyes |
| Elamipretide Eyes: Moderate | OLE: Severity of Ocular TEAEs | Visual impairment | 0 eyes |
| Elamipretide Eyes: Moderate | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications Corneal abrasian | 0 eyes |
| Elamipretide Eyes: Moderate | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Fall | 0 eyes |
| Elamipretide Eyes: Severe | OLE: Severity of Ocular TEAEs | Visual impairment | 0 eyes |
| Elamipretide Eyes: Severe | OLE: Severity of Ocular TEAEs | Eyelid irritation | 0 eyes |
| Elamipretide Eyes: Severe | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications Corneal abrasian | 0 eyes |
| Elamipretide Eyes: Severe | OLE: Severity of Ocular TEAEs | Eye irritation | 0 eyes |
| Elamipretide Eyes: Severe | OLE: Severity of Ocular TEAEs | Dry eye | 0 eyes |
| Elamipretide Eyes: Severe | OLE: Severity of Ocular TEAEs | Punctate keratitis | 0 eyes |
| Elamipretide Eyes: Severe | OLE: Severity of Ocular TEAEs | Eye disorders Cataract nuclear | 0 eyes |
| Elamipretide Eyes: Severe | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications: Fall | 0 eyes |
| Elamipretide Eyes: Severe | OLE: Severity of Ocular TEAEs | Injury, poisoning and procedural complications Eye contusion | 0 eyes |
| Elamipretide Eyes: Severe | OLE: Severity of Ocular TEAEs | Immune system disorders Seasonal allergy | 0 eyes |
| Elamipretide Eyes: Severe | OLE: Severity of Ocular TEAEs | Visual acuity reduced | 0 eyes |
| Elamipretide Eyes: Severe | OLE: Severity of Ocular TEAEs | Ocular hyperaemia | 0 eyes |
| Elamipretide Eyes: Severe | OLE: Severity of Ocular TEAEs | General disorders and administration site conditions Application site pain | 0 eyes |
Primary
Open Label Extension: Incidence of Ocular TEAEs
Open Label Extension: The incidence of ocular treatment emergent adverse events (TEAEs).
Time frame: Assessed at Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit)
Population: All participants for whom ocular AEs were measured. For the ocular AE Injury, poisoning and procedural complications: Fall, the subject fell and had a black eye. For ocular AE Immune system disorders Seasonal allergy, individual reported seasonal allergies affecting vision in both eyes. For General disorders and administration site conditions Application site pain, individuals reported burning in eyes, worsening of vision upon application of vehicle or elamipretide eyes.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension: Incidence of Ocular TEAEs | Eye disorders Ocular hyperaemia | 1 eyes |
| Double Masked Group Vehicle Eyes | Open Label Extension: Incidence of Ocular TEAEs | Eye disorders Punctate keratitis | 1 eyes |
| Double Masked Group Vehicle Eyes | Open Label Extension: Incidence of Ocular TEAEs | Eye disorders Dry eye | 1 eyes |
| Double Masked Group Vehicle Eyes | Open Label Extension: Incidence of Ocular TEAEs | Eye disorders Eyelid irritation | 1 eyes |
| Double Masked Group Vehicle Eyes | Open Label Extension: Incidence of Ocular TEAEs | Eye disorders Eye irritation | 2 eyes |
| Double Masked Group Vehicle Eyes | Open Label Extension: Incidence of Ocular TEAEs | Immune system disorders Seasonal allergy (worsening vision in eyes) | 1 eyes |
| Double Masked Group Vehicle Eyes | Open Label Extension: Incidence of Ocular TEAEs | General disorders and administration site conditions Application site pain (eyes burn, vision worse) | 3 eyes |
| Double Masked Group Vehicle Eyes | Open Label Extension: Incidence of Ocular TEAEs | Injury, poisoning and procedural complications: Corneal abrasian | 1 eyes |
| Double Masked Group Vehicle Eyes | Open Label Extension: Incidence of Ocular TEAEs | Eye disorders Visual impairment | 0 eyes |
| Double Masked Group Vehicle Eyes | Open Label Extension: Incidence of Ocular TEAEs | Injury, poisoning and procedural complications: Eye contusion | 0 eyes |
| Double Masked Group Vehicle Eyes | Open Label Extension: Incidence of Ocular TEAEs | Eye disorders Cataract nuclear | 1 eyes |
| Double Masked Group Vehicle Eyes | Open Label Extension: Incidence of Ocular TEAEs | Injury, poisoning and procedural complications: Fall (resulting in black eye) | 0 eyes |
| Double Masked Group Vehicle Eyes | Open Label Extension: Incidence of Ocular TEAEs | Eye disorders Visual acuity reduced | 1 eyes |
| Double Masked Group Elamipretide Eyes | Open Label Extension: Incidence of Ocular TEAEs | Injury, poisoning and procedural complications: Fall (resulting in black eye) | 1 eyes |
| Double Masked Group Elamipretide Eyes | Open Label Extension: Incidence of Ocular TEAEs | Eye disorders Eyelid irritation | 1 eyes |
| Double Masked Group Elamipretide Eyes | Open Label Extension: Incidence of Ocular TEAEs | Eye disorders Visual acuity reduced | 1 eyes |
| Double Masked Group Elamipretide Eyes | Open Label Extension: Incidence of Ocular TEAEs | Eye disorders Visual impairment | 2 eyes |
| Double Masked Group Elamipretide Eyes | Open Label Extension: Incidence of Ocular TEAEs | Eye disorders Punctate keratitis | 0 eyes |
| Double Masked Group Elamipretide Eyes | Open Label Extension: Incidence of Ocular TEAEs | General disorders and administration site conditions Application site pain (eyes burn, vision worse) | 5 eyes |
| Double Masked Group Elamipretide Eyes | Open Label Extension: Incidence of Ocular TEAEs | Eye disorders Cataract nuclear | 1 eyes |
| Double Masked Group Elamipretide Eyes | Open Label Extension: Incidence of Ocular TEAEs | Eye disorders Ocular hyperaemia | 1 eyes |
| Double Masked Group Elamipretide Eyes | Open Label Extension: Incidence of Ocular TEAEs | Eye disorders Dry eye | 1 eyes |
| Double Masked Group Elamipretide Eyes | Open Label Extension: Incidence of Ocular TEAEs | Immune system disorders Seasonal allergy (worsening vision in eyes) | 1 eyes |
| Double Masked Group Elamipretide Eyes | Open Label Extension: Incidence of Ocular TEAEs | Injury, poisoning and procedural complications: Corneal abrasian | 0 eyes |
| Double Masked Group Elamipretide Eyes | Open Label Extension: Incidence of Ocular TEAEs | Injury, poisoning and procedural complications: Eye contusion | 1 eyes |
| Double Masked Group Elamipretide Eyes | Open Label Extension: Incidence of Ocular TEAEs | Eye disorders Eye irritation | 1 eyes |
Primary
Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale
Open Label Extension Period: Best corrected visual acuity (BCVA) using the using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale by visit. ETDRS charts present a series of five letters of equal difficulty on each row, with standardized spacing between letters and rows; there is a total of 14 lines (70 letters), with letter size increasing further geometrically and equivalently in every line by a factor of 1.2589 (or 0.1 log unit), moving up the chart. Minimum score of zero, maximum score of 100. Change from baseline: a more negative score is worse outcome, a more positive score is better outcome. A lower score means less letters were read correctly (worse outcome) and a higher score means more letters were read correctly (better outcome).
Time frame: Assessed at each visit from Baseline from Week 68 to Week 160 (follow-up visit).
Population: All participants for whom BCVA was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Week 68 | 8.5 letters | Standard Deviation 14.16 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Week 84 | 10.6 letters | Standard Deviation 18.66 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Week 100 | 6.1 letters | Standard Deviation 13.86 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Week 104 | -3.5 letters | Standard Deviation 6.36 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Week 116 | 11.3 letters | Standard Deviation 14 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Week 132 | 11.6 letters | Standard Deviation 17.04 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Week 148 | 2.8 letters | Standard Deviation 2.5 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Week 160 | 1.7 letters | Standard Deviation 2.08 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Week 160 | 7.7 letters | Standard Deviation 4.16 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Week 68 | 6.00 letters | Standard Deviation 7.18 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Week 116 | 5.9 letters | Standard Deviation 6.71 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Week 84 | 5.4 letters | Standard Deviation 7.7 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Week 148 | 2.9 letters | Standard Deviation 4.05 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Week 100 | 6.1 letters | Standard Deviation 6.12 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Week 132 | 4.3 letters | Standard Deviation 8.36 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale | Week 104 | 2.5 letters | Standard Deviation 0.71 |
Secondary
Double Masked Change From Baseline in Retinal Ganglion Cell Layer Thickness
Change from baseline in Retinal Ganglion Cell Layer Thickness by SD-OCT From Baseline by Visit
Time frame: Assessed at Baseline and Week 52 (end-of-treatment visit)
Population: All participants for whom Retinal Ganglion Cell Layer Thickness was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked Change From Baseline in Retinal Ganglion Cell Layer Thickness | -1.5 µm | Standard Deviation 4 |
| Double Masked Group Elamipretide Eyes | Double Masked Change From Baseline in Retinal Ganglion Cell Layer Thickness | -3.6 µm | Standard Deviation 13.49 |
Secondary
Double Masked: Dilated Fundus Changes From Baseline: Vehicle Eye and Elamipretide Eyes
Double Masked: Dilated Fundus Changes from Baseline: Vehicle Eye and Elamipretide Eyes Incidence of Change from normal or Abnormal-clinically insignificant, to normal clinically significant
Time frame: Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit)
Population: All participants for whom dilated fundus was measured.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked: Dilated Fundus Changes From Baseline: Vehicle Eye and Elamipretide Eyes | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked: Dilated Fundus Changes From Baseline: Vehicle Eye and Elamipretide Eyes | 0 eyes |
Secondary
Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude
Double Masked Period: Change from baseline by visit in PhNR Peak to Trough Adjusted for a-wave Amplitude by Visit. Calculated as (b-wave amplitude - PhNR amplitude)/a-wave amplitude. A reduced amplitude ratio equals worse outcome.
Time frame: Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit.
Population: All participants for whom PhNR-ERG was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 16 | -0.53 Ratio | Standard Deviation 1.26 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 32 | -0.24 Ratio | Standard Deviation 0.82 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 44 | -0.68 Ratio | Standard Deviation 0.438 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 36 | -0.05 Ratio | Standard Deviation 0.687 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 20 | -0.32 Ratio | Standard Deviation 0.992 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 40 | 0.63 Ratio | Standard Deviation 0.546 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 12 | 0.08 Ratio | Standard Deviation 0.827 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 24 | -0.35 Ratio | Standard Deviation 0.856 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 48 | -0.61 Ratio | Standard Deviation 0.811 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 8 | -0.03 Ratio | Standard Deviation 0.655 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 52 | -0.61 Ratio | Standard Deviation 0.291 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 28 | -0.35 Ratio | Standard Deviation 0.819 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 56 (Follow up) | -1.22 Ratio | Standard Deviation 0.114 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 4 | 0.18 Ratio | Standard Deviation 0.392 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 56 (Follow up) | -0.87 Ratio | Standard Deviation 0.274 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 4 | -0.21 Ratio | Standard Deviation 1.1 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 8 | 1.08 Ratio | Standard Deviation 4.628 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 12 | 0.00 Ratio | Standard Deviation 0.962 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 16 | -0.01 Ratio | Standard Deviation 0.862 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 20 | 0.10 Ratio | Standard Deviation 1.022 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 24 | -0.29 Ratio | Standard Deviation 0.746 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 28 | -0.21 Ratio | Standard Deviation 1.358 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 32 | -0.15 Ratio | Standard Deviation 0.606 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 36 | -0.70 Ratio | Standard Deviation 1.162 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 40 | 0.02 Ratio | Standard Deviation 1.123 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 44 | -0.66 Ratio | Standard Deviation 1.016 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 48 | -0.31 Ratio | Standard Deviation 0.769 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 52 | -0.37 Ratio | Standard Deviation 0.403 |
Secondary
Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit
Letters are arranged on a 60 x 85 cm chart in sets of triplets which contain the same contrast, decreasing in log10 contrast from top to bottom and left to right as participant progresses from one triplet to the other. Each group of three letters is decreased in contrast by a factor 0.71 (1/√2) (log contrast 0.15) of the proceeding set. Participant reads letters, starting with highest contrast, until unable to read letters in a single group. Score is based on the contrast of the last group read correctly.The size of the letters on the chart subtend 0.5 degrees at 3m, The test is scored in LogCS units, where each set of triplets advances in steps of 0.15 log units ranging from LogCS of 0.00 (approx. 100% contrast) to LogCS 2.25 (approx. 0.56% contrast). Change from baseline: the more negative the number means poor outcome, the more positive the number means better outcome.
Time frame: Assessed at each visit from Baseline to Week 56 (follow-up visit)
Population: All participants for whom Contrast sensitivity was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Day 5 | 0.075 score on a scale | Standard Deviation 0.0802 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 8 | 0.075 score on a scale | Standard Deviation 0.1134 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 12 | 0.056 score on a scale | Standard Deviation 0.1116 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 16 | 0.075 score on a scale | Standard Deviation 0.1134 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 20 | 0.094 score on a scale | Standard Deviation 0.1116 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 24 | 0.075 score on a scale | Standard Deviation 0.1134 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 28 | 0.113 score on a scale | Standard Deviation 0.1747 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 48 | 0.188 score on a scale | Standard Deviation 0.2232 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 4 | 0.038 score on a scale | Standard Deviation 0.0694 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 32 | 0.113 score on a scale | Standard Deviation 0.1553 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 36 | 0.150 score on a scale | Standard Deviation 0.1604 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 40 | 0.206 score on a scale | Standard Deviation 0.1782 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 44 | 0.225 score on a scale | Standard Deviation 0.2268 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 52 | 0.225 score on a scale | Standard Deviation 0.2121 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 56 (Follow up) | 0.100 score on a scale | Standard Deviation 0.0866 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 44 | 0.066 score on a scale | Standard Deviation 0.2625 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Day 5 | -0.019 score on a scale | Standard Deviation 0.1078 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 4 | -0.028 score on a scale | Standard Deviation 0.1366 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 36 | 0.056 score on a scale | Standard Deviation 0.1806 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 8 | 0.047 score on a scale | Standard Deviation 0.142 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 56 (Follow up) | 0.050 score on a scale | Standard Deviation 0.0866 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 12 | 0.000 score on a scale | Standard Deviation 0.1342 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 48 | 0.084 score on a scale | Standard Deviation 0.1446 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 16 | -0.009 score on a scale | Standard Deviation 0.102 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 40 | 0.075 score on a scale | Standard Deviation 0.1975 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 20 | -0.009 score on a scale | Standard Deviation 0.102 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 32 | 0.028 score on a scale | Standard Deviation 0.1663 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 24 | 0.028 score on a scale | Standard Deviation 0.1663 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 52 | 0.075 score on a scale | Standard Deviation 0.2258 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change From Baseline in Contrast Sensitivity by Pelli-Robson Contrast Sensitivity Chart by Visit | Week 28 | -0.009 score on a scale | Standard Deviation 0.1393 |
Secondary
Double Masked Period: Change in Intraocular Pressure (IOP)
Double Masked Period: Change in intraocular Pressure (IOP) in mmHg from Baseline by Visit
Time frame: Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit)
Population: All participants for whom intraocular Pressure (IOP) was measured
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 16 | 0.4 mmHg | Standard Deviation 1.51 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 32 | 0.0 mmHg | Standard Deviation 2.62 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 4 | 0.0 mmHg | Standard Deviation 3.16 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 36 | -1.1 mmHg | Standard Deviation 1.55 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 20 | 0.4 mmHg | Standard Deviation 1.85 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 40 | 0.3 mmHg | Standard Deviation 2.25 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 12 | 0.5 mmHg | Standard Deviation 2.39 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 44 | -0.4 mmHg | Standard Deviation 2 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 24 | -0.1 mmHg | Standard Deviation 3.31 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 48 | 0.5 mmHg | Standard Deviation 3.66 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 8 | -0.4 mmHg | Standard Deviation 1.77 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 52 | -1.0 mmHg | Standard Deviation 1.2 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 28 | 0.3 mmHg | Standard Deviation 1.83 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 56 (follow up Visit) | -1.0 mmHg | Standard Deviation 1.2 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Day 5 | -0.3 mmHg | Standard Deviation 1.67 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 56 (follow up Visit) | -0.5 mmHg | Standard Deviation 2.66 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Day 5 | -0.2 mmHg | Standard Deviation 1.87 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 4 | -0.4 mmHg | Standard Deviation 2.16 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 8 | -0.5 mmHg | Standard Deviation 1.83 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 12 | -0.5 mmHg | Standard Deviation 2.53 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 16 | 0.6 mmHg | Standard Deviation 3.14 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 20 | -0.3 mmHg | Standard Deviation 1.66 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 24 | -0.4 mmHg | Standard Deviation 2.99 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 28 | 0.3 mmHg | Standard Deviation 1.88 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 32 | -0.3 mmHg | Standard Deviation 1.92 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 36 | -1.3 mmHg | Standard Deviation 1.96 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 40 | -0.6 mmHg | Standard Deviation 1.75 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 44 | -0.3 mmHg | Standard Deviation 1.85 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 48 | 0.8 mmHg | Standard Deviation 2.79 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Change in Intraocular Pressure (IOP) | Week 52 | -0.5 mmHg | Standard Deviation 2.66 |
Secondary
Double Masked Period: Color Discrimination - Number of Plates
Double Masked Period: Change from Baseline in Color Discrimination - Number of Plates from Baseline by Visit by Ishihara Test. Score range: 0-38. Higher number equals better color discrimination equals better outcome. Lower number equals worse color discrimination equals worse outcome.
Time frame: Assessed at each visit from Baseline to Week 56 (follow-up visit)
Population: All participants for whom Number of Plates was measured
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 16 | 0.0 Plates | Standard Deviation 0 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 32 | 0.8 Plates | Standard Deviation 1.16 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 4 | 0.0 Plates | Standard Deviation 0 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 36 | 0.8 Plates | Standard Deviation 1.16 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 20 | 0.5 Plates | Standard Deviation 0.93 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 40 | 0.5 Plates | Standard Deviation 0.76 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 12 | 0.3 Plates | Standard Deviation 0.71 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 44 | 1.0 Plates | Standard Deviation 1.07 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 24 | 0.3 Plates | Standard Deviation 0.71 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 48 | 1.3 Plates | Standard Deviation 1.39 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 8 | 0.1 Plates | Standard Deviation 0.35 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 52 | 1.6 Plates | Standard Deviation 1.6 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 28 | 0.9 Plates | Standard Deviation 1.36 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 56 (Follow up) | 1.7 Plates | Standard Deviation 2.89 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Color Discrimination - Number of Plates | Day 5 | 0.0 Plates | Standard Deviation 0 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 56 (Follow up) | 1.0 Plates | Standard Deviation 1 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Color Discrimination - Number of Plates | Day 5 | 0.1 Plates | Standard Deviation 0.57 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 4 | 0.1 Plates | Standard Deviation 0.5 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 8 | 0.3 Plates | Standard Deviation 0.58 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 12 | 0.2 Plates | Standard Deviation 0.75 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 16 | 0.4 Plates | Standard Deviation 0.89 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 20 | 0.3 Plates | Standard Deviation 0.6 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 24 | 0.5 Plates | Standard Deviation 0.97 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 28 | 0.8 Plates | Standard Deviation 1.39 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 32 | 0.7 Plates | Standard Deviation 1.3 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 36 | 0.8 Plates | Standard Deviation 1.05 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 40 | 0.6 Plates | Standard Deviation 0.81 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 44 | 0.9 Plates | Standard Deviation 1.31 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 48 | 1.1 Plates | Standard Deviation 1.34 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Color Discrimination - Number of Plates | Week 52 | 1.1 Plates | Standard Deviation 1.09 |
Secondary
Double Masked Period: Mean Retinal Nerve Fiber (RNFL) Layer Thickness
Change from baseline in retinal nerve fiber layer thickness by spectral domain optical coherence tomography (SD-OCT). RNFL measures the loss of retinal ganglion cell axons. RNFL thickness decreases as disease progresses. A positive number, or, absence of change from baseline reflects a good clinical outcome, a negative number reflects loss of thickness, or a bad outcome.
Time frame: Assessed at Baseline and Week 52 (end-of-treatment visit)
Population: All participants for whom Retinal Nerve Fiber Layer Thickness was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked Period: Mean Retinal Nerve Fiber (RNFL) Layer Thickness | -3.8 µm | Standard Deviation 7.52 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Mean Retinal Nerve Fiber (RNFL) Layer Thickness | -2.6 µm | Standard Deviation 4.76 |
Secondary
Double Masked Period: PhNR Amplitude
Double Masked Period: Change from baseline in PhNR Amplitude by Visit. Photopic negative response electroretinography (PhNR -ERG) assesses retinal cell function. A decrease in PhNR amplitude means worse outcome. Change from baseline: A more negative number means a better outcome, a more positive number means a worse outcome.
Time frame: Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit.
Population: All participants for whom PhNR Amplitude was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Amplitude | Week 4 | -6.4 µV | Standard Deviation 22.27 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Amplitude | Week 8 | -4.6 µV | Standard Deviation 26.8 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Amplitude | Week 12 | -11.6 µV | Standard Deviation 26.59 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Amplitude | Week 16 | -4.5 µV | Standard Deviation 10.26 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Amplitude | Week 20 | 0.5 µV | Standard Deviation 20.65 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Amplitude | Week 24 | -4.3 µV | Standard Deviation 21.51 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Amplitude | Week 28 | -7.1 µV | Standard Deviation 26.32 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Amplitude | Week 32 | -4.1 µV | Standard Deviation 25.11 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Amplitude | Week 36 | -8.5 µV | Standard Deviation 24.94 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Amplitude | Week 40 | -2.3 µV | Standard Deviation 23.16 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Amplitude | Week 44 | -9.8 µV | Standard Deviation 29.75 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Amplitude | Week 48 | -2.8 µV | Standard Deviation 22.76 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Amplitude | Week 52 | -4.0 µV | Standard Deviation 25.41 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Amplitude | Week 56 (Follow up) | -1.6 µV | Standard Deviation 16.71 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Amplitude | Week 44 | -1.5 µV | Standard Deviation 11.91 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Amplitude | Week 4 | 0.3 µV | Standard Deviation 22.05 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Amplitude | Week 32 | -3.1 µV | Standard Deviation 13.61 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Amplitude | Week 8 | -3.3 µV | Standard Deviation 27.28 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Amplitude | Week 52 | -3.3 µV | Standard Deviation 11.73 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Amplitude | Week 12 | -0.7 µV | Standard Deviation 14.9 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Amplitude | Week 36 | -3.4 µV | Standard Deviation 10.17 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Amplitude | Week 16 | -3.1 µV | Standard Deviation 17.35 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Amplitude | Week 48 | -3.7 µV | Standard Deviation 13.14 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Amplitude | Week 20 | -3.1 µV | Standard Deviation 19.58 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Amplitude | Week 40 | -4.4 µV | Standard Deviation 10.92 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Amplitude | Week 24 | -0.5 µV | Standard Deviation 16.15 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Amplitude | Week 56 (Follow up) | -5.7 µV | Standard Deviation 6.45 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Amplitude | Week 28 | -1.5 µV | Standard Deviation 13.92 |
Secondary
Double Masked Period: PhNR/B-wave Amplitude Ratio
Double Masked Period: Change from baseline in PhNR/b-wave amplitude ratio by Visit. Photopic negative response electroretinography (PhNR assesses retinal cell function. A decrease in PhNR amplitude means worse outcome.
Time frame: Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit.
Population: All participants for whom PhNR was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 56 (Follow up) | 0.009 ratio | Standard Deviation 0.267 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 20 | -0.018 ratio | Standard Deviation 0.2005 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 4 | -0.102 ratio | Standard Deviation 0.2225 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 8 | -0.036 ratio | Standard Deviation 0.3101 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 12 | -0.113 ratio | Standard Deviation 0.2415 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 16 | -0.059 ratio | Standard Deviation 0.1179 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 24 | -0.052 ratio | Standard Deviation 0.208 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 28 | -0.044 ratio | Standard Deviation 0.2281 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 32 | -0.033 ratio | Standard Deviation 0.2414 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 36 | -0.104 ratio | Standard Deviation 0.2479 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 40 | -0.007 ratio | Standard Deviation 0.219 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 44 | -0.138 ratio | Standard Deviation 0.3194 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 48 | -0.020 ratio | Standard Deviation 0.2366 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 52 | -0.044 ratio | Standard Deviation 0.2428 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 24 | 0.014 ratio | Standard Deviation 0.2131 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 56 (Follow up) | -0.063 ratio | Standard Deviation 0.1149 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 44 | 0.013 ratio | Standard Deviation 0.2206 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 20 | -0.066 ratio | Standard Deviation 0.2365 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 52 | -0.029 ratio | Standard Deviation 0.1299 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 28 | -0.024 ratio | Standard Deviation 0.1634 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 4 | -0.033 ratio | Standard Deviation 0.2793 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 40 | -0.046 ratio | Standard Deviation 0.1202 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 8 | -0.033 ratio | Standard Deviation 0.3385 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 32 | -0.031 ratio | Standard Deviation 0.1513 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 12 | 0.019 ratio | Standard Deviation 0.1721 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 48 | -0.059 ratio | Standard Deviation 0.1533 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 16 | -0.070 ratio | Standard Deviation 0.2285 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR/B-wave Amplitude Ratio | Week 36 | -0.013 ratio | Standard Deviation 0.1795 |
Secondary
Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline
Double Masked Period: Change from baseline in PhNR Peak to Trough Amplitude (Unadjusted) by Visit. Calculated as b-wave amplitude - PhNR amplitude. Photopic negative response electroretinography (PhNR-ERG) assesses retinal cell function. A decrease in amplitude is associated with worse outcomes.
Time frame: Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit.
Population: All participants for whom PhNR-ERG was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 4 | -2.72 µV | Standard Deviation 24.75 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 8 | 0.08 µV | Standard Deviation 18.496 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 12 | 15.16 µV | Standard Deviation 38.016 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 16 | 1.70 µV | Standard Deviation 32.016 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 20 | -2.62 µV | Standard Deviation 20.398 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 24 | -4.57 µV | Standard Deviation 25.171 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 28 | 6.38 µV | Standard Deviation 26.095 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 32 | -1.42 µV | Standard Deviation 28.294 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 36 | 10.94 µV | Standard Deviation 48.051 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 40 | 2.22 µV | Standard Deviation 43.149 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 44 | 11.74 µV | Standard Deviation 46.933 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 48 | -4.12 µV | Standard Deviation 34.581 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 52 | 2.73 µV | Standard Deviation 19.124 |
| Double Masked Group Vehicle Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 56 (Follow up) | -16.78 µV | Standard Deviation 19.472 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 44 | -3.46 µV | Standard Deviation 26.042 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 4 | -4.80 µV | Standard Deviation 31.025 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 32 | 4.86 µV | Standard Deviation 26.75 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 8 | -0.08 µV | Standard Deviation 31.421 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 52 | -2.15 µV | Standard Deviation 22.109 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 12 | 15.38 µV | Standard Deviation 33.898 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 36 | 5.57 µV | Standard Deviation 26.427 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 16 | 0.52 µV | Standard Deviation 25.629 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 48 | -6.86 µV | Standard Deviation 28.849 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 20 | -3.29 µV | Standard Deviation 21.498 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 40 | -3.74 µV | Standard Deviation 27.206 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 24 | -0.54 µV | Standard Deviation 30.47 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 56 (Follow up) | -11.80 µV | Standard Deviation 49.865 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: PhNR Peak to Trough Amplitude (Unadjusted) From Baseline | Week 28 | -10.18 µV | Standard Deviation 19.504 |
Secondary
Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude
Change from baseline in photopic negative response electroretinography (PhNR-ERG) a-wave amplitude by Visit. Photopic negative response electroretinography (PhNR-ERG) assesses retinal cell function. A decrease in the amplitude of the a-wave is associated with worse outcomes. Change from baseline in a-wave amplitude: a more positive number equals a decrease in amplitude, and therefore a worse outcome, a more negative number mean a better outcome.
Time frame: Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit.
Population: All participants for whom photopic negative response electroretinography PhNR-ERG was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 4 | 0.0 µV | Standard Deviation 6.61 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 8 | -0.2 µV | Standard Deviation 6.92 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 12 | -3.5 µV | Standard Deviation 11.09 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 16 | 1.1 µV | Standard Deviation 6.71 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 20 | 1.5 µV | Standard Deviation 4.64 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 24 | 2.0 µV | Standard Deviation 6.18 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 28 | 0.4 µV | Standard Deviation 5.11 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 32 | 2.0 µV | Standard Deviation 8.18 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 36 | -2.5 µV | Standard Deviation 11.62 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 40 | 2.2 µV | Standard Deviation 10.07 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 44 | 1.0 µV | Standard Deviation 11.05 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 48 | 3.4 µV | Standard Deviation 8.31 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 52 | 2.3 µV | Standard Deviation 5.58 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 56 (Follow up Visit) | 7.0 µV | Standard Deviation 2.93 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 44 | 3.2 µV | Standard Deviation 6.55 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 4 | 1.3 µV | Standard Deviation 6.81 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 32 | 0.3 µV | Standard Deviation 5.64 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 8 | 0.5 µV | Standard Deviation 7.5 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 52 | 2.0 µV | Standard Deviation 5.16 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 12 | -3.2 µV | Standard Deviation 10.18 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 36 | 0.2 µV | Standard Deviation 7 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 16 | 0.7 µV | Standard Deviation 6.3 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 48 | 2.9 µV | Standard Deviation 6.19 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 20 | 1.1 µV | Standard Deviation 4.71 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 40 | 1.5 µV | Standard Deviation 6.28 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 24 | 2.0 µV | Standard Deviation 6.56 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 56 (Follow up Visit) | 5.1 µV | Standard Deviation 8.71 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 28 | 2.5 µV | Standard Deviation 5.8 |
Secondary
Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude
Change from baseline in photopic negative response electroretinography (PhNR-ERG) b-wave amplitude by visit. Photopic negative response electroretinography (PhNR -ERG) assesses retinal cell function. A decrease in the amplitude of the b-wave is associated with worse outcomes. Change from baseline in b-wave amplitude: a more negative number equals a decrease in amplitude, and therefore a worse outcome, a more positive number mean a better outcome.
Time frame: Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit.
Population: All participants for whom PhNR-ERG was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 32 | -5.6 µV | Standard Deviation 13.57 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 4 | -9.1 µV | Standard Deviation 14.36 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 36 | 2.4 µV | Standard Deviation 29.87 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 12 | 3.5 µV | Standard Deviation 17.85 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 40 | -0.1 µV | Standard Deviation 23.58 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 8 | -4.6 µV | Standard Deviation 21.82 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 44 | 1.9 µV | Standard Deviation 21.35 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 28 | -0.7 µV | Standard Deviation 19.04 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 48 | -6.9 µV | Standard Deviation 24.08 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 16 | -2.8 µV | Standard Deviation 28.02 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 52 | -1.3 µV | Standard Deviation 13.97 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 24 | -8.9 µV | Standard Deviation 20.58 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 56 (Follow up) | -18.4 µV | Standard Deviation 28.05 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 20 | -2.2 µV | Standard Deviation 22.48 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 56 (Follow up) | -17.5 µV | Standard Deviation 49.57 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 24 | -1.0 µV | Standard Deviation 28.06 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 28 | -11.6 µV | Standard Deviation 17.75 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 4 | -4.5 µV | Standard Deviation 31.48 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 12 | 14.6 µV | Standard Deviation 27.23 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 16 | -2.6 µV | Standard Deviation 19.83 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 20 | -6.4 µV | Standard Deviation 29.35 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 32 | 1.8 µV | Standard Deviation 24.58 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 36 | 2.2 µV | Standard Deviation 22.06 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 40 | -8.1 µV | Standard Deviation 23.17 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 44 | -5.0 µV | Standard Deviation 21.01 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 48 | -10.5 µV | Standard Deviation 23.8 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 52 | -5.4 µV | Standard Deviation 18.99 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude | Week 8 | -3.4 µV | Standard Deviation 22.33 |
Secondary
Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III
Change from Baseline in visual field Mean Deviation(MD) as measured by Humphrey automated visual field testing stimulus III by visit. The Humphrey visual field test measures the entire area of peripheral vision that can be seen while the eye is focused on a central point. Mean deviation (MD) is the mean deviation in the patient's results compared to those expected from the age-matched normative database. Lower/More negative scores mean worse outcome, higher/more positive score means better outcome.
Time frame: Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit.
Population: All participants for whom Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III was measured
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 32 | 2.9 dB | Standard Deviation 4.27 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 36 | 2.6 dB | Standard Deviation 4.75 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 48 | 1.5 dB | Standard Deviation 3.66 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 4 | 1.1 dB | Standard Deviation 3.62 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 8 | 0.5 dB | Standard Deviation 3.88 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 12 | 0.4 dB | Standard Deviation 3.85 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 16 | 0.3 dB | Standard Deviation 1.87 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 20 | -0.4 dB | Standard Deviation 5.31 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 24 | 2.0 dB | Standard Deviation 3.33 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 28 | 1.7 dB | Standard Deviation 4.4 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 40 | 2.8 dB | Standard Deviation 5.98 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 44 | 1.8 dB | Standard Deviation 6.03 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 52 | 0.9 dB | Standard Deviation 4.18 |
| Double Masked Group Vehicle Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 56 (Follow up) | 3.7 dB | Standard Deviation 5.67 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 56 (Follow up) | 1.6 dB | Standard Deviation 3.53 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 32 | 1.4 dB | Standard Deviation 4.38 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 20 | 2.1 dB | Standard Deviation 4.23 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 44 | 2.8 dB | Standard Deviation 5.1 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 40 | 2.2 dB | Standard Deviation 3.55 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 48 | 1.4 dB | Standard Deviation 3.92 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 24 | 1.1 dB | Standard Deviation 2.74 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 4 | 2.1 dB | Standard Deviation 3.94 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 52 | 2.2 dB | Standard Deviation 3.3 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 8 | 1.6 dB | Standard Deviation 3.82 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 28 | 1.3 dB | Standard Deviation 4.86 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 12 | 0.9 dB | Standard Deviation 3.42 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 36 | 2.2 dB | Standard Deviation 3.55 |
| Double Masked Group Elamipretide Eyes | Double Masked Period: Visual Field Mean Deviation as Measured by Humphrey Automated Visual Field Testing Stimulus III | Week 16 | 1.9 dB | Standard Deviation 2.62 |
Secondary
Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye
Incidence of Change from normal or Abnormal, clinically insignificant to normal clinically significant
Time frame: Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit)
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Conjunctiva: Week 44 Normal to Abnormal Clinically significant | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Conjunctiva Week 4 Normal to Abnormal Clinically significant | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Cornea: Week 32 Normal to Abnormal Clinically significant | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Conjunctiva: Week 8 Normal to Abnormal Clinically significant | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Lids: Week 28 Normal to Abnormal Clinically significant | 0 eyes |
| Double Masked Group Vehicle Eyes | Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Conjunctiva: Week 16 Normal to Abnormal Clinically significant | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Cornea: Week 44 Normal to Abnormal Clinically significant | 1 eyes |
| Double Masked Group Vehicle Eyes | Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Conjunctiva: Week 28 Normal to Abnormal Clinically significant | 1 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Cornea: Week 44 Normal to Abnormal Clinically significant | 1 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Conjunctiva: Week 44 Normal to Abnormal Clinically significant | 1 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Lids: Week 28 Normal to Abnormal Clinically significant | 1 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Cornea: Week 32 Normal to Abnormal Clinically significant | 2 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Conjunctiva: Week 28 Normal to Abnormal Clinically significant | 1 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Conjunctiva Week 4 Normal to Abnormal Clinically significant | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Conjunctiva: Week 8 Normal to Abnormal Clinically significant | 0 eyes |
| Double Masked Group Elamipretide Eyes | Double Masked: Slit Lamp Changes From Baseline: Vehicle Eye and Elamipretide Eye | Conjunctiva: Week 16 Normal to Abnormal Clinically significant | 1 eyes |
Secondary
Double Masked: VFQ-39 Color Vision Score
Change from baseline in Visual Function Questionnaire (VFQ-39) Color Vision score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit)
Population: All participants for whom VFQ-39 Color Vision score was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Color Vision Score | Week 52 | 6.3 score on a scale | Standard Deviation 17.68 |
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Color Vision Score | Week 56 (Follow-up Visit) | 0.0 score on a scale | Standard Deviation 25 |
| Double Masked Group Elamipretide Eyes | Double Masked: VFQ-39 Color Vision Score | Week 52 | 6.3 score on a scale | Standard Deviation 12.5 |
Secondary
Double Masked: VFQ-39 Composite
Double Masked: Change from baseline in Visual Function Questionnaire (VFQ-39) Composite score by visit. National Eye Institute VFQ-39 measures health-related quality of life in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, peripheral vision, and composite score. For each domain: the lowest and highest possible scores are 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit)
Population: All participants for whom VFQ-39 was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Composite | Week 52 | 6.1 score on a scale | Standard Deviation 5.63 |
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Composite | Week 56 (Follow-up Visit) | 7.8 score on a scale | Standard Deviation 7.02 |
| Double Masked Group Elamipretide Eyes | Double Masked: VFQ-39 Composite | Week 52 | 5.7 score on a scale | Standard Deviation 8.07 |
Secondary
Double Masked: VFQ-39 Dependency Score
Change from baseline in Visual Function Questionnaire (VFQ-39) Dependency score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit)
Population: All participants for whom VFQ-39 Dependency score was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Dependency Score | Week 52 | 9.4 score on a scale | Standard Deviation 22.1 |
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Dependency Score | Week 56 (Follow-up Visit) | -8.3 score on a scale | Standard Deviation 23.66 |
| Double Masked Group Elamipretide Eyes | Double Masked: VFQ-39 Dependency Score | Week 52 | -2.6 score on a scale | Standard Deviation 24.85 |
Secondary
Double Masked: VFQ-39 Distance Activities Score
Change from baseline in Visual Function Questionnaire (VFQ-39) Distance Activities score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit)
Population: All participants for whom VFQ-39 Distance Activities was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Distance Activities Score | Week 52 | 9.4 score on a scale | Standard Deviation 12.94 |
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Distance Activities Score | Week 56 (Follow-up Visit) | 54.2 score on a scale | Standard Deviation 14.43 |
| Double Masked Group Elamipretide Eyes | Double Masked: VFQ-39 Distance Activities Score | Week 52 | 0.0 score on a scale | Standard Deviation 14.03 |
Secondary
Double Masked: VFQ-39 General Health Score
Change from baseline in Visual Function Questionnaire (VFQ-39) General Health score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit)
Population: All participants for whom VFQ-39 General Health score was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 General Health Score | Week 52 | -3.8 score on a scale | Standard Deviation 10.09 |
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 General Health Score | Week 56 (Follow-up Visit) | -5.8 score on a scale | Standard Deviation 6.29 |
| Double Masked Group Elamipretide Eyes | Double Masked: VFQ-39 General Health Score | Week 52 | -0.6 score on a scale | Standard Deviation 19.72 |
Secondary
Double Masked: VFQ-39 General Vision Score
Change from baseline in VFQ-39 General Vision Score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculate
Time frame: Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit)
Population: All participants for whom change from VFQ-39 General Health score was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 General Vision Score | Week 52 | 8.1 score on a scale | Standard Deviation 8.84 |
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 General Vision Score | Week 56 (Follow-up Visit) | 3.3 score on a scale | Standard Deviation 7.64 |
| Double Masked Group Elamipretide Eyes | Double Masked: VFQ-39 General Vision Score | Week 52 | 1.3 score on a scale | Standard Deviation 11.09 |
Secondary
Double Masked: VFQ-39 Mental Health Score
Change from baseline in Visual Function Questionnaire (VFQ-39) Mental Health score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit)
Population: All participants for whom VFQ-39 Mental Health was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Mental Health Score | Week 52 | 12.0 score on a scale | Standard Deviation 9.16 |
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Mental Health Score | Week 56 (Follow-up Visit) | 13.3 score on a scale | Standard Deviation 25.17 |
| Double Masked Group Elamipretide Eyes | Double Masked: VFQ-39 Mental Health Score | Week 52 | 19.7 score on a scale | Standard Deviation 32.47 |
Secondary
Double Masked: VFQ-39 Near Activities Score
Change from Baseline in VFQ-39 Near Activities Score from Baseline. National Eye Institute VFQ-39 measures health-related quality of life in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, peripheral vision, and composite score. For each domain: the lowest and highest possible scores are 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit)
Population: All participants for whom change from VFQ-39 Near Activities score was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Near Activities Score | Week 52 | 3.6 score on a scale | Standard Deviation 7.53 |
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Near Activities Score | Week 56 (Follow-up Visit) | 11.1 score on a scale | Standard Deviation 9.62 |
| Double Masked Group Elamipretide Eyes | Double Masked: VFQ-39 Near Activities Score | Week 52 | 9.2 score on a scale | Standard Deviation 9.41 |
Secondary
Double Masked: VFQ-39 Ocular Pain Score
Change from baseline in VFQ-39 Ocular Pain score. National Eye Institute VFQ-39 measures health-related quality of life in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, peripheral vision, and composite score. For each domain: the lowest and highest possible scores are 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit)
Population: All participants for whom change from VFQ-39 General Health score was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Ocular Pain Score | Week 52 | 1.6 score on a scale | Standard Deviation 12.39 |
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Ocular Pain Score | Week 56 (Follow-up Visit) | 12.5 score on a scale | Standard Deviation 12.5 |
| Double Masked Group Elamipretide Eyes | Double Masked: VFQ-39 Ocular Pain Score | Week 52 | 3.1 score on a scale | Standard Deviation 6.25 |
Secondary
Double Masked: VFQ-39 Peripheral Score
Change from baseline in Visual Function Questionnaire (VFQ-39) Peripheral Score . National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit)
Population: All participants for whom VFQ-39 Peripheral score was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Peripheral Score | Week 52 | 0.0 score on a scale | Standard Deviation 13.36 |
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Peripheral Score | Week 56 (Follow-up Visit) | 66.7 score on a scale | Standard Deviation 14.43 |
| Double Masked Group Elamipretide Eyes | Double Masked: VFQ-39 Peripheral Score | Week 52 | 6.3 score on a scale | Standard Deviation 12.5 |
Secondary
Double Masked: VFQ-39 Role Difficulties Score
Change from baseline in Visual Function Questionnaire (VFQ-39) Role Difficulties score National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit)
Population: All participants for whom VFQ-39 Role Difficulties was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Role Difficulties Score | Week 52 | 4.7 score on a scale | Standard Deviation 10.43 |
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Role Difficulties Score | Week 56 (Follow-up Visit) | 10.4 score on a scale | Standard Deviation 13.01 |
| Double Masked Group Elamipretide Eyes | Double Masked: VFQ-39 Role Difficulties Score | Week 52 | 14.1 score on a scale | Standard Deviation 9.38 |
Secondary
Double Masked: VFQ-39 Social Functioning Score.
Change from baseline in Visual Function Questionnaire (VFQ-39) Social Functioning score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 52 (end-of-treatment visit) and Week 56 (follow-up visit)
Population: All participants for whom VFQ-39 Social Functioning was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Social Functioning Score. | Week 52 | 6.3 score on a scale | Standard Deviation 10.68 |
| Double Masked Group Vehicle Eyes | Double Masked: VFQ-39 Social Functioning Score. | Week 56 (Follow-up Visit) | 16.7 score on a scale | Standard Deviation 8.34 |
| Double Masked Group Elamipretide Eyes | Double Masked: VFQ-39 Social Functioning Score. | Week 52 | 0.0 score on a scale | Standard Deviation 19.25 |
Secondary
OLE: Slit Lamp Examination (SLE)-Elamipretide Eye
Open Label Extension: Shift in Slit Lamp Examination from Baseline by Visit: number of eyes that changed from normal or abnormal not clinically significant, to abnormal clinically significant for Elamipretide Eyes
Time frame: Assessed at Baseline, Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit)
Population: All participants for who SLE was measured.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Conjunctiva 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Iris Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Anterior Chamber 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lids Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lens Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Anterior Chamber 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lens Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Conjunctiva 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lens Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Anterior Chamber 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lens Week 116 | 1 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lids Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lens Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Iris Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Conjunctiva 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Vitreous Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Iris Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Vitreous Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lids Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Vitreous Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Iris Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Vitreous Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Conjunctiva 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Retina Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lens Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Retina Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Conjunctiva 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Retina Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lens Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Macula Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Retina Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Cornea Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Retina Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Vitreous Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Retina Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Cornea Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Optic nerve Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Vitreous Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Optic nerve Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Vitreous Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lids Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Optic nerve Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Vitreous Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Optic nerve Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Optic nerve Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Cornea Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Retina Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Optic nerve Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Optic nerve Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Conjunctiva 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Optic nerve Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Retina Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Macula Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Cornea Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Macula Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lids Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Macula Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Cornea Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Macula Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Cornea Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Macula Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Cornea Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Macula Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Conjunctiva 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Choroid Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Cornea Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Choroid Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Anterior Chamber 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Choroid Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lids Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Choroid Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Anterior Chamber 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Choroid Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Anterior Chamber 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Choroid Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Iris Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Choroid Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lens Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Choroid Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Conjunctiva 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Periphery Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Iris Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Periphery Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Anterior Chamber 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Periphery Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Iris Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Periphery Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lids Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Periphery Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Iris Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Periphery Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Anterior Chamber 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Periphery Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Macula Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Periphery 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lids Eye Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Periphery 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lids Eye Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lids Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lids Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lids Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lids Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lids Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lids Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lids Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Conjunctiva 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Conjunctiva 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Conjunctiva 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Conjunctiva 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Conjunctiva 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Conjunctiva 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Conjunctiva 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Conjunctiva 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Cornea Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Cornea Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Cornea Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Cornea Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Anterior Chamber 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Anterior Chamber 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Anterior Chamber 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Anterior Chamber 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Anterior Chamber 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Anterior Chamber 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Iris Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Iris Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Iris Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Iris Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lens Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lens Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Vitreous Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Vitreous Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Vitreous Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Retina Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Retina Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Cornea Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Cornea Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Cornea Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Cornea Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Anterior Chamber 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Anterior Chamber 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Iris Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Iris Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Iris Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Optic nerve Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Iris Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Macula Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lens Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lens Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lens Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lens Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lens Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Lens Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Vitreous Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Vitreous Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Vitreous Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Vitreous Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Vitreous Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Retina Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Retina Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Retina Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Retina Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Retina Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Retina Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Optic nerve Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Macula Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Optic nerve Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Optic nerve Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Optic nerve Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Optic nerve Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Optic nerve Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Optic nerve Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Macula Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Macula Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Macula Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Macula Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Macula Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Macula Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Choroid Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Choroid Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Choroid Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Choroid Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Choroid Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Choroid Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Choroid Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Choroid Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Periphery Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Periphery Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Periphery Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Periphery Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Periphery Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Periphery Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Elamipretide Eye | Periphery Week 148 | 0 eyes |
Secondary
OLE: Slit Lamp Examination (SLE)-Vehicle Eye
Open Label Extension: Shift in Slit Lamp Examination from Baseline by Visit: number of eyes that changed from normal or abnormal not clinically significant, to abnormal clinically significant for Vehicle Eyes
Time frame: Assessed at Baseline, Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit)
Population: All participants for who SLE was measured.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lens Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Retina Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Retina Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Retina Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Periphery Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Periphery Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Periphery Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Periphery Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Retina Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Conjunctiva 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Conjunctiva 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Conjunctiva 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Conjunctiva 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Cornea Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Cornea Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Cornea Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Cornea Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Cornea Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Anterior Chamber 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Anterior Chamber 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Iris Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Iris Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Iris Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Iris Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lens Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lens Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lens Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lens Week 116 | 1 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lens Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lens Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lens Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Vitreous Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Vitreous Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Vitreous Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Retina Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Vitreous Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Vitreous Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Vitreous Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Vitreous Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Vitreous Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Retina Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Retina Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Retina Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Optic nerve Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Optic nerve Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Optic nerve Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Optic nerve Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Optic nerve Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Optic nerve Week132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Optic nerve Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Optic nerve Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Macula Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Macula Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Macula Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Macula Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Macula Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Macula Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Macula Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Macula Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Choroid Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Choroid Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Choroid Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Choroid Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Choroid Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Choroid Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Choroid Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Choroid Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Periphery Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Periphery Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Periphery Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Periphery 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lids Week 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lids Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lids Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lids Week 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Iris Week 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lids Eye Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lids Week 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lids Week 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lids Week 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Conjunctiva 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Conjunctiva 84 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Conjunctiva 100 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Conjunctiva 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Cornea Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Cornea Week 84 | 1 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Cornea Week 100 | 1 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Anterior Chamber 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Anterior Chamber 104 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Anterior Chamber 116 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Anterior Chamber 132 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Anterior Chamber 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Anterior Chamber 160 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Iris Week 68 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Iris Week 148 | 0 eyes |
| Double Masked Group Vehicle Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Iris Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Iris Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Optic nerve Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Retina Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Cornea Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Retina Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Macula Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Anterior Chamber 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Periphery Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Macula Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Periphery Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lids Eye Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Periphery Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Macula Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Periphery Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Vitreous Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Anterior Chamber 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Retina Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Macula Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Conjunctiva 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lids Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Conjunctiva 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Macula Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Conjunctiva 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Retina Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Macula Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Cornea Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lids Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Cornea Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Macula Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Cornea Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Anterior Chamber 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Cornea Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Macula Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Anterior Chamber 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lids Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Anterior Chamber 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Choroid Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Iris Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Iris Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Choroid Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Iris Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Conjunctiva 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Iris Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Choroid Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lens Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Anterior Chamber 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lens Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Choroid Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lens Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Conjunctiva 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lens Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Choroid Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lens Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Iris Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lens Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Choroid Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lens Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Conjunctiva 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lens Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Choroid Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Vitreous Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Anterior Chamber 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Vitreous Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Choroid Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Conjunctiva 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Vitreous Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Periphery Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Vitreous Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Conjunctiva 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Vitreous Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Periphery Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Vitreous Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Iris Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Vitreous Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Periphery Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Retina Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Cornea Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Retina Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Periphery 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Retina Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lids Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Retina Week 160 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Anterior Chamber 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Optic nerve Week 68 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lids Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Optic nerve Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Cornea Week 84 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Optic nerve Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lids Week 132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Optic nerve Week 104 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Iris Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Optic nerve Week 116 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Lids Week 148 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Optic nerve Week132 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Cornea Week 100 | 0 eyes |
| Double Masked Group Elamipretide Eyes | OLE: Slit Lamp Examination (SLE)-Vehicle Eye | Optic nerve Week 148 | 0 eyes |
Secondary
Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio
Open Label Extension Period: Change from baseline by visit PhNR/b Wave Amplitude Ratio by visit. A reduced amplitude ratio equals worse outcome.
Time frame: Assessed at each visit from Baseline from Week 68 to Week 160
Population: All participants for whom PhNR-ERG was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Week 68 | -0.097 Ratio | Standard Deviation 0.2453 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Week 148 | -0.035 Ratio | Standard Deviation 0.0717 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Week 160 | -0.155 Ratio | Standard Deviation 0.1075 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Week 84 | -0.021 Ratio | Standard Deviation 0.2678 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Week 100 | 0.012 Ratio | Standard Deviation 0.1523 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Week 104 | 0.019 Ratio | Standard Deviation 0.0066 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Week 116 | -0.034 Ratio | Standard Deviation 0.2112 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Week 132 | -0.055 Ratio | Standard Deviation 0.1957 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Week 132 | 0.032 Ratio | Standard Deviation 0.201 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Week 68 | -0.002 Ratio | Standard Deviation 0.1722 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Week 100 | -0.003 Ratio | Standard Deviation 0.0796 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Week 148 | -0.046 Ratio | Standard Deviation 0.0982 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Week 116 | 0.000 Ratio | Standard Deviation 0.0971 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Week 160 | -0.212 Ratio | Standard Deviation 0.2211 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Week 104 | -0.048 Ratio | Standard Deviation 0.0667 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR/b Wave Amplitude Ratio | Week 84 | -0.041 Ratio | Standard Deviation 0.1207 |
Secondary
Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude
Open Label Extension Period: Change from baseline by visit in PhNR Peak to Trough Adjusted for a-wave Amplitude by Visit. \[Calculated by b-wave amplitude - PhNR amplitude)/a-wave amplitude.\] Photopic negative response electroretinography (PhNR-ERG) assesses retinal cell function. A decrease in the amplitude of the a-wave is associated with worse outcomes. A reduced amplitude ratio equals worse outcome.
Time frame: Assessed at each visit from Baseline from Week 68 to Week 160
Population: All participants for whom PhNR-ERG was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 148 | -0.91 Ratio | Standard Deviation 0.609 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 104 | -0.10 Ratio | Standard Deviation 0.454 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 160 | -0.46 Ratio | Standard Deviation 0.457 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 68 | -0.31 Ratio | Standard Deviation 1.006 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 84 | 0.25 Ratio | Standard Deviation 1.7 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 100 | -0.30 Ratio | Standard Deviation 0.591 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 116 | -0.64 Ratio | Standard Deviation 1.189 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 132 | -0.77 Ratio | Standard Deviation 0.831 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 116 | -0.46 Ratio | Standard Deviation 0.673 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 84 | -0.09 Ratio | Standard Deviation 1.05 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 104 | -0.09 Ratio | Standard Deviation 0.103 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 148 | -0.16 Ratio | Standard Deviation 0.437 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 100 | -0.13 Ratio | Standard Deviation 0.407 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 160 | -0.92 Ratio | Standard Deviation 1.027 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 132 | -0.38 Ratio | Standard Deviation 0.329 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline by Visit PhNR Peak to Trough Adjusted for A-wave Amplitude | Week 68 | -0.70 Ratio | Standard Deviation 0.859 |
Secondary
Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit
Open Label Extension Period: Change from Baseline in Color Discrimination - Number of Plates from Baseline by Visit by Ishihara Test. Score range: 0-38. Higher number equals better color discrimination equals better outcome. Lower number equals worse color discrimination equals worse outcome.
Time frame: Assessed at each visit from Baseline from Week 68 to Week 160
Population: All participants for whom Color Discrimination - Number of Plates by Visit was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Week 104 | 0.0 plates | Standard Deviation 0 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Week 68 | 1.3 plates | Standard Deviation 1.28 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Week 84 | 1.9 plates | Standard Deviation 1.96 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Week 100 | 2.8 plates | Standard Deviation 3.65 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Week 116 | 2.4 plates | Standard Deviation 2.7 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Week 132 | 2.0 plates | Standard Deviation 1.83 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Week 148 | 1.0 plates | Standard Deviation 0.82 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Week 160 | 2.0 plates | Standard Deviation 2 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Week 160 | 1.3 plates | Standard Deviation 1.53 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Week 116 | 1.2 plates | Standard Deviation 1.92 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Week 68 | 1.4 plates | Standard Deviation 1.36 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Week 148 | 0.9 plates | Standard Deviation 0.35 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Week 84 | 1.6 plates | Standard Deviation 1.75 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Week 132 | 1.6 plates | Standard Deviation 1.33 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Week 100 | 2.0 plates | Standard Deviation 2.54 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Color Discrimination - Number of Plates by Visit | Week 104 | 0.5 plates | Standard Deviation 0.71 |
Secondary
Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit
Letters are arranged on a 60 x 85 cm chart in sets of triplets which contain the same contrast, decreasing in log10 contrast from top to bottom and left to right as participant progresses from one triplet to the other. Each group of three letters is decreased in contrast by a factor 0.71 (1/√2) (log contrast 0.15) of the proceeding set. Participant reads letters, starting with highest contrast, until unable to read letters in a single group. Score is based on the contrast of the last group read correctly. The size of the letters on the chart subtend 0.5 degrees at 3m, The test is scored in LogCS units, where each set of triplets advances in steps of 0.15 log units ranging from LogCS of 0.00 (approx. 100% contrast) to LogCS 2.25 (approx. 0.56% contrast). Change from baseline: the more negative the number means poor outcome, the more positive the number means better outcome.
Time frame: Assessed at each visit from Baseline from Week 68 to Week 160
Population: All participants for whom Contrast Sensitivity was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Week 68 | 0.225 score on a scale | Standard Deviation 0.2121 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Week 100 | 0.281 score on a scale | Standard Deviation 0.2034 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Week 104 | 0.075 score on a scale | Standard Deviation 0.1061 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Week 116 | 0.300 score on a scale | Standard Deviation 0.3 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Week 132 | 0.214 score on a scale | Standard Deviation 0.1909 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Week 84 | 0.300 score on a scale | Standard Deviation 0.1964 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Week 148 | 0.225 score on a scale | Standard Deviation 0.2598 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Week 160 | 0.150 score on a scale | Standard Deviation 0.2598 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Week 160 | 0.100 score on a scale | Standard Deviation 0.0866 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Week 84 | 0.122 score on a scale | Standard Deviation 0.1991 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Week 132 | 0.127 score on a scale | Standard Deviation 0.2796 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Week 100 | 0.118 score on a scale | Standard Deviation 0.2893 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Week 68 | 0.084 score on a scale | Standard Deviation 0.1972 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Week 104 | 0.075 score on a scale | Standard Deviation 0.1061 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Week 148 | 0.113 score on a scale | Standard Deviation 0.1553 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Contrast Sensitivity by Visit | Week 116 | 0.196 score on a scale | Standard Deviation 0.2626 |
Secondary
Open Label Extension Period: Change From Baseline in Retinal Ganglion Cell Layer Thickness
Open Label Extension Period: Change from baseline in Retinal Ganglion Cell Layer Thickness by SD-OCT by SD-OCT by visit
Time frame: Assessed at Baseline, Week 148,
Population: All participants for whom Change from baseline in Retinal Ganglion Cell Layer Thickness by SD-OCT was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Change From Baseline in Retinal Ganglion Cell Layer Thickness | -1.0 µm | — |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Change From Baseline in Retinal Ganglion Cell Layer Thickness | 3.4 µm | Standard Deviation 6.07 |
Secondary
Open Label Extension Period: Dilated Fundus Changes From Baseline: Vehicle Eye and Elamipretide Eyes
Incidence of Change from normal or Abnormal-clinically insignificant, to normal clinically significant
Time frame: Assessed at Baseline, Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit)
Population: All participants for whom dilated fundus was measured.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Dilated Fundus Changes From Baseline: Vehicle Eye and Elamipretide Eyes | 0 eyes |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Dilated Fundus Changes From Baseline: Vehicle Eye and Elamipretide Eyes | 0 eyes |
Secondary
Open Label Extension Period: Intraocular Pressure (IOP)
Change from baseline in Intraocular Pressure (IOP) in mmHg by Visit.
Time frame: Assessed at Baseline, Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit)
Population: All participants for whom intraocular pressure was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Intraocular Pressure (IOP) | Week 68 | -0.4 mmHg | Standard Deviation 1.6 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Intraocular Pressure (IOP) | Week 84 | 1.4 mmHg | Standard Deviation 3.29 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Intraocular Pressure (IOP) | Week 100 | -0.9 mmHg | Standard Deviation 2.03 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Intraocular Pressure (IOP) | Week 104 | 1.5 mmHg | Standard Deviation 2.12 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Intraocular Pressure (IOP) | Week 116 | 0.1 mmHg | Standard Deviation 3.18 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Intraocular Pressure (IOP) | Week 132 | 0.1 mmHg | Standard Deviation 1.35 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Intraocular Pressure (IOP) | Week 148 | 1.3 mmHg | Standard Deviation 2.06 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Intraocular Pressure (IOP) | Week 160 | 0.7 mmHg | Standard Deviation 0.58 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Intraocular Pressure (IOP) | Week 160 | -1.7 mmHg | Standard Deviation 1.53 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Intraocular Pressure (IOP) | Week 68 | 0.8 mmHg | Standard Deviation 2.49 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Intraocular Pressure (IOP) | Week 116 | -0.5 mmHg | Standard Deviation 2.63 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Intraocular Pressure (IOP) | Week 84 | 0.4 mmHg | Standard Deviation 2.37 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Intraocular Pressure (IOP) | Week 148 | -0.1 mmHg | Standard Deviation 2.36 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Intraocular Pressure (IOP) | Week 100 | -0.2 mmHg | Standard Deviation 2.22 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Intraocular Pressure (IOP) | Week 132 | 0.1 mmHg | Standard Deviation 1.8 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Intraocular Pressure (IOP) | Week 104 | -1.0 mmHg | Standard Deviation 1.41 |
Secondary
Open Label Extension Period: PhNR Amplitude
Open Label Extension Period: Change from baseline in PhNR Amplitude by Visit. Photopic negative response electroretinography (PhNR -ERG) assesses retinal cell function. A decrease in PhNR amplitude means worse outcome. Change from baseline: A more negative number means a better outcome, a more positive number means a worse outcome.
Time frame: Assessed at each visit from Baseline from Week 68 to Week 160
Population: All participants for whom PhNR Amplitude was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR Amplitude | Week 160 | -9.9 µV | Standard Deviation 4.31 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR Amplitude | Week 68 | -7.1 µV | Standard Deviation 22.91 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR Amplitude | Week 104 | 3.3 µV | Standard Deviation 1.03 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR Amplitude | Week 100 | 3.3 µV | Standard Deviation 16.25 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR Amplitude | Week 116 | -5.1 µV | Standard Deviation 20.89 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR Amplitude | Week 148 | -4.1 µV | Standard Deviation 8.75 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR Amplitude | Week 132 | -3.6 µV | Standard Deviation 20.98 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR Amplitude | Week 84 | -2.5 µV | Standard Deviation 25.45 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR Amplitude | Week 160 | -10.0 µV | Standard Deviation 11.64 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR Amplitude | Week 84 | -2.2 µV | Standard Deviation 11.96 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR Amplitude | Week 100 | 1.8 µV | Standard Deviation 6.5 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR Amplitude | Week 132 | 0.5 µV | Standard Deviation 10.75 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR Amplitude | Week 148 | -1.4 µV | Standard Deviation 4.23 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR Amplitude | Week 104 | -1.2 µV | Standard Deviation 6.72 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR Amplitude | Week 116 | 0.4 µV | Standard Deviation 9 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR Amplitude | Week 68 | 0.5 µV | Standard Deviation 16.6 |
Secondary
Open Label Extension Period: PhNR-ERG B-wave Amplitude
Change from baseline in photopic negative response electroretinography (PhNR-ERG) b-wave Amplitude by Visit. Photopic negative response electroretinography (PhNR -ERG) assesses retinal cell function. A decrease in the amplitude of the b-wave is associated with worse outcomes. Change from baseline in b-wave amplitude: a more negative number equals a decrease in amplitude, and therefore a worse outcome, a more positive number means a better outcome.
Time frame: Assessed at each visit from Baseline from Week 68 to Week 160
Population: All participants for whom PhNR-ERG was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Week 100 | -28.7 µV | Standard Deviation 23.23 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Week 68 | -15.4 µV | Standard Deviation 25.98 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Week 104 | -17.7 µV | Standard Deviation 5.23 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Week 116 | 17.1 µV | Standard Deviation 44.4 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Week 148 | -13.1 µV | Standard Deviation 30.88 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Week 84 | -6.0 µV | Standard Deviation 23.97 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Week 160 | -17.8 µV | Standard Deviation 14.15 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Week 132 | -17.1 µV | Standard Deviation 17.08 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Week 160 | -38.1 µV | Standard Deviation 25.2 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Week 116 | 4.9 µV | Standard Deviation 18.79 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Week 68 | -7.4 µV | Standard Deviation 17.1 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Week 84 | -13.1 µV | Standard Deviation 20.97 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Week 100 | -19.9 µV | Standard Deviation 18.04 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Week 132 | -1.3 µV | Standard Deviation 18.81 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Week 148 | -7.3 µV | Standard Deviation 18.84 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR-ERG B-wave Amplitude | Week 104 | -14.2 µV | Standard Deviation 4.62 |
Secondary
Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted)
Open Label Extension Period: Change from baseline in PhNR Peak to Trough Amplitude (Unadjusted) by Visit. b-wave amplitude - PhNR amplitude. Photopic negative response electroretinography (PhNR-ERG) assesses retinal cell function. A decrease in the amplitude of the a-wave is associated with worse outcomes.
Time frame: Assessed at each visit from Baseline from Week 68 to Week 160
Population: All participants for whom PhNR-ERG was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Week 104 | -21.01 µV | Standard Deviation 4.193 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Week 148 | -8.99 µV | Standard Deviation 35.662 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Week 116 | 22.26 µV | Standard Deviation 42.352 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Week 100 | -32.02 µV | Standard Deviation 23.994 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Week 132 | -13.56 µV | Standard Deviation 27.012 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Week 68 | -8.36 µV | Standard Deviation 36.178 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Week 160 | -7.87 µV | Standard Deviation 11.415 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Week 84 | -3.50 µV | Standard Deviation 13.206 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Week 160 | -28.17 µV | Standard Deviation 31.249 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Week 84 | -10.90 µV | Standard Deviation 22.928 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Week 100 | -21.64 µV | Standard Deviation 21.432 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Week 148 | -5.84 µV | Standard Deviation 19.242 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Week 68 | -7.92 µV | Standard Deviation 21.402 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Week 104 | -13.01 µV | Standard Deviation 11.34 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Week 116 | 4.52 µV | Standard Deviation 22.231 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: PhNR Peak to Trough Amplitude (Unadjusted) | Week 132 | -1.85 µV | Standard Deviation 15.341 |
Secondary
Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude
Change from baseline in photopic negative response electroretinography (PhNR-ERG) a-wave Amplitude by Visit. Photopic negative response electroretinography (PhNR-ERG) assesses retinal cell function. A decrease in the amplitude of the a-wave is associated with worse outcomes. Change from baseline in a-wave amplitude: a more positive number equals a decrease in amplitude, and therefore a worse outcome, a more negative number mean a better outcome.
Time frame: Assessed at each visit from Baseline from Week 68 to Week 160
Population: All participants for whom photopic negative response electroretinography (PhNR-ERG) was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 104 | 5.3 µV | Standard Deviation 3.27 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 100 | 8.0 µV | Standard Deviation 5.69 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 116 | -2.7 µV | Standard Deviation 9.39 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 68 | 3.2 µV | Standard Deviation 9.24 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 148 | 5.4 µV | Standard Deviation 8.92 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 132 | 5.7 µV | Standard Deviation 7.07 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 160 | 2.9 µV | Standard Deviation 2.82 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 84 | -7.1 µV | Standard Deviation 19.27 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 160 | 2.82 µV | Standard Deviation 7.6 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 100 | 4.9 µV | Standard Deviation 4.41 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 132 | 1.4 µV | Standard Deviation 3.99 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 68 | 4.0 µV | Standard Deviation 6 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 104 | 3.0 µV | Standard Deviation 1.71 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 116 | 0.3 µV | Standard Deviation 5.93 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 148 | 1.5 µV | Standard Deviation 3.84 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude | Week 84 | 1.8 µV | Standard Deviation 6.81 |
Secondary
Open Label Extension Period: Retinal Nerve Fiber Layer Thickness
Open Label Extension Period: Change from baseline in Retinal Nerve Fiber Layer Thickness by SD-OCT by visit. Change from baseline in retinal nerve fiber layer thickness by spectral domain optical coherence tomography (SD-OCT). RNFL measures the loss of retinal ganglion cell axons. RNFL thickness decreases as disease progresses. A positive number, or, absence of change from baseline reflects a good clinical outcome, a negative number reflects loss of thickness, or a bad outcome.
Time frame: Assessed at Baseline, Week 148
Population: All participants for whom Change in Retinal Nerve Fiber Layer Thickness by SD-OCT was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Retinal Nerve Fiber Layer Thickness | -1.0 µm | — |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Retinal Nerve Fiber Layer Thickness | 1.4 µm | Standard Deviation 2.7 |
Secondary
Open Label Extension Period: VFQ-39 Color Vision Score
Change from baseline in Visual Function Questionnaire (VFQ-39) Color Vision score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 160 (End of OLE Period)
Population: All participants for whom Change in Visual Function Questionnaire (VFQ-39) Color Vision Score was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: VFQ-39 Color Vision Score | -7.1 score on a scale | Standard Deviation 18.9 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: VFQ-39 Color Vision Score | 8.3 score on a scale | Standard Deviation 14.43 |
Secondary
Open Label Extension Period: VFQ-39 Composite Score
Change from baseline in Visual Function Questionnaire (VFQ-39) Composite score.National Eye Institute VFQ-39 measures health-related quality of life in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, peripheral vision, and composite score. For each domain: the lowest and highest possible scores are 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 160 (End of OLE Period)
Population: All participants for whom change in VFQ-39 composite score was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: VFQ-39 Composite Score | 5.6 score on a scale | Standard Deviation 11.3 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: VFQ-39 Composite Score | 13.9 score on a scale | Standard Deviation 8.66 |
Secondary
Open Label Extension Period:VFQ-39 Dependency Score
Change from baseline in Visual Function Questionnaire (VFQ-39 ) Dependency Score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 160 (End of OLE Period)
Population: All participants for whom Change in Visual Function Questionnaire (VFQ-39) Dependency Score was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period:VFQ-39 Dependency Score | 16.1 score on a scale | Standard Deviation 13.91 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period:VFQ-39 Dependency Score | 27.1 score on a scale | Standard Deviation 3.61 |
Secondary
Open Label Extension Period: VFQ-39 Distance Activities Score
Change in Visual Function Questionnaire (VFQ-39) Distance Activities Score.National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 160 (End of OLE Period)
Population: All participants for whom Change in Visual Function Questionnaire (VFQ-39) Distance Activities score was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: VFQ-39 Distance Activities Score | 12.5 score on a scale | Standard Deviation 20.56 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: VFQ-39 Distance Activities Score | 13.9 score on a scale | Standard Deviation 6.36 |
Secondary
Open Label Extension Period: VFQ-39 General Health Score
Change from baseline in Visual Function Questionnaire (VFQ-39) General Health score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 160 (End of OLE Period)
Population: All participants for whom Change from baseline in Visual Function Questionnaire (VFQ-39) General Health score was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: VFQ-39 General Health Score | -6.8 score on a scale | Standard Deviation 10.58 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: VFQ-39 General Health Score | 6.7 score on a scale | Standard Deviation 11.55 |
Secondary
Open Label Extension Period: VFQ-39 General Vision Score
Change from baseline in VFQ-39 General Vision score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculate
Time frame: Assessed at Baseline, Week 160 (End of OLE Period)
Population: All participants for whom Change in Visual Function Questionnaire (VFQ-39) General Vision was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: VFQ-39 General Vision Score | 9.3 score on a scale | Standard Deviation 13.36 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: VFQ-39 General Vision Score | 6.7 score on a scale | Standard Deviation 11.55 |
Secondary
Open Label Extension Period: VFQ-39 Mental Health Score
Change from baseline in Visual Function Questionnaire (VFQ-39) Mental Health Score . National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 160 (End of OLE Period)
Population: All participants for whom Change in Visual Function Questionnaire (VFQ-39) Mental Health score was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: VFQ-39 Mental Health Score | 16.4 score on a scale | Standard Deviation 22.31 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: VFQ-39 Mental Health Score | 29.2 score on a scale | Standard Deviation 29.83 |
Secondary
Open Label Extension Period: VFQ-39 Near Activities
Change from Baseline in Visual Function Questionnaire (VFQ-39) Near Activities score.. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; score ranges from 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 160 (End of OLE Period)
Population: All participants for whom change in VFQ-39 Near Activities score was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: VFQ-39 Near Activities | 8.5 score on a scale | Standard Deviation 11.03 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: VFQ-39 Near Activities | 16.7 score on a scale | Standard Deviation 16.67 |
Secondary
Open Label Extension Period: VFQ-39 Ocular Pain Score
Change from baseline in VFQ-39 Ocular Pain score by visit. National Eye Institute VFQ-39 measures health-related quality of life in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, peripheral vision, and composite score. For each domain: the lowest and highest possible scores are 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 160 (End of OLE Period)
Population: All participants for whom Change in Visual Function Questionnaire (VFQ-39) Ocular Pain was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: VFQ-39 Ocular Pain Score | -16.1 score on a scale | Standard Deviation 20.04 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: VFQ-39 Ocular Pain Score | -4.2 score on a scale | Standard Deviation 7.22 |
Secondary
Open Label Extension Period: VFQ-39 Role Difficulties Score
Change from baseline in Visual Function Questionnaire (VFQ-39) Role Difficulties Score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 160 (End of OLE Period)
Population: All participants for whom Change in Visual Function Questionnaire (VFQ-39) Role Difficulties score was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: VFQ-39 Role Difficulties Score | 8.0 score on a scale | Standard Deviation 12.87 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: VFQ-39 Role Difficulties Score | 37.5 score on a scale | Standard Deviation 16.54 |
Secondary
Open Label Extension Period: VFQ-39 Social Functioning Score
Change from baseline in Visual Function Questionnaire (VFQ-39) Social Functioning Score. National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 160 (End of OLE Period)
Population: All participants for whom Change in Visual Function Questionnaire (VFQ-39) Social Functioning score was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: VFQ-39 Social Functioning Score | 8.3 score on a scale | Standard Deviation 11.78 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: VFQ-39 Social Functioning Score | 5.6 score on a scale | Standard Deviation 17.35 |
Secondary
Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline
Change from Baseline in visual field Mean Deviation(MD) as measured by Humphrey automated visual field testing stimulus III by visit. The Humphrey visual field test measures the entire area of peripheral vision that can be seen while the eye is focused on a central point. Mean deviation (MD) is the mean deviation in the patient's results compared to those expected from the age-matched normative database. Lower/More negative scores mean worse outcome, higher/more positive score means better outcome.
Time frame: Assessed at each visit from Baseline from Week 68 to Week 160
Population: All participants for whom Visual Field Mean Deviation (dB) measured by Humphrey from Baseline was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Week 68 | 3.3 dB | Standard Deviation 5.59 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Week 84 | 5.1 dB | Standard Deviation 6.66 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Week 100 | 3.1 dB | Standard Deviation 6.85 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Week 104 | 0.9 dB | Standard Deviation 0.97 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Week 116 | 4.4 dB | Standard Deviation 5.48 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Week 132 | 4.7 dB | Standard Deviation 8.43 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Week 148 | 5.5 dB | Standard Deviation 8.16 |
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Week 160 | 6.3 dB | Standard Deviation 13.67 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Week 160 | 11.5 dB | Standard Deviation 16.98 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Week 68 | 4.0 dB | Standard Deviation 6.66 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Week 116 | 4.7 dB | Standard Deviation 6.21 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Week 84 | 4.4 dB | Standard Deviation 6.53 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Week 148 | 5.6 dB | Standard Deviation 9.51 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Week 100 | 5.5 dB | Standard Deviation 8.04 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Week 132 | 4.0 dB | Standard Deviation 6.62 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Visual Field Mean Deviation (dB) Measured by Humphrey From Baseline | Week 104 | 2.9 dB | Standard Deviation 0.99 |
Secondary
Open Label Extension Period: Visual Function Questionnaire (VFQ-39) Peripheral Score
Change from baseline in Visual Function Questionnaire (VFQ-39) Peripheral Score . National Eye Institute VFQ-39 score measures health-related quality of life of subjects with visual impairment, in 12 domains: general vision, ocular pain, near activities, distance activities, vision-specific social functioning, vision-specific mental health, vision-specific role difficulties, vision-specific dependency, driving, color vision, and peripheral vision and 1 composite score. Each domain is converted to a 0 to 100 scale; the lowest and highest possible scores are set at 0 and 100 points, respectively. Higher score means higher functioning. Scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score. For the composite score, the vision-targeted sub-scale scores are averaged, excluding the general health questions. Domain scores from each cohort are averaged and the change from baseline per domain is calculated.
Time frame: Assessed at Baseline, Week 160 (End of OLE Period)
Population: All participants for whom Change in Visual Function Questionnaire (VFQ-39) Peripheral Score was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Double Masked Group Vehicle Eyes | Open Label Extension Period: Visual Function Questionnaire (VFQ-39) Peripheral Score | 0.0 score on a scale | Standard Deviation 32.27 |
| Double Masked Group Elamipretide Eyes | Open Label Extension Period: Visual Function Questionnaire (VFQ-39) Peripheral Score | 0.0 score on a scale | Standard Deviation 25 |