Age-Related Macular Degeneration
Conditions
Brief summary
This is an open-label, Phase 1 single-center study in approximately 40 subjects who have 1 eye with intermediate AMD, including a high-risk drusen without geographic atrophy (GA) subgroup and a noncentral GA subgroup. Eligible subjects will receive 40 mg of elamipretide administered as a once daily 1.0 mL subcutaneous injection for 12 weeks.
Interventions
DRUGElamipretide
40 mg dose of elamipretide administered once daily as a 1.0mL SC injection.
Sponsors
Stealth BioTherapeutics Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Both arms received 40 mg dose of elamipretide administered once daily as a 1.0mL SC injection.
Eligibility
Sex/Gender
ALL
Age
55 Years to No maximum
Healthy volunteers
No
Inclusion criteria
For this study, only 1 eye of an eligible subject will be included and designated as the study eye. However, all specified ophthalmic testing will be performed on both eyes at each time point. A potential subject must meet the following criteria to be eligible for inclusion in the study: Intermediate AMD - noncentral GA disease group: 1. Adults ≥ 55 years of age with 1 eye with intermediate AMD - noncentral GA. 2. No evidence of choroidal neovascularization (active or prior history) in the study eye. 3. Geographic atrophy may be multifocal, but the cumulative GA lesion size must be: 1. ≥ 1.27 mm2 (approximately ≥ 0.5 DA) and ≤ 10.16 mm2 (approximately ≤ 4 DA). 2. Must reside completely within the FAF imaging field (field 2 to 30-degree image centered on the fovea). 4. Presence of measurable hyperautofluorescence adjacent to the discrete foci of GA. OR Intermediate AMD - high-risk drusen without GA disease group: 5. ≥ 55 years of age with one eye with intermediate AMD - high-risk drusen without GA. 6. High-risk drusen is defined as presence of either at least 1 large (≥ 125 µm) druse or multiple medium-size (between 63 and 124 µm) drusen. General (both disease groups): 7. Able to provide informed consent and willing to comply with all study visits and examinations. 8. Women of childbearing potential who are not pregnant or nursing and have a negative serum pregnancy test at screening. 9. Best-corrected visual acuity assessed by ETDRS letters ≥ 55 letters (Snellen equivalent ≥ 20/70). 10. Low-luminance visual acuity deficit (defined as difference between BCVA and LL visual acuity) \> 5 letters. 11. Has at least two Low-Luminance Questionnaire sub scale results, in which one of the abnormal subscales is either general dim light vision or dim light reading. 12. The fellow eye may have intermediate AMD without noncentral GA (i.e., high-risk drusen), intermediate AMD with noncentral GA, NV AMD, or central GA. Ongoing treatment with antiangiogenic therapies in the fellow eye is allowable. 13. No evidence of visually significant cataract OR pseudophakia without evidence of posterior capsular opacity. 14. Sufficiently clear ocular media, adequate pupillary dilation, fixation to permit quality fundus imaging, and able to cooperate sufficiently for adequate ophthalmic visual function testing and anatomic assessment. 15. Able to administer SC study drug solution as demonstrated at screening or able to have a care provider or appropriate designee who can administer the study drug (i.e., a capable family member or home health nursing aide). 16. If of childbearing potential or in a relationship with a partner of childbearing potential, are able to abstain from sex or use acceptable contraception during the study and for 3 months after dosing. 1. For men: Abstinence is only acceptable if it is in line with the preferred and usual lifestyle of the subject. The subject also agrees to use an acceptable method of contraception should they become sexually active. Subjects must use a condom with spermicide from the date of informed consent until at least 3 months after the last dose of study drug. Periodic abstinence (e.g.calendar, ovulation, symptothermal, or postovulation methods) and withdrawal are not acceptable methods of contraception. 2. For women: abstinence is only acceptable when it is in line with the preferred and usual lifestyle of the subject. The subject agrees to use an acceptable method of contraception should they become sexually active. Maintenance of a monogamous relationship with a male partner who has been surgically sterilized by vasectomy (the vasectomy procedure must have been conducted at least 60 days before the Screening visit or confirmed via sperm analysis), barrier method (e.g., condom or occlusive cap) with spermicidal foam/gel/film/cream AND either hormonal contraception (oral, implanted, or injectable) or an intrauterine device or system are acceptable methods. 17. Ability and willingness to undertake all scheduled visits and assessments.
Exclusion criteria
A subject with study eye who meets any of the following criteria will be excluded from the study: Ocular conditions - study eye 1. Age-related macular degeneration with any evidence of central GA (i.e., involving the fovea). 2. Atrophic retinal disease because of causes other than AMD. 3. Presence or diagnosis of exudative AMD or choroidal neovascularization in the study eye. 4. History of diabetic retinopathy (a history of diabetes mellitus without retinopathy is not a criterion for exclusion). 5. Presence of vitreous hemorrhage. 6. History of retinal detachment or macular hole (stage 3 or 4) in the study eye. 7. Presence of macular pucker. 8. History of uncontrolled glaucoma, defined as advanced cup-to-disc ratio \> 0.7 and IOP \> 25, with or without topical antihypertensive eye drops; treatment of ocular hypertension or controlled glaucoma are not criteria for exclusion. 9. History of advanced guttae indicative of Fuchs endothelial dystrophy. 10. Presence of visually significant cataract OR presence of significant posterior capsular opacity in the setting of Pseudophakia. 11. Presence of significant keratopathy that would cause scattering of light or alter visual function, especially in LL conditions. 12. Ocular incisional surgery (including cataract surgery) in the study eye within 3 months (i.e. 90 days) before Day 1. 13. Aphakia. 14. History of vitrectomy surgery, submacular surgery, or any vitreoretinal surgery. 15. Prior treatment with Visudyne ® (verteporfin), external-beam radiation therapy (for intraocular conditions), or transpupillary thermotherapy. 16. History of prophylactic subthreshold laser treatment for retinal disease. 17. Previous intravitreal drug delivery (e.g., intravitreal corticosteroid injection, anti-angiogenic drugs, or device implantation) in the study eye. Ocular conditions - either eye 18. Active uveitis and/or vitritis (grade trace or above) in either eye. 19. History of idiopathic or autoimmune-associated uveitis in either eye. 20. Active, infectious conjunctivitis, keratitis, scleritis, or endophthalmitis in either eye. Systemic conditions 21. Known to be immunocompromised or receiving systemic immunosuppression. 22. 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. 23. Estimated glomerular filtration rate \< 30 mL/minute, by MDRD. 24. Presence or history of clinically significant allergy disease requiring treatment, as judged by the Investigator. Hay fever is allowed unless it is active. General 25. Participation in other investigational drug or device clinical trials within 30 days before enrollment, or planning to participate in any other investigational drug or device clinical trials within 30 days of study completion. 26. History of allergy to fluorescein that is not amenable to treatment. 27. Inability to comply with study or follow-up procedures. 28. Inability to obtain color fundus photograph, FAF, and fluorescein angiography of sufficient quality to be analyzed and interpreted. 29. History of allergic reaction to the investigational drug or any of its components. 30. Current use of or likely need for any excluded medication.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Day 0 (Baseline to Day 7, and to Weeks 4, 8, 12, 16, 20, 24, and 28. | Change from Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) at 4 meters, letters from Baseline to Day 7, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Mean Dark Adaptometry | Baseline (Day 0) and Week 24 | Change from Baseline in Mean Dark Adaptometry from Baseline to Week 24- at 0% 25%, 50%, 75% Bleach Level. Dark adaptometry, used to evaluate night blindness by measuring the absolute thresholds of rod sensitivity, was performed within 14 days before Day 0 and at all in-person visits except for Day 7. Dark adaptation is the delayed recovery of light sensitivity in darkness following prior light exposure (photobleaching). The observed and change from baseline recovery scores (in minutes) for each bleach level (0%, 25%, 50%, and 75%) were summarized descriptively for each eye at each visit and were presented in a listing. Shorter recovery times are better than longer recovery times. |
| Mean Treatment Compliance (%) of Administration of Subcutaneous Elamipretide | Baseline through Week 28 | Mean percentage of treatment compliance of administration of subcutaneous elamipretide. The number of injections (diary) and vials used was used to compute % compliance over the duration of the subject's participation in the trial. Percentage can range from 0-100%, where 0% means the participant followed the correct dosing 0% of the time, and 100% compliance means the participant followed the correct dosing 100% of the time, with a higher % meaning a better outcome. |
| Mean Number of Home Health Visits to Administer Elamipretide | Baseline (Day 0) through Week 24 | Mean Number of Home Health Visits Necessary for Participant or Caregiver to Learn How to Administer Elamipretide |
| Change From Baseline in Mean Area of Geographic Atrophy by Fundus Autofluorescence | Baseline (Day 0) to Week 24 | Change from Baseline in Mean Area of Geographic Atrophy (GA) by Fundus Autofluorescence (FAF) at Week 24. Fluorescein angiography (FA) was used to examine the circulation of the retina and choroid using fluorescein dye and a specialized camera to trace the dye. Fundus autofluorescence imaging of the retinal pigment epithelium and neurosensory retina was performed within 14 days of Day 0 and at every visit with the exception of Day 0 and Day 7. Atrophy is characterized by loss of the retinal pigment epithelium (RPE), overlying photoreceptors and underlying choriocapillaris. Greater area affected means a worse outcome than smaller area affected. |
| Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT) | Baseline to Week 24 | Change from Baseline in Mean Area of Geographic Atrophy by Sector as measured by Spectral-Domain Optical Coherence Tomography (SD-OCT) at Week 24 |
| Change From Baseline in Mean Reading Acuity Test: With Standard Luminance | Baseline and Weeks 4, 8, 12, 16 20, 24 and 28 | Change from Baseline in Mean Reading Acuity Test: Mean Critical Print Size with Standard Luminance in Weeks 4, 8, 12, 16, 20, 24 and 28 as measured by the Logarithm of the Minimum Angle of Resolution LogMAR chart. Scores range from -0.3 to 1.0, where higher number means worse acuity/worse outcome, a lower number means better acuity/better outcome. |
| Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Baseline to Day 7, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28. | Change from Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) from Baseline (Day 0) to Day 7, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28. |
| Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Assessed at baseline (Day 0), Week 12, Week 24 and Week 28 | Change from baseline (Day 0) at Week 12, 24 and 28. National Eye Institute Visual Function Questionnaire-39 (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. |
| Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Baseline (Day 0), Week 12, and Week 24 and Week 28 | Change from baseline (Day 0) at Week 12, Week 24, and Week 28 in LLQ score. The LLQ is a 32-item questionnaire with six subscales related to low luminance settings: driving, mobility, extreme lighting, general dim lighting, and peripheral vision. Each question is scored on a scale ranging from 0, or maximal difficulty, to 100, or no difficulty in low luminance settings. Higher scores mean better functioning. The questions are assigned to different subscales and are averaged to generate one score per subscale. After weighting each subscale for the number of questions, the weighted subscales are averaged to generate a composite LLQ score. |
| Change From Baseline in Mean Mesopic Light Sensitivity | Baseline (Day 0) and Weeks 4,8,12,16,20,and 24 | Change from baseline in mean mesopic light sensitivity for Weeks, 4, 8, 12, 16, 20, and 24 as assessed by microperimetry for number of loci \<25dB and \<14dB. Mesopic microperimetry, used to measure retinal sensitivity, was performed within 14 days before Day 0 and at all in-person visits except for Day 7. |
| Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Baseline (Day 0) and Week 24 | Change from baseline in mean retinal pigment epithelium - drusen complex (RPEDC) thickness as Measured by SD-OCT by sector at Week 24 |
| Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Baseline (Day 0) to Week 24 | Change from baseline in mean Retinal Pigment Epithelium-Drusen Complex volume at Week 24 by sector |
| Change From Baseline in Mean Reading Acuity Test: Low Luminance | Assessed at screening, baseline, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28 | Change from Baseline in Mean Reading Acuity Test: Mean Critical Print Size with Low Luminance in Weeks 4, 8, 12, 16, 20, 24 and 28 as measured by the Logarithm of the Minimum Angle of Resolution LogMAR chart. Scores range from -0.3 to 1.0, where higher number means worse acuity/worse outcome, a lower number means better acuity/better outcome. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| High-Risk Drusen (HRD) The HRD cohort was defined as the presence of either at least 1 large (≥125 μm) druse or multiple medium-size (63-124 μm) drusen in one eye. Subjects had to have 1 eye with intermediate AMD (high-risk drusen \[HRD\] without geographic atrophy \[GA\]). | 21 |
| NCGA (Noncentral GA) The NCGA cohort was defined as evidence of GA with cumulative area ≥1.27 mm2 (approximately 0.5 disc area) by fundus autofluorescence (FAF) that spared the fovea (defined as retinal pigment epithelium and outer retina intact by spectral-domain optical coherence tomography \[SD-OCT\]). Subjects had to have 1 eye with intermediate AMD with noncentral GA \[NCGA\] | 19 |
| Total | 40 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 1 | 2 |
| Overall Study | Lack of Efficacy | 0 | 1 |
| Overall Study | Lost to follow up | 1 | 0 |
| Overall Study | Withdrawal by Subject | 1 | 1 |
Baseline characteristics
| Characteristic | NCGA (Noncentral GA) | Total | High-Risk Drusen (HRD) |
|---|---|---|---|
| Age, Continuous | 76.0 years STANDARD_DEVIATION 8.22 | 73.3 years STANDARD_DEVIATION 8.67 | 70.9 years STANDARD_DEVIATION 8.54 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 2 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 18 Participants | 38 Participants | 20 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 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 | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 19 Participants | 40 Participants | 21 Participants |
| Sex: Female, Male Female | 11 Participants | 24 Participants | 13 Participants |
| Sex: Female, Male Male | 8 Participants | 16 Participants | 8 Participants |
| Smoking Status Current smoker | 0 Participants | 0 Participants | 0 Participants |
| Smoking Status Former smoker | 11 Participants | 19 Participants | 8 Participants |
| Smoking Status Never smoker | 8 Participants | 21 Participants | 13 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 21 | 0 / 19 |
| other Total, other adverse events | 21 / 21 | 19 / 19 |
| serious Total, serious adverse events | 1 / 21 | 1 / 19 |
Outcome results
Primary
Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA)
Change from Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) at 4 meters, letters from Baseline to Day 7, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28.
Time frame: Day 0 (Baseline to Day 7, and to Weeks 4, 8, 12, 16, 20, 24, and 28.
Population: All participants for whom Standard Luminance BCVA was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Day 7 | 0.810 letters | Standard Deviation 4.8746 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Week 28 | 1.333 letters | Standard Deviation 10.4881 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Week 4 | 2.762 letters | Standard Deviation 5.5759 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Week 8 | 1.950 letters | Standard Deviation 4.4066 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Week 12 | 1.150 letters | Standard Deviation 5.5845 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Week 16 | 2.789 letters | Standard Deviation 5.8933 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Week 20 | 3.842 letters | Standard Deviation 4.1401 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Week 24 | 3.579 letters | Standard Deviation 6.3972 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Week 8 | 1.556 letters | Standard Deviation 6.2987 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Week 4 | 1.833 letters | Standard Deviation 5.0904 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Week 24 | 4.600 letters | Standard Deviation 5.0681 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Week 16 | 2.813 letters | Standard Deviation 5.5883 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Week 28 | 3.667 letters | Standard Deviation 5.8023 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Day 7 | 0.526 letters | Standard Deviation 4.1281 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Week 12 | 1.438 letters | Standard Deviation 5.4156 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) | Week 20 | 1.438 letters | Standard Deviation 5.7847 |
Secondary
Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT)
Change from Baseline in Mean Area of Geographic Atrophy by Sector as measured by Spectral-Domain Optical Coherence Tomography (SD-OCT) at Week 24
Time frame: Baseline to Week 24
Population: All participants for whom geographic atrophy by SD-OCT was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT) | Center Sector | 0.02 mm^2 | Standard Deviation 0.048 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT) | Outer Inferior | 0.05 mm^2 | Standard Deviation 0.146 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT) | Outer Temporal | 0.08 mm^2 | Standard Deviation 0.129 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT) | Inner Superior | 0.05 mm^2 | Standard Deviation 0.096 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT) | Inner Nasal | 0.04 mm^2 | Standard Deviation 0.059 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT) | Inner Inferior | 0.02 mm^2 | Standard Deviation 0.073 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT) | Inner Temporal | 0.05 mm^2 | Standard Deviation 0.097 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT) | Outer Superior | 0.08 mm^2 | Standard Deviation 0.116 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT) | Outer Nasal | 0.06 mm^2 | Standard Deviation 0.114 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT) | Total ETDRS grid | 0.45 mm^2 | Standard Deviation 0.607 |
Secondary
Change From Baseline in Mean Area of Geographic Atrophy by Fundus Autofluorescence
Change from Baseline in Mean Area of Geographic Atrophy (GA) by Fundus Autofluorescence (FAF) at Week 24. Fluorescein angiography (FA) was used to examine the circulation of the retina and choroid using fluorescein dye and a specialized camera to trace the dye. Fundus autofluorescence imaging of the retinal pigment epithelium and neurosensory retina was performed within 14 days of Day 0 and at every visit with the exception of Day 0 and Day 7. Atrophy is characterized by loss of the retinal pigment epithelium (RPE), overlying photoreceptors and underlying choriocapillaris. Greater area affected means a worse outcome than smaller area affected.
Time frame: Baseline (Day 0) to Week 24
Population: All participants for whom Geographic Atrophy by Fundus Autofluorescence was measured at Week 24.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Area of Geographic Atrophy by Fundus Autofluorescence | Week 12 | 0.264 mm^2 | Standard Deviation 0.1641 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Area of Geographic Atrophy by Fundus Autofluorescence | Week 24 | 0.503 mm^2 | Standard Deviation 0.4914 |
Secondary
Change From Baseline in Mean Dark Adaptometry
Change from Baseline in Mean Dark Adaptometry from Baseline to Week 24- at 0% 25%, 50%, 75% Bleach Level. Dark adaptometry, used to evaluate night blindness by measuring the absolute thresholds of rod sensitivity, was performed within 14 days before Day 0 and at all in-person visits except for Day 7. Dark adaptation is the delayed recovery of light sensitivity in darkness following prior light exposure (photobleaching). The observed and change from baseline recovery scores (in minutes) for each bleach level (0%, 25%, 50%, and 75%) were summarized descriptively for each eye at each visit and were presented in a listing. Shorter recovery times are better than longer recovery times.
Time frame: Baseline (Day 0) and Week 24
Population: All participants for whom Dark Adaptometry was measured at Baseline and Week 24 at 0% 25%, 50%, 75% Bleach Level.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Dark Adaptometry | 0% Bleach Level | -11.553 minutes | Standard Deviation 10.3229 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Dark Adaptometry | 25% Bleach Level | 0.409 minutes | Standard Deviation 4.2798 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Dark Adaptometry | 50% Bleach Level | 0.000 minutes | Standard Deviation 0 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Dark Adaptometry | 75% Bleach Level | 1.562 minutes | Standard Deviation 5.3936 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Dark Adaptometry | 75% Bleach Level | -3.048 minutes | Standard Deviation 9.1675 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Dark Adaptometry | 0% Bleach Level | 3.543 minutes | Standard Deviation 5.014 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Dark Adaptometry | 50% Bleach Level | -3.263 minutes | Standard Deviation 7.9935 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Dark Adaptometry | 25% Bleach Level | 8.3302 minutes | Standard Deviation 0 |
Secondary
Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA)
Change from Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) from Baseline (Day 0) to Day 7, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28.
Time frame: Baseline to Day 7, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28.
Population: All participants for whom Standard Luminance BCVA was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Week 12 | 2.150 letters | Standard Deviation 6.6986 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Day 7 | 2.714 letters | Standard Deviation 5.3399 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Week 16 | 5.158 letters | Standard Deviation 7.3126 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Week 28 | 2.556 letters | Standard Deviation 14.2095 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Week 20 | 5.632 letters | Standard Deviation 7.4923 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Week 4 | 2.762 letters | Standard Deviation 4.8673 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Week 24 | 5.632 letters | Standard Deviation 7.7832 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Week 8 | 2.800 letters | Standard Deviation 6.2205 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Week 24 | 5.400 letters | Standard Deviation 7.854 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Week 28 | 3.733 letters | Standard Deviation 8.1545 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Day 7 | 1.105 letters | Standard Deviation 8.4518 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Week 8 | 4.167 letters | Standard Deviation 4.2183 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Week 12 | 4.688 letters | Standard Deviation 4.5858 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Week 16 | 4.875 letters | Standard Deviation 5.9203 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Week 20 | 5.438 letters | Standard Deviation 5.8875 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA) | Week 4 | 5.000 letters | Standard Deviation 5.5413 |
Secondary
Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score
Change from baseline (Day 0) at Week 12, Week 24, and Week 28 in LLQ score. The LLQ is a 32-item questionnaire with six subscales related to low luminance settings: driving, mobility, extreme lighting, general dim lighting, and peripheral vision. Each question is scored on a scale ranging from 0, or maximal difficulty, to 100, or no difficulty in low luminance settings. Higher scores mean better functioning. The questions are assigned to different subscales and are averaged to generate one score per subscale. After weighting each subscale for the number of questions, the weighted subscales are averaged to generate a composite LLQ score.
Time frame: Baseline (Day 0), Week 12, and Week 24 and Week 28
Population: All participants for whom Low-Luminance visual function by the Visual Function Questionnaire was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 12 Driving or riding in car | 12.2 score on a scale | Standard Deviation 16.41 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 24 Dim light reading | 15.8 score on a scale | Standard Deviation 14.34 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 28 Dim light reading | 12.2 score on a scale | Standard Deviation 19.7 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 24 General dim light vision | 15.6 score on a scale | Standard Deviation 15.17 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 12 General dim light vision | 11.9 score on a scale | Standard Deviation 13.26 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 24 Light transitions and glare | 17.4 score on a scale | Standard Deviation 13.98 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 12 Control questions | 10.5 score on a scale | Standard Deviation 15.47 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 24 Mobility | 9.6 score on a scale | Standard Deviation 20.27 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 12 Light transitions and glare | 9.3 score on a scale | Standard Deviation 15.07 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 24 Other Activities of Daily Living (ADLs) | 17.8 score on a scale | Standard Deviation 17.95 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 24 Control questions | 7.2 score on a scale | Standard Deviation 12.56 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 24 Peripheral vision | 17.8 score on a scale | Standard Deviation 20.12 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 28 Control Questions | 3.2 score on a scale | Standard Deviation 18.44 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 12 Mobility | 6.7 score on a scale | Standard Deviation 11.66 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 28 Driving or riding in car | 10.8 score on a scale | Standard Deviation 23.85 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 28 General dim light vision | 11.7 score on a scale | Standard Deviation 19.43 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 12 Other Activities of Daily Living (ADLs) | 11.5 score on a scale | Standard Deviation 18.36 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 28 Light transitions and glare | 15.0 score on a scale | Standard Deviation 14.75 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 12 Dim light reading | 9.7 score on a scale | Standard Deviation 12.58 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 28 Mobility | 7.4 score on a scale | Standard Deviation 23.02 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 28 Other Activities of Daily Living | 12.2 score on a scale | Standard Deviation 23.82 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 12 Peripheral vision | 16.3 score on a scale | Standard Deviation 19.49 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 28 Peripheral Vision | 13.9 score on a scale | Standard Deviation 20.06 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 24 Driving or riding in car | 16.4 score on a scale | Standard Deviation 18.77 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 24 Light transitions and glare | 6.7 score on a scale | Standard Deviation 13.71 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 24 Driving or riding in car | 4.2 score on a scale | Standard Deviation 14.11 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 24 Peripheral vision | 5.8 score on a scale | Standard Deviation 24.03 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 28 Dim light reading | 7.1 score on a scale | Standard Deviation 12.47 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 28 Driving or riding in car | 4.3 score on a scale | Standard Deviation 14.34 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 28 Mobility | -1.1 score on a scale | Standard Deviation 14.39 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 12 Control questions | -2.3 score on a scale | Standard Deviation 9.86 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 12 Dim light reading | 4.3 score on a scale | Standard Deviation 13.05 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 12 Driving or riding in car | 3.9 score on a scale | Standard Deviation 10.92 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 12 General dim light vision | 6.9 score on a scale | Standard Deviation 12.62 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 12 Light transitions and glare | 6.3 score on a scale | Standard Deviation 13.35 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 12 Other Activities of Daily Living (ADLs) | 10.2 score on a scale | Standard Deviation 15.29 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 12 Peripheral vision | 3.9 score on a scale | Standard Deviation 17.51 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 24 Control questions | 0.7 score on a scale | Standard Deviation 12.47 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 24 Dim light reading | 7.1 score on a scale | Standard Deviation 11.3 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 24 General dim light vision | 7.7 score on a scale | Standard Deviation 12.1 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 12 Mobility | 6.3 score on a scale | Standard Deviation 10.32 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 24 Mobility | 2.8 score on a scale | Standard Deviation 16.57 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 24 Other Activities of Daily Living (ADLs) | 10.4 score on a scale | Standard Deviation 20.82 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 28 Control Questions | 0.2 score on a scale | Standard Deviation 14.25 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 28 General dim light vision | 3.3 score on a scale | Standard Deviation 10.73 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 28 Light transitions and glare | 5.3 score on a scale | Standard Deviation 14.07 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 28 Other Activities of Daily Living | 3.8 score on a scale | Standard Deviation 13.73 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Low Luminance Questionnaire (LLQ) Score | Week 28 Peripheral Vision | 5.0 score on a scale | Standard Deviation 15.53 |
Secondary
Change From Baseline in Mean Mesopic Light Sensitivity
Change from baseline in mean mesopic light sensitivity for Weeks, 4, 8, 12, 16, 20, and 24 as assessed by microperimetry for number of loci \<25dB and \<14dB. Mesopic microperimetry, used to measure retinal sensitivity, was performed within 14 days before Day 0 and at all in-person visits except for Day 7.
Time frame: Baseline (Day 0) and Weeks 4,8,12,16,20,and 24
Population: All participants for whom mean mesopic light sensitivity was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <25dB Week 4 | 0.6 loci | Standard Deviation 6.08 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <25dB Week 8 | 1.3 loci | Standard Deviation 8.21 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <25dB Week 12 | 0.5 loci | Standard Deviation 7.87 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <25dB Week 16 | 0.9 loci | Standard Deviation 8.56 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <25dB Week 20 | -1.2 loci | Standard Deviation 5.88 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <25dB Week 24 | -0.5 loci | Standard Deviation 8.27 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <14dB Week 4 | 0.3 loci | Standard Deviation 0.3 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <14dB Week 8 | -1.4 loci | Standard Deviation 3.65 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <14dB Week 12 | 1.3 loci | Standard Deviation 10.05 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <14dB Week 16 | -2.5 loci | Standard Deviation 2.38 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <14dB Week 20 | -0.2 loci | Standard Deviation 6.14 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <14dB Week 24 | -0.3 loci | Standard Deviation 8.3 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <14dB Week 20 | 1.5 loci | Standard Deviation 4.88 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <25dB Week 4 | 0.6 loci | Standard Deviation 1.79 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <14dB Week 4 | 1.7 loci | Standard Deviation 1.7 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <25dB Week 8 | 0.7 loci | Standard Deviation 1.94 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <14dB Week 16 | 1.5 loci | Standard Deviation 5.01 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <25dB Week 12 | 0.1 loci | Standard Deviation 3.09 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <14dB Week 8 | 1.7 loci | Standard Deviation 4.89 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <25dB Week 16 | 0.1 loci | Standard Deviation 2.35 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <14dB Week 24 | 3.3 loci | Standard Deviation 5.82 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <25dB Week 20 | -0.1 loci | Standard Deviation 1.44 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <14dB Week 12 | 3.2 loci | Standard Deviation 5.9 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Mesopic Light Sensitivity | Number of loci <25dB Week 24 | -0.1 loci | Standard Deviation 3.45 |
Secondary
Change From Baseline in Mean Reading Acuity Test: Low Luminance
Change from Baseline in Mean Reading Acuity Test: Mean Critical Print Size with Low Luminance in Weeks 4, 8, 12, 16, 20, 24 and 28 as measured by the Logarithm of the Minimum Angle of Resolution LogMAR chart. Scores range from -0.3 to 1.0, where higher number means worse acuity/worse outcome, a lower number means better acuity/better outcome.
Time frame: Assessed at screening, baseline, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24, and Week 28
Population: All participants for whom Mean Critical Print Size (logMAR) with Low Luminance was measured at baseline and Week 4 through Week 28.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Reading Acuity Test: Low Luminance | Week 12 | -0.21 units on a scale | Standard Deviation 0.177 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Reading Acuity Test: Low Luminance | Week 20 | -0.29 units on a scale | Standard Deviation 0.184 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Reading Acuity Test: Low Luminance | Week 8 | -0.16 units on a scale | Standard Deviation 0.179 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Reading Acuity Test: Low Luminance | Week 24 | -0.28 units on a scale | Standard Deviation 0.174 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Reading Acuity Test: Low Luminance | Week 16 | -0.25 units on a scale | Standard Deviation 0.209 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Reading Acuity Test: Low Luminance | Week 28 | -0.24 units on a scale | Standard Deviation 0.238 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Reading Acuity Test: Low Luminance | Week 4 | -0.11 units on a scale | Standard Deviation 0.17 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Reading Acuity Test: Low Luminance | Week 28 | -0.10 units on a scale | Standard Deviation 0.194 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Reading Acuity Test: Low Luminance | Week 4 | -0.09 units on a scale | Standard Deviation 0.088 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Reading Acuity Test: Low Luminance | Week 8 | -0.10 units on a scale | Standard Deviation 0.253 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Reading Acuity Test: Low Luminance | Week 12 | -0.20 units on a scale | Standard Deviation 0.236 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Reading Acuity Test: Low Luminance | Week 16 | -0.16 units on a scale | Standard Deviation 0.232 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Reading Acuity Test: Low Luminance | Week 20 | -0.14 units on a scale | Standard Deviation 0.171 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Reading Acuity Test: Low Luminance | Week 24 | -0.11 units on a scale | Standard Deviation 0.209 |
Secondary
Change From Baseline in Mean Reading Acuity Test: With Standard Luminance
Change from Baseline in Mean Reading Acuity Test: Mean Critical Print Size with Standard Luminance in Weeks 4, 8, 12, 16, 20, 24 and 28 as measured by the Logarithm of the Minimum Angle of Resolution LogMAR chart. Scores range from -0.3 to 1.0, where higher number means worse acuity/worse outcome, a lower number means better acuity/better outcome.
Time frame: Baseline and Weeks 4, 8, 12, 16 20, 24 and 28
Population: All participants for whom Mean Critical Print Size (logMAR) with Standard Luminance was measured
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Reading Acuity Test: With Standard Luminance | Week 12 | -0.10 units on a scale | Standard Deviation 0.156 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Reading Acuity Test: With Standard Luminance | Week 16 | -0.11 units on a scale | Standard Deviation 0.151 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Reading Acuity Test: With Standard Luminance | Week 4 | -0.04 units on a scale | Standard Deviation 0.121 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Reading Acuity Test: With Standard Luminance | Week 8 | -0.08 units on a scale | Standard Deviation 0.111 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Reading Acuity Test: With Standard Luminance | Week 20 | -0.12 units on a scale | Standard Deviation 0.126 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Reading Acuity Test: With Standard Luminance | Week 24 | -0.11 units on a scale | Standard Deviation 0.152 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Reading Acuity Test: With Standard Luminance | Week 28 | -0.04 units on a scale | Standard Deviation 0.201 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Reading Acuity Test: With Standard Luminance | Week 20 | -0.01 units on a scale | Standard Deviation 0.109 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Reading Acuity Test: With Standard Luminance | Week 12 | -0.02 units on a scale | Standard Deviation 0.098 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Reading Acuity Test: With Standard Luminance | Week 8 | -0.02 units on a scale | Standard Deviation 0.115 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Reading Acuity Test: With Standard Luminance | Week 16 | -0.06 units on a scale | Standard Deviation 0.115 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Reading Acuity Test: With Standard Luminance | Week 24 | -0.02 units on a scale | Standard Deviation 0.126 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Reading Acuity Test: With Standard Luminance | Week 4 | -0.03 units on a scale | Standard Deviation 0.091 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Reading Acuity Test: With Standard Luminance | Week 28 | 0.01 units on a scale | Standard Deviation 0.173 |
Secondary
Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT
Change from baseline in mean retinal pigment epithelium - drusen complex (RPEDC) thickness as Measured by SD-OCT by sector at Week 24
Time frame: Baseline (Day 0) and Week 24
Population: All participants for whom RPEDC) thickness as Measured by SD-OCT by sector at Baseline and Week 24
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Inner Superior | 1.29 um | Standard Deviation 3.699 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Inner temporal | 1.00 um | Standard Deviation 3.68 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Center | 0.57 um | Standard Deviation 5.147 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Outer Superior | 0.34 um | Standard Deviation 2.279 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Inner Nasal | 1.43 um | Standard Deviation 3.143 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Outer Nasal | 0.34 um | Standard Deviation 2.301 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Outer Temporal | 0.25 um | Standard Deviation 2.672 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Outer Inferior | -0.19 um | Standard Deviation 2.356 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Total ETDRS | 0.39 um | Standard Deviation 1.885 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Inner Inferior | 0.72 um | Standard Deviation 4.393 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Total ETDRS | -0.25 um | Standard Deviation 1.59 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Outer Temporal | -0.04 um | Standard Deviation 1.594 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Center | 0.38 um | Standard Deviation 4.137 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Inner Superior | -0.85 um | Standard Deviation 2.389 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Inner Nasal | -0.83 um | Standard Deviation 2.801 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Inner Inferior | -0.82 um | Standard Deviation 3.798 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Inner temporal | -0.34 um | Standard Deviation 2.897 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Outer Superior | -0.10 um | Standard Deviation 1.58 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Outer Nasal | -0.09 um | Standard Deviation 1.919 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium - Drusen Complex (RPEDC) Thickness as Measured by SD-OCT | Outer Inferior | -0.04 um | Standard Deviation 2.023 |
Secondary
Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume
Change from baseline in mean Retinal Pigment Epithelium-Drusen Complex volume at Week 24 by sector
Time frame: Baseline (Day 0) to Week 24
Population: All participants for whom the retinal pigment epithelium-drusen complex volume was measured
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Inner nasal | 0.00 mm^3 | Standard Deviation 0.005 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Outer superior | 0.00 mm^3 | Standard Deviation 0.012 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Inner superior | 0.00 mm^3 | Standard Deviation 0.006 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Outer nasal | 0.00 mm^3 | Standard Deviation 0.012 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Outer inferior | -0.00 mm^3 | Standard Deviation 0.012 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Inner inferior | 0.00 mm^3 | Standard Deviation 0.007 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Outer temporal | 0.00 mm^3 | Standard Deviation 0.014 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Center | 0.00 mm^3 | Standard Deviation 0.004 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Total ETDRS | 0.01 mm^3 | Standard Deviation 0.054 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Inner temporal | 0.00 mm^3 | Standard Deviation 0.006 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Total ETDRS | -0.01 mm^3 | Standard Deviation 0.046 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Center | 0.00 mm^3 | Standard Deviation 0.003 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Inner superior | -0.00 mm^3 | Standard Deviation 0.004 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Inner nasal | -0.00 mm^3 | Standard Deviation 0.004 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Inner inferior | -0.00 mm^3 | Standard Deviation 0.006 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Inner temporal | -0.00 mm^3 | Standard Deviation 0.005 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Outer superior | -0.00 mm^3 | Standard Deviation 0.008 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Outer inferior | -0.00 mm^3 | Standard Deviation 0.011 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Outer temporal | -0.00 mm^3 | Standard Deviation 0.009 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in Mean Retinal Pigment Epithelium-Drusen Complex Volume | Outer nasal | -0.00 mm^3 | Standard Deviation 0.01 |
Secondary
Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance
Change from baseline (Day 0) at Week 12, 24 and 28. National Eye Institute Visual Function Questionnaire-39 (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 (Day 0), Week 12, Week 24 and Week 28
Population: All participants for whom the VFQ-39 was measured at Week 12 and Week 24.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Dependency | 1.7 score on a scale | Standard Deviation 17.91 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Composite | 9.2 score on a scale | Standard Deviation 9.19 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Distance Activities | 5.7 score on a scale | Standard Deviation 16.92 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 General Vision | 8.7 score on a scale | Standard Deviation 12.78 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Driving | 12.3 score on a scale | Standard Deviation 12.54 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Color Vision | 11.8 score on a scale | Standard Deviation 22.62 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 General Health | 4.2 score on a scale | Standard Deviation 10.18 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Mental Health | 9.5 score on a scale | Standard Deviation 17.55 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 General Vision | 8.6 score on a scale | Standard Deviation 16.25 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Distance Activities | 7.0 score on a scale | Standard Deviation 8.13 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Mental Health | 9.4 score on a scale | Standard Deviation 19.84 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Near Activities | 9.8 score on a scale | Standard Deviation 11 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Near Activities | 6.0 score on a scale | Standard Deviation 20.17 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Ocular Pain | 5.9 score on a scale | Standard Deviation 19.26 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Ocular Pain | 7.6 score on a scale | Standard Deviation 20.17 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Dependency | 2.3 score on a scale | Standard Deviation 14.61 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Peripheral Vision | 15.3 score on a scale | Standard Deviation 21.25 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Peripheral Vision | 17.1 score on a scale | Standard Deviation 16.78 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Role Difficulties | 4.2 score on a scale | Standard Deviation 21 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Social Functioning | 5.6 score on a scale | Standard Deviation 17.85 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Color Vision | 8.8 score on a scale | Standard Deviation 18.63 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Composite | 7.6 score on a scale | Standard Deviation 7.47 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Mental Health | 9.3 score on a scale | Standard Deviation 13.79 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Dependency | 1.9 score on a scale | Standard Deviation 10.94 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Role Difficulties | 6.9 score on a scale | Standard Deviation 11.58 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Distance Activities | 8.4 score on a scale | Standard Deviation 11.11 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Driving | 10.0 score on a scale | Standard Deviation 12.85 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Social Functioning | 6.6 score on a scale | Standard Deviation 10.96 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 General Health | 1.4 score on a scale | Standard Deviation 8.98 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Social Functioning | 7.5 score on a scale | Standard Deviation 8.51 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 General Vision | 5.3 score on a scale | Standard Deviation 11.18 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Color Vision | 12.5 score on a scale | Standard Deviation 17.68 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Driving | 14.5 score on a scale | Standard Deviation 14.66 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Near Activities | 8.2 score on a scale | Standard Deviation 10.43 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Composite | 7.9 score on a scale | Standard Deviation 14.04 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Ocular Pain | 7.5 score on a scale | Standard Deviation 20.84 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Peripheral Vision | 12.5 score on a scale | Standard Deviation 19.02 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 General Health | -0.3 score on a scale | Standard Deviation 9.01 |
| AMD-High-Risk Drusen (HRD) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Role Difficulties | 5.9 score on a scale | Standard Deviation 12.25 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 General Vision | 3.7 score on a scale | Standard Deviation 12.6 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Ocular Pain | 3.1 score on a scale | Standard Deviation 9.68 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Color Vision | 11.7 score on a scale | Standard Deviation 26.5 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Driving | 0.6 score on a scale | Standard Deviation 10.46 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Near Activities | 5.6 score on a scale | Standard Deviation 13.84 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Role Difficulties | 5.4 score on a scale | Standard Deviation 20.03 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Social Functioning | 3.3 score on a scale | Standard Deviation 10.82 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Role Difficulties | 5.9 score on a scale | Standard Deviation 26.37 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Social Functioning | 5.2 score on a scale | Standard Deviation 18.48 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Composite | 7.0 score on a scale | Standard Deviation 9.38 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Dependency | 6.7 score on a scale | Standard Deviation 10.94 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Distance Activities | 4.7 score on a scale | Standard Deviation 16.13 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 General Health | 3.3 score on a scale | Standard Deviation 10.21 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 General Vision | 6.3 score on a scale | Standard Deviation 13.69 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Mental Health | 7.7 score on a scale | Standard Deviation 9.8 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Ocular Pain | 5.0 score on a scale | Standard Deviation 14.02 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Peripheral Vision | 15.0 score on a scale | Standard Deviation 18.42 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Role Difficulties | 5.8 score on a scale | Standard Deviation 17.27 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 24 Social Functioning | 5.6 score on a scale | Standard Deviation 15.64 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Color Vision | 5.0 score on a scale | Standard Deviation 28.66 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Composite | 4.3 score on a scale | Standard Deviation 10.46 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Dependency | -1.3 score on a scale | Standard Deviation 20.07 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Distance Activities | 7.1 score on a scale | Standard Deviation 13.95 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Driving | 0.8 score on a scale | Standard Deviation 9.47 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 General Health | 4.0 score on a scale | Standard Deviation 9.2 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Color Vision | 10.9 score on a scale | Standard Deviation 20.35 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Mental Health | 5.7 score on a scale | Standard Deviation 10.5 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Near Activities | 1.7 score on a scale | Standard Deviation 12.63 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Ocular Pain | 0.0 score on a scale | Standard Deviation 14.94 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 28 Peripheral Vision | 13.3 score on a scale | Standard Deviation 20.85 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Composite | 5.5 score on a scale | Standard Deviation 8.9 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Dependency | 2.0 score on a scale | Standard Deviation 14.02 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Distance Activities | 4.6 score on a scale | Standard Deviation 12.19 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Driving | 0.6 score on a scale | Standard Deviation 13.38 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 General Health | 1.9 score on a scale | Standard Deviation 9.33 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 General Vision | 7.2 score on a scale | Standard Deviation 14.02 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Mental Health | 9.1 score on a scale | Standard Deviation 14.63 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Near Activities | 2.4 score on a scale | Standard Deviation 11.51 |
| AMD-NCGA (Noncentral GA) | Change From Baseline in National Eye Institute Visual Function Questionnaire-39 (VFQ-39) Score Standard Luminance | Week 12 Peripheral Vision | 7.8 score on a scale | Standard Deviation 19.83 |
Secondary
Mean Number of Home Health Visits to Administer Elamipretide
Mean Number of Home Health Visits Necessary for Participant or Caregiver to Learn How to Administer Elamipretide
Time frame: Baseline (Day 0) through Week 24
Population: All participants and caregivers whom administered elamipretide injections.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| AMD-High-Risk Drusen (HRD) | Mean Number of Home Health Visits to Administer Elamipretide | 2.2 visits | Standard Deviation 0.54 |
| AMD-NCGA (Noncentral GA) | Mean Number of Home Health Visits to Administer Elamipretide | 2.5 visits | Standard Deviation 1.02 |
Secondary
Mean Treatment Compliance (%) of Administration of Subcutaneous Elamipretide
Mean percentage of treatment compliance of administration of subcutaneous elamipretide. The number of injections (diary) and vials used was used to compute % compliance over the duration of the subject's participation in the trial. Percentage can range from 0-100%, where 0% means the participant followed the correct dosing 0% of the time, and 100% compliance means the participant followed the correct dosing 100% of the time, with a higher % meaning a better outcome.
Time frame: Baseline through Week 28
Population: All participants for whom compliance was measured.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| AMD-High-Risk Drusen (HRD) | Mean Treatment Compliance (%) of Administration of Subcutaneous Elamipretide | 98.4 % of compliance in study dosing | Standard Deviation 4.04 |
| AMD-NCGA (Noncentral GA) | Mean Treatment Compliance (%) of Administration of Subcutaneous Elamipretide | 97.3 % of compliance in study dosing | Standard Deviation 6.7 |