Barth Syndrome
Conditions
Keywords
Barth Syndrome, elamipretide, MTP-131, Stealth, Stealth BT, BTHS
Brief summary
A randomized, double-blind cross over trial to evaluate the safety, efficacy, and tolerability of elamipretide in subjects with Barth syndrome.
Detailed description
A phase 2 randomized, double-blind, placebo-controlled crossover trial to evaluate the safety, tolerability, and efficacy of subcutaneous injections of elamipretide in subjects with genetically confirmed Barth syndrome followed by open-label treatment extension. Part 1 was a randomized, double-blind, placebo-controlled, crossover trial to assess safety, tolerability, and efficacy single daily subcutaneous (SC) doses of 40 mg elamipretide administered for 12 weeks in subjects with Barth syndrome. Part 2: This was an open-label extension trial to assess the long-term safety, tolerability, and longitudinal trends in efficacy single daily SC doses of 40 mg elamipretide for up to 192 weeks.
Interventions
DRUGElamipretide
40 mg daily subcutaneous injection for 12 weeks
DRUGPlacebo
daily subcutaneous injection for 12 weeks
Sponsors
Stealth BioTherapeutics Inc.
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Intervention model description
28 weeks, double-blinded crossover followed by a 168-week open label long term safety & tolerability trial.
Eligibility
Sex/Gender
MALE
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Genetically confirmed Barth Syndrome * Male aged 12 and above * At the screening visit, eGFR must meet the following: 1. Body weight \>30 kg AND eGFR ≥ 90mL/min at screening 2. Body weight \>40kg AND eGFR ≥60 but \<90mL/min/ 1.73m² at screening * Ambulatory and impaired during the baseline 6MWT * On stable medication for 30 days prior to the baseline visit
Exclusion criteria
* Participated in another interventional clinical trial within 30 days of or is currently enrolled in a non-interventional clinical trial at the baseline visit potentially confounding with this trial * Prior or current medical condition that would prevent the subject from safely participating in the trial * Undergone any inpatient hospitalizations within 30 days of the baseline visit * Is undergoing an apparent pubertal growth spurt * Has uncontrolled hypertension * History of substance abused within the year before the baseline visit or is likely to be uncompliant * History of heart transplantation or current placement on the waiting list for a heart transplant * For subjects with an ICD: known occurrence of ICD discharge in the 3 months prior to the baseline visit * For subjects without an ICD: expected to undergo an implantation of an ICD during the conduct of the study * Currently receiving treatment with chemotherapeutic agents or immunosuppressant agents or has received prior radiation therapy to the chest * Recipient of stem cell or gene therapy or is currently being treated by a therapeutic investigational device
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit | Pre-dose to Week 12 (end of treatment) | Average distance walked in meters during the 6-minute walk test (6MWT) at end of treatment, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period. |
| Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Pre-dose, Weeks 1, 4, 8, and 12 (end of treatment) | Total Fatigue Score (Q1, Q2, and Q4) Based on the BarTH Syndrome Symptom Assessment (BTHS-SA) by visit at predose, Weeks 1, 4, 8, and 12 (end of treatment). The BTHS-SA is 3-question fatigue assessment using a 0 to 4-point scale for each question where no fatigue =0 and very severe fatigue =4, with scores from all 3 questions combined and averaged for all subjects; a lower score means better outcome, higher score means a worse outcome. Combined Min score=0, max score =12. Weekly values are based on the weekly average of the last 7 days before the site or visiting nurse visit for both periods. Results from each 12-week period were combined, where end of treatment for Period 1 =Visit 5 (week 12), and End of Period 2 = Visit 10 (week 12) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192 | Change from Baseline (mean) in Total Fatigue Score (Q1, Q2, and Q4) Based on the BarTH Syndrome Symptom Assessment (BTHS-SA) by visit. The BTHS-SA is 3-question fatigue assessment using a 0-4 point scale for each question where no fatigue =0 and very severe fatigue =4, with scores from all 3 questions combined and averaged for all subjects by visit; a lower score means better outcome, higher score means a worse outcome. Change from baseline: an increase in scores would mean worse outcome, a decrease in scores means better outcome. Combined Min score=-12, max score =12. Weekly values are based on the weekly average of the last 7 days before the site or visiting nurse visit for both periods. Baseline= Period 1 Predose. |
| Part 1: Muscle Strength as Measured by HHD by Visit | Pre-dose to Week 12 (end of treatment) | Muscle strength as measured by handheld dynamometry (HHD) in Newtons, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period. Handheld dynamometry assesses the average strength of the knee extensors of both legs. |
| Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192 | Muscle strength as measured by handheld dynamometry (HHD) in Newtons. Handheld dynamometry assesses the average strength of the knee extensors of both legs. Change from baseline where Baseline= Part 1 Period 1 Pre-dose: the greater positive change, the better the outcome, the smaller positive change (or negative change) the number the worse the outcome. |
| Part 2: Change From Baseline: 5X Sit to Stand by Visit | Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192 | Change in baseline in time (in seconds), where Baseline= Part 1 Period 1 Pre-dose to complete the Five Times Sit-To-Stand by visit. More time means worse outcome, less time means better outcome. |
| Part 1: SWAY Application Balance Assessments by Visit | Pre-dose to Week 12 (end of treatment) | SWAY application balance assessment consists of five stances each performed for 10 seconds and scores postural stability on a scale from 0-100 with higher scores indicating better postural stability from Pre-dose to Week 12 (end of treatment), where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period. |
| Part 1: 5XSST by Visit | Pre-dose to Week 12 (end of treatment) | Time (in seconds) to complete the Five Times Sit-To-Stand, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period. |
| Part 1: Patient Global Impression Scales of Symptoms | Pre-dose, Week 1, Week 12 (end of treatment) | Patient-reported health status over the past week, by visit, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period. Scores range from 0-40 for adolescents and 0-50 for adults. PGI Scale is as follows: 0=None, 1=Mild, 2=Moderate, 3=Severe, and 4=Very Severe. Higher score means worse health status, means worse outcome; lower score means better health status, means better outcome. |
| Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192 | Part 2: Change from Baseline: Patient Global Impression Scales of Symptoms by Visit Patient-reported health status over the past week where Baseline= Part 1 Period 1 Pre-dose, administered at Week 12 for Period 1 and Period 2. Scores range from 0-40 for adolescents and 0-50 for adults. PGI Scale is as follows: 1=None, 2=Mild, 3=Moderate, 4=Severe, and 5=Very Severe. Change from baseline: Higher score means worse health status, means worse outcome; lower score means better health status, means better outcome. |
| Part 1: Part 1: Clinician Global Impression | Pre-dose to Week 12 (end of treatment) | Clinician Global Impression Scale by visit: Clinician-reported overall health status at end of treatment, administered at Week 12 for Period 1 and Period 2. Scores range from 0-6 for both adolescents and adults. PGI Scale is as follows: 1= Very much Better, 2= Moderately Better, 3= A Little Better, 4= No Change, and 5= A Little Worse 6= Very much Worse. Higher score means worse health status, means worse outcome; lower score means better health status, means better outcome. End of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period. |
| Part 2: Change From Baseline: CGI Symptom Scale | Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192 | Change from Baseline: CGI Symptom Scale by visit where Baseline= Part 1 Period 1 Pre-dose. Clinician Global Impression Scale: Clinician-reported overall health status at end of treatment, administered at Week 12 for Period 1 and Period 2. Scores range from 0-6 for both adolescents and adults. PGI Scale is as follows: 1= Very much Better, 2= Moderately Better, 3= A Little Better, 4= No Change, and 5= A Little Worse 6= Very much Worse. Change from baseline: higher score means worse health status, means worse outcome; lower score means better health status, means better outcome. |
| Part 2: Change From Baseline: SWAY Application Balance Assessment | Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192 | Change from Baseline: SWAY Application Balance Assessment,where Baseline= Part 1 Period 1 Pre-doseSWAY application balance assessment consists of five stances each performed for 10 seconds and scores postural stability on a scale from 0-100 with higher scores indicating better postural stability. An increase from baseline is a better outcome and a decrease in score is a worse outcome. |
| Part 2: Distance Walked During the 6MWT | Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192 | Change from baseline for distance walked in meters during the 6-minute walk test (6MWT) by visit where Baseline= Part 1 Period 1 Pre-dose |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Experimental: Elamipretide (Treatment AB) Patients will be randomized to receive 12 weeks of elamipretide in one of the two treatment periods. All subjects will receive 12 weeks of elamipretide, 4 weeks of washout, and 12 weeks of placebo and 4 weeks of follow up (for those not continuing to Part 2).
Elamipretide: 40 mg daily subcutaneous injection for 12 weeks Placebo: daily subcutaneous injection for 12 weeks | 6 |
| Experimental Placebo (Treatment BA) Patients will be randomized to receive 12 weeks of placebo in one of the two treatment periods. All subjects will receive 12 weeks of placebo, 4 weeks of washout, and 12 weeks of elamipretide, and 4 weeks of follow up (for those not continuing to Part 2).
Placebo: daily subcutaneous injection for 12 weeks Elamipretide: 40 mg daily subcutaneous injection for 12 weeks | 6 |
| Total | 12 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Part 2 Open Label Extension | Adverse Event | 2 | 0 |
Baseline characteristics
| Characteristic | Experimental: Elamipretide (Treatment AB) | Experimental Placebo (Treatment BA) | Total |
|---|---|---|---|
| Age, Continuous Part 1 | 23.3 years STANDARD_DEVIATION 9.07 | 15.7 years STANDARD_DEVIATION 3.33 | 19.5 years STANDARD_DEVIATION 7.65 |
| Age, Continuous PART 2: OLE | 21.8 years STANDARD_DEVIATION 9.23 | 16.2 years STANDARD_DEVIATION 3.42 | 19.0 years STANDARD_DEVIATION 7.2 |
| Body Mass Index Part 1 | 18.23 kg/m^2 STANDARD_DEVIATION 3.677 | 16.88 kg/m^2 STANDARD_DEVIATION 4.179 | 17.56 kg/m^2 STANDARD_DEVIATION 3.818 |
| Body Mass Index Part 2 OLE | 18.14 kg/m^2 STANDARD_DEVIATION 4.103 | 17.54 kg/m^2 STANDARD_DEVIATION 4.313 | 17.84 kg/m^2 STANDARD_DEVIATION 3.981 |
| Race (NIH/OMB) Part 1 American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Part 1 Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Part 1 Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Part 1 More than one race | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Part 1 Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Part 1 Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Part 1 White | 6 Participants | 5 Participants | 11 Participants |
| Race (NIH/OMB) Part 2 (OLE) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Part 2 (OLE) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Part 2 (OLE) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Part 2 (OLE) More than one race | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Part 2 (OLE) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Part 2 (OLE) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Part 2 (OLE) White | 5 Participants | 4 Participants | 9 Participants |
| Region of Enrollment United States | 6 participants | 6 participants | 12 participants |
| Sex: Female, Male Part 1 Female | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Part 1 Male | 6 Participants | 6 Participants | 12 Participants |
| Sex: Female, Male Part 2 (OLE) Female | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Part 2 (OLE) Male | 5 Participants | 5 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 / 5 | 0 / 5 | 0 / 6 | 0 / 6 |
| other Total, other adverse events | 5 / 5 | 5 / 5 | 12 / 12 | 12 / 12 |
| serious Total, serious adverse events | 1 / 5 | 4 / 5 | 0 / 12 | 0 / 12 |
Outcome results
Primary
Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit
Average distance walked in meters during the 6-minute walk test (6MWT) at end of treatment, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period.
Time frame: Pre-dose to Week 12 (end of treatment)
Population: All participants for whom 6MWT was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Experimental: Elamipretide | Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit | Pre-dose | 400.1 meters | Standard Deviation 55.07 |
| Experimental: Elamipretide | Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit | Week 12 (End of Treatment | 443.1 meters | Standard Deviation 65.43 |
| Placebo | Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit | Pre-dose | 412.6 meters | Standard Deviation 60.16 |
| Placebo | Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit | Week 12 (End of Treatment | 443.9 meters | Standard Deviation 77.1 |
Primary
Part 1: Total Fatigue Score Based on the BTHS-SA by Visit.
Total Fatigue Score (Q1, Q2, and Q4) Based on the BarTH Syndrome Symptom Assessment (BTHS-SA) by visit at predose, Weeks 1, 4, 8, and 12 (end of treatment). The BTHS-SA is 3-question fatigue assessment using a 0 to 4-point scale for each question where no fatigue =0 and very severe fatigue =4, with scores from all 3 questions combined and averaged for all subjects; a lower score means better outcome, higher score means a worse outcome. Combined Min score=0, max score =12. Weekly values are based on the weekly average of the last 7 days before the site or visiting nurse visit for both periods. Results from each 12-week period were combined, where end of treatment for Period 1 =Visit 5 (week 12), and End of Period 2 = Visit 10 (week 12)
Time frame: Pre-dose, Weeks 1, 4, 8, and 12 (end of treatment)
Population: All participants for whom Total Fatigue Score was measured
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Experimental: Elamipretide | Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Week 1 | 6.837 score on a scale | Standard Deviation 2.1114 |
| Experimental: Elamipretide | Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Week 8 | 6.089 score on a scale | Standard Deviation 1.8344 |
| Experimental: Elamipretide | Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Week 4 | 6.220 score on a scale | Standard Deviation 2.3325 |
| Experimental: Elamipretide | Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Week 12 | 6.305 score on a scale | Standard Deviation 2.0612 |
| Experimental: Elamipretide | Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Pre-dose | 7.672 score on a scale | Standard Deviation 1.936 |
| Placebo | Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Week 12 | 6.240 score on a scale | Standard Deviation 1.6384 |
| Placebo | Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Pre-dose | 7.433 score on a scale | Standard Deviation 1.3848 |
| Placebo | Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Week 1 | 6.994 score on a scale | Standard Deviation 1.7093 |
| Placebo | Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Week 4 | 6.485 score on a scale | Standard Deviation 1.4447 |
| Placebo | Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. | Week 8 | 6.196 score on a scale | Standard Deviation 1.7815 |
Secondary
Part 1: 5XSST by Visit
Time (in seconds) to complete the Five Times Sit-To-Stand, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period.
Time frame: Pre-dose to Week 12 (end of treatment)
Population: All participants for whom 5XSST was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Experimental: Elamipretide | Part 1: 5XSST by Visit | Pre-dose | 12.965 seconds | Standard Deviation 3.3366 |
| Experimental: Elamipretide | Part 1: 5XSST by Visit | Week 12 | 14.023 seconds | Standard Deviation 5.4508 |
| Placebo | Part 1: 5XSST by Visit | Pre-dose | 13.710 seconds | Standard Deviation 5.3088 |
| Placebo | Part 1: 5XSST by Visit | Week 12 | 13.749 seconds | Standard Deviation 6.1024 |
Secondary
Part 1: Muscle Strength as Measured by HHD by Visit
Muscle strength as measured by handheld dynamometry (HHD) in Newtons, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period. Handheld dynamometry assesses the average strength of the knee extensors of both legs.
Time frame: Pre-dose to Week 12 (end of treatment)
Population: All participants for whom muscle strength as measured by handheld dynamometry was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Experimental: Elamipretide | Part 1: Muscle Strength as Measured by HHD by Visit | Pre-dose | 131.236 Newtons | Standard Deviation 29.9004 |
| Experimental: Elamipretide | Part 1: Muscle Strength as Measured by HHD by Visit | Week 12 | 135.934 Newtons | Standard Deviation 52.2796 |
| Placebo | Part 1: Muscle Strength as Measured by HHD by Visit | Week 12 | 129.253 Newtons | Standard Deviation 34.2395 |
| Placebo | Part 1: Muscle Strength as Measured by HHD by Visit | Pre-dose | 123.100 Newtons | Standard Deviation 24.6762 |
Secondary
Part 1: Part 1: Clinician Global Impression
Clinician Global Impression Scale by visit: Clinician-reported overall health status at end of treatment, administered at Week 12 for Period 1 and Period 2. Scores range from 0-6 for both adolescents and adults. PGI Scale is as follows: 1= Very much Better, 2= Moderately Better, 3= A Little Better, 4= No Change, and 5= A Little Worse 6= Very much Worse. Higher score means worse health status, means worse outcome; lower score means better health status, means better outcome. End of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period.
Time frame: Pre-dose to Week 12 (end of treatment)
Population: All participants for whom Patient Global Impression Scales of Symptoms was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Experimental: Elamipretide | Part 1: Part 1: Clinician Global Impression | Predose | 1.8 Score on a scale | Standard Deviation 0.45 |
| Experimental: Elamipretide | Part 1: Part 1: Clinician Global Impression | 12 weeks | 1.6 Score on a scale | Standard Deviation 0.51 |
| Placebo | Part 1: Part 1: Clinician Global Impression | Predose | 1.4 Score on a scale | Standard Deviation 0.51 |
| Placebo | Part 1: Part 1: Clinician Global Impression | 12 weeks | 1.6 Score on a scale | Standard Deviation 0.51 |
Secondary
Part 1: Patient Global Impression Scales of Symptoms
Patient-reported health status over the past week, by visit, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period. Scores range from 0-40 for adolescents and 0-50 for adults. PGI Scale is as follows: 0=None, 1=Mild, 2=Moderate, 3=Severe, and 4=Very Severe. Higher score means worse health status, means worse outcome; lower score means better health status, means better outcome.
Time frame: Pre-dose, Week 1, Week 12 (end of treatment)
Population: All participants for whom Patient Global Impression Scales of Symptoms was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Experimental: Elamipretide | Part 1: Patient Global Impression Scales of Symptoms | Predose | 1.7 Score on a scale | Standard Deviation 0.65 |
| Experimental: Elamipretide | Part 1: Patient Global Impression Scales of Symptoms | Week 1 | 1.7 Score on a scale | Standard Deviation 0.89 |
| Experimental: Elamipretide | Part 1: Patient Global Impression Scales of Symptoms | Week 12 | 1.4 Score on a scale | Standard Deviation 0.79 |
| Placebo | Part 1: Patient Global Impression Scales of Symptoms | Predose | 1.8 Score on a scale | Standard Deviation 0.83 |
| Placebo | Part 1: Patient Global Impression Scales of Symptoms | Week 1 | 1.5 Score on a scale | Standard Deviation 1 |
| Placebo | Part 1: Patient Global Impression Scales of Symptoms | Week 12 | 1.6 Score on a scale | Standard Deviation 0.67 |
Secondary
Part 1: SWAY Application Balance Assessments by Visit
SWAY application balance assessment consists of five stances each performed for 10 seconds and scores postural stability on a scale from 0-100 with higher scores indicating better postural stability from Pre-dose to Week 12 (end of treatment), where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period.
Time frame: Pre-dose to Week 12 (end of treatment)
Population: All participants for who SWAY application balance was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Experimental: Elamipretide | Part 1: SWAY Application Balance Assessments by Visit | Pre-dose | 70.752 Score on a scale | Standard Deviation 19.7199 |
| Experimental: Elamipretide | Part 1: SWAY Application Balance Assessments by Visit | Week 12 | 78.698 Score on a scale | Standard Deviation 15.1554 |
| Placebo | Part 1: SWAY Application Balance Assessments by Visit | Pre-dose | 68.820 Score on a scale | Standard Deviation 24.7478 |
| Placebo | Part 1: SWAY Application Balance Assessments by Visit | Week 12 | 71.353 Score on a scale | Standard Deviation 20.2515 |
Secondary
Part 2: Change From Baseline: 5X Sit to Stand by Visit
Change in baseline in time (in seconds), where Baseline= Part 1 Period 1 Pre-dose to complete the Five Times Sit-To-Stand by visit. More time means worse outcome, less time means better outcome.
Time frame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192
Population: All participants for whom 5X Sit to Stand was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Experimental: Elamipretide | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Week 72 | -4.770 seconds | Standard Deviation 3.2043 |
| Experimental: Elamipretide | Part 2: Change From Baseline: 5X Sit to Stand by Visit | End of Part 1 | -0.773 seconds | Standard Deviation 2.5323 |
| Experimental: Elamipretide | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Week 12 | -1.505 seconds | Standard Deviation 2.1843 |
| Experimental: Elamipretide | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Week 24 | -2.533 seconds | Standard Deviation 2.7507 |
| Experimental: Elamipretide | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Week 36 | -3.700 seconds | Standard Deviation 3.7649 |
| Experimental: Elamipretide | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Week 48 | -4.780 seconds | Standard Deviation 2.982 |
| Experimental: Elamipretide | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Week 96 | -8.060 seconds | — |
| Experimental: Elamipretide | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Week 168 | -4.320 seconds | Standard Deviation 4.0307 |
| Experimental: Elamipretide | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Week 192 | -6.850 seconds | — |
| Placebo | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Week 192 | -0.390 seconds | Standard Deviation 1.5839 |
| Placebo | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Week 48 | -0.082 seconds | Standard Deviation 2.6625 |
| Placebo | Part 2: Change From Baseline: 5X Sit to Stand by Visit | End of Part 1 | 0.400 seconds | Standard Deviation 2.7824 |
| Placebo | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Week 72 | -0.454 seconds | Standard Deviation 1.6369 |
| Placebo | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Week 12 | 0.384 seconds | Standard Deviation 3.7167 |
| Placebo | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Week 168 | -0.986 seconds | Standard Deviation 2.1857 |
| Placebo | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Week 24 | -1.160 seconds | Standard Deviation 2.8668 |
| Placebo | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Week 96 | -0.397 seconds | Standard Deviation 3.6036 |
| Placebo | Part 2: Change From Baseline: 5X Sit to Stand by Visit | Week 36 | -0.344 seconds | Standard Deviation 2.7035 |
Secondary
Part 2: Change From Baseline: CGI Symptom Scale
Change from Baseline: CGI Symptom Scale by visit where Baseline= Part 1 Period 1 Pre-dose. Clinician Global Impression Scale: Clinician-reported overall health status at end of treatment, administered at Week 12 for Period 1 and Period 2. Scores range from 0-6 for both adolescents and adults. PGI Scale is as follows: 1= Very much Better, 2= Moderately Better, 3= A Little Better, 4= No Change, and 5= A Little Worse 6= Very much Worse. Change from baseline: higher score means worse health status, means worse outcome; lower score means better health status, means better outcome.
Time frame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192
Population: All participants for whom Patient Global Impression Scales of Symptoms was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Experimental: Elamipretide | Part 2: Change From Baseline: CGI Symptom Scale | Week 96 | -1.0 score on a scale | Standard Deviation 0 |
| Experimental: Elamipretide | Part 2: Change From Baseline: CGI Symptom Scale | Week 120 | -1.7 score on a scale | Standard Deviation 0.58 |
| Experimental: Elamipretide | Part 2: Change From Baseline: CGI Symptom Scale | Week 144 | -1.3 score on a scale | Standard Deviation 0.58 |
| Experimental: Elamipretide | Part 2: Change From Baseline: CGI Symptom Scale | Week 168 | -1.3 score on a scale | Standard Deviation 0.58 |
| Experimental: Elamipretide | Part 2: Change From Baseline: CGI Symptom Scale | Week 192 | -1.0 score on a scale | — |
| Experimental: Elamipretide | Part 2: Change From Baseline: CGI Symptom Scale | Week 12 | -0.4 score on a scale | Standard Deviation 0.55 |
| Experimental: Elamipretide | Part 2: Change From Baseline: CGI Symptom Scale | Week 24 | -1.3 score on a scale | Standard Deviation 0.96 |
| Experimental: Elamipretide | Part 2: Change From Baseline: CGI Symptom Scale | Week 36 | -1.3 score on a scale | Standard Deviation 0.58 |
| Experimental: Elamipretide | Part 2: Change From Baseline: CGI Symptom Scale | Week 48 | -1.3 score on a scale | Standard Deviation 0.58 |
| Experimental: Elamipretide | Part 2: Change From Baseline: CGI Symptom Scale | Week 72 | -1.3 score on a scale | Standard Deviation 0.58 |
| Placebo | Part 2: Change From Baseline: CGI Symptom Scale | Week 36 | -0.2 score on a scale | Standard Deviation 0.84 |
| Placebo | Part 2: Change From Baseline: CGI Symptom Scale | Week 96 | -0.7 score on a scale | Standard Deviation 0.58 |
| Placebo | Part 2: Change From Baseline: CGI Symptom Scale | Week 12 | 0.0 score on a scale | Standard Deviation 0 |
| Placebo | Part 2: Change From Baseline: CGI Symptom Scale | Week 120 | -1.0 score on a scale | Standard Deviation 0.71 |
| Placebo | Part 2: Change From Baseline: CGI Symptom Scale | Week 72 | -0.6 score on a scale | Standard Deviation 0.55 |
| Placebo | Part 2: Change From Baseline: CGI Symptom Scale | Week 144 | -0.6 score on a scale | Standard Deviation 0.55 |
| Placebo | Part 2: Change From Baseline: CGI Symptom Scale | Week 24 | -0.4 score on a scale | Standard Deviation 0.55 |
| Placebo | Part 2: Change From Baseline: CGI Symptom Scale | Week 168 | -0.8 score on a scale | Standard Deviation 0.84 |
| Placebo | Part 2: Change From Baseline: CGI Symptom Scale | Week 48 | -0.4 score on a scale | Standard Deviation 0.55 |
| Placebo | Part 2: Change From Baseline: CGI Symptom Scale | Week 192 | -1.5 score on a scale | Standard Deviation 0.71 |
Secondary
Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit
Muscle strength as measured by handheld dynamometry (HHD) in Newtons. Handheld dynamometry assesses the average strength of the knee extensors of both legs. Change from baseline where Baseline= Part 1 Period 1 Pre-dose: the greater positive change, the better the outcome, the smaller positive change (or negative change) the number the worse the outcome.
Time frame: Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192
Population: All participants for whom Muscle Strength by HHD (Newtons) was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Experimental: Elamipretide | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Week 48 | 69.613 Newtons | Standard Deviation 20.8788 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Week 12 | 33.606 Newtons | Standard Deviation 24.7304 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Week 24 | 54.073 Newtons | Standard Deviation 29.7699 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Week 36 | 68.798 Newtons | Standard Deviation 22.8892 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Week 72 | 62.089 Newtons | Standard Deviation 33.4753 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Week 96 | 93.745 Newtons | — |
| Experimental: Elamipretide | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Week 168 | 57.751 Newtons | Standard Deviation 17.7509 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Week 192 | 42.368 Newtons | — |
| Placebo | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Week 192 | 32.140 Newtons | Standard Deviation 17.9287 |
| Placebo | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Week 72 | 32.071 Newtons | Standard Deviation 31.7087 |
| Placebo | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Week 12 | 42.147 Newtons | Standard Deviation 34.9498 |
| Placebo | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Week 168 | 61.897 Newtons | Standard Deviation 19.1573 |
| Placebo | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Week 24 | 54.068 Newtons | Standard Deviation 37.306 |
| Placebo | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Week 96 | 38.848 Newtons | Standard Deviation 18.1422 |
| Placebo | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Week 36 | 48.351 Newtons | Standard Deviation 31.1331 |
| Placebo | Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit | Week 48 | 45.704 Newtons | Standard Deviation 56.8895 |
Secondary
Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit
Part 2: Change from Baseline: Patient Global Impression Scales of Symptoms by Visit Patient-reported health status over the past week where Baseline= Part 1 Period 1 Pre-dose, administered at Week 12 for Period 1 and Period 2. Scores range from 0-40 for adolescents and 0-50 for adults. PGI Scale is as follows: 1=None, 2=Mild, 3=Moderate, 4=Severe, and 5=Very Severe. Change from baseline: Higher score means worse health status, means worse outcome; lower score means better health status, means better outcome.
Time frame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192
Population: All participants for whom Patient Global Impression Scales of Symptoms was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Experimental: Elamipretide | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 36 | -0.7 score on a scale | Standard Deviation 1.15 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 96 | -0.5 score on a scale | Standard Deviation 2.12 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 24 | -0.3 score on a scale | Standard Deviation 0.5 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 120 | -0.7 score on a scale | Standard Deviation 1.15 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 48 | -0.7 score on a scale | Standard Deviation 1.15 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 144 | -1.0 score on a scale | Standard Deviation 1.41 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 12 | -0.2 score on a scale | Standard Deviation 1.1 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 168 | -0.7 score on a scale | Standard Deviation 1.15 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 72 | -0.7 score on a scale | Standard Deviation 1.15 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 192 | -2.0 score on a scale | — |
| Experimental: Elamipretide | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | End of Part 1 | 0.0 score on a scale | Standard Deviation 0 |
| Placebo | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 192 | -0.5 score on a scale | Standard Deviation 0.71 |
| Placebo | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | End of Part 1 | -0.4 score on a scale | Standard Deviation 0.55 |
| Placebo | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 12 | -0.6 score on a scale | Standard Deviation 0.55 |
| Placebo | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 24 | -0.4 score on a scale | Standard Deviation 0.55 |
| Placebo | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 36 | -0.6 score on a scale | Standard Deviation 0.55 |
| Placebo | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 48 | 0.0 score on a scale | Standard Deviation 0 |
| Placebo | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 72 | -0.4 score on a scale | Standard Deviation 0.55 |
| Placebo | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 96 | -0.3 score on a scale | Standard Deviation 0.58 |
| Placebo | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 120 | -0.6 score on a scale | Standard Deviation 0.55 |
| Placebo | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 144 | -0.8 score on a scale | Standard Deviation 0.45 |
| Placebo | Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit | Week 168 | -0.6 score on a scale | Standard Deviation 0.55 |
Secondary
Part 2: Change From Baseline: SWAY Application Balance Assessment
Change from Baseline: SWAY Application Balance Assessment,where Baseline= Part 1 Period 1 Pre-doseSWAY application balance assessment consists of five stances each performed for 10 seconds and scores postural stability on a scale from 0-100 with higher scores indicating better postural stability. An increase from baseline is a better outcome and a decrease in score is a worse outcome.
Time frame: Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192
Population: All participants for whom SWAY Application Balance Assessment was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Experimental: Elamipretide | Part 2: Change From Baseline: SWAY Application Balance Assessment | Week 12 | -3.152 score on a scale | Standard Deviation 29.8419 |
| Experimental: Elamipretide | Part 2: Change From Baseline: SWAY Application Balance Assessment | Week 24 | 12.190 score on a scale | Standard Deviation 9.6799 |
| Experimental: Elamipretide | Part 2: Change From Baseline: SWAY Application Balance Assessment | Week 36 | 0.807 score on a scale | Standard Deviation 14.9493 |
| Experimental: Elamipretide | Part 2: Change From Baseline: SWAY Application Balance Assessment | Week 48 | 5.773 score on a scale | Standard Deviation 7.8814 |
| Experimental: Elamipretide | Part 2: Change From Baseline: SWAY Application Balance Assessment | Week 72 | 10.420 score on a scale | Standard Deviation 1.7501 |
| Experimental: Elamipretide | Part 2: Change From Baseline: SWAY Application Balance Assessment | Week 96 | 12.840 score on a scale | — |
| Experimental: Elamipretide | Part 2: Change From Baseline: SWAY Application Balance Assessment | Week 168 | 21.127 score on a scale | Standard Deviation 14.1854 |
| Experimental: Elamipretide | Part 2: Change From Baseline: SWAY Application Balance Assessment | Week 192 | 15.100 score on a scale | — |
| Placebo | Part 2: Change From Baseline: SWAY Application Balance Assessment | Week 192 | 13.080 score on a scale | Standard Deviation 16.3483 |
| Placebo | Part 2: Change From Baseline: SWAY Application Balance Assessment | Week 12 | 10.464 score on a scale | Standard Deviation 19.1971 |
| Placebo | Part 2: Change From Baseline: SWAY Application Balance Assessment | Week 72 | 15.240 score on a scale | Standard Deviation 14.3557 |
| Placebo | Part 2: Change From Baseline: SWAY Application Balance Assessment | Week 24 | 6.664 score on a scale | Standard Deviation 9.5896 |
| Placebo | Part 2: Change From Baseline: SWAY Application Balance Assessment | Week 168 | 19.644 score on a scale | Standard Deviation 18.6821 |
| Placebo | Part 2: Change From Baseline: SWAY Application Balance Assessment | Week 36 | 9.484 score on a scale | Standard Deviation 11.3834 |
| Placebo | Part 2: Change From Baseline: SWAY Application Balance Assessment | Week 96 | 18.433 score on a scale | Standard Deviation 21.3323 |
| Placebo | Part 2: Change From Baseline: SWAY Application Balance Assessment | Week 48 | 15.260 score on a scale | Standard Deviation 16.254 |
Secondary
Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit
Change from Baseline (mean) in Total Fatigue Score (Q1, Q2, and Q4) Based on the BarTH Syndrome Symptom Assessment (BTHS-SA) by visit. The BTHS-SA is 3-question fatigue assessment using a 0-4 point scale for each question where no fatigue =0 and very severe fatigue =4, with scores from all 3 questions combined and averaged for all subjects by visit; a lower score means better outcome, higher score means a worse outcome. Change from baseline: an increase in scores would mean worse outcome, a decrease in scores means better outcome. Combined Min score=-12, max score =12. Weekly values are based on the weekly average of the last 7 days before the site or visiting nurse visit for both periods. Baseline= Period 1 Predose.
Time frame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192
Population: All participants for whom Total Fatigue Score was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Experimental: Elamipretide | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 192 | -5.286 score on a scale | — |
| Experimental: Elamipretide | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 24 | -1.500 score on a scale | Standard Deviation 2.0253 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 168 | -2.333 score on a scale | Standard Deviation 4.0515 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 36 | -2.667 score on a scale | Standard Deviation 3.6821 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Baseline | -2.425 score on a scale | Standard Deviation 2.0849 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 48 | -1.000 score on a scale | Standard Deviation 2.9172 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 144 | -2.667 score on a scale | Standard Deviation 2.2961 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 72 | -2.667 score on a scale | Standard Deviation 3.4827 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 12 | -1.800 score on a scale | Standard Deviation 2.4838 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 96 | -3.500 score on a scale | Standard Deviation 2.5254 |
| Experimental: Elamipretide | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 120 | -3.000 score on a scale | Standard Deviation 2.9172 |
| Placebo | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 96 | -0.357 score on a scale | Standard Deviation 1.5763 |
| Placebo | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 120 | -0.114 score on a scale | Standard Deviation 1.0309 |
| Placebo | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 144 | -0.314 score on a scale | Standard Deviation 2.0067 |
| Placebo | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 168 | -0.514 score on a scale | Standard Deviation 0.7309 |
| Placebo | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 192 | -1.107 score on a scale | Standard Deviation 0.5556 |
| Placebo | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Baseline | -1.540 score on a scale | Standard Deviation 1.3653 |
| Placebo | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 12 | -1.314 score on a scale | Standard Deviation 1.4237 |
| Placebo | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 24 | -0.314 score on a scale | Standard Deviation 1.0816 |
| Placebo | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 36 | -1.714 score on a scale | Standard Deviation 1.0583 |
| Placebo | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 48 | -0.314 score on a scale | Standard Deviation 1.065 |
| Placebo | Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit | Week 72 | -1.114 score on a scale | Standard Deviation 1.1301 |
Secondary
Part 2: Distance Walked During the 6MWT
Change from baseline for distance walked in meters during the 6-minute walk test (6MWT) by visit where Baseline= Part 1 Period 1 Pre-dose
Time frame: Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192
Population: All participants for whom distance walked in 6MWT was measured.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Experimental: Elamipretide | Part 2: Distance Walked During the 6MWT | Part 2: Week 12 | 45.4 meters | Standard Deviation 55.73 |
| Experimental: Elamipretide | Part 2: Distance Walked During the 6MWT | Part 2: Week 24 | 79.8 meters | Standard Deviation 23.54 |
| Experimental: Elamipretide | Part 2: Distance Walked During the 6MWT | Part 2: Week 36 | 68.0 meters | Standard Deviation 31.19 |
| Experimental: Elamipretide | Part 2: Distance Walked During the 6MWT | Part 2: Week 48 | 87.3 meters | Standard Deviation 25.58 |
| Experimental: Elamipretide | Part 2: Distance Walked During the 6MWT | Part 2: Week 72 | 121.7 meters | Standard Deviation 20.74 |
| Experimental: Elamipretide | Part 2: Distance Walked During the 6MWT | Part 2: Week 96 | 128.0 meters | — |
| Experimental: Elamipretide | Part 2: Distance Walked During the 6MWT | Part 2: Week 168 | 89.3 meters | Standard Deviation 32.75 |
| Experimental: Elamipretide | Part 2: Distance Walked During the 6MWT | Part 2: Week 192 | 118.0 meters | — |
| Placebo | Part 2: Distance Walked During the 6MWT | Part 2: Week 192 | 125.0 meters | Standard Deviation 28.28 |
| Placebo | Part 2: Distance Walked During the 6MWT | Part 2: Week 12 | 75.6 meters | Standard Deviation 87.25 |
| Placebo | Part 2: Distance Walked During the 6MWT | Part 2: Week 72 | 97.8 meters | Standard Deviation 117.53 |
| Placebo | Part 2: Distance Walked During the 6MWT | Part 2: Week 24 | 100.4 meters | Standard Deviation 94.61 |
| Placebo | Part 2: Distance Walked During the 6MWT | Part 2: Week 168 | 100.2 meters | Standard Deviation 78.1 |
| Placebo | Part 2: Distance Walked During the 6MWT | Part 2: Week 36 | 112.6 meters | Standard Deviation 119.35 |
| Placebo | Part 2: Distance Walked During the 6MWT | Part 2: Week 96 | 63.7 meters | Standard Deviation 69.14 |
| Placebo | Part 2: Distance Walked During the 6MWT | Part 2: Week 48 | 103.4 meters | Standard Deviation 102.02 |