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Trial Outcomes & Findings for Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM) (NCT NCT02976038)

NCT ID: NCT02976038

Last Updated: 2021-12-17

Results Overview

Change From Baseline in Distance Walked (meters) on the Six-Minute Walk Test by Visit

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

28 participants

Primary outcome timeframe

Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

Results posted on

2021-12-17

Participant Flow

Participant milestones

Participant milestones
Measure
Elamipretide
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Overall Study
STARTED
28
Overall Study
COMPLETED
0
Overall Study
NOT COMPLETED
28

Reasons for withdrawal

Reasons for withdrawal
Measure
Elamipretide
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Overall Study
Physician Decision
22
Overall Study
Withdrawal by Subject
3
Overall Study
Lost to Follow-up
2
Overall Study
Adverse Event
1

Baseline Characteristics

Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Elamipretide
n=28 Participants
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Age, Continuous
47.8 years
STANDARD_DEVIATION 11.30 • n=5 Participants
Sex: Female, Male
Female
24 Participants
n=5 Participants
Sex: Female, Male
Male
4 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
1 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
27 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
Race (NIH/OMB)
White
27 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
1 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Baseline weight
65.93 kg
STANDARD_DEVIATION 14.614 • n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

Population: All participants for whom Change From Baseline in Distance Walked (meters) on the Six-Minute Walk Test by Visit was measured.

Change From Baseline in Distance Walked (meters) on the Six-Minute Walk Test by Visit

Outcome measures

Outcome measures
Measure
Elamipretide
n=28 Participants
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Slight Problem
Participant reports symptom is a "slight problem"
Moderate Problem
Participant reports symptom is a "moderate problem"
Severe Problem
Participant reports symptom is a "severe problem"
Extreme Problem
Participant reports symptom is an "extreme problem"
Distance Walked on Six Minute Walk Test
Week 13
3.6 meters
Standard Deviation 52.68
—
—
—
—
Distance Walked on Six Minute Walk Test
Week 26
-8.6 meters
Standard Deviation 56.33
—
—
—
—
Distance Walked on Six Minute Walk Test
Week 52
-17.6 meters
Standard Deviation 70.60
—
—
—
—
Distance Walked on Six Minute Walk Test
Week 78
-26.2 meters
Standard Deviation 73.17
—
—
—
—
Distance Walked on Six Minute Walk Test
Week 104
-20.9 meters
Standard Deviation 77.78
—
—
—
—
Distance Walked on Six Minute Walk Test
Week 130
-18.9 meters
Standard Deviation 76.94
—
—
—
—
Distance Walked on Six Minute Walk Test
Week 156
-35.0 meters
Standard Deviation NA
Only 1 participant.
—
—
—
—
Distance Walked on Six Minute Walk Test
End of Trial (Week 160)
-54.0 meters
Standard Deviation 70.30
—
—
—
—

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

Population: All participants for whom EQ-5D-5L was measured. Trial was terminated early.

EuroQol 5 dimensions 5 levels (EQ-5D-5L) by visit. Count of participants per arm, or severity level of dimension. Measures 5 dimensions: mobility, self-care, usual activities, pain/discomfort, anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. Score ranges from 5-25 with lower score=better outcome, and higher score means a worse outcome.

Outcome measures

Outcome measures
Measure
Elamipretide
n=28 Participants
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Slight Problem
n=28 Participants
Participant reports symptom is a "slight problem"
Moderate Problem
n=28 Participants
Participant reports symptom is a "moderate problem"
Severe Problem
n=28 Participants
Participant reports symptom is a "severe problem"
Extreme Problem
n=28 Participants
Participant reports symptom is an "extreme problem"
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Baseline: : Mobility
2 Participants
10 Participants
11 Participants
5 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 104 Anxiety/Depression
10 Participants
7 Participants
4 Participants
0 Participants
1 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 104 : Mobility
4 Participants
5 Participants
10 Participants
2 Participants
1 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 104 : Pain/Discomfort
5 Participants
8 Participants
9 Participants
0 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 104 : Self-Care
15 Participants
4 Participants
1 Participants
2 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 104 : Usual Activities
3 Participants
13 Participants
4 Participants
2 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 130 Anxiety/Depression
7 Participants
8 Participants
3 Participants
4 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 130 : Mobility
7 Participants
4 Participants
8 Participants
3 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 130 : Pain/Discomfort
7 Participants
6 Participants
5 Participants
4 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 130 : Self-Care
14 Participants
5 Participants
1 Participants
2 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 130 : Usual Activities
6 Participants
8 Participants
6 Participants
1 Participants
1 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 156 Anxiety/Depression
0 Participants
0 Participants
1 Participants
0 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 156 : Mobility
1 Participants
0 Participants
0 Participants
0 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 156 : Pain/Discomfort
0 Participants
0 Participants
1 Participants
0 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 156 : Self-Care
1 Participants
0 Participants
0 Participants
0 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 156 : Usual Activities
0 Participants
1 Participants
0 Participants
0 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
End of Study (Week 160) Anxiety/Depression
9 Participants
6 Participants
4 Participants
1 Participants
1 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
End of Study (Week 160) : Mobility
3 Participants
5 Participants
9 Participants
4 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
21End of Study (Week 160) : Pain/Discomfort
4 Participants
7 Participants
8 Participants
2 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
End of Study (Week 160) : Self-Care
9 Participants
8 Participants
3 Participants
1 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
End of Study (Week 160) : Usual Activities
5 Participants
7 Participants
6 Participants
2 Participants
1 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Baseline: Anxiety/Depression
12 Participants
8 Participants
4 Participants
3 Participants
1 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Baseline: : Pain/Discomfort
4 Participants
5 Participants
15 Participants
4 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Baseline: : Self-Care
12 Participants
10 Participants
5 Participants
0 Participants
1 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Baseline: : Usual Activities
5 Participants
7 Participants
12 Participants
4 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 13 Anxiety/Depression
16 Participants
6 Participants
5 Participants
1 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 13 : Mobility
7 Participants
7 Participants
12 Participants
1 Participants
1 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 13 : Pain/Discomfort
4 Participants
12 Participants
8 Participants
3 Participants
1 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 13 : Self-Care
18 Participants
7 Participants
2 Participants
0 Participants
1 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 13 : Usual Activities
6 Participants
15 Participants
5 Participants
0 Participants
2 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 26 Anxiety/Depression
16 Participants
6 Participants
5 Participants
0 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 26 : Mobility
5 Participants
6 Participants
12 Participants
4 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 26 : Pain/Discomfort
8 Participants
11 Participants
5 Participants
2 Participants
1 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 26 : Self-Care
17 Participants
7 Participants
3 Participants
0 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 26 : Usual Activities
6 Participants
13 Participants
7 Participants
1 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 56 Anxiety/Depression
14 Participants
5 Participants
4 Participants
1 Participants
1 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 56 : Mobility
6 Participants
6 Participants
4 Participants
9 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 56 : Pain/Discomfort
6 Participants
8 Participants
9 Participants
2 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 56 : Self-Care
16 Participants
6 Participants
2 Participants
1 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 56: Usual Activities
8 Participants
9 Participants
5 Participants
3 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 78 Anxiety/Depression
12 Participants
6 Participants
3 Participants
2 Participants
1 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 78 : Mobility
5 Participants
8 Participants
8 Participants
2 Participants
1 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 78 : Pain/Discomfort
4 Participants
14 Participants
5 Participants
1 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 78 : Self-Care
15 Participants
6 Participants
2 Participants
1 Participants
0 Participants
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
Week 78 : Usual Activities
5 Participants
12 Participants
6 Participants
1 Participants
0 Participants

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

Population: All participants for whom EQ VAS was measured. Trial was terminated early.

Euroquol Visual Analog Scale (EQ VAS): A patient-reported measure. The visual scale is numbered from 0 to 100, participants are directed as follows: "100 means the best health you can imagine, 0 means the worst health you can imagine. Mark an X on the scale to indicate how your health is TODAY." Higher scores mean better outcome, lower scores mean worse outcome.

Outcome measures

Outcome measures
Measure
Elamipretide
n=28 Participants
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Slight Problem
Participant reports symptom is a "slight problem"
Moderate Problem
Participant reports symptom is a "moderate problem"
Severe Problem
Participant reports symptom is a "severe problem"
Extreme Problem
Participant reports symptom is an "extreme problem"
Euroquol Visual Analog Scale
Baseline
65.29 score on a scale
Standard Deviation 17.83
—
—
—
—
Euroquol Visual Analog Scale
Week 13
65.44 score on a scale
Standard Deviation 22.68
—
—
—
—
Euroquol Visual Analog Scale
Week 26
62.70 score on a scale
Standard Deviation 21.23
—
—
—
—
Euroquol Visual Analog Scale
Week 52
70.52 score on a scale
Standard Deviation 21.27
—
—
—
—
Euroquol Visual Analog Scale
Week 78
64.88 score on a scale
Standard Deviation 22.18
—
—
—
—
Euroquol Visual Analog Scale
Week 104
67.23 score on a scale
Standard Deviation 21.75
—
—
—
—
Euroquol Visual Analog Scale
Week 130
72.18 score on a scale
Standard Deviation 21.38
—
—
—
—
Euroquol Visual Analog Scale
Week 156
60.00 score on a scale
Standard Deviation NA
Only 1 participant, so there is no standard deviation
—
—
—
—
Euroquol Visual Analog Scale
End of Trial (Week 160)
60.48 score on a scale
Standard Deviation 23.28
—
—
—
—

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

Population: All participants for whom Neuro-Qol Fatigue was measured. Trial was terminated early.

Change from baseline in NeuroQOL Fatigue Questionnaire by visit. Questionnaire is comprised of 8 questions measuring participants incidence of fatigue affecting activities of daily living. Participants respond by choosing one of the following: 1=never, 2=rarely, 3=sometimes, 4=often, 5=always, with a lower score meaning a better outcome with fatigue affecting participant less, and high score meaning a worse outcome, with fatigue affecting the participant more. Total raw scores range from 8-40. Scores were converted to percentages: range0-100%. Change from baseline score: negative number means improved from baseline, positive number means worsened compared to baseline.

Outcome measures

Outcome measures
Measure
Elamipretide
n=28 Participants
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Slight Problem
Participant reports symptom is a "slight problem"
Moderate Problem
Participant reports symptom is a "moderate problem"
Severe Problem
Participant reports symptom is a "severe problem"
Extreme Problem
Participant reports symptom is an "extreme problem"
NeuroQOL Fatigue Questionnaire
Week 13
-8.39 score on a scale
Standard Deviation 16.43
—
—
—
—
NeuroQOL Fatigue Questionnaire
Week 26
-9.33 score on a scale
Standard Deviation 16.40
—
—
—
—
NeuroQOL Fatigue Questionnaire
Week 52
-10.88 score on a scale
Standard Deviation 17.48
—
—
—
—
NeuroQOL Fatigue Questionnaire
Week 78
-6.79 score on a scale
Standard Deviation 17.07
—
—
—
—
NeuroQOL Fatigue Questionnaire
Week 104
-8.27 score on a scale
Standard Deviation 18.61
—
—
—
—
NeuroQOL Fatigue Questionnaire
Week 130
-6.77 score on a scale
Standard Deviation 15.69
—
—
—
—
NeuroQOL Fatigue Questionnaire
Week 156
13.00 score on a scale
Standard Deviation NA
Only 1 participant was measured.
—
—
—
—
NeuroQOL Fatigue Questionnaire
End of Study (Week 21 up to Week 160)
-2.67 score on a scale
Standard Deviation 17.38
—
—
—
—

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

Population: All participants for whom PGA was measured. Trial was terminated early.

Patient Global Assessment \[PGA\] Score by Visit, count of participants. Patient-reported current health status by visit. PGA Scale is as follows: 1=Excellent, 2=Very good, 3=Good, 4=Fair, and 5=Poor. Total score ranges from 1-5. Higher score means worse health status, means worse outcome.

Outcome measures

Outcome measures
Measure
Elamipretide
n=28 Participants
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Slight Problem
Participant reports symptom is a "slight problem"
Moderate Problem
Participant reports symptom is a "moderate problem"
Severe Problem
Participant reports symptom is a "severe problem"
Extreme Problem
Participant reports symptom is an "extreme problem"
Patient Global Assessment : Categorical
Baseline · Very Good
2 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 13 · Fair
6 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 13 · Good
12 Participants
—
—
—
—
Patient Global Assessment : Categorical
Baseline · Excellent
1 Participants
—
—
—
—
Patient Global Assessment : Categorical
Baseline · Fair
11 Participants
—
—
—
—
Patient Global Assessment : Categorical
Baseline · Good
9 Participants
—
—
—
—
Patient Global Assessment : Categorical
Baseline · Poor
5 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 13 · Excellent
0 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 13 · Poor
2 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 13 · Very Good
6 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 26 · Excellent
1 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 26 · Fair
10 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 26 · Good
8 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 26 · Poor
3 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 26 · Very Good
5 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 52 · Excellent
0 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 52 · Fair
3 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 52 · Good
13 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 52 · Poor
4 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 52 · Very Good
5 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 78 · Excellent
0 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 78 · Fair
7 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 78 · Good
10 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 78 · Poor
4 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 78 · Very Good
3 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 104 · Excellent
0 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 104 · Fair
7 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 104 · Good
8 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 104 · Poor
2 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 104 · Very Good
5 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 130 · Excellent
1 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 130 · Fair
6 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 130 · Good
10 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 130 · Poor
4 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 130 · Very Good
1 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 156 · Excellent
0 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 156 · Fair
1 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 156 · Good
0 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 156 · Poor
0 Participants
—
—
—
—
Patient Global Assessment : Categorical
Week 156 · Very Good
0 Participants
—
—
—
—
Patient Global Assessment : Categorical
End of Study End of Study (Week 21 up to Week 160) · Excellent
0 Participants
—
—
—
—
Patient Global Assessment : Categorical
End of Study End of Study (Week 21 up to Week 160) · Fair
8 Participants
—
—
—
—
Patient Global Assessment : Categorical
End of Study End of Study (Week 21 up to Week 160) · Good
9 Participants
—
—
—
—
Patient Global Assessment : Categorical
End of Study End of Study (Week 21 up to Week 160) · Poor
3 Participants
—
—
—
—
Patient Global Assessment : Categorical
End of Study End of Study (Week 21 up to Week 160) · Very Good
1 Participants
—
—
—
—

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

Population: All participants for whom Change From Baseline in Patient Global Assessment was measured.

Patient Global Assessment \[PGA\] Score by Visit, Patient-reported current health status by visit. PGA Scale is as follows: 1=Excellent, 2=Very good, 3=Good, 4=Fair, and 5=Poor. Total score ranges from 1-5. Higher score means worse health status, lower score means worse outcome.

Outcome measures

Outcome measures
Measure
Elamipretide
n=28 Participants
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Slight Problem
Participant reports symptom is a "slight problem"
Moderate Problem
Participant reports symptom is a "moderate problem"
Severe Problem
Participant reports symptom is a "severe problem"
Extreme Problem
Participant reports symptom is an "extreme problem"
Patient Global Assessment: Continuous Variable
Week 104
3.27 score on a scale
Standard Deviation 0.94
—
—
—
—
Patient Global Assessment: Continuous Variable
Baseline
3.61 score on a scale
Standard Deviation 0.99
—
—
—
—
Patient Global Assessment: Continuous Variable
Week 13
3.15 score on a scale
Standard Deviation 0.88
—
—
—
—
Patient Global Assessment: Continuous Variable
Week 26
3.15 score on a scale
Standard Deviation 0.88
—
—
—
—
Patient Global Assessment: Continuous Variable
Week 52
3.24 score on a scale
Standard Deviation 0.97
—
—
—
—
Patient Global Assessment: Continuous Variable
Week 78
3.50 score on a scale
Standard Deviation 0.93
—
—
—
—
Patient Global Assessment: Continuous Variable
Week 130
3.50 score on a scale
Standard Deviation 1.01
—
—
—
—
Patient Global Assessment: Continuous Variable
Week 156
4.00 score on a scale
Standard Deviation NA
Only 1 participant was measured. There is no standard deviation.
—
—
—
—
Patient Global Assessment: Continuous Variable
End of Trial (Week 160)
3.62 score on a scale
Standard Deviation 0.80
—
—
—
—

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

Population: All individuals for whom Work Limitations was measured. Trial was terminated early.

Work Limitations Questionnaire by visit. Comprised of 25 items, in 4 categories, or demands: Mental/Interpersonal, Output Tasks, Physical Tasks, and Time Management, measuring the degree to which employed individuals are experiencing limitations on-the-job due to their health problems, and health-related productivity loss. 1= All of the time (100%), 2= most of the time, 3=some of the time (about 50%), 4=slight bit of time, 5=none of the time (0%), 6=does not apply to my job. (Presenteeism). The WLQ items ask respondents to rate their level of difficulty or ability to perform specific job demands. Scale score range from 0 (limited none of the time=better outcome) to 100 (limited all of the time= worse outcome) and represent the reported amount of time in the prior two weeks respondents were limited on-the-job.

Outcome measures

Outcome measures
Measure
Elamipretide
n=28 Participants
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Slight Problem
Participant reports symptom is a "slight problem"
Moderate Problem
Participant reports symptom is a "moderate problem"
Severe Problem
Participant reports symptom is a "severe problem"
Extreme Problem
Participant reports symptom is an "extreme problem"
Work Limitations Questionnaire
Mental/Interpersonal: Baseline
67.59 score on a scale
Standard Deviation 15.58
—
—
—
—
Work Limitations Questionnaire
Mental/Interpersonal: Week 13
71.33 score on a scale
Standard Deviation 20.00
—
—
—
—
Work Limitations Questionnaire
Mental/Interpersonal: Week 26
72.00 score on a scale
Standard Deviation 18.54
—
—
—
—
Work Limitations Questionnaire
Mental/Interpersonal: Week 52
75.06 score on a scale
Standard Deviation 19.37
—
—
—
—
Work Limitations Questionnaire
Mental/Interpersonal: Week 78
70.62 score on a scale
Standard Deviation 20.02
—
—
—
—
Work Limitations Questionnaire
Mental/Interpersonal: Week 104
68.57 score on a scale
Standard Deviation 17.42
—
—
—
—
Work Limitations Questionnaire
Mental/Interpersonal: Week 130
66.11 score on a scale
Standard Deviation 24.77
—
—
—
—
Work Limitations Questionnaire
Mental/Interpersonal: End of Trial (Week 160)
74.72 score on a scale
Standard Deviation 19.66
—
—
—
—
Work Limitations Questionnaire
Output Task: Baseline
69.67 score on a scale
Standard Deviation 20.99
—
—
—
—
Work Limitations Questionnaire
Output Task: Week 13
75.20 score on a scale
Standard Deviation 21.89
—
—
—
—
Work Limitations Questionnaire
Output Task: Week 26
75.20 score on a scale
Standard Deviation 18.16
—
—
—
—
Work Limitations Questionnaire
Output Task: Week 52
71.56 score on a scale
Standard Deviation 18.05
—
—
—
—
Work Limitations Questionnaire
Output Task: Week 78
61.50 score on a scale
Standard Deviation 20.28
—
—
—
—
Work Limitations Questionnaire
Output Task: Week 104
59.33 score on a scale
Standard Deviation 25.85
—
—
—
—
Work Limitations Questionnaire
Output Task: Week 130
73.00 score on a scale
Standard Deviation 22.50
—
—
—
—
Work Limitations Questionnaire
Output Task: End of Trial (Week 160)
71.00 score on a scale
Standard Deviation 19.91
—
—
—
—
Work Limitations Questionnaire
Physical Task: Baseline
56.39 score on a scale
Standard Deviation 23.37
—
—
—
—
Work Limitations Questionnaire
Physical Task Week 13
58.67 score on a scale
Standard Deviation 17.37
—
—
—
—
Work Limitations Questionnaire
Physical Task Week 26
55.00 score on a scale
Standard Deviation 11.57
—
—
—
—
Work Limitations Questionnaire
Physical Task Week 52
52.59 score on a scale
Standard Deviation 14.32
—
—
—
—
Work Limitations Questionnaire
Physical Task Week 78
57.78 score on a scale
Standard Deviation 11.55
—
—
—
—
Work Limitations Questionnaire
Physical Task Week 104
56.67 score on a scale
Standard Deviation 25.53
—
—
—
—
Work Limitations Questionnaire
Physical Task Week 130
58.33 score on a scale
Standard Deviation 23.03
—
—
—
—
Work Limitations Questionnaire
Physical Task: End of Trial (Week 160)
58.75 score on a scale
Standard Deviation 23.03
—
—
—
—
Work Limitations Questionnaire
Time Management Baseline
61.60 score on a scale
Standard Deviation 23.26
—
—
—
—
Work Limitations Questionnaire
Time Management Week 13
72.40 score on a scale
Standard Deviation 21.54
—
—
—
—
Work Limitations Questionnaire
Time Management Week 26
68.00 score on a scale
Standard Deviation 24.25
—
—
—
—
Work Limitations Questionnaire
Time Management Week 52
68.89 score on a scale
Standard Deviation 25.83
—
—
—
—
Work Limitations Questionnaire
Time Management Week 78
72.89 score on a scale
Standard Deviation 21.43
—
—
—
—
Work Limitations Questionnaire
Time Management Week 104
56.67 score on a scale
Standard Deviation 27.76
—
—
—
—
Work Limitations Questionnaire
Time Management Week 130
57.71 score on a scale
Standard Deviation 29.11
—
—
—
—
Work Limitations Questionnaire
Time Management End of Trial (Week 160)
68.57 score on a scale
Standard Deviation 24.92
—
—
—
—

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

Population: All participants for whom PhGA was measured. Trial was terminated early.

Physician Global Assessment- Mean Score by visit. PGA Scale asks physician to rate participant's current health status as follows: 1=Excellent, 2=Very good, 3=Good, 4=Fair, and 5=Poor. Total score ranges from 1-5. Higher score means lower health status, worse outcome; lower score means better health status, better outcome.

Outcome measures

Outcome measures
Measure
Elamipretide
n=28 Participants
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Slight Problem
Participant reports symptom is a "slight problem"
Moderate Problem
Participant reports symptom is a "moderate problem"
Severe Problem
Participant reports symptom is a "severe problem"
Extreme Problem
Participant reports symptom is an "extreme problem"
Physician Global Assessment Score (PhGA) by Visit
Baseline
3.32 score on a scale
Standard Deviation 0.61
—
—
—
—
Physician Global Assessment Score (PhGA) by Visit
Week 13
3.11 score on a scale
Standard Deviation 0.80
—
—
—
—
Physician Global Assessment Score (PhGA) by Visit
Week 26
3.00 score on a scale
Standard Deviation 0.73
—
—
—
—
Physician Global Assessment Score (PhGA) by Visit
Week 52
2.72 score on a scale
Standard Deviation 0.84
—
—
—
—
Physician Global Assessment Score (PhGA) by Visit
Week 78
3.25 score on a scale
Standard Deviation 0.79
—
—
—
—
Physician Global Assessment Score (PhGA) by Visit
Week 104
3.18 score on a scale
Standard Deviation 0.85
—
—
—
—
Physician Global Assessment Score (PhGA) by Visit
Week 130
3.18 score on a scale
Standard Deviation 0.73
—
—
—
—
Physician Global Assessment Score (PhGA) by Visit
Week 156
5.00 score on a scale
Standard Deviation NA
Only 1 participant was measured.
—
—
—
—
Physician Global Assessment Score (PhGA) by Visit
End of Study (Week 160)
3.40 score on a scale
Standard Deviation 0.60
—
—
—
—

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

Population: All participants for whom PhGA was measured. Trial was terminated early.

Physician Global Assessment \[PhGA\] Score by Visit, Physician-reported current health status by visit. PhGA Scale is as follows: 1=Excellent, 2=Very good, 3=Good, 4=Fair, and 5=Poor. Total score ranges from 1-5. Higher score means worse health status, means worse outcome.

Outcome measures

Outcome measures
Measure
Elamipretide
n=28 Participants
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Slight Problem
n=28 Participants
Participant reports symptom is a "slight problem"
Moderate Problem
n=28 Participants
Participant reports symptom is a "moderate problem"
Severe Problem
n=28 Participants
Participant reports symptom is a "severe problem"
Extreme Problem
n=28 Participants
Participant reports symptom is an "extreme problem"
Physician Global Assessment (PhGA): Categorical
End of Study (Week 160)
0 Participants
6 Participants
13 Participants
1 Participants
0 Participants
Physician Global Assessment (PhGA): Categorical
Baseline
0 Participants
8 Participants
18 Participants
1 Participants
1 Participants
Physician Global Assessment (PhGA): Categorical
Week 13
0 Participants
7 Participants
13 Participants
1 Participants
6 Participants
Physician Global Assessment (PhGA): Categorical
Week 26
1 Participants
6 Participants
16 Participants
0 Participants
4 Participants
Physician Global Assessment (PhGA): Categorical
Week 52
1 Participants
2 Participants
12 Participants
1 Participants
9 Participants
Physician Global Assessment (PhGA): Categorical
Week 78
0 Participants
8 Participants
11 Participants
1 Participants
4 Participants
Physician Global Assessment (PhGA): Categorical
Week 104
0 Participants
4 Participants
12 Participants
2 Participants
4 Participants
Physician Global Assessment (PhGA): Categorical
Week 130
0 Participants
5 Participants
13 Participants
1 Participants
3 Participants
Physician Global Assessment (PhGA): Categorical
Week 156
0 Participants
0 Participants
0 Participants
1 Participants
0 Participants

SECONDARY outcome

Timeframe: Baseline, Week 13, 26, 52, 104, 156, End of Study (Week 160)

Population: All participants for which PMMSA was measured. Trial was terminated early.

The PMMSA is a patient-reported outcome measure, assessing a participant's "worst feeling" of 10 symptoms during the last 24 hours: tiredness at rest, tiredness during activities, muscle weakness at rest, muscle weakness during activities, balance, vision, abdominal discomfort, muscle pain, numbness, and headache using a 4-point scale: 1=Not at all, 2=Mild, 3=Moderate, and 4=Severe. Max Score =40= worse outcome, Min score= 10= better outcome on scale. PMMSA Individual Items shows number or participants with mild, moderate, severe or no symptoms at all.

Outcome measures

Outcome measures
Measure
Elamipretide
n=28 Participants
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Slight Problem
Participant reports symptom is a "slight problem"
Moderate Problem
Participant reports symptom is a "moderate problem"
Severe Problem
Participant reports symptom is a "severe problem"
Extreme Problem
Participant reports symptom is an "extreme problem"
PMMSA Individual Items
Week 104: Abdominal Discomfort · Mild
7 Participants
—
—
—
—
PMMSA Individual Items
Week 104: Abdominal Discomfort · Not At All
10 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Vision Problems · Severe
4 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Muscle Weakness during Activities · Mild
12 Participants
—
—
—
—
PMMSA Individual Items
Week 52:Vision Problems · Not At All
5 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Vision Problems · Not At All
6 Participants
—
—
—
—
PMMSA Individual Items
Week 104: Abdominal Discomfort · Moderate
4 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Muscle Weakness during Activities · Moderate
8 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Muscle Weakness during Activities · Not At All
3 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Abdominal Discomfort · Moderate
1 Participants
—
—
—
—
PMMSA Individual Items
Week 130:Muscle Weakness at Rest · Mild
9 Participants
—
—
—
—
PMMSA Individual Items
Week 130:Numbness · Severe
0 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Balance Problems · Not At All
0 Participants
—
—
—
—
PMMSA Individual Items
Week 104: Abdominal Discomfort · Severe
1 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Balance Problems · Mild
10 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Balance Problems · Moderate
7 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Balance Problems · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Vision Problems · Not At All
7 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Vision Problems · Severe
5 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Abdominal Discomfort · Mild
10 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Abdominal Discomfort · Moderate
2 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Abdominal Discomfort · Not At All
10 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Abdominal Discomfort · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Balance Problems · Mild
10 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Balance Problems · Moderate
9 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Balance Problems · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Balance Problems · Severe
5 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Headache · Mild
10 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Headache · Moderate
4 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Headache · Not At All
10 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Headache · Severe
1 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Muscle Pain · Mild
8 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Muscle Pain · Moderate
6 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Muscle Pain · Not At All
9 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Muscle Pain · Severe
2 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Muscle Weakness at Rest · Mild
8 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Muscle Weakness at Rest · Moderate
8 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Muscle Weakness at Rest · Not At All
6 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Muscle Weakness at Rest · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Muscle Weakness during Activities · Moderate
7 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Muscle Weakness during Activities · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Muscle Weakness during Activities · Severe
5 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Numbness · Mild
4 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Numbness · Moderate
5 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Numbness · Not At All
13 Participants
—
—
—
—
PMMSA Individual Items
Week 52: Numbness · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 52:Tiredness at Rest · Mild
12 Participants
—
—
—
—
PMMSA Individual Items
Week 52:Tiredness at Rest · Moderate
8 Participants
—
—
—
—
PMMSA Individual Items
Week 52:Tiredness at Rest · Not At All
4 Participants
—
—
—
—
PMMSA Individual Items
Week 52:Tiredness at Rest · Severe
1 Participants
—
—
—
—
PMMSA Individual Items
Week 52:Tiredness during Activities · Mild
12 Participants
—
—
—
—
PMMSA Individual Items
Week 52:Tiredness during Activities · Moderate
10 Participants
—
—
—
—
PMMSA Individual Items
Week 52:Tiredness during Activities · Not At All
0 Participants
—
—
—
—
PMMSA Individual Items
Week 52:Tiredness during Activities · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 52:Vision Problems · Mild
8 Participants
—
—
—
—
PMMSA Individual Items
Week 52:Vision Problems · Moderate
9 Participants
—
—
—
—
PMMSA Individual Items
Week 52:Vision Problems · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Abdominal Discomfort · Mild
11 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Abdominal Discomfort · Moderate
2 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Abdominal Discomfort · Not At All
9 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Abdominal Discomfort · Severe
2 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Balance Problems · Mild
7 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Balance Problems · Moderate
13 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Balance Problems · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Balance Problems · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Headache · Mild
6 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Headache · Moderate
3 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Headache · Not At All
14 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Headache · Severe
1 Participants
—
—
—
—
PMMSA Individual Items
Week 78:Muscle Pain · Mild
10 Participants
—
—
—
—
PMMSA Individual Items
Week 78:Muscle Pain · Moderate
5 Participants
—
—
—
—
PMMSA Individual Items
Week 78:Muscle Pain · Not At All
7 Participants
—
—
—
—
PMMSA Individual Items
Week 78:Muscle Pain · Severe
2 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Muscle Weakness at Rest · Mild
10 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Muscle Weakness at Rest · Moderate
9 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Muscle Weakness at Rest · Not At All
5 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Muscle Weakness at Rest · Severe
0 Participants
—
—
—
—
PMMSA Individual Items
Week 78:Muscle Weakness during Activities · Mild
8 Participants
—
—
—
—
PMMSA Individual Items
Week 78:Muscle Weakness during Activities · Moderate
9 Participants
—
—
—
—
PMMSA Individual Items
Week 78:Muscle Weakness during Activities · Not At All
2 Participants
—
—
—
—
PMMSA Individual Items
Week 78:Muscle Weakness during Activities · Severe
5 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Numbness · Mild
7 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Numbness · Moderate
3 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Numbness · Not At All
12 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Numbness · Severe
2 Participants
—
—
—
—
PMMSA Individual Items
Week 78:Tiredness at Rest · Mild
11 Participants
—
—
—
—
PMMSA Individual Items
Week 78:Tiredness at Rest · Moderate
8 Participants
—
—
—
—
PMMSA Individual Items
Week 78:Tiredness at Rest · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Week 78:Tiredness at Rest · Severe
4 Participants
—
—
—
—
PMMSA Individual Items
Week 78:Tiredness during Activities · Mild
8 Participants
—
—
—
—
PMMSA Individual Items
Week 78:Tiredness during Activities · Moderate
10 Participants
—
—
—
—
PMMSA Individual Items
Week 78:Tiredness during Activities · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Week 78:Tiredness during Activities · Severe
5 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Vision Problems · Mild
10 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Vision Problems · Moderate
5 Participants
—
—
—
—
PMMSA Individual Items
Week 78: Vision Problems · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Balance Problems · Severe
4 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Headache · Mild
5 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Headache · Moderate
2 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Headache · Not At All
14 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Headache · Severe
1 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Muscle Pain · Mild
10 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Muscle Pain · Moderate
7 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Muscle Pain · Not At All
5 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Muscle Pain · Severe
0 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Muscle Weakness at Rest · Mild
10 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Muscle Weakness at Rest · Moderate
6 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Muscle Weakness at Rest · Not At All
4 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Muscle Weakness at Rest · Severe
1 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Muscle Weakness during Activities · Mild
8 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Muscle Weakness during Activities · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Numbness · Mild
5 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Numbness · Moderate
2 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Numbness · Not At All
12 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Numbness · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 104: Tiredness at Rest · Mild
11 Participants
—
—
—
—
PMMSA Individual Items
Week 104: Tiredness at Rest · Moderate
6 Participants
—
—
—
—
PMMSA Individual Items
Week 104: Tiredness at Rest · Not At All
2 Participants
—
—
—
—
PMMSA Individual Items
Week 104: Tiredness at Rest · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Tiredness during Activities · Mild
10 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Tiredness during Activities · Moderate
9 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Tiredness during Activities · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Week 104:Tiredness during Activities · Severe
2 Participants
—
—
—
—
PMMSA Individual Items
Week 104: Vision Problems · Mild
7 Participants
—
—
—
—
PMMSA Individual Items
Week 104: Vision Problems · Moderate
5 Participants
—
—
—
—
PMMSA Individual Items
Week 104: Vision Problems · Not At All
5 Participants
—
—
—
—
PMMSA Individual Items
Week 104: Vision Problems · Severe
5 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Abdominal Discomfort · Mild
8 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Abdominal Discomfort · Not At All
9 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Abdominal Discomfort · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Balance Problems · Mild
8 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Balance Problems · Moderate
7 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Balance Problems · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Balance Problems · Severe
5 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Headache · Mild
8 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Headache · Moderate
2 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Headache · Not At All
11 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Headache · Severe
0 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Muscle Pain · Mild
7 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Muscle Pain · Moderate
4 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Muscle Pain · Not At All
7 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Muscle Pain · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 130:Muscle Weakness at Rest · Moderate
7 Participants
—
—
—
—
PMMSA Individual Items
Week 130:Muscle Weakness at Rest · Not At All
4 Participants
—
—
—
—
PMMSA Individual Items
Week 130:Muscle Weakness at Rest · Severe
1 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Muscle Weakness during Activities · Mild
8 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Muscle Weakness during Activities · Moderate
8 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Muscle Weakness during Activities · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Muscle Weakness during Activities · Severe
4 Participants
—
—
—
—
PMMSA Individual Items
Week 130:Numbness · Mild
7 Participants
—
—
—
—
PMMSA Individual Items
Week 130:Numbness · Moderate
3 Participants
—
—
—
—
PMMSA Individual Items
Week 130:Numbness · Not At All
11 Participants
—
—
—
—
PMMSA Individual Items
Week 130:Tiredness at Rest · Mild
9 Participants
—
—
—
—
PMMSA Individual Items
Week 130:Tiredness at Rest · Moderate
9 Participants
—
—
—
—
PMMSA Individual Items
Week 130:Tiredness at Rest · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Week 130:Tiredness at Rest · Severe
2 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Tiredness during Activities · Mild
9 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Tiredness during Activities · Moderate
10 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Tiredness during Activities · Not At All
0 Participants
—
—
—
—
PMMSA Individual Items
Week 130: Tiredness during Activities · Severe
2 Participants
—
—
—
—
PMMSA Individual Items
Week 130:Vision Problems · Mild
7 Participants
—
—
—
—
PMMSA Individual Items
Week 130:Vision Problems · Moderate
7 Participants
—
—
—
—
PMMSA Individual Items
Week 130:Vision Problems · Not At All
4 Participants
—
—
—
—
PMMSA Individual Items
Week 130:Vision Problems · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 156: Abdominal Discomfort · Mild
1 Participants
—
—
—
—
PMMSA Individual Items
Week 156: Abdominal Discomfort · Moderate
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156: Abdominal Discomfort · Not At All
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156: Abdominal Discomfort · Severe
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156:Balance Problems · Mild
1 Participants
—
—
—
—
PMMSA Individual Items
Week 156:Balance Problems · Moderate
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156:Balance Problems · Not At All
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156:Balance Problems · Severe
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156 Headache · Mild
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156 Headache · Moderate
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156 Headache · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Week 156 Headache · Severe
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156 Muscle Pain · Mild
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156 Muscle Pain · Moderate
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156 Muscle Pain · Not At All
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156 Muscle Pain · Severe
1 Participants
—
—
—
—
PMMSA Individual Items
Week 156: Muscle Weakness at Rest · Mild
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156: Muscle Weakness at Rest · Moderate
1 Participants
—
—
—
—
PMMSA Individual Items
Week 156: Muscle Weakness at Rest · Not At All
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156: Muscle Weakness at Rest · Severe
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156: Muscle Weakness during Activities · Mild
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156: Muscle Weakness during Activities · Moderate
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156: Muscle Weakness during Activities · Not At All
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156: Muscle Weakness during Activities · Severe
1 Participants
—
—
—
—
PMMSA Individual Items
Week 156: Numbness · Mild
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156: Numbness · Moderate
1 Participants
—
—
—
—
PMMSA Individual Items
Week 156: Numbness · Not At All
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156: Numbness · Severe
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156:Tiredness at Rest · Mild
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156:Tiredness at Rest · Moderate
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156:Tiredness at Rest · Not At All
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156:Tiredness at Rest · Severe
1 Participants
—
—
—
—
PMMSA Individual Items
Week 156:Tiredness during Activities · Mild
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156:Tiredness during Activities · Moderate
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156:Tiredness during Activities · Not At All
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156:Tiredness during Activities · Severe
1 Participants
—
—
—
—
PMMSA Individual Items
Week 156:Vision Problems · Mild
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156:Vision Problems · Moderate
0 Participants
—
—
—
—
PMMSA Individual Items
Week 156:Vision Problems · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Week 156:Vision Problems · Severe
0 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Abdominal Discomfort · Mild
8 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Abdominal Discomfort · Moderate
2 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Abdominal Discomfort · Not At All
6 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Abdominal Discomfort · Severe
4 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Balance Problems · Mild
9 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Balance Problems · Moderate
8 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Balance Problems · Severe
4 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Headache · Mild
9 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Headache · Moderate
5 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Headache · Not At All
7 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Headache · Severe
0 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Muscle Pain · Mild
5 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Muscle Pain · Moderate
9 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Muscle Pain · Not At All
4 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Muscle Pain · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Muscle Weakness at Rest · Mild
8 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Muscle Weakness at Rest · Moderate
7 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Muscle Weakness at Rest · Not At All
4 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Muscle Weakness at Rest · Severe
2 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160): Muscle Weakness during Activities · Mild
3 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160): Muscle Weakness during Activities · Moderate
11 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160): Muscle Weakness during Activities · Not At All
2 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160): Muscle Weakness during Activities · Severe
5 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Numbness · Mild
5 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Numbness · Moderate
5 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Numbness · Not At All
11 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Numbness · Severe
0 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Tiredness at Rest · Mild
7 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Tiredness at Rest · Moderate
9 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Tiredness at Rest · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Tiredness at Rest · Severe
4 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Tiredness during Activities · Mild
4 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Tiredness during Activities · Moderate
10 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Tiredness during Activities · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160) Tiredness during Activities · Severe
6 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160): Vision Problems · Mild
3 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160): Vision Problems · Moderate
9 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160): Vision Problems · Not At All
6 Participants
—
—
—
—
PMMSA Individual Items
End of Study: (Week 160): Vision Problems · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 13:Abdominal Discomfort · Mild
6 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Abdominal Discomfort · Mild
11 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Abdominal Discomfort · Moderate
5 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Abdominal Discomfort · Not At All
8 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Abdominal Discomfort · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Balance Problems · Mild
5 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Balance Problems · Moderate
16 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Balance Problems · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Balance Problems · Severe
5 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Headache · Mild
4 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Headache · Moderate
6 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Headache · Not At All
16 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Headache · Severe
1 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Muscle Pain · Mild
6 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Muscle Pain · Moderate
12 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Muscle Pain · Not At All
5 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Muscle Pain · Severe
4 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Muscle Weakness at Rest · Mild
12 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Muscle Weakness at Rest · Moderate
10 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Muscle Weakness at Rest · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Muscle Weakness at Rest · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Baseline:Muscle Weakness during Activities · Mild
2 Participants
—
—
—
—
PMMSA Individual Items
Baseline:Muscle Weakness during Activities · Moderate
18 Participants
—
—
—
—
PMMSA Individual Items
Baseline:Muscle Weakness during Activities · Not At All
0 Participants
—
—
—
—
PMMSA Individual Items
Baseline:Muscle Weakness during Activities · Severe
7 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Numbness · Mild
11 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Numbness · Moderate
2 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Numbness · Not At All
11 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Numbness · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Tiredness at Rest · Mild
6 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Tiredness at Rest · Moderate
13 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Tiredness at Rest · Not At All
0 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Tiredness at Rest · Severe
8 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Tiredness during Activities · Mild
5 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Tiredness during Activities · Moderate
13 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Tiredness during Activities · Not At All
0 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Tiredness during Activities · Severe
9 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Vision Problems · Mild
8 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Vision Problems · Moderate
9 Participants
—
—
—
—
PMMSA Individual Items
Baseline: Vision Problems · Not At All
6 Participants
—
—
—
—
PMMSA Individual Items
Week 13:Abdominal Discomfort · Moderate
8 Participants
—
—
—
—
PMMSA Individual Items
Week 13:Abdominal Discomfort · Not At All
11 Participants
—
—
—
—
PMMSA Individual Items
Week 13:Abdominal Discomfort · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Balance Problems · Mild
11 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Balance Problems · Moderate
9 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Balance Problems · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Balance Problems · Severe
7 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Headache · Mild
10 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Headache · Moderate
2 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Headache · Not At All
14 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Headache · Severe
2 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Muscle Pain · Mild
11 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Muscle Pain · Moderate
7 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Muscle Pain · Not At All
7 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Muscle Pain · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Muscle Weakness at Rest · Mild
13 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Muscle Weakness at Rest · Moderate
6 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Muscle Weakness at Rest · Not At All
7 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Muscle Weakness at Rest · Severe
2 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Muscle Weakness during Activities · Mild
10 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Muscle Weakness during Activities · Moderate
9 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Muscle Weakness during Activities · Not At All
3 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Muscle Weakness during Activities · Severe
6 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Numbness · Mild
6 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Numbness · Moderate
6 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Numbness · Not At All
15 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Numbness · Severe
1 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Tiredness at Rest · Mild
14 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Tiredness at Rest · Moderate
10 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Tiredness at Rest · Not At All
2 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Tiredness at Rest · Severe
2 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Tiredness during Activities · Mild
12 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Tiredness during Activities · Moderate
11 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Tiredness during Activities · Not At All
0 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Tiredness during Activities · Severe
5 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Vision Problems · Mild
11 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Vision Problems · Moderate
7 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Vision Problems · Not At All
8 Participants
—
—
—
—
PMMSA Individual Items
Week 13: Vision Problems · Severe
2 Participants
—
—
—
—
PMMSA Individual Items
Week 26:Abdominal Discomfort · Mild
7 Participants
—
—
—
—
PMMSA Individual Items
Week 26:Abdominal Discomfort · Moderate
5 Participants
—
—
—
—
PMMSA Individual Items
Week 26:Abdominal Discomfort · Not At All
13 Participants
—
—
—
—
PMMSA Individual Items
Week 26:Abdominal Discomfort · Severe
2 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Balance Problems · Mild
11 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Balance Problems · Moderate
9 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Balance Problems · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Balance Problems · Severe
6 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Headache · Mild
13 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Headache · Moderate
1 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Headache · Not At All
12 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Headache · Severe
1 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Muscle Pain · Mild
11 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Muscle Pain · Moderate
7 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Muscle Pain · Not At All
7 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Muscle Pain · Severe
2 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Muscle Weakness at Rest · Mild
13 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Muscle Weakness at Rest · Moderate
4 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Muscle Weakness at Rest · Not At All
7 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Muscle Weakness at Rest · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Muscle Weakness during Activities · Mild
10 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Muscle Weakness during Activities · Moderate
11 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Muscle Weakness during Activities · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Muscle Weakness during Activities · Severe
5 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Numbness · Mild
7 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Numbness · Moderate
3 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Numbness · Not At All
14 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Numbness · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Tiredness at Rest · Mild
15 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Tiredness at Rest · Moderate
8 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Tiredness at Rest · Not At All
1 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Tiredness at Rest · Severe
3 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Tiredness during Activities · Mild
12 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Tiredness during Activities · Moderate
10 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Tiredness during Activities · Not At All
0 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Tiredness during Activities · Severe
5 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Vision Problems · Mild
6 Participants
—
—
—
—
PMMSA Individual Items
Week 26: Vision Problems · Moderate
9 Participants
—
—
—
—

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

Population: All participants for whom PMMSA was measured. Study terminated early.

Change from baseline in the Total Fatigue score Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) where participants report their "worst feeling" of 4 symptoms: Tiredness at rest, Tiredness during activities, Muscle weakness at rest, Muscle weakness during activities, at the end of each treatment period. Total fatigue score is sum of Q1 to Q4 only of PMMSA) and is scored as follows: 1=Not at all, 2=Mild, 3=Moderate, and 4=Severe. Total score range is 4-16; lower score means less fatigue and better outcome, higher score means more fatigue and worse outcome. Raw scores were transformed into percentages: 0-100%. Change from baseline: the lower the score, the better the outcome,=less fatigue; the higher the score, means worse outcome= more fatigue.

Outcome measures

Outcome measures
Measure
Elamipretide
n=28 Participants
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Slight Problem
Participant reports symptom is a "slight problem"
Moderate Problem
Participant reports symptom is a "moderate problem"
Severe Problem
Participant reports symptom is a "severe problem"
Extreme Problem
Participant reports symptom is an "extreme problem"
Total Fatigue Score_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 130
-12.92 score on a scale
Standard Deviation 31.12
—
—
—
—
Total Fatigue Score_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 156
25.00 score on a scale
Standard Deviation NA
No standard deviation because only 1 participant was measured.
—
—
—
—
Total Fatigue Score_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
End of Trial (Week 160)
-8.33 score on a scale
Standard Deviation 33.00
—
—
—
—
Total Fatigue Score_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 13
-15.12 score on a scale
Standard Deviation 29.33
—
—
—
—
Total Fatigue Score_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 26
-15.06 score on a scale
Standard Deviation 29.72
—
—
—
—
Total Fatigue Score_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 52
-17.01 score on a scale
Standard Deviation 25.36
—
—
—
—
Total Fatigue Score_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 78
-13.41 score on a scale
Standard Deviation 31.05
—
—
—
—
Total Fatigue Score_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 104
-18.65 score on a scale
Standard Deviation 30.15
—
—
—
—

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

Population: All participants for whom PMMSA was measured. Study terminated early.

Change from baseline in the Total Fatigue during Activities score Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) where participants report their "worst feeling" of 2 symptoms: Tiredness during activities, Muscle weakness during activities, at the end of each treatment period. Total fatigue score is sum of Q2 and Q4 only of PMMSA) and is scored as follows: 1=Not at all, 2=Mild, 3=Moderate, and 4=Severe. Total score range is 2-8; lower score means less fatigue and better outcome, higher score means more fatigue and worse outcome. Raw scores were transformed into percentages: 0-100%. Change from baseline: the lower the score, the better the outcome,=less fatigue; the higher the score, means worse outcome= more fatigue.

Outcome measures

Outcome measures
Measure
Elamipretide
n=28 Participants
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Slight Problem
Participant reports symptom is a "slight problem"
Moderate Problem
Participant reports symptom is a "moderate problem"
Severe Problem
Participant reports symptom is a "severe problem"
Extreme Problem
Participant reports symptom is an "extreme problem"
Total Fatigue Score During Activities_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 13
-14.20 score on a scale
Standard Deviation 29.13
—
—
—
—
Total Fatigue Score During Activities_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 26
-14.10 score on a scale
Standard Deviation 28.55
—
—
—
—
Total Fatigue Score During Activities_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 52
-17.36 score on a scale
Standard Deviation 25.76
—
—
—
—
Total Fatigue Score During Activities_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 78
-14.49 score on a scale
Standard Deviation 32.30
—
—
—
—
Total Fatigue Score During Activities_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 104
-20.63 score on a scale
Standard Deviation 28.34
—
—
—
—
Total Fatigue Score During Activities_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 130
-15.00 score on a scale
Standard Deviation 31.94
—
—
—
—
Total Fatigue Score During Activities_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Week 156
33.33 score on a scale
Standard Deviation NA
Only 1 participant was measured. There is no standard deviation.
—
—
—
—
Total Fatigue Score During Activities_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
End of Trial (Week 160)
-8.33 score on a scale
Standard Deviation 31.30
—
—
—
—

Adverse Events

Elamipretide

Serious events: 8 serious events
Other events: 28 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Elamipretide
n=28 participants at risk
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Gastrointestinal disorders
Pancreatitis acute
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Breast cancer stage I
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Nervous system disorders
Hemiparesis
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Nervous system disorders
Syncope
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Vascular disorders
Cerebrovascular accident
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Congenital, familial and genetic disorders
Hereditary optic atrophy
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Gastrointestinal disorders
Dysphagia
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Injury, poisoning and procedural complications
Ankle fracture
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Investigations
Troponin increased
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Renal and urinary disorders
Renal colic
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Urosepsis
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Renal and urinary disorders
Acute kidney injury
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Bacteraemia
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Urinary tract infection
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Cellulitis
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Cardiac disorders
Bradycardia
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Gastrointestinal disorders
Intestinal obstruction
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Hepatobiliary disorders
Cholelithiasis
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Injury, poisoning and procedural complications
Tibia fracture
3.6%
1/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Pneumonia
3.6%
1/28 • Baseline through end of study for up to 260 weeks

Other adverse events

Other adverse events
Measure
Elamipretide
n=28 participants at risk
Open-label once daily subcutaneous injection of 40mg elamipretide elamipretide: 40 mg, subcutaneous injections (in the abdomen) daily for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
Injury, poisoning and procedural complications
Thermal burn
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Investigations
Blood lactic acid increased
14.3%
4/28 • Baseline through end of study for up to 260 weeks
Investigations
Alanine aminotransferase increased
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Investigations
Eosinophil count increased
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Investigations
Weight increased
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Metabolism and nutrition disorders
Hypokalaemia
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Musculoskeletal and connective tissue disorders
Arthralgia
14.3%
4/28 • Baseline through end of study for up to 260 weeks
Musculoskeletal and connective tissue disorders
Pain in extremity
14.3%
4/28 • Baseline through end of study for up to 260 weeks
Musculoskeletal and connective tissue disorders
Back pain
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Musculoskeletal and connective tissue disorders
Muscle fatigue
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Musculoskeletal and connective tissue disorders
Muscular weakness
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Nervous system disorders
Headache
25.0%
7/28 • Baseline through end of study for up to 260 weeks
Nervous system disorders
Dizziness
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Nervous system disorders
Hypoaesthesia
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Nervous system disorders
Migraine
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Nervous system disorders
Neuralgia
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Nervous system disorders
Dizziness postural
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Nervous system disorders
Dystonia
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Nervous system disorders
Syncope
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Nervous system disorders
Tremor
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Psychiatric disorders
Anxiety
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Psychiatric disorders
Depression
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Psychiatric disorders
Adjustment disorder with depressed mood
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Respiratory, thoracic and mediastinal disorders
Cough
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Respiratory, thoracic and mediastinal disorders
Throat irritation
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Respiratory, thoracic and mediastinal disorders
Apnoea
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Respiratory, thoracic and mediastinal disorders
Nasal congestion
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Skin and subcutaneous tissue disorders
Rash
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Skin and subcutaneous tissue disorders
Urticaria
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Skin and subcutaneous tissue disorders
Acne
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Skin and subcutaneous tissue disorders
Dermal cyst
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Skin and subcutaneous tissue disorders
Pruritus
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Cardiac disorders
Tachycardia
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Gastrointestinal disorders
Vomiting
17.9%
5/28 • Baseline through end of study for up to 260 weeks
Gastrointestinal disorders
Abdominal distension
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Gastrointestinal disorders
Abdominal pain
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Gastrointestinal disorders
Abdominal pain upper
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Gastrointestinal disorders
Diarrhoea
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Gastrointestinal disorders
Abdominal discomfort
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Gastrointestinal disorders
Constipation
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Gastrointestinal disorders
Haemorrhoids
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Gastrointestinal disorders
Impaired gastric emptying
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Gastrointestinal disorders
Nausea
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Gastrointestinal disorders
Injection site pruritus
75.0%
21/28 • Baseline through end of study for up to 260 weeks
General disorders
Injection site pruritus
64.3%
18/28 • Baseline through end of study for up to 260 weeks
General disorders
Injection site mass
46.4%
13/28 • Baseline through end of study for up to 260 weeks
General disorders
Injection site urticaria
46.4%
13/28 • Baseline through end of study for up to 260 weeks
General disorders
Injection site pain
35.7%
10/28 • Baseline through end of study for up to 260 weeks
General disorders
Injection site swelling
25.0%
7/28 • Baseline through end of study for up to 260 weeks
General disorders
Chest pain
21.4%
6/28 • Baseline through end of study for up to 260 weeks
General disorders
Fatigue
21.4%
6/28 • Baseline through end of study for up to 260 weeks
General disorders
Injection site bruising
14.3%
4/28 • Baseline through end of study for up to 260 weeks
General disorders
Injection site rash
14.3%
4/28 • Baseline through end of study for up to 260 weeks
General disorders
Pyrexia
10.7%
3/28 • Baseline through end of study for up to 260 weeks
General disorders
Chills
7.1%
2/28 • Baseline through end of study for up to 260 weeks
General disorders
Injection site haemorrhage
7.1%
2/28 • Baseline through end of study for up to 260 weeks
General disorders
Injection site induration
7.1%
2/28 • Baseline through end of study for up to 260 weeks
General disorders
Peripheral swelling
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Nasopharyngitis
28.6%
8/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Gastroenteritis viral
21.4%
6/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Urinary tract infection
14.3%
4/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Sinusitis
14.3%
4/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Upper respiratory tract infection
14.3%
4/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Influenza
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Bronchitis
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Ear infection
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Fungal infection
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Hordeolum
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Pneumonia
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Postoperative wound infection
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Vaginal infection
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Injury, poisoning and procedural complications
Fall
35.7%
10/28 • Baseline through end of study for up to 260 weeks
Injury, poisoning and procedural complications
Concussion
14.3%
4/28 • Baseline through end of study for up to 260 weeks
Injury, poisoning and procedural complications
Contusion
14.3%
4/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Ligament sprain
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Infections and infestations
Skin laceration
10.7%
3/28 • Baseline through end of study for up to 260 weeks
Injury, poisoning and procedural complications
Joint dislocation
7.1%
2/28 • Baseline through end of study for up to 260 weeks
Injury, poisoning and procedural complications
Muscle strain
7.1%
2/28 • Baseline through end of study for up to 260 weeks

Additional Information

Jim Carr, Pharm.D. Chief Clinical Development Officer

Stealth BioTherapeutics, Inc

Phone: 1-617-600-6888

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place

Restriction type: GT60