A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension (MMPOWER-3)
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|ClinicalTrials.gov Identifier: NCT03323749|
Recruitment Status : Terminated (Part1,double blind portion of the trial did not meet the primary end points)
First Posted : October 27, 2017
Results First Posted : April 2, 2021
Last Update Posted : January 24, 2022
|Condition or disease||Intervention/treatment||Phase|
|Primary Mitochondrial Myopathy||Combination Product: elamipretide Combination Product: placebo comparator Combination Product: elamipretide open label treatment||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||218 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Phase 3 Randomized, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Myopathy Followed by an Open-Label Treatment Extension|
|Actual Study Start Date :||October 9, 2017|
|Actual Primary Completion Date :||February 10, 2020|
|Actual Study Completion Date :||February 10, 2020|
Experimental: Part 1: Elamipretide
40 mg (0.5mL) elamipretide subcutaneous (SC) daily
Combination Product: elamipretide
40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
Other Name: MTP-131
Placebo Comparator: Part 1: Placebo
Placebo SC daily
Combination Product: placebo comparator
40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
Other Name: Placebo
Experimental: Part 2: Elamipretide open label
Elamepretide 40 mg (0.5 mL) SC daily
Combination Product: elamipretide open label treatment
40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system
- Six-minute Walk Test (6MWT) [ Time Frame: Baseline to 24 weeks ]Change From Baseline in Distance Walked (meters) on the Six-Minute Walk Test by Visit
- Total Fatigue Score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) [ Time Frame: Baseline to 24 weeks ]Change from Baseline in Total fatigue score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) by visit. Each individual item score ranges from 1 (none) to 4 (severe). The total fatigue score ranges from 4-16. Lower values represent a better outcome. The total fatigue score is the sum of question 1 through question 4 on the Primary Mitochondrial Myopathy Symptom Assessment.
- Fatigue During Activities Score on the Primary Mitochondrial Disease Symptom Assessment (PMMSA). [ Time Frame: Baseline to 24 weeks ]Change from baseline in Fatigue During Activities. Fatigue During Activities is the sum of question 2 (tiredness during activities) and question 4 (muscle weakness during activities.) The four response options are: 1=Not at all, 2=Mild, 3=Moderate, and 4=Severe. Raw scores for each subject range from 2-8. A lower score means a better outcome, with less fatigue. A higher score means a worse outcome, with more fatigue.
- Neuro-QoL Fatigue Activities of Daily Living [ Time Frame: Baseline to 24 weeks ]Change From Baseline in Neuro-QoL Fatigue Activities of Daily Living by Visit. Each individual item score ranges from 1-5. Total raw score for the entire item bank ranges from 19-95. Raw scores will be calibrated using Item Response Theory Model. Lower values represent a better outcome. Individual items will be summed to calculate total scores.
- Change From Baseline in the Most Bothersome Symptom Score on the Primary Mitochondrial Myopathy Symptoms Assessment [ Time Frame: Baseline to 24 weeks ]The item score rangers from 1 (none) to 4 (severe). Lower values represent a better outcome. The most bothersome score is the average of the identified most bothersome symptom of the Primary Mitochondrial Myopathy Symptom Assessment by each subject.
- Neuro-QoL Fatigue Short Form Score [ Time Frame: 24 Weeks ]Change From Baseline in Neuro-QoL Fatigue - Short Form: Total T-Scores by Visit. The Neuro-QoL Fatigue Short Form is comprised of the sum of the first 8 questions of the Neuro-QoL Item Bank v1.0 - Fatigue. Each question is scored as following: 1=Never, 2=Rarely, 3=Sometimes, 4=Often, and 5=Always. The questions include: I felt exhausted, I felt that I had no energy, I felt fatigued, I was too tired to do my household chores, I was too tired to leave the house, I was frustrated by being too tired to do the things I wanted to do, I felt tired, and I had to limit my social activity because I was tired. T-scores are calculated from the short form scoring table provided by the instrument authors (Neuro-QoL User Manual, 2015). T-score distributions rescale raw scores into standardized scores with a mean of 50 and a standard deviation (SD) of 10. Change from baseline: Negative numbers mean less fatigue, better outcome, positive score means more fatigue, worse outcome.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03323749