Six-minute Walk Test (6MWT)
| Group | Value (meters) | Spread |
|---|---|---|
| Elamipretide | 16.243 | 42.0071 |
| Placebo | 7.811 | 39.5737 |
Trial · Interventional
TerminatedPhase 3Results postedRegistry dataShort name MMPOWER-3
This trial was stopped early.
Reason given at the registry: Part1,double blind portion of the trial did not meet the primary end points
Results were posted for this trial.
United States, Canada, Denmark, Germany, Hungary, Italy, United Kingdom
1 trial · 1 stopped early · 1 with posted results
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
This is a multicenter phase 3 randomized, double-blind, parallel-group, placebo-controlled trial to evaluate the safety and efficacy of daily subcutaneous injections of elamipretide in subjects with primary mitochondrial myopathy. This will be followed by an open-label treatment extension.
Experimental
Part 1: Elamipretide
40 mg (0.5mL) elamipretide subcutaneous (SC) daily
Placebo comparator
Part 1: Placebo
Placebo SC daily
Experimental
Part 2: Elamipretide open label
Elamepretide 40 mg (0.5 mL) SC daily
Outcomes the sponsor listed as primary.
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.
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
| Group | Value (meters) | Spread |
|---|---|---|
| Elamipretide | 16.243 | 42.0071 |
| Placebo | 7.811 | 39.5737 |
| Group | Value (score on a scale) | Spread |
|---|---|---|
| Elamipretide | -1.01 | 1.714 |
| Placebo | -1.08 | 1.865 |
| Group | Value (score on a scale) | Spread |
|---|---|---|
| Elamipretide | -0.48 | 0.890 |
| Placebo | -0.60 | 0.998 |
| Group | Value (score on a scale) | Spread |
|---|---|---|
| Elamipretide | -2.5 | 5.33 |
| Placebo | -2.7 | 5.31 |
| Group | Value (score on a scale) | Spread |
|---|---|---|
| Elamipretide | -0.21 | 0.492 |
| Placebo | -0.24 | 0.535 |
| Group | Value (score on a scale) | Spread |
|---|---|---|
| Elamipretide | -3.37 | 5.077 |
| Placebo | -2.89 | 5.915 |
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| Double-Blind ElamipretideDouble-blind period: elamipretide: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system, then 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 | 0 / 109 | 5 / 109 | 107 / 109 |
| Double-Blind PlaceboPlacebo SC daily Double-blind period: placebo comparator: Placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system. Open-label period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system. | 0 / 109 | 3 / 109 | 83 / 109 |
| Open-Label ElamipretideDouble blind period: elamipretide: 40mg (0.5mL) of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system, then 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 | 0 / 93 | 12 / 93 | 88 / 93 |
| Open-Label PlaceboDouble-blind period: placebo comparator: Placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system. Open-label period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system. | 0 / 103 | 9 / 103 | 102 / 103 |
Each cell reads participants affected / participants at risk, as the registry posted them.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies that cite this trial.
2 studies on this page
Indexing hint: Randomized controlled trial · 2024
Karaa et al. 2024Orphanet journal of rare diseasescited by 11
PMID 39574155PMC11583740DOI 10.1186/s13023-024-03421-5PUB-39574155
Indexing hint: Randomized controlled trial · 2023
Karaa et al. 2023Neurologycited by 66
PMID 37268435PMC10382259DOI 10.1212/wnl.0000000000207402PUB-37268435
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