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Study · Randomized controlled trial · n = 30 · 2020

11 results

A randomized crossover trial of elamipretide in adults with primary mitochondrial myopathy.

Karaa A, Haas R, Goldstein A, Vockley J, Cohen BH

Journal of cachexia, sarcopenia and muscle25 Feb 2020Cited by 57 (Europe PMC)

Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: CC BY-NC. Results still quote short attributed sentences.

Results from this study11

11 results from studies that gave the peptide. 11 results from people.

  1. 01HumanRandomized controlled trialEfficacy outcome

    In adults with primary mitochondrial myopathy, elamipretide did not significantly increase 6-minute walk distance versus placebo (difference 19.8 m).

    In plain wordsIn adults with a mitochondrial muscle disease, elamipretide did not significantly increase 6-minute walk distance compared with placebo.

    Outcome
    No significant change6-min walk test distance
    Study

    n = 30

    Comparator
    Placebo
    Analysis
    Primary
    Outcome group
    Exercise and physical function · Musculoskeletal
    Studied dose
    40 mg/day · subcutaneous · 4 weeksAs given in this study (human).

    Measurements

    6MWT difference vs placebo
    19.8 m
    elamipretide 40 mg/day

    Source sentences

    Summary
    The distance walked on the 6MWT by the elamipretide-treated participants was 398.3 (±134.16) meters compared with 378.5 (±125.10) meters in the placebo-treated group, a difference of 19.8 m (95% confidence interval, -2.8, 42.5; P = 0.0833).
    2 context sentences
    Summary · context
    MMPOWER-2 was a randomized, double-blind, placebo-controlled, crossover trial that enrolled participants (N = 30) with genetically confirmed primary mitochondrial myopathy.
    Summary · context
    Participants were randomly assigned (1:1) to 40 mg/day SC elamipretide for 4 weeks followed by placebo SC for 4 weeks, separated by a 4-week washout period, or the opposite sequence.
  2. 02HumanRandomized controlled trialEfficacy outcome

    In adults with primary mitochondrial myopathy, elamipretide reduced total fatigue (PMMSA Total Fatigue) versus placebo.

    In plain wordsIn adults with a mitochondrial muscle disease, elamipretide reduced reported fatigue more than placebo.

    Outcome
    DecreasePMMSA Total Fatigue score
    Study

    n = 30

    Comparator
    Placebo
    Outcome group
    Patient-reported outcomes · Musculoskeletal
    Studied dose
    40 mg/day · subcutaneous · 4 weeksAs given in this study (human).

    Source sentences

    Summary
    participants treated with elamipretide reported less fatigue and muscle complaints compared with placebo (P = 0.0006
    3 context sentences
    Summary · context
    The results of the Primary Mitochondrial Myopathy Symptom Assessment Total Fatigue and Total Fatigue During Activities scores
    Summary · context
    MMPOWER-2 was a randomized, double-blind, placebo-controlled, crossover trial that enrolled participants (N = 30) with genetically confirmed primary mitochondrial myopathy.
    Summary · context
    Participants were randomly assigned (1:1) to 40 mg/day SC elamipretide for 4 weeks followed by placebo SC for 4 weeks, separated by a 4-week washout period, or the opposite sequence.
  3. 03HumanRandomized controlled trialEfficacy outcome

    In adults with primary mitochondrial myopathy, elamipretide reduced fatigue during activities versus placebo.

    In plain wordsIn adults with a mitochondrial muscle disease, elamipretide reduced reported fatigue during activities more than placebo.

    Outcome
    DecreasePMMSA Total Fatigue During Activities score
    Study

    n = 30

    Comparator
    Placebo
    Outcome group
    Patient-reported outcomes · Musculoskeletal
    Studied dose
    40 mg/day · subcutaneous · 4 weeksAs given in this study (human).

    Source sentences

    Summary
    The results of the Primary Mitochondrial Myopathy Symptom Assessment Total Fatigue and Total Fatigue During Activities scores showed that participants treated with elamipretide reported less fatigue and muscle complaints compared with placebo (P = 0.0006 and P = 0.0018, respectively).
    2 context sentences
    Summary · context
    MMPOWER-2 was a randomized, double-blind, placebo-controlled, crossover trial that enrolled participants (N = 30) with genetically confirmed primary mitochondrial myopathy.
    Summary · context
    Participants were randomly assigned (1:1) to 40 mg/day SC elamipretide for 4 weeks followed by placebo SC for 4 weeks, separated by a 4-week washout period, or the opposite sequence.
  4. 04HumanRandomized controlled trialEfficacy outcome

    In adults with primary mitochondrial myopathy, elamipretide reduced symptoms on the Neuro-QoL Fatigue Short Form versus placebo.

    In plain wordsIn adults with a mitochondrial muscle disease, elamipretide reduced fatigue scores on a quality-of-life scale compared with placebo.

    Outcome
    DecreaseNeuro-QoL Fatigue Short Form
    Study

    n = 30

    Comparator
    Placebo
    Outcome group
    Patient-reported outcomes · Musculoskeletal
    Studied dose
    40 mg/day · subcutaneous · 4 weeksAs given in this study (human).

    Source sentences

    Summary
    Additionally, the Neuro-QoL Fatigue Short Form and Patient Global Assessment showed reductions in symptoms (P = 0.0115 and P = 0.0421, respectively).
    2 context sentences
    Summary · context
    MMPOWER-2 was a randomized, double-blind, placebo-controlled, crossover trial that enrolled participants (N = 30) with genetically confirmed primary mitochondrial myopathy.
    Summary · context
    Participants were randomly assigned (1:1) to 40 mg/day SC elamipretide for 4 weeks followed by placebo SC for 4 weeks, separated by a 4-week washout period, or the opposite sequence.
  5. 05HumanRandomized controlled trialEfficacy outcome

    In adults with primary mitochondrial myopathy, elamipretide reduced symptoms on the Patient Global Assessment versus placebo.

    In plain wordsIn adults with a mitochondrial muscle disease, patients rated their overall symptoms as lower with elamipretide than with placebo.

    Outcome
    DecreasePatient Global Assessment
    Study

    n = 30

    Comparator
    Placebo
    Outcome group
    Patient-reported outcomes · Musculoskeletal
    Studied dose
    40 mg/day · subcutaneous · 4 weeksAs given in this study (human).

    Source sentences

    Summary
    Additionally, the Neuro-QoL Fatigue Short Form and Patient Global Assessment showed reductions in symptoms (P = 0.0115 and P = 0.0421, respectively).
    2 context sentences
    Summary · context
    MMPOWER-2 was a randomized, double-blind, placebo-controlled, crossover trial that enrolled participants (N = 30) with genetically confirmed primary mitochondrial myopathy.
    Summary · context
    Participants were randomly assigned (1:1) to 40 mg/day SC elamipretide for 4 weeks followed by placebo SC for 4 weeks, separated by a 4-week washout period, or the opposite sequence.
  6. 06HumanRandomized controlled trialEfficacy outcome

    In adults with primary mitochondrial myopathy, elamipretide caused no statistically significant change in the Physician Global Assessment versus placebo.

    In plain wordsIn adults with a mitochondrial muscle disease, doctors' overall ratings did not change significantly with elamipretide compared with placebo.

    Outcome
    No significant changePhysician Global Assessment
    Study

    n = 30

    Comparator
    Placebo
    Outcome group
    Patient-reported outcomes · Musculoskeletal
    Studied dose
    40 mg/day · subcutaneous · 4 weeksAs given in this study (human).

    Source sentences

    Summary
    In this 4-week treatment period, no statistically significant change was observed in the Physician Global Assessment (P = 0.0636), the Triple Timed Up and Go (P = 0.8423) test, and wrist/hip accelerometry (P = 0.9345 and P = 0.7326, respectively).
    2 context sentences
    Summary · context
    MMPOWER-2 was a randomized, double-blind, placebo-controlled, crossover trial that enrolled participants (N = 30) with genetically confirmed primary mitochondrial myopathy.
    Summary · context
    Participants were randomly assigned (1:1) to 40 mg/day SC elamipretide for 4 weeks followed by placebo SC for 4 weeks, separated by a 4-week washout period, or the opposite sequence.
  7. 07HumanRandomized controlled trialEfficacy outcome

    In adults with primary mitochondrial myopathy, elamipretide caused no statistically significant change in the Triple Timed Up and Go test versus placebo.

    In plain wordsIn adults with a mitochondrial muscle disease, elamipretide did not change a timed walking and standing test compared with placebo.

    Outcome
    No significant changeTriple Timed Up and Go test
    Study

    n = 30

    Comparator
    Placebo
    Outcome group
    Exercise and physical function · Musculoskeletal
    Studied dose
    40 mg/day · subcutaneous · 4 weeksAs given in this study (human).

    Source sentences

    Summary
    In this 4-week treatment period, no statistically significant change was observed in the Physician Global Assessment (P = 0.0636), the Triple Timed Up and Go (P = 0.8423) test, and wrist/hip accelerometry (P = 0.9345 and P = 0.7326, respectively).
    2 context sentences
    Summary · context
    MMPOWER-2 was a randomized, double-blind, placebo-controlled, crossover trial that enrolled participants (N = 30) with genetically confirmed primary mitochondrial myopathy.
    Summary · context
    Participants were randomly assigned (1:1) to 40 mg/day SC elamipretide for 4 weeks followed by placebo SC for 4 weeks, separated by a 4-week washout period, or the opposite sequence.
  8. 08HumanRandomized controlled trialEfficacy outcome

    In adults with primary mitochondrial myopathy, elamipretide caused no statistically significant change in wrist or hip accelerometry versus placebo.

    In plain wordsIn adults with a mitochondrial muscle disease, elamipretide did not change activity measured by body-worn sensors compared with placebo.

    Outcome
    No significant changeWrist and hip accelerometry
    Study

    n = 30

    Comparator
    Placebo
    Outcome group
    Exercise and physical function · Musculoskeletal
    Studied dose
    40 mg/day · subcutaneous · 4 weeksAs given in this study (human).

    Source sentences

    Summary
    In this 4-week treatment period, no statistically significant change was observed in the Physician Global Assessment (P = 0.0636), the Triple Timed Up and Go (P = 0.8423) test, and wrist/hip accelerometry (P = 0.9345 and P = 0.7326, respectively).
    2 context sentences
    Summary · context
    MMPOWER-2 was a randomized, double-blind, placebo-controlled, crossover trial that enrolled participants (N = 30) with genetically confirmed primary mitochondrial myopathy.
    Summary · context
    Participants were randomly assigned (1:1) to 40 mg/day SC elamipretide for 4 weeks followed by placebo SC for 4 weeks, separated by a 4-week washout period, or the opposite sequence.
  9. 09HumanRandomized controlled trialSafety outcome

    In adults with primary mitochondrial myopathy, injection site reactions were the most commonly reported adverse events with elamipretide, mostly mild.

    In plain wordsIn adults with a mitochondrial muscle disease, injection site reactions, mostly mild, were the most common side effects of elamipretide.

    Outcome
    Not applicableInjection site reactions
    Study

    n = 30

    Outcome group
    Adverse events · Whole body
    Studied dose
    40 mg/day · subcutaneous · 4 weeksAs given in this study (human).

    Measurements

    Injection site reactions
    80%
    elamipretide

    Source sentences

    Summary
    Injection site reactions were the most commonly reported adverse events with elamipretide (80%), the majority of which were mild.
    2 context sentences
    Summary · context
    MMPOWER-2 was a randomized, double-blind, placebo-controlled, crossover trial that enrolled participants (N = 30) with genetically confirmed primary mitochondrial myopathy.
    Summary · context
    Participants were randomly assigned (1:1) to 40 mg/day SC elamipretide for 4 weeks followed by placebo SC for 4 weeks, separated by a 4-week washout period, or the opposite sequence.
  10. 10HumanRandomized controlled trialSafety outcome

    In adults with primary mitochondrial myopathy, no serious adverse events were reported with elamipretide.

    In plain wordsIn adults with a mitochondrial muscle disease, no serious side effects were reported with elamipretide.

    Outcome
    No significant changeSerious adverse events
    Study

    n = 30

    Outcome group
    Adverse events · Whole body
    Studied dose
    40 mg/day · subcutaneous · 4 weeksAs given in this study (human).

    Source sentences

    Summary
    No serious adverse events or deaths were reported.
    2 context sentences
    Summary · context
    MMPOWER-2 was a randomized, double-blind, placebo-controlled, crossover trial that enrolled participants (N = 30) with genetically confirmed primary mitochondrial myopathy.
    Summary · context
    Participants were randomly assigned (1:1) to 40 mg/day SC elamipretide for 4 weeks followed by placebo SC for 4 weeks, separated by a 4-week washout period, or the opposite sequence.
  11. 11HumanRandomized controlled trialSafety outcome

    In adults with primary mitochondrial myopathy, no deaths were reported in the trial of elamipretide.

    In plain wordsIn adults with a mitochondrial muscle disease, no deaths were reported in the elamipretide trial.

    Outcome
    No significant changeDeaths
    Study

    n = 30

    Outcome group
    Mortality · Whole body
    Studied dose
    40 mg/day · subcutaneous · 4 weeksAs given in this study (human).

    Source sentences

    Summary
    No serious adverse events or deaths were reported.
    2 context sentences
    Summary · context
    MMPOWER-2 was a randomized, double-blind, placebo-controlled, crossover trial that enrolled participants (N = 30) with genetically confirmed primary mitochondrial myopathy.
    Summary · context
    Participants were randomly assigned (1:1) to 40 mg/day SC elamipretide for 4 weeks followed by placebo SC for 4 weeks, separated by a 4-week washout period, or the opposite sequence.

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

  1. Version 2Current

    9 Oct 2026, 18:17 UTC

    Study results updated

  2. Version 1

    9 Oct 2026, 02:47 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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