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Study · Randomized controlled trial · n = 218 · 2023

3 results

Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial.

Karaa A, Bertini E, Carelli V, Cohen BH, Enns GM, Falk MJ, Goldstein A, Gorman GS, Haas R, Hirano M, Klopstock T, Koenig MK and 20 more

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Kornblum C, Lamperti C, Lehman A, Longo N, Molnar MJ, Parikh S, Phan H, Pitceathly RDS, Saneto R, Scaglia F, Servidei S, Tarnopolsky M, Toscano A, Van Hove JLK, Vissing J, Vockley J, Finman JS, Brown DA, Shiffer JA, Mancuso M

Neurology2 Jun 2023Cited by 66 (Europe PMC)

PMID37268435 (opens in a new tab)PMCIDPMC10382259 (opens in a new tab)DOI10.1212/wnl.0000000000207402 (opens in a new tab)PepBankPUB-37268435
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Summary

Background and objectivesPrimary mitochondrial myopathies (PMMs) encompass a group of genetic disorders that impair mitochondrial oxidative phosphorylation, adversely affecting physical function, exercise capacity, and quality of life (QoL). Current PMM standards of care address symptoms, with limited clinical impact, constituting a significant therapeutic unmet need. We present data from MMPOWER-3, a pivotal, phase-3, randomized, double-blind, placebo-controlled clinical trial that evaluated the efficacy and safety of elamipretide in participants with genetically confirmed PMM.MethodsAfter screening, eligible participants were randomized 1:1 to receive either 24 weeks of elamipretide at a dose of 40 mg/d or placebo subcutaneously. Primary efficacy endpoints included change from baseline to week 24 on the distance walked on the 6-minute walk test (6MWT) and total fatigue on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA). Secondary endpoints included most bothersome symptom score on the PMMSA, NeuroQoL Fatigue Short-Form scores, and the patient global impression and clinician global impression of PMM symptoms.ResultsParticipants (N = 218) were randomized (n = 109 elamipretide; n = 109 placebo). The m0ean age was 45.6 years (64% women; 94% White). Most of the participants (n = 162 [74%]) had mitochondrial DNA (mtDNA) alteration, with the remainder having nuclear DNA (nDNA) defects. At screening, the most frequent bothersome PMM symptom on the PMMSA was tiredness during activities (28.9%). At baseline, the mean distance walked on the 6MWT was 336.7 ± 81.2 meters, the mean score for total fatigue on the PMMSA was 10.6 ± 2.5, and the mean T score for the Neuro-QoL Fatigue Short-Form was 54.7 ± 7.5. The study did not meet its primary endpoints assessing changes in the 6MWT and PMMSA total fatigue score (TFS). Between the participants receiving elamipretide and those receiving placebo, the difference in the least squares mean (SE) from baseline to week 24 on distance walked on the 6MWT was -3.2 (95% CI -18.7 to 12.3; p = 0.69) meters, and on the PMMSA, the total fatigue score was -0.07 (95% CI -0.10 to 0.26; p = 0.37). Elamipretide treatment was well-tolerated with most adverse events being mild to moderate in severity.DiscussionSubcutaneous elamipretide treatment did not improve outcomes in the 6MWT and PMMSA TFS in patients with PMM. However, this phase-3 study demonstrated that subcutaneous elamipretide is well-tolerated.Trial registration informationTrial registered with clinicaltrials.gov, Clinical Trials Identifier: NCT03323749; submitted on October 12, 2017; first patient enrolled October 9, 2017.Clinicaltrialsgov/ct2/show/NCT03323749?term = elamipretide&draw = 2&rank = 9.Classification of evidenceThis study provides Class I evidence that elamipretide does not improve the 6MWT or fatigue at 24 weeks compared with placebo in patients with primary mitochondrial myopathy.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study3

3 results from studies that gave the peptide. 3 results from people.

  1. 01HumanRandomized controlled trialEfficacy outcome

    In adults with primary mitochondrial myopathy, elamipretide did not significantly change 6-minute walk distance from baseline to week 24 versus placebo.

    In plain wordsIn adults with a mitochondrial muscle disease, elamipretide did not improve walking distance more than placebo.

    Peptide
    Elamipretide (SS-31)
    Outcome
    No significant change6MWT distance change from baseline to week 24
    Study

    n = 218

    Comparator
    Placebo
    Analysis
    Primary
    Outcome group
    Exercise and physical function · Musculoskeletal
    Studied dose
    40 mg/d · subcutaneous · 24 weeksAs given in this study (human).

    Measurements

    6MWT least squares mean difference
    −3.2 m
    elamipretide vs placebo, week 24

    Source sentences

    Summary
    the difference in the least squares mean (SE) from baseline to week 24 on distance walked on the 6MWT was -3.2 (95% CI -18.7 to 12.3; p = 0.69) meters
    2 context sentences
    Summary · context
    After screening, eligible participants were randomized 1:1 to receive either 24 weeks of elamipretide at a dose of 40 mg/d or placebo subcutaneously.
    Summary · context
    Participants (N = 218) were randomized (n = 109 elamipretide; n = 109 placebo).
  2. 02HumanRandomized controlled trialEfficacy outcome

    In adults with primary mitochondrial myopathy, elamipretide did not significantly change PMMSA total fatigue score from baseline to week 24 versus placebo.

    In plain wordsIn adults with a mitochondrial muscle disease, elamipretide did not reduce reported fatigue more than placebo.

    Peptide
    Elamipretide (SS-31)
    Outcome
    No significant changePMMSA total fatigue score change from baseline to week 24
    Study

    n = 218

    Comparator
    Placebo
    Analysis
    Primary
    Outcome group
    Exercise and physical function · Whole body
    Studied dose
    40 mg/d · subcutaneous · 24 weeksAs given in this study (human).

    Measurements

    PMMSA total fatigue score difference
    −0.07 score
    elamipretide vs placebo, week 24

    Source sentences

    Summary
    on the PMMSA, the total fatigue score was -0.07 (95% CI -0.10 to 0.26; p = 0.37).
    2 context sentences
    Summary · context
    After screening, eligible participants were randomized 1:1 to receive either 24 weeks of elamipretide at a dose of 40 mg/d or placebo subcutaneously.
    Summary · context
    Participants (N = 218) were randomized (n = 109 elamipretide; n = 109 placebo).
  3. 03HumanRandomized controlled trialSafety outcome

    In adults with primary mitochondrial myopathy, elamipretide was well tolerated, with most adverse events mild to moderate in severity.

    In plain wordsIn adults with a mitochondrial muscle disease, elamipretide was well tolerated, and most side effects were mild or moderate.

    Peptide
    Elamipretide (SS-31)
    Outcome
    Not applicableTolerability and adverse event severity
    Study

    n = 218

    Outcome group
    Adverse events · Whole body
    Studied dose
    40 mg/d · subcutaneous · 24 weeksAs given in this study (human).

    Source sentences

    Summary
    Elamipretide treatment was well-tolerated with most adverse events being mild to moderate in severity.
    2 context sentences
    Summary · context
    After screening, eligible participants were randomized 1:1 to receive either 24 weeks of elamipretide at a dose of 40 mg/d or placebo subcutaneously.
    Summary · context
    Participants (N = 218) were randomized (n = 109 elamipretide; n = 109 placebo).

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

Registered trials1

Trials this study reports on, or that list this study as a reference. Registry data from ClinicalTrials.gov.

1 trial · 1 stopped early · 1 with posted results · source ClinicalTrials.gov (opens in a new tab)

TerminatedPhase 3Results postedNCT03323749

A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension

Stealth BioTherapeutics Inc.n = 2189 Oct 2017 to 10 Feb 2020

Primary Mitochondrial Myopathy

Trial pageClinicalTrials.govUpdated 24 Jan 2022

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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