Human study PLS-00107

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

Randomized controlled trial · 218 participants enrolled

Version 1 · published November 7, 2026 · evidence through November 7, 2026 · research snapshot 2026.1.1 · methodology ev-1 · Automated assessment by AI (claude-opus-5-5 and claude-sonnet-5-5), not reviewed by a person.

Only the abstract was available. Essential methods may be missing, which limits what can be judged.

Reports of this study

Every publication, registration and abstract describing this one study. Participants are counted once.

Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial.primary · Neurology · 2023-06-02 · PMID 37268435 · DOI

Design and participants

Design: Randomized controlled trial
Population: Participants with genetically confirmed primary mitochondrial myopathy, most with mtDNA alterations and the rest with nDNA defects. Mean age 45.6 years, 64% women, 94% White.
Conditions: Primary mitochondrial myopathy
Planned enrollment (registry): Not reported
Enrolled: 218
Randomized: 218
Completed follow-up: Not reported
Funding: PubMed publication type lists Research Support, Non-U.S. Gov't. The funder is not named in the record.
Authors’ conflicts: Not stated in the record.

Intervention and comparator

What each group received. Related peptides, analogs and combinations are never treated as the compound itself.

Elamipretide (intervention) · elamipretide · Elamipretide given subcutaneously once daily · subcutaneous · 24 weeks · 109 assigned
Placebo (comparator) · Placebo given subcutaneously · placebo · subcutaneous · 24 weeks · 109 assigned

What the authors reported

As stated in the sources. This is the authors’ account, not Peptide Library’s assessment.

6-minute walk test distance

primary outcome · patient-important · Week 24 · Change from baseline in distance walked on the 6MWT (meters)

Elamipretide vs Placebo: mean difference -3.2 (95% CI -18.7 to 12.3), p = 0.69 — The between-group difference in least squares mean change in 6MWT distance was not significant.

PMMSA total fatigue score

primary outcome · patient-important · Week 24 · Change from baseline in total fatigue score on the Primary Mitochondrial Myopathy Symptom Assessment

Elamipretide vs Placebo: mean difference -0.07 (95% CI -0.1 to 0.26), p = 0.37 — The between-group difference in least squares mean change in PMMSA total fatigue score was not significant.

PMMSA most bothersome symptom score

secondary outcome · patient-important · Most bothersome symptom score on the PMMSA

No results reported.

Neuro-QoL Fatigue Short-Form score

secondary outcome · patient-important · Neuro-QoL Fatigue Short-Form T score

No results reported.

Patient global impression of PMM symptoms

secondary outcome · patient-important · Patient global impression

No results reported.

Clinician global impression of PMM symptoms

secondary outcome · patient-important · Clinician global impression

No results reported.

Adverse events

unclear outcome · safety · Over 24 weeks of treatment · Adverse events and their severity

Elamipretide: narrative — Elamipretide was well tolerated, with most adverse events mild to moderate in severity.

Adverse events

Adverse events (unspecified), mostly mild to moderate · Elamipretide: Not reported participants

Monitoring: The abstract reports adverse events and their severity. No other monitoring details are given.

The authors concluded (paraphrased): Subcutaneous elamipretide did not improve 6MWT distance or PMMSA total fatigue compared with placebo at 24 weeks in primary mitochondrial myopathy, but it was well tolerated.

Peptide Library’s assessment

An automated assessment, made by AI separately from the authors’ conclusions and not reviewed by a person. How risk of bias is assessed.

Limitations

abstract only: Only the abstract is available, so details of allocation, blinding, attrition and analysis populations cannot be checked.
selective reporting: Secondary endpoints (most bothersome symptom, Neuro-QoL fatigue, patient and clinician global impressions) are named but no results are given in the abstract.
short follow up: Treatment and assessment lasted only 24 weeks, which may be short for a chronic genetic myopathy.
other: Safety is described only narratively (well tolerated, mostly mild to moderate AEs), without counts by arm or serious event data.

6-minute walk test distance: Some concerns (Cochrane RoB 2)

Risk of bias in one result of a randomized trial, judged in five domains.

Domain judgments
Randomization process	Some concerns	1:1 randomization is stated, but sequence generation and allocation concealment are not described in the abstract.
Deviations from intended interventions	Some concerns	The trial is described as double-blind and placebo-controlled, but adherence and the analysis population are not reported in the abstract.
Missing outcome data	Some concerns	The numbers completing and analysed at week 24 are not reported in the abstract.
Measurement of the outcome	Some concerns	The 6MWT is effort-dependent. Assessor blinding is implied by double-blinding but not detailed.
Selection of the reported result	Some concerns	This is a prespecified primary endpoint with a reported estimate and CI, but no protocol or SAP was available to verify the analysis.

The trial is a double-blind RCT, but abstract-only reporting leaves allocation, attrition and analysis details unverified.

Version 1 · published November 7, 2026 · evidence through November 7, 2026 · research snapshot 2026.1.1 · methodology ev-1 · RoB 2 (2019-08-22) · Automated assessment by AI (claude-opus-5-5 and claude-sonnet-5-5), not reviewed by a person.

PMMSA total fatigue score: Some concerns (Cochrane RoB 2)

Risk of bias in one result of a randomized trial, judged in five domains.

Domain judgments
Randomization process	Some concerns	1:1 randomization is stated, but allocation concealment and sequence generation are not described in the abstract.
Deviations from intended interventions	Some concerns	The trial is double-blind and placebo-controlled, but deviations and the analysis population are not described.
Missing outcome data	Some concerns	Completion and analysed numbers for the fatigue score at week 24 are not given in the abstract.
Measurement of the outcome	Some concerns	The score is patient-reported. Blinding is stated as double-blind, but there are no further details on maintaining the blind.
Selection of the reported result	Some concerns	This is a prespecified primary endpoint reported with CI, but the analysis plan could not be checked from the abstract.

The trial is a blinded RCT, but abstract-only information leaves several domains unverifiable.

Adverse events: High risk of bias (Cochrane RoB 2)

Risk of bias in one result of a randomized trial, judged in five domains.

Domain judgments
Randomization process	Some concerns	Randomization is stated, but the concealment method is not described in the abstract.
Deviations from intended interventions	Some concerns	The trial is placebo-controlled and double-blind. Exposure and adherence data are not reported in the abstract.
Missing outcome data	Some concerns	The number of participants contributing safety data is not reported.
Measurement of the outcome	Some concerns	The methods for collecting and grading adverse events are not described in the abstract.
Selection of the reported result	High risk of bias	Safety is summarised only narratively, with no counts by arm, so selective presentation cannot be excluded.

The safety result is a narrative statement without arm-level data in the abstract, so its reporting carries high risk of bias.

Claims this study informs

Improves function in Barth syndrome: not eligible (wrong population) for “Muscle strength, exercise capacity and fatigue”
Improves exercise capacity in primary mitochondrial myopathy: eligible for “Six-minute walk distance and fatigue”, direct evidence
Is safe for human administration: eligible for “Adverse events, including injection-site reactions, and discontinuations”, indirect evidence

Low certainty Six-minute walk distance and fatigue
Insufficient information to assess Adverse events, including injection-site reactions, and discontinuations

Where each value comes from

adverse_events: PMID 37268435, abstract (Results final sentence) · extracted by AI (automated, not verified by a person) · abstract only
arms: PMID 37268435, abstract (Methods sentence 1) · extracted by AI (automated, not verified by a person) · abstract only
author_conclusion: PMID 37268435, abstract (Discussion sentences 1-2) · extracted by AI (automated, not verified by a person) · abstract only
design: PMID 37268435, abstract (Background and objectives, final sentence; Methods sentence 1) · extracted by AI (automated, not verified by a person) · abstract only
funding: PMID 37268435, abstract (PubMed publication types list) · extracted by AI (automated, not verified by a person) · abstract only
limitations: PMID 37268435, abstract (Methods and Results sections overall) · extracted by AI (automated, not verified by a person) · abstract only
outcomes: PMID 37268435, abstract (Methods sentences 2-3) · extracted by AI (automated, not verified by a person) · abstract only
participants: PMID 37268435, abstract (Results sentence 1) · extracted by AI (automated, not verified by a person) · abstract only
population: PMID 37268435, abstract (Results sentences 2-3) · extracted by AI (automated, not verified by a person) · abstract only
results: PMID 37268435, abstract (Results sentences 6-8) · extracted by AI (automated, not verified by a person) · abstract only

Version 1 · published November 7, 2026 · evidence through November 7, 2026 · research snapshot 2026.1.1 · methodology ev-1 · Automated assessment by AI (claude-opus-5-5 and claude-sonnet-5-5), not reviewed by a person.

These assessments describe published research. Records marked automated were produced by AI under the published methodology and have not been reviewed by a person; they can contain errors. None of this is medical advice, not a statement about any product’s safety for human use, and not a regulatory approval. Peptide Library’s vendor and partner relationships play no part in them (editorial independence).