Human study PLS-00116

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

Randomized controlled trial · 30 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.

A randomized crossover trial of elamipretide in adults with primary mitochondrial myopathy.primary · Journal of cachexia, sarcopenia and muscle · 2020-02-25 · PMID 32096613 · DOI

Design and participants

Design: Randomized controlled trial
Population: Adults with genetically confirmed primary mitochondrial myopathy
Conditions: Primary mitochondrial myopathy
Planned enrollment (registry): Not reported
Enrolled: 30
Randomized: 30
Completed follow-up: Not reported
Funding: Not stated in the abstract. The PubMed publication type lists non-U.S. government research support.
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 period (intervention) · elamipretide · Elamipretide given by daily subcutaneous injection during one crossover period; sequence randomized 1:1 against placebo with a 4-week washout · subcutaneous · 4 weeks · Not reported assigned
Placebo period (comparator) · Placebo given by subcutaneous injection during the other crossover period · placebo · subcutaneous · 4 weeks · Not reported 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 · End of each 4-week treatment period · Distance walked on the 6MWT (meters)

Elamipretide period: mean 398.3 — Elamipretide-treated participants walked 398.3 m (SD 134.16)
Placebo period: mean 378.5 — Placebo-treated participants walked 378.5 m (SD 125.10)
Elamipretide period vs Placebo period: mean difference 19.8 (95% CI -2.8 to 42.5), p = 0.0833 — Difference of 19.8 m favoring elamipretide, not statistically significant

PMMSA Total Fatigue score

secondary outcome · patient-important · End of each 4-week treatment period · Primary Mitochondrial Myopathy Symptom Assessment Total Fatigue score

Elamipretide period vs Placebo period: narrative, p = 0.0006 — Less fatigue reported with elamipretide than with placebo

PMMSA Total Fatigue During Activities score

secondary outcome · patient-important · End of each 4-week treatment period · Primary Mitochondrial Myopathy Symptom Assessment Total Fatigue During Activities score

Elamipretide period vs Placebo period: narrative, p = 0.0018 — Less fatigue and fewer muscle complaints during activities with elamipretide than with placebo

Neuro-QoL Fatigue Short Form

secondary outcome · patient-important · End of each 4-week treatment period · Neuro-QoL Fatigue Short Form score

Elamipretide period vs Placebo period: narrative, p = 0.0115 — Reduction in fatigue symptoms with elamipretide compared with placebo

Patient Global Assessment

secondary outcome · patient-important · End of each 4-week treatment period · Patient Global Assessment

Elamipretide period vs Placebo period: narrative, p = 0.0421 — Reduction in symptoms with elamipretide compared with placebo

Physician Global Assessment

secondary outcome · patient-important · End of each 4-week treatment period · Physician Global Assessment

Elamipretide period vs Placebo period: narrative, p = 0.0636 — No statistically significant change versus placebo

Triple Timed Up and Go test

secondary outcome · patient-important · End of each 4-week treatment period · Triple Timed Up and Go functional test

Elamipretide period vs Placebo period: narrative, p = 0.8423 — No statistically significant change versus placebo

Wrist and hip accelerometry

secondary outcome · surrogate · End of each 4-week treatment period · Wrist and hip accelerometry activity measures

Elamipretide period vs Placebo period: narrative, p = 0.9345 — Wrist accelerometry showed no statistically significant change versus placebo
Elamipretide period vs Placebo period: narrative, p = 0.7326 — Hip accelerometry showed no statistically significant change versus placebo

Adverse events

secondary outcome · safety · During treatment periods · Reported adverse events, serious adverse events and deaths

Elamipretide period: proportion — Injection site reactions were the most common adverse events with elamipretide, mostly mild
Elamipretide period vs Placebo period: narrative — No serious adverse events or deaths were reported

Adverse events

Injection site reactions · Elamipretide period: Not reported participants
Serious adverse events (serious) · Elamipretide period: 0 participants
Death (serious) · Elamipretide period: 0 participants

Monitoring: Safety was a stated study aim. The abstract reports adverse events, serious adverse events and deaths but gives no details of how monitoring was done.

The authors concluded (paraphrased): Four weeks of daily subcutaneous elamipretide gave a clinically meaningful but not statistically significant 6MWT gain. Secondary and patient-reported fatigue outcomes suggested benefit, supporting the Phase 3 MMPOWER-3 trial.

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

short follow up: Each treatment period lasted only 4 weeks, which is too short to judge sustained effects in a chronic myopathy.
small or imprecise: Only 30 participants were enrolled, and the 95% CI for the primary 6MWT difference crosses zero (-2.8 to 42.5).
selective reporting: Benefit is claimed from multiple secondary endpoints with nominal p-values, with no multiplicity adjustment described, while the primary endpoint was not significant.
inadequate blinding: Injection site reactions occurred in 80% of participants on elamipretide, which could reveal treatment allocation for self-reported fatigue outcomes.
abstract only: Only the abstract is available, so randomization methods, carryover analysis, attrition and analyzed numbers cannot be checked.
other: Crossover design with a 4-week washout; the abstract does not say whether carryover or period effects were assessed.

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