Human study PLS-00111

Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathy.

Randomized controlled trial · 36 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.

Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathy.primary · Neurology · 2018-03-02 · PMID 29500292 · DOI · corrected

Design and participants

Design: Randomized controlled trial
Population: Adults with genetically confirmed primary mitochondrial myopathy (PMM)
Conditions: Primary mitochondrial myopathy
Setting: Multicenter phase I/II trial; sites not described in the abstract
Planned enrollment (registry): Not reported
Enrolled: 36
Randomized: 36
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 highest dose (intervention) · elamipretide · Elamipretide given as a 2-hour intravenous infusion at the highest of three dose levels, given in a dose-escalating sequence · intravenous · 5 days of treatment · Not reported assigned
Elamipretide middle dose (intervention) · elamipretide · Elamipretide given as a 2-hour intravenous infusion at the middle of three dose levels · intravenous · 5 days of treatment · Not reported assigned
Elamipretide lowest dose (intervention) · elamipretide · Elamipretide given as a 2-hour intravenous infusion at the lowest of three dose levels · intravenous · 5 days of treatment · Not reported assigned
Placebo (comparator) · Placebo given as a 2-hour intravenous infusion · placebo · intravenous · 5 days of treatment · Not reported assigned

What the authors reported

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

Change in 6-minute walk test distance

primary outcome · patient-important · Day 5 · Distance walked in the 6-minute walk test (6MWT), change from baseline

Elamipretide highest dose vs Placebo: change from baseline 64.5, p = 0.053 — Highest-dose group walked a mean of 64.5 m farther at day 5, versus a 20.4 m change with placebo
Placebo: change from baseline 20.4 — Placebo group changed by 20.4 m at day 5
Elamipretide highest dose vs Placebo: narrative, p = 0.014 — Dose-dependent increase in 6MWT distance across elamipretide doses
Elamipretide highest dose vs Placebo: change from baseline 51.2, p = 0.0297 — Covariate-adjusted model: change of 51.2 m with the highest dose versus 3.0 m with placebo
Placebo: change from baseline 3 — Covariate-adjusted change of 3.0 m in the placebo group

Cardiopulmonary exercise testing parameters

secondary outcome · surrogate · After 5 days of treatment · Changes in cardiopulmonary exercise testing parameters

Elamipretide highest dose vs Placebo: narrative — No significant differences observed in other efficacy endpoints

Participant-reported symptoms

secondary outcome · patient-important · After 5 days of treatment · Changes in participant-reported symptoms

Elamipretide highest dose vs Placebo: narrative — No significant differences observed in other efficacy endpoints

Serum and urinary biomarkers

secondary outcome · surrogate · After 5 days of treatment · Changes in serum and urinary biomarkers

Elamipretide highest dose vs Placebo: narrative — No significant differences observed in other efficacy endpoints

Safety and tolerability

secondary outcome · safety · During 5-day treatment period · Safety and tolerability endpoints

Elamipretide highest dose vs Placebo: narrative — No significant differences in safety endpoints; authors report no increased safety concerns

Pharmacokinetics

secondary outcome · surrogate · During treatment · Pharmacokinetic parameters of elamipretide

No results reported.

Adverse events

None reported. That is not evidence of safety.

Monitoring: Safety and tolerability were measured. Specific adverse events and monitoring procedures are not described in the abstract.

The authors concluded (paraphrased): Elamipretide improved exercise performance (6MWT distance) after 5 days in adults with primary mitochondrial myopathy without increased safety concerns. The authors say larger, longer trials are needed.

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

small or imprecise: Only 36 participants were split across three dose groups and placebo. The unadjusted primary comparison was not significant (p = 0.053).
short follow up: Efficacy was assessed after only 5 days of treatment, so durability and longer-term safety are unknown.
selective reporting: Significance was reached only in a dose-trend analysis and a covariate-adjusted model. Other endpoints are reported only as 'no significant differences', without data.
abstract only: Only the abstract is available. Arm sizes, attrition, allocation methods and adverse event details are not reported.
other: The PubMed record is flagged as corrected. The nature of the correction is not given in the record.
funding or conflicts: Funding and conflicts of interest are not stated in the record.

Change in 6-minute walk test distance: High risk of bias (Cochrane RoB 2)