Human study PLS-00241

Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)

Other design · 102 participants enrolled

Version 1 · published November 8, 2026 · evidence through November 8, 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.

Reports of this study

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

Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)registry entry · NCT05162768 (COMPLETED, no results posted, planned enrollment 102)

Design and participants

Design: Other design
Population: Subjects with primary mitochondrial disease caused by nuclear DNA mutations (nPMD). Age and other eligibility details are not stated in the record.
Conditions: Mitochondrial Myopathies, Mitochondrial Pathology, Mitochondrial DNA Mutation, Mitochondrial Diseases, Mitochondrial DNA Deletion, Mitochondrial DNA Depletion, Mitochondrial Metabolism Defect, Mitochondrial Complex I Deficiency
Planned enrollment (registry): 102
Enrolled: 102
Randomized: Not reported
Completed follow-up: Not reported

Intervention and comparator

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

Elamipretide (intervention) · elamipretide · Elamipretide, also listed under its code MTP-131 · Not reported assigned
Placebo (comparator) · Placebo · placebo · Not reported assigned

What the authors reported

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

Safety

unclear outcome · safety · Not specified in the record; safety is named only in the study title.

No results reported.

Adverse events

None reported. That is not evidence of safety, and how safety was monitored isn’t described.

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 a registry entry is available, with has_results false. No outcome data, participant flow or publication is in the record.
other: The record does not state the allocation method or blinding, so randomization cannot be confirmed despite the placebo arm and Phase 3 label.
selective reporting: Registered outcome measures are not listed in the record, and no results have been posted for this completed study.

No risk-of-bias assessment has been published for this study’s results.

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: not eligible (insufficient data) for “Six-minute walk distance and fatigue”
Is safe for human administration: not eligible (insufficient data) for “Adverse events, including injection-site reactions, and discontinuations”

Where each value comes from

arms: NCT NCT05162768, registry (interventions field) · extracted by AI (automated, not verified by a person)
design: NCT NCT05162768, registry (study_type and phases fields) · extracted by AI (automated, not verified by a person)
limitations: NCT NCT05162768, registry (has_results and status fields) · extracted by AI (automated, not verified by a person)
outcomes: NCT NCT05162768, title (study title (efficacy and safety)) · extracted by AI (automated, not verified by a person)
participants: NCT NCT05162768, registry (enrollment and enrollment_type fields) · extracted by AI (automated, not verified by a person)
population: NCT NCT05162768, title (study title) · extracted by AI (automated, not verified by a person)

Version 1 · published November 8, 2026 · evidence through November 8, 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).