Human study PLS-00236

Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)

Single-arm trial · 28 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.

Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)registry entry · NCT02976038 (TERMINATED, results posted, planned enrollment 28)

Design and participants

Design: Single-arm trial
Population: Subjects with genetically confirmed primary mitochondrial myopathy (PMM) entering an open-label extension
Conditions: Primary Mitochondrial Disease, Primary Mitochondrial Myopathy
Planned enrollment (registry): 28
Enrolled: 28
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 (open-label) (intervention) · elamipretide · Elamipretide given open-label in an extension trial · 28 assigned

What the authors reported

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

Long-term safety and tolerability

unclear outcome · safety

No results reported.

Adverse events

None reported. That is not evidence of safety.

Monitoring: The study aims to characterize long-term safety and tolerability. No monitoring procedures are described in the record.

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

no control group: Open-label extension with a single elamipretide arm and no placebo or concurrent comparator, per the registry and title.
abstract only: Only the ClinicalTrials.gov registration is available. No publication, abstract or result values are in the record.
small or imprecise: The registry gives an actual enrollment of 28 participants, which is too small to reliably detect uncommon adverse events.
incomplete follow up: The registry status is TERMINATED, so the planned long-term follow-up may not have been completed.
selection bias: Extension participants presumably came from a prior trial, which may enrich for people who tolerated the drug. Entry criteria are not shown in the record.

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 (wrong outcome) 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 NCT02976038, registry (interventions field) · extracted by AI (automated, not verified by a person)
design: NCT NCT02976038, registry (study_type and phases fields; title (open-label extension)) · extracted by AI (automated, not verified by a person)
limitations: NCT NCT02976038, registry (status and enrollment fields) · extracted by AI (automated, not verified by a person)
outcomes: NCT NCT02976038, title (study title (long-term safety and tolerability)) · extracted by AI (automated, not verified by a person)
participants: NCT NCT02976038, registry (enrollment and enrollment_type fields) · extracted by AI (automated, not verified by a person)
population: NCT NCT02976038, title (study title; registry conditions field) · 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).