Study record
MMPOWER-3: elamipretide Phase 3 in primary mitochondrial myopathy — failed primary (Karaa et al., 2023)
Human RCT — functional endpoint (failed)
○ Evidence tier 3 — Human RCT — functional endpoint
Record verify: primary source pending
| Design | Randomized, double-blind, placebo-controlled Phase 3 trial (functional endpoints; negative) |
| DOI | 10.1212/WNL.0000000000207402 |
| Citation status | doi verified via Crossref 2026-07-12 (Neurology 2023;101(3), Karaa et al., 'Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy' = MMPOWER-3 primary results). Barth-syndrome FDA accelerated approval (FORZINITY, Sept 2025) verified via FDA press release + Stealth BioTherapeutics announcement (websearch 2026-07-12). PMID + NCT: needs_primary_fulltext. |
Five-qualifier claim
| Species / population | Adults with genetically confirmed primary mitochondrial myopathy — a DISEASE population. |
| Exposure, route, schedule | Subcutaneous elamipretide daily for 24 weeks. |
| Comparator / duration | Placebo-controlled, randomized, double-blind; 24-week primary readout. |
| Endpoint / numeric result | Did NOT meet its co-primary endpoints (6-minute walk distance and a mitochondrial-myopathy symptom assessment). |
| What it did NOT establish | A failed Phase 3 in a disease population; NOT an aging trial and NOT evidence of any healthy-aging benefit. Separately, elamipretide later received FDA accelerated approval for Barth syndrome (a different, rare disease) in 2025 — also not an aging indication. |
Interventions
Primary reference
https://doi.org/10.1212/WNL.0000000000207402
Exact identifier confirmed against the primary record via Crossref/ClinicalTrials.gov where stated in the citation status; anything marked needs_primary_fulltext is not yet confirmed.