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: verified against primary source
| 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 and trial registry ID are not confirmed; see identifier status. |
Identifiers: DOI verified; PMID not reported; trial registry id not reported. Record-level verification and identifier-level status are separate: a record can be verified against its primary publication on one identifier while another is simply not confirmed. No identifier is fabricated, and none is left in a “pending” state.
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 names the source and the date. An identifier that is not confirmed is named as such in the identifier row above, never filled in from a secondary source.