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

Studied

A randomized, double-blind trial compared intravenous elamipretide with placebo. The primary outcome was change in six-minute walking distance after five days.

Who / model

The study included 36 adults with genetically confirmed primary mitochondrial myopathy.

Studied dose & schedule

Dose and schedule have not yet been extracted for this summary.

Found

Walking distance increased by 64.5 metres at the highest dose versus 20.4 metres with placebo; this unadjusted comparison did not reach statistical significance. Dose-response and adjusted analyses favored elamipretide. Other efficacy and safety outcomes showed no significant differences.

Limits

The authors called for larger trials with longer treatment periods. These five-day results do not establish sustained benefit or long-term safety.

Read summary source ↗ · Prepared 9/22/2026 · AI-assisted, source-based summary