Karaa 2018 — Elamipretide Dose-Escalation Trial in Primary Mitochondrial Myopathy

Neurology·March 1, 2018

Amel Karaa, Michio Hirano, Bruce H. Cohen, et al.

Summary

This early trial reported improvement in 6-minute walk distance with elamipretide in primary mitochondrial myopathy, supporting later phase 2/3 development.

Study Details

Study Design

Randomized double-blind placebo-controlled dose-escalation trial

Indication

Primary mitochondrial myopathy

Intervention

Intravenous elamipretide vs placebo

Species

Human

Sample Size

36 subjects

Risk of Bias Assessment

Small early-phase trial