A randomized crossover trial of elamipretide in adults with primary mitochondrial myopathy.

ABSTRACT:

Background

This study aims to evaluate the effect of subcutaneous (SC) elamipretide dosing on exercise performance using the 6 min walk test (6MWT), patient-reported outcomes measuring fatigue, functional assessments, and safety to guide the development of the Phase 3 trial.

Methods

MMPOWER-2 was a randomized, double-blind, placebo-controlled, crossover trial that enrolled participants (N = 30) with genetically confirmed primary mitochondrial myopathy. Participants were randomly assigned (1:1) to 40 mg/day SC elamipretide for 4 weeks followed by placebo SC for 4 weeks, separated by a 4-week washout period, or the opposite sequence. The primary endpoint was the distance walked on the 6MWT.

Results

The distance walked on the 6MWT by the elamipretide-treated participants

SUBMITTER: Karaa A 

PROVIDER: S-EPMC7432581 | biostudies-literature | 2020 Aug

REPOSITORIES: biostudies-literature