Study record · Elamipretide
MMPOWER-2 crossover trial
Treatment difference in 6MWT was 19.8 m (P=.0833); selected patient-reported fatigue measures showed signals.
Key result
Treatment difference in 6MWT was 19.8 m (P=.0833); selected patient-reported fatigue measures showed signals.
Study details
Study designRandomized double-blind placebo-controlled crossover study
PhasePhase II
PopulationPrimary mitochondrial myopathy
Sample size30
RouteSubcutaneous
Regimen / exposure40 mg once daily
Duration4 weeks per treatment period
ComparatorPlacebo
Primary endpoint / question6-minute walk test and fatigue
Safety resultInjection-site reactions occurred in about 80% during elamipretide exposure.
Key limitationsSmall crossover study; primary functional difference was not statistically significant.
Registry / identifierPMID 32096613