Study record · Elamipretide
MMPOWER acute dose study in primary mitochondrial myopathy
Highest-dose group improved 64.5 m versus 20.4 m with placebo; nominal P=.053, with dose-response signal and adjusted analyses favoring high dose.
Key result
Highest-dose group improved 64.5 m versus 20.4 m with placebo; nominal P=.053, with dose-response signal and adjusted analyses favoring high dose.
Study details
Study designRandomized placebo-controlled dose-ranging study
PhasePhase II
PopulationPrimary mitochondrial myopathy
Sample size36
RouteIntravenous infusion
Regimen / exposure0.01, 0.1 or 0.25 mg/kg/hour for 2 hours
Duration5 consecutive days
ComparatorPlacebo
Primary endpoint / question6-minute walk test / exercise performance
Safety resultShort exposure; generally tolerated in study.
Key limitationsSmall exploratory study; short duration; nominal primary comparison did not cross conventional significance threshold.
Registry / identifierPMID 29500292