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Human evidence record · Elamipretide

MMPOWER-2 crossover trial

Treatment difference in 6MWT was 19.8 m (P=.0833); selected patient-reported fatigue measures showed signals.

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Human evidence record

What this study tells us

Participants were assigned to comparison groups by chance, usually with placebo and/or blinding to reduce bias. This is a strong design for testing cause and effect, but the population, duration and endpoints still limit what can be concluded.

Who was studied?

Primary mitochondrial myopathy

How many?

n=30

What happened?

Treatment difference in 6MWT was 19.8 m (P=.0833); selected patient-reported fatigue measures showed signals.

What stops us overclaiming?

Small crossover study; primary functional difference was not statistically significant.

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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