Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial.
Amel KaraaEnrico Silvio BertiniValerio CarelliBruce H CohenGregory M EnnsMarni J FalkAmy GoldsteinGráinne Siobhan GormanRichard HaasMichio HiranoThomas KlopstockMary Kay KoenigCornelia KornblumCostanza LampertiAnna LehmanNicola LongoMaria Judit MolnarSumit ParikhHan PhanRobert D S PitceathlyRussell SanetoFernando ScagliaSerenella ServideiMark TarnopolskyAntonio ToscanoJohan L K Van HoveJohn VissingJerry VockleyJeffrey S FinmanDavid A BrownJames A ShifferMichelangelo Mancusonull nullPublished in: Neurology (2023)
Trial registered with clinicaltrials.gov, Clinical Trials Identifier: NCT03323749; submitted on October 12, 2017;first patient enrolled October 9, 2017. https://clinicaltrials.gov/ct2/show/NCT03323749?term=elamipretide&draw=2&rank=9 CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that elamipretide does not improve the 6 minute walk test or fatigue at 24 weeks compared to placebo in patients with primary mitochondrial myopathy.