Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathy
Karaa A, Haas R, Goldstein A, et al. Neurology 2018;90:e1212-e1221.
What it found
At the highest dose, people walked 64.5 m further on the 6-minute walk test vs 20.4 m on placebo (p = 0.053, not significant), with a dose-related trend. No other efficacy or safety differences were seen.
Limitations
Small, very short trial. The main result missed statistical significance and was not confirmed by the later phase 3 trial.
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