A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism
Reid Thompson W, Hornby B, Manuel R, et al. Genetics in Medicine 2021;23:471-478.
What it found
In the placebo-controlled crossover part, neither main goal (6-minute walk distance and a symptom score) was met. In the open-label extension, the 8 people who reached 36 weeks walked on average 95.9 m further than at the extension's start, with gains in knee muscle strength and some heart measures.
Limitations
Very small trial in a rare disease. The improvements came in the open-label part, without a placebo comparison. One author was employed by the developer.
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