Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER
Thompson WR, Manuel R, Abbruscato A, et al. (2024). Genetics in Medicine.
Summary
Reports the 168-week open-label extension of TAZPOWER, a 28-week randomised placebo-controlled trial of elamipretide in Barth syndrome, a rare genetic mitochondrial disease. Ten patients entered the extension and eight reached week 168. Injection-site reactions were the most common adverse event. Six-minute walk distance improved from the extension baseline at every time point (about 96 metres cumulatively at week 168), fatigue scores were below baseline, and cardiac volumes and the cardiolipin ratio trended toward improvement. An uncontrolled extension in a very small group; three authors are employees of the sponsor.