- recruitingPhase III/IV
A Phase IIIb/IV, randomized, double-blind, parallel-group, placebo-controlled, trial to evaluate the efficacy and safety of daily subcutaneous injections of elamipretide in patients with genetically confirmed Barth syndrome
This study is looking into a new drug, elamipretide, for boys and men with Barth syndrome. It aims to see how safe and effective daily injections of this drug are compared to a dummy treatment, hoping to improve energy levels in cells.
United Kingdom · England · Australia - not yet recruitingPHASE4
Clinical Trial in Patients With Barth Syndrome- 4TAZPower
This study is looking at a new medicine called elamipretide for boys and men with Barth Syndrome. We want to see if it helps improve muscle strength and is safe to use. It's already approved in the US for this condition.
United States · United Kingdom