Phase 3b4 Study of Daily Elamipretide Injections to Treat Barth Syndrome in Males Aged 5 to 55 Years

What this Trial Is About

This trial studies males aged 5 to 55 years with genetically confirmed Barth Syndrome BTHS, a rare condition affecting muscles and heart function. It is a Phase 3b4 randomized, double-blind, placebo-controlled clinical trial designed to confirm the effectiveness and safety of elamipretide, a drug approved in the US under the name FORZINITY11, for improving muscle strength in both adults and children with BTHS weighing at least 30 kg. The study aims to verify its impact on knee extensor muscle strength, an important clinical measure. Participants will receive either a daily subcutaneous injection of elamipretide or a matching placebo for 72 weeks. The study compares these two groups to evaluate the drugs pharmacokinetics, safety, and efficacy over this period. Elamipretide is administered via sterile multi-dose vials, and the trial follows a parallel design with random assignment to treatment arms. During the trial, participants undergo various assessments including echocardiograms to measure heart function, functional tests, and evaluations of muscle strength. Researchers will monitor safety, drug effects, and adherence throughout the 72-week treatment. The main outcome is effectiveness at 72 weeks, with additional secondary endpoints also evaluated. The study is sponsored by Stealth BioTherapeutics Inc. and continues to monitor participants until late 2029.

Clinical Trial in Patients With Barth Syndrome- 4TAZPower