Barth syndrome – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Thu, 30 Jul 2026 11:51:40 +0000 en-US hourly 1 https://wordpress.org/?v=7.0.2 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Barth syndrome – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Efficacy and Safety of Daily Subcutaneous Elamipretide Trihydrochloride Injections in Patients with Barth Syndrome (Phase 3b/4 Randomized Trial) https://clinicaltrials.eu/trial/efficacy-and-safety-of-daily-subcutaneous-elamipretide-trihydrochloride-injections-in-patients-with-barth-syndrome-phase-3b-4-randomized-trial/ Thu, 30 Jul 2026 11:51:40 +0000 https://clinicaltrials.eu/trial/efficacy-and-safety-of-daily-subcutaneous-elamipretide-trihydrochloride-injections-in-patients-with-barth-syndrome-phase-3b-4-randomized-trial/ Barth Syndrome is a rare inherited condition that can cause heart problems and weak muscles. The study is testing a medication called Elamipretide, which is given as a daily subcutaneous injection (a small shot placed just under the skin). Some participants will receive the medication while others will receive a placebo, which looks the same but does not contain the active drug.

The purpose of the study is to find out whether daily treatment with Elamipretide can improve heart and muscle function and be safe to use. Participants will be asked to receive the assigned injection each day for a period of about one and a half years, with regular clinic visits to monitor health, record any side effects, and perform simple walking and strength tests.

During the visits, researchers will measure how far a person can walk in six minutes, how quickly they can stand up and sit down several times, and the strength of certain leg muscles using a handheld device. These tests help show any changes in the ability to move around and perform daily activities, as well as overall disease severity as judged by both the participant and the doctor.

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