This study is for men aged 5 to 55 years old with Barth Syndrome, a genetic condition. It's testing a drug called elamipretide (also known as FORZINITY™) given as a shot under the skin once a day, or a placebo (an inactive shot that looks like the drug). The main goal is to see if elamipretide can improve muscle strength over 72 weeks. Elamipretide is already approved in the United States to help with muscle strength in Barth Syndrome, and this study aims to confirm its effectiveness. You would need to provide informed consent to participate.

Study design

This is a Phase 3b/4, randomized, double-blind, parallel-group, placebo-controlled study involving 48 participants.

What's involved

You would receive daily subcutaneous (under the skin) injections of either elamipretide or placebo for 72 weeks.

Compensation

Not stated in the trial record.

Follow-up

The primary efficacy endpoint is measured at 72 weeks, indicating the duration of the main treatment period.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.