Barth Syndrome Heart Treatment Study
Part of Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This trial tests a medicine called elamipretide (4TAZ) in boys with Barth Syndrome, a genetic condition affecting the heart and muscles. The goal is to see if this medicine can help improve heart function and how the body uses energy.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have been genetically diagnosed with Barth Syndrome (a mutation in the TAZ gene)
- You are a male aged 5 years or older
- Your heart's pumping strength (ejection fraction) is at least 50%
- If you take heart medications, you've been on the same doses for at least 3 months
- You or a caregiver can give yourself or receive injections of the study medicine
- You have not been in another drug trial in the past 30 days
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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