Clinical Trial in Patients With Barth Syndrome- 4TAZPower
Recruiting now · Phase 4 · Has a placebo group
Conditions studied: Barth Syndrome
In brief
Phase 3b/4, randomized, double-blind, parallel-group, placebo-controlled clinical trial to evaluate the efficacy, safety, and pharmacokinetics of a once daily SC injection of elamipretide in subjects with genetically confirmed BTHS for 72 weeks. The primary trial objective is to confirm the efficacy of elamipretide which is approved in the United States(FORZINITY™) under the accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint.
Key facts
- Study ID
- NCT07531251
- Run by
- Stealth BioTherapeutics Inc.
- People needed
- 48
- Starts
- 2026-07-02
- Expected to finish
- 2029-11-30
- Last updated by the study team
- 2026-07-13
Who can join
Age: 5 and older, up to 55. Sex: male. Healthy volunteers: not accepted.
Where it is running
- Bristol Royal Hospital for Children Upper Maudlin Street Paul O'Gorman Building — Bristol, United Kingdom (enrolling)
- Trial Not Offered in the U.S — Needham, Massachusetts, United States
- Metabolics and Genetics in Canada (MAGIC) — Calgary, Alberta, Canada
Full record on ClinicalTrials.gov
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