Clinical Trial in Patients With Barth Syndrome- 4TAZPower

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.

NCT Number	NCT07531251
Lead Sponsor	Stealth BioTherapeutics Inc.
Conditions	Barth Syndrome
Enrollment	48 participants
Start Date	2026-07-02
Primary Completion	2029-09-30 (estimated)
Study Completion	2029-11-30 (estimated)
Updated on ClinicalTrials.gov	2026-07-13