Clinical Trial in Patients With Barth Syndrome- 4TAZPower

Summary

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.

Timeline

Start

2026-07-02

Primary completion

2029-09-30

Completion

2029-11-30

Drugs

Indications