Clinical Trial: Ph3b/4 Trial in Patients With Barth Syndrome

U.S., April 16 -- ClinicalTrials.gov registry received information related to the study (NCT07531251) titled 'Ph3b/4 Trial in Patients With Barth Syndrome' on March 12.

Brief 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.

Study Start Date: June 30

Study Type: INTERVENTIONAL

Condition: ...