Clinical Trial: Study to Evaluate Pharmacokinetics, Safety, and Tolerability of Subcutaneous Injections of Elamipretide in Pediatric Subjects With Barth Syndrome

U.S., Nov. 11 -- ClinicalTrials.gov registry received information related to the study (NCT07927972) titled 'Study to Evaluate Pharmacokinetics, Safety, and Tolerability of Subcutaneous Injections of Elamipretide in Pediatric Subjects With Barth Syndrome' on Oct. 23.

Brief Summary: This Phase 1, open-label trial will enroll approximately 6 subjects who have BTHS weighing between 7 and 30 kg. Subjects will receive a weight-based dose of elamipretide daily via SC injection for 4 weeks. PK samples will be collected on Day 1 and at week 4 to determine PK profile in BTHS patients weighing between 7 and 30kg.

Study Start Date: Nov., 2026

Study Type: INTERVENTIONAL

Condition: Barth Syndrome

Intervention: DRUG: Elamipretide

SC injection dai...