ClinicaliQ Trial Snapshot
- Clinical Trial in Patients With Barth Syndrome- 4TAZPower — Recruiting • Phase IV • Cardiology / Cardiovascular • NCT07531251.
- Intervention being tested: Once-daily subcutaneous injection of elamipretide in genetically confirmed Barth syndrome, evaluated over 72 weeks in a Phase 3b/4 randomised, double-blind, placebo-controlled design to assess efficacy, safety, and pharmacokinetics.
- Patient eligibility overview: The trial enrolls subjects with genetically confirmed Barth syndrome (BTHS), a rare X-linked mitochondrial disorder affecting cardiolipin metabolism; specific eligibility criteria would include confirmed genetic diagnosis and disease severity thresholds.
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- Quick orientation before opening the registry record.
- Checking recruitment status, phase and sponsor at a glance.
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What This Trial Is Studying
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.
Eligibility Snapshot
- Key Inclusion Criteria: 1. Willing and able to provide signed informed consent form (ICF) prior to participation in any trial-related procedures. If applicable, informed consent in writing from parent(s) or legally-acceptable representative(s) and, informed assent from subject (if age appropriate according to local requirements) should be provided. 2. Agrees to adhere to the trial requirements for the length of the trial. 3. Must have genetically confirmed Barth Syndrome (pathogenic variant in the TAZ gene) 4. Male aged ≥ 5 years at time of the Screening Visit 5. Left Ventricular Ejection fraction of ≥ 50% by 3-D Echocardiogram at the Screening Visit. 6. For subjects with a medical history of cardiomyopathy, must be on a stable regimen (unchanged and constant) of background heart failure medications for at least 3 months prior to the Screening Visit. 7. Able to administer Investigational Medicinal Product (IMP) or have an appropriate designee who can administer the IMP (i.e., a capable family member or a caregiver). 8. Subjects with female partners of childbearing potential must be willing to use a highly effective method of contraception (e.g., abstinence, dual method of contraception) from the date they sign the ICF until 28 days after the last dose of…
Use the source registry for the full inclusion and exclusion criteria before discussing referral or enrolment.