NCT07531251 Clinical Trial in Patients With Barth Syndrome- 4TAZPower
| NCT ID | NCT07531251 |
| Status | Recruiting |
| Phase | Phase 4 |
| Sponsor | Stealth BioTherapeutics Inc. |
| Condition | Barth Syndrome |
| Study Type | INTERVENTIONAL |
| Enrollment | 48 participants |
| Start Date | 2026-07-02 |
| Primary Completion | 2029-09-30 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 4 studies follow an already-approved treatment in real-world conditions to monitor long-term safety and effectiveness.
This trial targets 48 participants in total. It began in 2026-07-02 with a primary completion date of 2029-09-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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.
Eligibility Criteria
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 IMP. Key Exclusion Criteria: 1. Unable to perform the required functional tests or undergo echocardiography. 2. History of solid organ transplant, except successful cardiac transplantation \> 12 months prior to screening, if, in the opinion of the Investigator, there is no evidence of organ rejection and post-transplant pharmacotherapy, is stable, and does not pose additional safety risk to participant. 3. Patients with an implantable cardioverter defibrillator (ICD) and with a known occurrence of ICD discharge in the 3 months prior to the Screening Visit. 4. Current placement on the waiting list for heart transplantation. 5. Hospitalization for heart failure within 6 months prior to the Screening Visit. 6. Any disease or medical condition that in the opinion of the Investigator would prevent the subject from successfully participating in the trial and reliably completing the assessments or might confound trial results. 7. Has a history of a systemic eosinophilic illness 8. Estimated Glomerular Filtration Rate (eGFR) of \< 30 mL/min at the Screening Visit (using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) 2021 formula for subjects \>16 years of age and the Schwartz 2009 formula for subjects 5-16 years of age). 9. Active malignancy or any other cancer from which the subject has been cancer-free for \< 2 years. Localized squamous or non-invasive basal cell skin carcinomas are allowed, if appropriately treated prior to Screening. 10. Participation in other investigational drug or device clinical trials within 30 days or 5 half-lives (whichever is longer) of Screening; or is currently enrolled in a non-interventional clinical trial that, in the opinion of the Investigator, may be potentially confounding to the results of the current trial. 11. History of allergic reaction to the IMP or any of its components. 12. Prior participation in any elamipretide trial or expanded access programs.
Contact & Investigator
Rekha Sathyanarayana
STUDY DIRECTOR
Stealth BioTherapeutics Inc.
Frequently Asked Questions
Who can join the NCT07531251 clinical trial?
This trial is open to male participants only, aged 5 Years or older, up to 55 Years, studying Barth Syndrome. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07531251 trial and what does that mean for participants?
Phase 4 studies are conducted after a treatment has been approved. They monitor long-term safety and real-world effectiveness in a broader patient population.
Is NCT07531251 currently recruiting?
Yes, NCT07531251 is actively recruiting participants. Contact the research team at rekha.sathyanarayana@stealthbt.com for enrollment information.
Where is the NCT07531251 trial being conducted?
This trial is being conducted at Needham, United States, Calgary, Canada, Bristol, United Kingdom.
Who is sponsoring the NCT07531251 clinical trial?
NCT07531251 is sponsored by Stealth BioTherapeutics Inc.. The principal investigator is Rekha Sathyanarayana at Stealth BioTherapeutics Inc.. The trial plans to enroll 48 participants.