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NCT07531251: 4TAZPower
Ph3b/4 Trial in Patients With Barth Syndrome
Phase 4 trial testing Elamipretide in Barth Syndrome in 48 participants. Not yet recruiting.
30 September 2029
Quick facts
| Lead sponsor | Stealth BioTherapeutics Inc. |
|---|---|
| Phase | Phase 4 |
| Status | Not yet recruiting |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | quadruple |
| Primary purpose | treatment |
| Enrollment | 48 |
| Start date | 30 June 2026 |
| Primary completion | 30 September 2029 |
| Estimated completion | 30 November 2029 |
| Sites | 1 location across United Kingdom |
Drugs / interventions tested
- Elamipretide (ELAMIPRETIDE) — full drug profile →
- Placebo
Conditions studied
- Barth Syndrome — all drugs for Barth Syndrome →
Sponsor
Stealth BioTherapeutics Inc. — full company profile →
Who can join
Adults 5 to 55, male only, with Barth Syndrome. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
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.
Publications & conference data
No peer-reviewed publications indexed yet for this trial.
Verify or expand the search:
- PubMed search for NCT07531251
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of Elamipretide
Trials testing the same drug.
- NCT05162768 — Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mu · Phase 3 · completed
- NCT05168774 — FRDA Investigator Initiated Study (IIS) With Elamipretide · Phase 1, PHASE2 · completed
- NCT03098797 — A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome · Phase 2, PHASE3 · completed
- NCT02848313 — An Open-Label, Phase 1 Clinical Study to Evaluate the Safety and Tolerability of Subcutaneous Elamipretide in Subjects W · Phase 1 · completed
- NCT02805790 — Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study · Phase 2 · completed
Other Stealth BioTherapeutics Inc. trials
Trials by the same sponsor.
- NCT05162768 — Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mu · Phase 3 · completed
- NCT03891875 — ReCLAIM-2 Study to Evaluate Safety,Efficacy & Pharmacokinetics of Elamipretide in Subjects With AMD With Non-central GA · Phase 2 · completed
- NCT03323749 — A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension · Phase 3 · terminated
- NCT03098797 — A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome · Phase 2, PHASE3 · completed
- NCT03048617 — An Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300) · completed
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT07531251 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Stealth BioTherapeutics Inc.
- Last refreshed: 15 April 2026
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT07531251.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing