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4TAZPower trial of elamipretide in genetically confirmed Barth syndrome
2025-523837-25-00Phase 4Authorised
- Sponsor
- Stealth BioTherapeutics
- Start
- Jul 2026
- Modality
- Recombinant Proteins and Enzymes, Peptides
- Countries
- 8
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Overview
Phase 4, randomised, double-blind trial of semaglutide in Barth syndrome (BTHS) (2 arms), sponsored by Stealth BioTherapeutics. Primary endpoint: Change from baseline to Week 72 in composite normalized functional score. Planned start Jul 2026; status: authorised.
02
Drugs
03
Conditions
Barth syndrome (BTHS)
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