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Elamipretide versus placebo in genetically confirmed Barth syndrome
ISRCTN84705575Phase 4Recruiting
- Sponsor
- Stealth BioTherapeutics
- Start
- Apr 2026
- Modality
- Peptides
01
Overview
Phase 4, randomised, double-blind trial in Barth syndrome (BTHS) comparing Elamipretide with Placebo (2 arms). Started Apr 2026; status: recruiting.
02
Drugs
elamipretide
03
Conditions
Barth syndrome (BTHS)
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