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Phase 3b/4, Randomized, Double-Blind, Parallel-Group, Placebo-Controlled, Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Patients With Genetically Confirmed Barth Syndrome

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Trial Profile

Phase 3b/4, Randomized, Double-Blind, Parallel-Group, Placebo-Controlled, Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Patients With Genetically Confirmed Barth Syndrome

Status: Not yet recruiting
Phase of Trial: Phase III/IV

Latest Information Update: 14 May 2026

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At a glance

  • Drugs Elamipretide (Primary)
  • Indications Barth syndrome
  • Focus Therapeutic Use
  • Acronyms 4TAZPower
  • Sponsors Stealth BioTherapeutics

Most Recent Events

  • 30 Apr 2026 According to Stealth BioTherapeutics media release, company has initiated the first site in its Phase 4 trial, which is in Bristol, UK.
  • 21 Apr 2026 New trial record

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