4TAZPower: Phase 3b/4 Trial of Elamipretide in Barth Syndrome.
Research type
Research Study
Full title
A 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
IRAS ID
1013337
Contact name
Jennifer Yates
Contact email
Sponsor organisation
Stealth BioTherapeutics Inc.
ISRCTN Number
ISRCTN84705575
Research summary
Barth syndrome is a rare genetic disorder that affects multiple body systems, particularly the heart, muscles, and immune system. It is caused by mutations in the TAZ gene, which is located on the X chromosome. This condition is almost exclusively diagnosed in males, while females can be carriers without showing symptoms.
In the United States, elamipretide (Forzinity™), has gained accelerated approval in patients with Barth syndrome weighing at least 30kg, pending the outcome of this confirmatory trial. There are currently no available approved therapies for Barth syndrome outside of the United States.
The study medication elamipretide is a protective agent specific to the mitochondria of cells (where energy is produced). The information gained in this study will help confirm how well elamipretide works to treat Barth Syndrome, and how well patients tolerate it. The study medication will be given as an under-the-skin (subcutaneous) injection in the stomach area or thigh.
This study will enroll approximately 48 males ≥ 5 years of age located outside of the United States, including the United Kingdom, European Union, and Australia, with genetically confirmed Barth syndrome. For 72 weeks of treatment, half of the participants will receive elamipretide once daily. The other half of the participants will receive the placebo (inactive substance) once daily.
This is a Phase3b/4 randomised, double-blind, placebo-controlled study. Randomised means that a computer program will randomly assign the treatment each participant receives. Double-blind means none of the participants, researchers, study doctors, or other study staff will know what treatment each participant receives. Placebo-controlled means that some participants will receive a placebo (something that looks identical to study medication but contains no active ingredients).
The Sponsor of the study is Stealth BioTherapeutics Inc.REC name
South West - Central Bristol Research Ethics Committee
REC reference
26/SW/0009
Date of REC Opinion
24 Feb 2026
REC opinion
Further Information Favourable Opinion