Clinical Trial in Patients With Barth Syndrome- 4TAZPower

What Was Tested

In this phase 4 trial, researchers set out to test SS-31 (Elamipretide).

It had two arms (groups): Elamipretide (active comparator) and Placebo (placebo comparator).

The registry classes the interventions as drug.

Who Took Part

48 planned participants were the target.

It was open to men only aged from 5 years up to 55 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Agrees to adhere to the trial requirements for the length of the trial. Must have genetically confirmed Barth Syndrome (pathogenic variant in the TAZ gene). Male aged ≥ 5 years at time of the Screening Visit. Able to administer Investigational Medicinal Product (IMP) or have an appropriate designee who can administer the IMP (i.e., a capable family member or a caregiver).

The full record lists 8 inclusion and 12 exclusion criteria; among the exclusions: unable to perform the required functional tests or undergo echocardiography; patients with an implantable cardioverter defibrillator (icd) and with a known occurrence of icd discharge in the 3 months prior to the screening visit; current placement on the waiting list for heart transplantation.

How the Study Was Run

Design: randomized, parallel-group and quadruple-blind.

The participants were allocated by chance, so the SS-31 (Elamipretide) group and the comparison group should start out alike.

Groups ran in parallel: each of the participants stayed in one arm throughout.

As a double-blind SS-31 (Elamipretide) study, the assignments were hidden from both participants and investigators until the end.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2026-07-02 with completion listed for 2029-09-30.

It ran at 3 sites in United States (1), Canada (1) and United Kingdom (1).

Its stated primary purpose is treatment.

What This Means in Plain Language

Put simply, this is a phase 4, randomized trial of SS-31 (Elamipretide) in 48 people.

It is still recruiting; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the SS-31 (Elamipretide) compound page, all human trials.