Clinical Trial in Patients With Barth Syndrome- 4TAZPower
ConditionBarth Syndrome
Trial statusNot yet recruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexMale
Age5-55
SponsorStealth BioTherapeutics Inc.
About this trial
Phase 3b/4, randomized, double-blind, parallel-group, placebo-controlled clinical trial to evaluate the efficacy, safety, and pharmacokinetics of a once daily SC injection of elamipretide in subjects with genetically confirmed BTHS for 72 weeks. The primary trial objective is to confirm the efficacy of elamipretide which is approved in the United States(FORZINITY™) under the accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint.
Eligibility criteria
Qualifiers
Willing and able to provide signed informed consent form (ICF) prior to participation in any trial-related procedures. If applicable, informed consent in writing from parent(s) or legally-acceptable representative(s) and, informed assent from subject (if age appropriate according to local requirements) should be provided.
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
Disqualifiers
Unable to perform the required functional tests or undergo echocardiography.
History of solid organ transplant, except successful cardiac transplantation > 12 months prior to screening, if, in the opinion of the Investigator, there is no evidence of organ rejection and post-transplant pharmacotherapy, is stable, and does not pose additional safety risk to participant.
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.
Trial design
Treatments tested in this trial
- Elamipretide
- Placebo
Treatment groups
48 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov