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New treatment option for Barth Syndrome
Official title Clinical Trial in Patients With Barth Syndrome- 4TAZPower
ClinicalTrials.gov ID: NCT07531251
What this study is testing
What is Elamipretide?
Elamipretide is an investigational medicine, given as an once-daily injection under the skin, being studied as a potential treatment for barth syndrome.
Also referred to as elamipretide hydrochloride.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 4: studies an already-approved treatment
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 5 to 55, men only
You may be able to join if
- Willing and able to provide signed informed consent form (ICF) prior to participation in any trial-related procedures. If applicable, informed...
- 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
- Left Ventricular Ejection fraction of ≥ 50% by 3-D Echocardiogram at the Screening Visit.
You likely can't join if
- 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...
- Patients with an implantable cardioverter defibrillator (ICD) and with a known occurrence of ICD discharge in the 3 months prior to the Screening...
- Current placement on the waiting list for heart transplantation.
- Hospitalization for heart failure within 6 months prior to the Screening Visit.
- Any disease or medical condition that in the opinion of the Investigator would prevent the subject from successfully participating in the trial and...
See the full eligibility criteria
- 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)...
- 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
- Left Ventricular Ejection fraction of ≥ 50% by 3-D Echocardiogram at the Screening Visit.
- For people with a medical history of cardiomyopathy, must be on a stable regimen (unchanged and constant) of background heart failure medications for at least 3 months prior to 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).
- people with female partners of childbearing potential must be willing to use a highly effective method of contraception (e.g., abstinence, dual method of contraception) from the date they sign the ICF until 28 days...
- 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...
- 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.
- Hospitalization for heart failure within 6 months prior to the Screening Visit.
- Any disease or medical condition that in the opinion of the Investigator would prevent the subject from successfully participating in the trial and reliably completing the assessments or might confound trial results.
- Has a history of a systemic eosinophilic illness
- Estimated Glomerular Filtration Rate (eGFR) of \ 16 years of age and the Schwartz 2009 formula for people 5-16 years of age).
- Active malignancy or any other cancer from which the subject has been cancer-free for \< 2 years. Localized squamous or non-invasive basal cell skin carcinomas are allowed, if appropriately treated prior to Screening.
- Participation in other investigational drug or device clinical trials within 30 days or 5 half-lives (whichever is longer) of Screening; or is currently enrolled in a non-treatment clinical trial that, in the opinion of...
- History of allergic reaction to the IMP or any of its components.
- Prior participation in any elamipretide trial or expanded access programs.
The study team makes the final eligibility decision.
Where it's taking place
- Needham, Massachusetts, United States
- Calgary, Alberta, Canada
- Bristol, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 5 years to 55 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Needham, Massachusetts, United States; Calgary, Alberta, Canada; Bristol, United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.