Testing Elamipretide against a placebo for barth syndrome
Official title: Clinical Trial in Patients With Barth Syndrome- 4TAZPower
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
- Phase 4
- 2 groups
- One site, in Calgary
- Recruiting
Interventions
- Medication
Elamipretide
sub cutaneous injection
- Medication
Placebo
sub cutaneous injection
Canadian Sites (1)
1 listed, none recruiting
- Not yet recruiting
Metabolics and Genetics in Canada (MAGIC)
Calgary, Alberta
Eligibility Criteria
See who this study is looking for20 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +Willing and able to provide signed informed consent formInformed consentThe process of being told what taking part involves, then choosing freely.Read more → (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 VisitScreeningThe checks done before joining, to see whether a study fits.Read more →
- +Left Ventricular Ejection fraction of ≥ 50% by 3-D Echocardiogram at the Screening Visit.
- +For subjects 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).
- +Subjects 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 after the last dose of IMP.
Exclusion
- −Estimated Glomerular Filtration Rate (eGFR) of \< 30 mL/min at the Screening VisitScreeningThe checks done before joining, to see whether a study fits.Read more → (using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) 2021 formula for subjects \>16 years of age and the Schwartz 2009 formula for subjects 5-16 years of age).
- −History of allergic reaction to the IMP or any of its components.
- −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 InvestigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more →, 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.
- −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
- −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 enrolledEnrolmentThe number of participants a study plans to include, or has included.Read more → in a non-interventionalInterventional studyA study where participants are given something to see what happens.Read more → clinical trial that, in the opinion of the Investigator, may be potentially confounding to the results of the current trial.
- −Prior participation in any elamipretide trial or expanded access programs.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 4Phase 4A study of a treatment that is already approved and in use.Read more → — a study of a treatment already approved for use, following it in everyday practice.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 2 groups in this study.
Group A, the comparison group, receives Elamipretide.
Registry label: A: Elamipretide
Group B, the placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group, receives Placebo.
Registry label: B: Placebo
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
One group receives a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → — a dummy treatment with no active medicine in it.
One group receives an existing treatment, so the two can be compared.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists an age range of 5 years to 55 years.
The study lists male participants only.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 48 people.
The study is currently expected to finish around November 2029.
The main measurement is taken over: 72 weeks.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Primary Efficacy End PointEndpointThe specific thing a study measures to answer its question.Read more → — measured over 72 weeks.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT07531251 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About ElamipretideDrug
sub cutaneous injection
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About PlaceboDrug
sub cutaneous injection
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: an age range of 5 years to 55 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
Yes. This study includes a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group — a dummy treatment with no active medicine in it.
One group receives an existing treatment so the two can be compared.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists an age range of 5 years to 55 years.
It lists male participants only.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: 72 weeks.
The study as a whole is currently expected to finish around 2029-11-30.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 48 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists one location: Calgary, Alberta, Canada.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
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.
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.