Current partner codePEPTIDESDE
NCT07531251·Phase 4·INTERVENTIONAL

Clinical Trial in Patients With Barth Syndrome- 4TAZPower

Status

Recruiting

Phase

Phase 4

Enrollment

48

Locations

3

Results

Not posted

Publications

0

Study summary

What the protocol is 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.

Full detailed description

The SPIBA-401 trial is a post marketing Phase 3b/4, randomized, double-blind, parallel-group, placebo-controlled clinical trial to evaluate the efficacy, safety, and pharmacokinetics of a once daily subcutaneous (SC) injection of elamipretide in subjects with genetically confirmed Barth syndrome (BTHS) for 72 weeks. The primary trial objective is to confirm the efficacy of elamipretide which is approved in the United States under the name FORZINITY™ as a mitochondrial cardiolipin binder indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg. This indication is approved in the United States under accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint.

Interventions

Treatment arms and agents.

DRUG

Elamipretide

sub cutaneous injection

DRUG

Placebo

sub cutaneous injection

Timeline

From registration to results.

  1. First posted

    Apr 15, 2026

  2. Study start

    Jul 2, 2026

  3. Primary completion

    Sep 30, 2029

  4. Study completion

    Nov 30, 2029

  5. Results posted

    Not reported

  6. Registry updated

    Jul 13, 2026

Outcomes

What the study measures.

Primary outcomes

Primary Efficacy End Point

Time frame · 72 weeks

-change in the composite normalized score of the three functional tests: Six-minute walk test (6MWT), the triple-timed up and go test (3TUG), and the Five times sit-to-stand test (5XSST) from baseline to 72 weeks of treatment

Secondary outcomes

Secondary Efficacy End Point 1

Time frame · 72 weeks

\- Change in 6Minute Walk Test from Baseline to Week 72

Secondary Efficacy End Point 2

Time frame · 72 weeks

-Change in 3Timed Up and Go Test from Baseline to Week 72

Secondary Efficacy End Point 3

Time frame · 72 weeks

-Change in 5XSit to Stand Test from Baseline to Week 72

Secondary Efficacy End Point 4

Time frame · 72 weeks

-Change in Patient Global Impression of Severity Scale (PGI-S) score from Baseline to Week 72

Secondary Efficacy End Point 5

Time frame · 72 weeks

Change in Clinician Global Impression of Severity Scale (CGI-S) score from Baseline to Week 72

Secondary Efficacy End Point

Time frame · 72 weeks

Change in knee extensor muscle strength as measured by handheld dynamometry (HHD)from Baseline to Week 72

Secondary Efficacy End Point 6

Time frame · 72 weeks

-Change in hip flexor muscle strength as measured by HHD from Baseline to Week 72

Eligibility

Who can take part.

Minimum age
5 Years
Maximum age
55 Years
Sex
MALE
Healthy volunteers
No

Key Inclusion Criteria: 1. 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. 2. Agrees to adhere to the trial requirements for the length of the trial. 3. Must have genetically confirmed Barth Syndrome (pathogenic variant in the TAZ gene) 4. Male aged ≥ 5 years at time of the Screening Visit 5. Left Ventricular Ejection fraction of ≥ 50% by 3-D Echocardiogram at the Screening Visit. 6. 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. 7. 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). 8. 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. Key Exclusion Criteria: 1. Unable to perform the required functional tests or undergo echocardiography. 2. 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. 3. Patients with an implantable cardioverter defibrillator (ICD) and with a known occurrence of ICD discharge in the 3 months prior to the Screening Visit. 4. Current placement on the waiting list for heart transplantation. 5. Hospitalization for heart failure within 6 months prior to the Screening Visit. 6. 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. 7. Has a history of a systemic eosinophilic illness 8. Estimated Glomerular Filtration Rate (eGFR) of \< 30 mL/min at the Screening Visit (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). 9. 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. 10. 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-interventional clinical trial that, in the opinion of the Investigator, may be potentially confounding to the results of the current trial. 11. History of allergic reaction to the IMP or any of its components. 12. Prior participation in any elamipretide trial or expanded access programs.

Study locations

3 registered sites.

Canada · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.

Trial Not Offered in the U.S

Needham, Massachusetts, United States

Metabolics and Genetics in Canada (MAGIC)

Calgary, Alberta, Canada

Bristol Royal Hospital for Children Upper Maudlin Street Paul O'Gorman Building

Bristol, United Kingdom

Publications

Results and literature.

No PMID-linked publications were present in this registry snapshot.

Primary links

Continue at the source.

Related trials

More studies on Elamipretide.

Related PeptideStat pages

Put the record in context.

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