Study to Evaluate Pharmacokinetics, Safety, and Tolerability of Subcutaneous Injections of Elamipretide in Pediatric Subjects With Barth Syndrome

What this study involves

The registry does not currently label this study as recruiting. Ask about its status before planning a screening visit.

Study type

Interventional

Phase

Phase 1

Age range

Not specified - Not specified

Sex eligible

Male

Healthy volunteers

Not accepted

Participants

6 Estimated

These are starting points, not an eligibility decision. The research team must review the full requirements.

About the research

This Phase 1, open-label trial will enroll approximately 6 subjects who have BTHS weighing between 7 and 30 kg. Subjects will receive a weight-based dose of elamipretide daily via SC injection for 4 weeks. PK samples will be collected on Day 1 and at week 4 to determine PK profile in BTHS patients weighing between 7 and 30kg.

Who may be able to participate?

The list below is the sponsor’s full registry wording. It can include conditions, prior treatment, test results and other requirements. A match in search does not mean you qualify.

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 ≥ 1 year and weighing between 7 and 30 kg at time of the Baseline Visit
5. 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.
6. Able to administer IMP or have an appropriate designee who can administer the IMP (i.e., a capable family member or a caregiver).

Exclusion Criteria:

1. 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, the subject is stable, and participation in the trial does not pose additional safety risk.
2. Current placement on the waiting list for heart transplantation.
3. Hospitalization for heart failure within 6 months prior to the Screening/Baseline Visit
4. 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.
5. eGFR of \< 30 mL/min at the Screening Visit (for purposes of Screening, eGFR can be confirmed locally at the clinical site. Historical values within 3 months prior to Screening can be accepted).
6. 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/Baseline.
7. Participation in other investigational drug or device clinical trials within 30 days or 5 half-lives (whichever is longer) of Screening/Baseline; 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. NOTE: Subjects currently treated with elamipretide through an EAP are eligible to participate in the SPIBA-101 study.
8. History of allergic reaction to the IMP or any of its components.

Treatment, groups and participation

The registry may describe treatments and study duration without a complete visit calendar. Ask the study team for the exact schedule, procedures, risks, costs and alternatives. Keep your usual care plan unless your treating clinician tells you otherwise.

Assignment

Not applicable

Masking

No masking

What researchers are measuring

Outcome measures describe what is being studied. They are not proof that a treatment works.

Primary measures

Secondary measures

Research locations (0)

The overall study status and a local site’s status can differ. Confirm that location’s availability before traveling.

No locations are listed. Use the official study contact.

Study team and timeline

Lead sponsor

Stealth BioTherapeutics Inc.

Study start

2026-11 Estimated

Primary completion

2027-05 Estimated

Study completion

2027-06 Estimated

First posted

Nov 10, 2026 Actual

Last updated

Nov 10, 2026 Actual

Official study contacts