Health TrialsbyHealthPanel

Clinical Trial in Patients With Barth Syndrome- 4TAZPower

ConditionBarth Syndrome
Trial statusRecruiting
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

This trial does not accept healthy volunteers

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

Design model

Parallel

Treatments tested in this trial

  • Elamipretide

    Drug

    sub cutaneous injection

  • Placebo

    Drug

    sub cutaneous injection

Treatment groups

48 Participants
are divided into 2 treatment groups
Group A: ElamipretideActive comparator 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Primary Efficacy End Point

-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

Time frame
72 weeks

Secondary outcomes

1

Secondary Efficacy End Point 1

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

Time frame
72 weeks
2

Secondary Efficacy End Point 2

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

Time frame
72 weeks
3

Secondary Efficacy End Point 3

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

Time frame
72 weeks
4

Secondary Efficacy End Point 4

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

Time frame
72 weeks

Other outcomes

1

Safety End Point Adverse Event Reporting

-Report the # of subjects with adverse events and incidence and severity of such adverse events (AEs)

Time frame
72weeks

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Stealth BioTherapeutics Inc.

Lead sponsor

Source ClinicalTrials.gov
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