Trial · Interventional
RecruitingPhase 4Registry dataClinical Trial in Patients With Barth Syndrome- 4TAZPower
Short name 4TAZPower
Overview
- Status
- Recruiting
- Phase
- Phase 4
- Enrollment
- 48 participants (estimated)
- Sponsor
- Stealth BioTherapeutics Inc. · Industry
- Design
- Randomized · Quadruple masking
- Countries
3 countries
United States, Canada, United Kingdom
- Start
- 2 Jul 2026
- Primary completion
- 30 Sept 2029 (estimated)
- Completion
- 30 Nov 2029 (estimated)
- First posted
- 15 Apr 2026
- Last update posted
- 17 Sept 2026
- Conditions
- Barth Syndrome
Timeline
1 trial · 1 ongoing
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
- Ph 4
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
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.
Interventions
- ElamipretideDrug
- PlaceboDrug
Arms2
Active comparator
Elamipretide
Elamipretide - aqueous, sterile 5.0 mL single-patient, ready to use, multi-dose glass vial containing 3.5 mL of elamipretide solution (elamipretide \[80 mg/mL\],
Placebo comparator
Placebo
Matching Placebo-aqueous, sterile 5.0 mL single-patient, ready to use, multi-dose glass vial containing will be composed of 3.5mL of sodium chloride, phosphate buffer, and benzyl alcohol similar to excipients in active drug but without the active drug substance
Primary outcomes1
Outcomes the sponsor listed as primary.
Primary Efficacy End Point
Time frame · 72 weeks
Registry description
-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(7)
Secondary Efficacy End Point 1
Time frame · 72 weeks
Registry description
\- Change in 6Minute Walk Test from Baseline to Week 72
Secondary Efficacy End Point 2
Time frame · 72 weeks
Registry description
-Change in 3Timed Up and Go Test from Baseline to Week 72
Secondary Efficacy End Point 3
Time frame · 72 weeks
Registry description
-Change in 5XSit to Stand Test from Baseline to Week 72
Secondary Efficacy End Point 4
Time frame · 72 weeks
Registry description
-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
Registry description
Change in Clinician Global Impression of Severity Scale (CGI-S) score from Baseline to Week 72
Secondary Efficacy End Point
Time frame · 72 weeks
Registry description
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
Registry description
-Change in hip flexor muscle strength as measured by HHD from Baseline to Week 72
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
No results are posted for this trial.
A trial that is still running, or that stopped early, may post results later.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies0
Studies that cite this trial.
No study cites this trial.
Eligibility
Who could join, as posted.
- Sex
- Male
- Age
- from 5 Years up to 55 Years
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
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