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4TAZPower: A 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

Status
Not yet recruiting
Phases
Phase 3Phase 4
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2025-523837-25-00
Enrollment
11
Registered
2026-07-27
Start date
Unknown
Completion date
Unknown
Last updated
2026-07-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Barth Syndrome

Brief summary

Change from Baseline to Week 72 in a composite normalized score of the following three functional tests: 6MWT, 3TUG, and 5XSST

Detailed description

Change from Baseline to Week 72 in the 6MWT, Change from Baseline to Week 72 in the 3TUG, Change from Baseline to Week 72 in the 5XSST, Change from Baseline to Week 72 in the Patient Global Impression of Severity Scale (PGI-S), Change from Baseline to Week 72 in the Clinician Global Impression of Severity Scale (CGI-S), Change from Baseline to Week 72 in knee extensor muscle strength as measured by handheld dynamometry (HHD), Change from Baseline to Week 72 in hip flexor muscle strength as measured by HHD

Interventions

DRUGElamipretide
DRUGElamipretide injection placebo

Sponsors

Stealth Biotherapeutics Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
Male
Age
0 Years to No maximum

Design outcomes

Primary

MeasureTime frame
Change from Baseline to Week 72 in a composite normalized score of the following three functional tests: 6MWT, 3TUG, and 5XSST—

Secondary

MeasureTime frame
Change from Baseline to Week 72 in the 6MWT, Change from Baseline to Week 72 in the 3TUG, Change from Baseline to Week 72 in the 5XSST, Change from Baseline to Week 72 in the Patient Global Impression of Severity Scale (PGI-S), Change from Baseline to Week 72 in the Clinician Global Impression of Severity Scale (CGI-S), Change from Baseline to Week 72 in knee extensor muscle strength as measured by handheld dynamometry (HHD), Change from Baseline to Week 72 in hip flexor muscle strength as measured by HHD—

Outcome results

None listed

Source: EU CTIS · Data processed: Jul 29, 2026