Clinical Trial in Patients With Barth Syndrome- 4TAZPower

Part of paid clinical trials in Needham, Massachusetts.

Sponsor
Stealth BioTherapeutics Inc.
Study ID
NCT07531251
Phase
PHASE4
Status
Not Yet Recruiting

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Conditions

  • Barth Syndrome

Eligibility Criteria

Sex
MALE
Age
5 Years - 55 Years
Healthy Volunteers
Not accepted

Interventions

  • Elamipretide — DRUG
    sub cutaneous injection
  • Placebo — DRUG
    sub cutaneous injection

Study Details

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.

Key Dates

Start date
Jun 30, 2026
Status verified
May 2026
Primary completion
Sep 30, 2029
Completion
Nov 30, 2029

Study Design

Enrollment
48 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • 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 Outcome Measure

Primary Efficacy End Point [ Time Frame: 72 weeks ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Trial Not Offered in the U.SNeedhamMassachusetts02494-

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