Clinical Trial in Patients With Barth Syndrome- 4TAZPower

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

First listed

Apr 15, 2026

Start date

Jul 2, 2026

Status verified

Jul 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

• Rekha Sathyanarayana
  617-762-2579
  [email protected]
• Lani Glennon
  [email protected]

Locations (1)

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