NCT07531251

Ph3b/4 Trial in Patients With Barth Syndrome

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
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 48 (estimated)
• Sponsor: Stealth BioTherapeutics Inc. · Industry
• Sex: Male
• Age: 5 Years – 55 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

The SPIBA-401 trial is a post marketing Phase 3b/4, randomized, double-blind, parallel-group, placebo-controlled clinical trial to evaluate the efficacy, safety, and pharmacokinetics of a once daily subcutaneous (SC) injection of elamipretide in subjects with genetically confirmed Barth syndrome (BTHS) for 72 weeks. The primary trial objective is to confirm the efficacy of elamipretide which is approved in the United States under the name FORZINITY™ as a mitochondrial cardiolipin binder indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg. This indication is approved in the United States under accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint.

Conditions

• Barth Syndrome

Interventions

Type	Name	Description
DRUG	Elamipretide	sub cutaneous injection
DRUG	Placebo	sub cutaneous injection

Timeline

• Start date: 2026-06-30
• Primary completion: 2029-09-30
• Completion: 2029-11-30
• First posted: 2026-04-15
• Last updated: 2026-04-15

Locations

1 site across 1 country: United Kingdom

Regulatory

• FDA-regulated drug study