NCT03098797

A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Crossover Trial to Evaluate the Safety and Efficacy of Subcutaneous Injections of Elamipretide in Subjects With Genetically Confirmed Barth Syndrome Followed by Open-Label Treatment

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Stealth BioTherapeutics Inc. · Industry
• Sex: Male
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

A randomized, double-blind cross over trial to evaluate the safety, efficacy, and tolerability of elamipretide in subjects with Barth syndrome.

Detailed description

A phase 2 randomized, double-blind, placebo-controlled crossover trial to evaluate the safety, tolerability, and efficacy of subcutaneous injections of elamipretide in subjects with genetically confirmed Barth syndrome followed by open-label treatment extension. Part 1 was a randomized, double-blind, placebo-controlled, crossover trial to assess safety, tolerability, and efficacy single daily subcutaneous (SC) doses of 40 mg elamipretide administered for 12 weeks in subjects with Barth syndrome. Part 2: This was an open-label extension trial to assess the long-term safety, tolerability, and longitudinal trends in efficacy single daily SC doses of 40 mg elamipretide for up to 192 weeks.

Conditions

• Barth Syndrome

Interventions

Type	Name	Description
DRUG	Elamipretide	40 mg daily subcutaneous injection for 12 weeks
DRUG	Placebo	daily subcutaneous injection for 12 weeks

Timeline

• Start date: 2017-07-05
• Primary completion: 2018-10-05
• Completion: 2021-10-11
• First posted: 2017-04-04
• Last updated: 2024-04-16
• Results posted: 2024-04-16

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study