Clinical Trial: A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Crossover Trial to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Injections of Elamipretide (MTP-131) in Subjects With Genetica
Brief Summary: A randomized double-blind cross over trial to evaluate the safety, efficacy and tolerability of elamipretide in subjects with Barth Syndrome.
Detailed Summary:
Sponsor: Stealth BioTherapeutics Inc.
Current Primary Outcome: Change in distance walked during the 6-minute walk test (6MWT) [ Time Frame: 12 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Change in muscle strength as measured by handheld dynamometry [ Time Frame: 12 weeks ]
- Change in five times sit-to-stand test [ Time Frame: 12 weeks ]
- Change in 2-D and 3-D echocardiographic measurements [ Time Frame: 12 weeks ]
- Change in accelerometry counts [ Time Frame: 12 weeks ]
- Change in SWAY application balance assessments [ Time Frame: 12 weeks ]
- Change in Patient Reported Outcomes [ Time Frame: 12 weeks ]
- Change in Clinician Global Impression [ Time Frame: 12 weeks ]
- Change in biomarkers [ Time Frame: 12 weeks ]
- Number of participants with treatment related adverse events [ Time Frame: 12 weeks ]
Original Secondary Outcome: Same as current
Information By: Stealth BioTherapeutics Inc.
Dates:
Date Received: March 21, 2017
Date Started: May 1, 2017
Date Completion: October 2018
Last Updated: May 8, 2017
Last Verified: May 2017
