Condition: Barth Syndrome · Sponsor: Stealth BioTherapeutics Inc.
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.
This description comes directly from the study's public registry record.
Rekha Sathyanarayana · 617-762-2579 · rekha.sathyanarayana@stealthbt.com
Lani Glennon · lani.glennon@parexel.com
Always discuss trial participation with your own doctor first.
| Trial Not Offered in the U.S | Needham, Massachusetts, United States | Terminated |
| Metabolics and Genetics in Canada (MAGIC) | Calgary, Alberta, Canada | Not Yet Recruiting |
| Bristol Royal Hospital for Children Upper Maudlin Street Paul O'Gorman Building | Bristol, United Kingdom | Recruiting |
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Source record: clinicaltrials.gov/study/NCT07531251