CompletedPhase 2/3

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

United States12 participantsStarted 2017-07-05

Plain-language summary

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

Age range12 Years

SexMALE

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Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit

Timeframe: Pre-dose to Week 12 (end of treatment)

Part 1: Total Fatigue Score Based on the BTHS-SA by Visit.

Timeframe: Pre-dose, Weeks 1, 4, 8, and 12 (end of treatment)

NCT IDNCT03098797

SponsorStealth BioTherapeutics Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2018-10-05

Results submitted2023-11-28