Thompson 2021 — Elamipretide Phase 2/3 Barth Syndrome Trial

Genetics in Medicine

W. Reid Thompson, et al.

Summary

Elamipretide was associated with improvements in Barth syndrome symptoms after randomized and open-label treatment phases.

Study Details

Study Design

Phase 2/3 randomized clinical trial followed by open-label extension

Indication

Barth syndrome

Intervention

Daily elamipretide

Species

Human

Sample Size

12 subjects

Risk of Bias Assessment

Very small rare-disease trial; mixed regulatory interpretation