Clinical Trial in Patients With Barth Syndrome- 4TAZPower
Source · ClinicalTrials.gov: elamipretide
TLDR
ClinicalTrials.gov lists 4TAZPower as recruiting for a 72-week, Phase 3b/4 placebo-controlled trial of elamipretide in patients with genetically confirmed Barth syndrome. The listing says the trial aims to confirm efficacy following U.S. accelerated approval of FORZINITY based on improved knee extensor muscle strength.
- 01The trial is randomized, double-blind, and parallel-group.
- 02Researchers will evaluate efficacy, safety, and pharmacokinetics.
- 03The listing identifies knee extensor muscle strength as the intermediate clinical endpoint supporting accelerated approval.
Why it matters Editorial
The trial is intended to provide confirmatory efficacy evidence for elamipretide in Barth syndrome following accelerated approval.
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ClinicalTrials.gov: elamipretide
ClinicalTrials.gov: elamipretide
ClinicalTrials.gov: elamipretide