Trial Profile

A Phase 2, Open-label, Fixed-dose Study to Assess the Efficacy of Sodium Phenylbutyrate (ACER-001) in Treating Pediatric and Adult Patients With Medium Chain Acyl-CoA Dehydrogenase (MCAD) Deficiency Resulting From the Prevalent ACADM c.985 A>G (K304E) Mutation

Status: Recruiting

Phase of Trial: Phase II

Latest Information Update: 12 May 2025

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At a glance

04 May 2025 Planned End Date changed from 1 Jan 2027 to 1 Feb 2027.
04 May 2025 Planned primary completion date changed from 1 Sep 2026 to 1 Nov 2026.
04 May 2025 Planned initiation date changed from 1 Apr 2025 to 1 Jul 2025.

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