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A Phase 2, Open-Label, Fixed Dose Study to Evaluate the Use of Sodium Phenylbutyrate (ACER-001) in the Treatment of Pediatric and Adult Patients With MCAD Deficiency Caused by the Common ACADM c.985 A>G (K304E) Mutation

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Trial Profile

A Phase 2, Open-Label, Fixed Dose Study to Evaluate the Use of Sodium Phenylbutyrate (ACER-001) in the Treatment of Pediatric and Adult Patients With MCAD Deficiency Caused by the Common ACADM c.985 A>G (K304E) Mutation

Status: Recruiting
Phase of Trial: Phase II

Latest Information Update: 09 Apr 2025

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At a glance

  • Drugs Sodium phenylbutyrate (Primary)
  • Indications Medium chain acyl CoA dehydrogenase deficiency
  • Focus Adverse reactions

Most Recent Events

  • 04 Apr 2025 Planned End Date changed from 1 Dec 2025 to 1 Dec 2026.
  • 04 Apr 2025 Planned primary completion date changed from 1 Jun 2025 to 1 Jun 2026.
  • 08 Jan 2024 Planned End Date changed from 30 Jun 2025 to 1 Dec 2025.

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