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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: 02 Apr 2024

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At a glance

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

    • Most Recent Events

    • 08 Jan 2024 Planned End Date changed from 30 Jun 2025 to 1 Dec 2025.
    • 08 Jan 2024 Planned primary completion date changed from 31 Dec 2024 to 1 Jun 2025.
    • 08 Jan 2024 Planned initiation date (estimated date of first participant enrollment) changed from 1 Dec 2023 to 1 Apr 2024.

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