Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

This is a medical research study to test a medication in patients 4 years of age and older with a disease called medium-chain acyl-CoA dehydrogenase deficiency (MCADD) caused by the common ACADM c.985 A\>G (K304E) mutation. The medication is sodium phenylbutyrate (ACER-001), which is currently FDA approved for the treatment of Urea Cyle Disorders. Previous research suggests that sodium phenylbutyrate may also be effective in the treatment MCADD. This study will investigate the safety and efficacy (how well it works) of sodium phenylbutyrate in patients with MCADD.

NCT Number	NCT06773026
Lead Sponsor	Jerry Vockley, MD, PhD Collaborators: Zevra Therapeutics
Conditions	Medium-chain Acyl-CoA Dehydrogenase Deficiency
Enrollment	24 participants
Start Date	2025-06-30
Primary Completion	2027-01 (estimated)
Study Completion	2027-07 (estimated)
Updated on ClinicalTrials.gov	2026-06-08