Clinical Trial: Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Study Status: Completed
Recruit Status: RECRUITING
Condition: Medium-chain Acyl-CoA Dehydrogenase Deficiency
Study Type: INTERVENTIONAL
Official Title: 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
Brief Summary:
This is a medical research study to test a medication in patients 10 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…
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