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drugset / Trial / NCT06069375

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

NCT06069375 ↗

Phase 2 Suspended 24 enrolled Jerry Vockley, MD, PhD Acer Therapeutics Inc. · collab
Non-randomizedParallel-groupOpen-labelTreatment

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 works) of sodium phenylbutyrate in patients with MCADD.

Timeline

Start
2024-04-01
Primary completion
2028-06
Completion
2028-12

Drugs

EvaluationDrugModalityDoseRoute
Subject Sodium Phenylbutyrate Small molecule 3000 mg/m2 —
Subject Sodium Phenylbutyrate Small molecule 4000 mg/m2 —

Indications

medium chain acyl-CoA dehydrogenase deficiency