Overview

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

Status:
Recruiting

Trial end date:
2025-12-01

Target enrollment:

Participant gender:

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.

Phase:

Phase 2

Details

Lead Sponsor:

Jerry Vockley, MD, PhD

Collaborator:

Acer Therapeutics Inc.

Treatments:

4-phenylbutyric acid