Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Recruiting
Acer Therapeutics Inc.
Phase 2
2024-04-01
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.
Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Recruiting
Jerry Vockley, MD, PhD
Phase 2
2024-04-01
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.
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