Condition: Medium-chain Acyl-CoA Dehydrogenase Deficiency · Sponsor: Jerry Vockley, MD, PhD
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.
This description comes directly from the study's public registry record.
Elizabeth McCracken · 412-692-5662 · elizabeth.mccracken@chp.edu
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| UPMC Children's Hospital of Pittsburgh | Pittsburgh, Pennsylvania, United States | Recruiting |
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Source record: clinicaltrials.gov/study/NCT06773026