Health Trials

Medium Chain Acyl Coa Dehydrogenase Deficiency

2

Review clinical trials related to Medium Chain Acyl Coa Dehydrogenase Deficiency. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

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

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.

Participants needed: 24
Trial details
Phase: Phase 2Age: 4+Biological sex: AllType: InterventionalSponsor: Jerry Vockley, MD, PhDUpdated: Jun 8, 2026Locations: 1
Eligibility criteria

A diagnosis of MCADD and molecular confirmation of at least one copy of the comm... [+6]

Use of any investigational drug within 30 days of Day 1. [+12]

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Status: Recruiting

Metabolic Effects of Medium-Chain Fatty Acids in Patients With Medium-Chain Acyl-CoA Dehydrogenase Deficiency and Healthy Individuals

The aim of this project is to investigate the physiological effects of intake of long-chain fatty acids (LCFA) and medium-chain fatty acids (MCFA) in patients with Medium-Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) and healthy individuals

Participants needed: 30
Trial details
Age: 18-80Biological sex: AllType: InterventionalSponsor: University of CopenhagenUpdated: Mar 10, 2026Locations: 1
Eligibility criteria

Male or female [+4]

Diabetes [+7]

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