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Clinical trial

A Phase 2, Open-label, Fixed-dose Study to Assess the Efficacy of Sodium Phenylbutyrate (ACER-001) in Treating Pediatric and Adult Patients With Medium Chain Acyl-CoA Dehydrogenase (MCAD) Deficiency Resulting From the Prevalent ACADM c.985 A>G (K304E) Mutation

NCT06773026 is registered in ClinicalTrials.gov with Jerry Vockley, MD, PhD as lead sponsor.

NCT NCT06773026
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 2, Open-label, Fixed-dose Study to Assess the Efficacy of Sodium Phenylbutyrate (ACER-001) in Treating Pediatric and Adult Patients With Medium Chain Acyl-CoA Dehydrogenase (MCAD) Deficiency Resulting From the Prevalent ACADM c.985 A>G (K304E) Mutation
Lead sponsor
Jerry Vockley, MD, PhD
Phase
Phase 2
Overall status
Recruiting
Start date
June 30, 2025
Completion date
July 31, 2027
Enrollment
24 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

medium-chain acyl-coa dehydrogenase deficiency