NCT06773026View on ClinicalTrials.gov

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

PHASE2RecruitingINTERVENTIONAL
Enrollment

24

Participants

Timeline

Start Date

June 30, 2025

Primary Completion Date

January 31, 2027

Study Completion Date

July 31, 2027

Conditions
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Interventions
DRUG

Sodium phenylbutyrate

Open-label design with doses of sodium phenylbutyrate at 4.0 g/m2/day

Trial Locations (1)

15224

RECRUITING

UPMC Children's Hospital of Pittsburgh, Pittsburgh

Sponsors

Collaborators (1)

Zevra Therapeutics
All Listed Sponsors
collaborator

Zevra Therapeutics

INDUSTRY

lead

Jerry Vockley, MD, PhD

OTHER

NCT06773026 - Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) | Biotech Hunter | Biotech Hunter