NCT06069375View on ClinicalTrials.gov

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

PHASE2SuspendedINTERVENTIONAL

Enrollment

24

Participants

Timeline

Start Date

April 1, 2024

Primary Completion Date

June 30, 2026

Study Completion Date

December 31, 2026

Conditions

Medium-chain Acyl-CoA Dehydrogenase Deficiency

Interventions

DRUG

Sodium phenylbutyrate

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

Trial Locations (1)

15224

UPMC Children's Hospital of Pittsburgh, Pittsburgh

Sponsors

Collaborators (1)

Acer Therapeutics Inc.

All Listed Sponsors

collaborator

Acer Therapeutics Inc.

INDUSTRY

lead

Jerry Vockley, MD, PhD

OTHER

NCT06069375 - Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) | Biotech Hunter | Biotech Hunter