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

NCT06773026 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2025-07-11

No results posted yet for this study

Summary

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.

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

Sponsors & Collaborators

  • Zevra Therapeutics

    collaborator INDUSTRY

  • Jerry Vockley, MD, PhD

    lead OTHER

Principal Investigators

  • Gerard Vockley, MD, PhD · UPMC Children's Hospital of Pittsburgh

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-06-30
Primary Completion
2027-01-31
Completion
2027-07-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06773026 on ClinicalTrials.gov