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

NCT06069375 · Status: SUSPENDED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2025-09-26

No results posted yet for this study

Summary

This is a medical research study to test a medication in patients 10 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 up to 4.0 g/m2/day

Sponsors & Collaborators

  • Acer Therapeutics Inc.

    collaborator INDUSTRY

  • Jerry Vockley, MD, PhD

    lead OTHER

Principal Investigators

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

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
10 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-04-01
Primary Completion
2026-06-30
Completion
2026-12-31
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 NCT06069375 on ClinicalTrials.gov