MedTrace Logo

An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)

NCT07125066 · Status: ENROLLING_BY_INVITATION · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2026-06-08

No results posted yet for this study

Summary

This is an individual patient research protocol to treat a patient diagnosed with Combined D,L-2-hydroxyglutaric aciduria (C-2HGA) with ACER-001.

Conditions

  • Combined D,L-2-hydroxyglutaric Aciduria

Interventions

DRUG

Sodium phenylbutyrate

open-label design with doses of sodium phenylbutyrate

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

Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-07-30
Primary Completion
2027-07-31
Completion
2027-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

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 NCT07125066 on ClinicalTrials.gov