Clinical Trial

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

Name: An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA) — clinical trial record (NCT07125066)
Creator: DataLookout
License: https://creativecommons.org/licenses/by/4.0/

Enrolling by Invitation Phase 1

View on ClinicalTrials.gov →

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.

Trial Details

NCT Number	NCT07125066
Lead Sponsor	Jerry Vockley, MD, PhD Collaborators: Zevra Therapeutics
Conditions	Combined D,L-2-hydroxyglutaric Aciduria
Enrollment	1 participants
Start Date	2025-07-30
Primary Completion	2027-07 (estimated)
Study Completion	2027-12 (estimated)
Updated on ClinicalTrials.gov	2026-06-08