Overview

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

Status:
ENROLLING_BY_INVITATION
Trial end date:
2027-12-01
Target enrollment:
Participant gender:
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.
Phase:
PHASE1
Details
Lead Sponsor:
Jerry Vockley, MD, PhD
Collaborator:
Zevra Therapeutics
Treatments:
4-phenylbutyric acid