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ENROLLING BY INVITATION
NCT07125066
PHASE1
An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)
Sponsor: Jerry Vockley, MD, PhD
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.
Official title: An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2-hydroxyglutaric Aciduria (C-2HGA)
Key Details
Gender
MALE
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
1
Start Date
2025-07-30
Completion Date
2027-12
Last Updated
2025-08-15
Healthy Volunteers
No
Conditions
Interventions
DRUG
Sodium phenylbutyrate
open-label design with doses of sodium phenylbutyrate
Locations (1)
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States