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

U.S., Aug. 16 -- ClinicalTrials.gov registry received information related to the study (NCT07125066) titled 'An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)' on July 30.

Brief 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.

Study Start Date: July 30

Study Type: INTERVENTIONAL

Condition: Combined D,L-2-hydroxyglutaric Aciduria

Intervention: DRUG: Sodium phenylbutyrate

open-label design with doses of sodium phenylbutyrate

Recruitment Status: ENROLLING_BY_INVITATION

Sponsor: Jerry Vockley, MD, PhD

Information provided by (Responsible Party): Jerry Vockley, MD, Ph...