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

Enrolling by invitation · Phase 1

Conditions studied: Combined D,L-2-hydroxyglutaric Aciduria

In brief

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

Key facts

Study ID

NCT07125066

Run by

Jerry Vockley, MD, PhD

People needed

1

Starts

2025-07-30

Expected to finish

2027-12-01

Last updated by the study team

2026-06-08

Who can join

Age: any. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Single patient with Combined D,L-2-hydroxyglutaric aciduria
• Parental permission must be given at the start of the protocol.
• Parents willing to adhere to the protocol

You may not qualify if…

• Refusal or withdrawal of consent.
• Drug Intolerance.

Where it is running

• Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States

Full record on ClinicalTrials.gov

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