drugset
drugset / Trial / NCT07125066

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

NCT07125066 ↗

Phase 1 Enrolling by invitation 1 enrolled Jerry Vockley, MD, PhD Zevra Therapeutics · collab
NaSingle-groupOpen-labelTreatment

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.

Timeline

Start
2025-07-30
Primary completion
2027-07
Completion
2027-12

Drugs

EvaluationDrugModalityDoseRoute
Subject Sodium Phenylbutyrate Small molecule 12 g —

Indications

D,L-2-hydroxyglutaric aciduria