Trial Profile

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

Status: Recruiting

Phase of Trial: Phase I

Latest Information Update: 22 Aug 2025

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At a glance

Drugs Sodium phenylbutyrate (Primary)
Indications Inborn urea cycle disorders
Focus Adverse reactions

Most Recent Events

15 Aug 2025 Status changed to recruiting.
20 May 2016 New trial record

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