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ACER-001 for Combined D,L-2-hydroxyglutaric Aciduria
NCT07125066Phase 1Enrolling by invitation
- Start
- Jul 2025
- Primary completion
- Jul 2027
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of sodium phenylbutyrate in Combined D,L-2-hydroxyglutaric aciduria (D,L-2-HGA). Primary endpoint: Number of treatment related adverse events at 2 years. Started Jul 2025; status: enrolling by invitation.
02
Drugs
sodium phenylbutyrate
03
Conditions
Combined D,L-2-hydroxyglutaric aciduria (D,L-2-HGA)
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