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N-carbamylglutamate for Ureagenesis in Inborn Errors of Metabolism
NCT01341379Phase 2Withdrawn
- Sponsor
- Children's Hospital of Philadelphia
- Start
- Dec 2010
- Primary completion
- Aug 2012
- Modality
- Small Molecules
01
Overview
A Phase 2 trial of carglumic acid in N-acetylglutamate synthetase deficiency, propionic acidemia (PA), methylmalonic acidemia (MMA), carbamoyl phosphate synthetase I deficiency (CPS1D), ornithine transcarbamylase deficiency, sponsored by Children's Hospital of Philadelphia. Planned start Dec 2010; status: withdrawn.
02
Drugs
carglumic acid
03
Conditions
N-acetylglutamate synthetase deficiencypropionic acidemia (PA)methylmalonic acidemia (MMA)carbamoyl phosphate synthetase I deficiency (CPS1D)ornithine transcarbamylase deficiency
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