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NCG for Acute Hyperammonemia in Organic Acidemias and Urea Cycle Disorders
NCT01599286Phase 2Completed
- Start
- Sep 2012
- Primary completion
- Apr 2019
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, randomised, double-blind trial of carglumic acid in propionic acidemia (PA), methylmalonic acidemia (MMA), carbamoyl phosphate synthetase I deficiency (CPS1D) and 1 more (2 arms). Primary endpoint: Time to the Primary Outcome (Earlier of Ammonia <50 µmol/L or Hospital Discharge) at 7 days. Started Sep 2012; status: completed.
02
Drugs
carglumic acid
03
Conditions
propionic acidemia (PA)methylmalonic acidemia (MMA)carbamoyl phosphate synthetase I deficiency (CPS1D)ornithine transcarbamylase deficiency
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