Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Short-term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
Brief Summary:
The overall objective of this drug trial is to determine whether treatment of acute hyperammonemia with N-carbamyl-L-glutamate (NCG, Carglumic acid, NCG) in propionic acidemia (PA), methylmalonic acidemia (MMA), late-onset CPS1 deficiency (CPSD) and late-onset Ornithine transcarbamylase deficiency (OTCD) accelerates the resolution of hyperammonemia efficiently and safely.
The primary goal is to determine if the study drug (NCG) efficiently reduces ammonia levels following a hyperammonemia episode(s).
Secondly, the investigators want to know if treatment with this study drug (NCG) efficiently improves neurologic function, reduces plasma glutamine levels and lessens the duration of hospitalization after each hyperammonemic episode.