Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
Status:
Completed
Trial end date:
2020-04-30
Target enrollment:
Participant gender:
Summary
The overall objective of this drug trial is to determine whether the treatment of acute
hyperammonemia with N-carbamyl-L-glutamate (NCG, Carglumic acid) 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 episode of hyperammonemia.
Phase:
Phase 2
Details
Lead Sponsor:
Mendel Tuchman
Collaborators:
Boston Children's Hospital Boston Children’s Hospital Children's Hospital Colorado Children's Hospital of Philadelphia Children's National Research Institute Children's Research Institute Icahn School of Medicine at Mount Sinai Stanford University University Hospitals Cleveland Medical Center University of California, Los Angeles University of Pittsburgh