drugset / Trial / NCT01599286
Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
Phase 2
Completed
35 enrolled
Mendel Tuchman
Boston Children's Hospital · collabChildren's Hospital Colorado · collabChildren's Hospital of Philadelphia · collabChildren's National Research Institute · collabIcahn School of Medicine at Mount Sinai · collabStanford University · collabUniversity Hospitals Cleveland Medical Center · collabUniversity of California, Los Angeles · collabUniversity of Pittsburgh · collab
RandomizedParallel-groupQuadruple-blindTreatment
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.
Timeline
- Start
- 2012-09-01
- Primary completion
- 2019-04-30
- Completion
- 2020-04-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Carglumic Acid | Unknown | 150 mg/kg | Oral |
| Subject | Carglumic Acid | Unknown | 3300 mg/m2 | Oral |