Trial ID or NCT#

NCT01599286

Status

NOT RECRUITING

Purpose

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.

Official Title

Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia

Eligibility Criteria

Ages Eligible for Study: Younger than 99 Years
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No

Investigator(s)

Gregory Enns
Geneticist
Professor of Pediatrics (Genetics) at the Lucile Salter Packard Children's Hospital

Contact us to find out if this trial is right for you.

CONTACT

Jennafer Dotson
(650) 724-1881