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

PHASE2CompletedINTERVENTIONAL
Enrollment

35

Participants

Timeline

Start Date

September 1, 2012

Primary Completion Date

April 30, 2019

Study Completion Date

April 30, 2020

Conditions
Propionic Acidemia, Type I and/or Type IIMethylmalonic AcidemiaCarbamoyl-Phosphate Synthase I Deficiency DiseaseOrnithine Carbamoyltransferase Deficiency
Interventions
DRUG

Carbaglu

"Carbaglu Chemical Composition: N-carbamoyl-L-glutamic acid (NCG)~The daily dose will be 150 mg/kg/ day or 3.3 g/m2/day for patients \>15 kg and will be administered for 7 days or until discharge, whichever is sooner. The doses are to be divided into 2 equal doses and administered orally or enterally by nasogastric or gastrostomy tube. Standard of care will prevail when choosing the mode of drug administration.~The tablets must be dispersed in a minimum of 2.5-10 ml of water and ingested immediately or administered by fast-push through a syringe via a nasogastric or gastrostomy tube. The suspension has a slightly acidic taste."

DRUG

Placebo

Placebo that looks/tastes the same as NCG and is administered on the same schedule as the NCG intervention

Trial Locations (9)

10029

Mount Sinai School of Medicine, New York

15224

University of Pittsburgh, Pittsburgh

19104

The Children's Hospital of Philadelphia (CHOP), Philadelphia

20010

Children's National Medical Center, Washington D.C.

44106

University Hospitals Cleveland Medical Center, Cleveland

80045

The Children's Hospital of Colorado, Aurora

90095

University of California Los Angeles, Los Angeles

94304

Lucile Packard Children's Hospital at Stanford, Palo Alto

02115

Children's Hospital Boston, Boston

All Listed Sponsors
collaborator

Children's National Research Institute

OTHER

collaborator

Boston Children's Hospital

OTHER

collaborator

University Hospitals Cleveland Medical Center

OTHER

collaborator

University of California, Los Angeles

OTHER

collaborator

Children's Hospital of Philadelphia

OTHER

collaborator

Stanford University

OTHER

collaborator

Icahn School of Medicine at Mount Sinai

OTHER

collaborator

University of Pittsburgh

OTHER

collaborator

Children's Hospital Colorado

OTHER

lead

Mendel Tuchman

OTHER