Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate
|ClinicalTrials.gov Identifier: NCT01341379|
Recruitment Status : Withdrawn (Lack of funding)
First Posted : April 25, 2011
Last Update Posted : May 29, 2014
|Condition or disease||Intervention/treatment||Phase|
|Urea Cycle Disorders, Inborn Inborn Errors of Metabolism Propionic Acidemia Methylmalonic Acidemia Carbamyl Phosphate Synthetase Deficiency||Drug: N-carbamylglutamate||Phase 2|
- To determine whether brief (3 day) treatment with N-carbamylglutamate can improve ureagenesis in adult healthy controls and patients with the following inborn errors of metabolism: N-acetylglutamate synthetase deficiency, propionic acidemia, methylmalonic acidemia, carbamylphosphate synthase deficiency, ornithine transcarbamylase deficiency and the syndrome of hyperammonemia, hypoglycemia and hyperinsulinemia (HHH Syndrome).
- To determine if such treatment improves other indicators of abnormal nitrogen metabolism such as elevated blood levels of glutamine, glycine and alanine.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate|
|Study Start Date :||December 2010|
|Actual Primary Completion Date :||August 2012|
|Actual Study Completion Date :||August 2012|
|Experimental: N-carbamylglutamate (Carbaglu)||
N-carbamylglutamate: 200 mg/kg/day for 3 days, divided into 4 daily oral doses
Other Name: Carbaglu
- Rate of ureagenesis [ Time Frame: 3 days ]Goal is to determine whether a 3 day trial of N-carbamylglutamate increases ureagenesis in patients with urea cycle defects and other inborn errors of metabolism.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01341379
|Principal Investigator:||Marc Yudkoff, MD||Children's Hospital of Philadelphia|