Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate
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|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Recruitment Status : Withdrawn (Lack of funding)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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01341379
|Principal Investigator:||Marc Yudkoff, MD||Children's Hospital of Philadelphia|