Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate
Status:
Withdrawn
Trial end date:
2012-08-01
Target enrollment:
Participant gender:
Summary
Hyperammonemia, which can cause brain damage, occurs in many different kinds of inborn errors
of metabolism. The investigators propose to determine if short-term (3 day) treatment with
N-carbamylglutamate can diminish hyperammonemia by enhancing ureagenesis in these patients.
The investigators propose here a short-term (3 day) trial. If it succeeds, the investigators
would consider more extensive long-term studies of the drug.
Phase:
Phase 2
Details
Lead Sponsor:
Children's Hospital of Philadelphia
Collaborator:
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)