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Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate

NCT01341379 · Status: WITHDRAWN · Phase: PHASE2 · Type: INTERVENTIONAL

Last updated 2014-05-29

No results posted yet for this study

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.

Conditions

  • Urea Cycle Disorders, Inborn
  • Inborn Errors of Metabolism
  • Propionic Acidemia
  • Methylmalonic Acidemia
  • Carbamyl Phosphate Synthetase Deficiency

Interventions

DRUG

N-carbamylglutamate

N-carbamylglutamate: 200 mg/kg/day for 3 days, divided into 4 daily oral doses

Sponsors & Collaborators

Principal Investigators

  • Marc Yudkoff, MD · Children's Hospital of Philadelphia

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2010-12-31
Primary Completion
2012-08-31
Completion
2012-08-31

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01341379 on ClinicalTrials.gov