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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 19 February 2015
Main ID:  NCT01341379
Date of registration: 22/04/2011
Prospective Registration: No
Primary sponsor: Children's Hospital of Philadelphia
Public title: Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate
Scientific title: Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate
Date of first enrolment: December 2010
Target sample size: 0
Recruitment status: Withdrawn
URL:  http://clinicaltrials.gov/show/NCT01341379
Study type:  Interventional
Study design:  Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment  
Phase:  Phase 2
Countries of recruitment
United States
Contacts
Name:     Marc Yudkoff, MD
Address: 
Telephone:
Email:
Affiliation:  Children's Hospital of Philadelphia
Key inclusion & exclusion criteria

Inclusion Criteria:

- Age range: males or females, ages 3 years - 70 years

- Condition(s): N-acetylglutamate synthetase deficiency, propionic acidemia,
methylmalonic acidemia, carbamylphosphate synthase deficiency, ornithine
transcarbamylase deficiency and the syndrome of hyperammonemia, hypoglycemia and
hyperinsulinemia.

- In addition, healthy volunteers will be studied (ages 18 years - 50 years).

Exclusion Criteria:

- Acutely ill on day of study (fever, evidence of hyperammonemia - ataxia, worsening
obtundation, focal neurologic signs, seizures, increased intracranial pressure,
vomiting, signs of acute respiratory or enteric illness, headache, confusion,
disorientation, acute personality change).

- Girls 11 years of age must have a negative urine/serum pregnancy test within 1 week
prior to testing unless having a menstrual period during week of test

- Lactating females

- Hyperammonemia probably refractory to N-carbamylglutamate: other urea cycle disorders
(UCD), lysinuric protein intolerance, mitochondrial disorders, congenital lactic
acidemia, fatty acid oxidation defects or primary liver disease.

- Amount of blood necessary for study exceeds safe limits.

- Any investigational drug use within 30 days prior to enrollment.

- Parents/guardians or subjects who, in the opinion of the PI, may be non-compliant
with study schedules or procedures.

- Subjects who do not meet all the enrollment criteria may not be enrolled. Any
violations of these criteria will be reported in accordance with Institutional Review
Board (IRB) Policies and Procedures.



Age minimum: 3 Years
Age maximum: 70 Years
Gender: Both
Health Condition(s) or Problem(s) studied
Carbamyl Phosphate Synthetase Deficiency
Inborn Errors of Metabolism
Methylmalonic Acidemia
Propionic Acidemia
Urea Cycle Disorders, Inborn
Intervention(s)
Drug: N-carbamylglutamate
Primary Outcome(s)
Rate of ureagenesis [Time Frame: 3 days]
Secondary Outcome(s)
Secondary ID(s)
10-007806
R01HD058567
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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