Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate (NCT01341379) | Clinical Trial Compass
WithdrawnPhase 2
Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate
Stopped: Lack of funding
0Started 2010-12
Plain-language 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.
Who can participate
Age range3 Years – 70 Years
SexALL
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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 acco…