NCT00004307

Study of Treatment and Metabolism in Patients With Urea Cycle Disorders

Therapeutic and Metabolic Studies of Urea Cycle Disorders: Part A: Nitrogen Flux and Ureagenesis; Part B (Closed): Phase I Adenovirus Vector-Mediated Gene Therapy for Ornithine Transcarbamylase Deficiency

• Status: Unknown
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 66 (planned)
• Sponsor: National Center for Research Resources (NCRR) · NIH
• Sex: All
• Age: 6 Months – 64 Years
• Healthy volunteers: Accepted

Summary

RATIONALE: The urea cycle is the process in which nitrogen is removed from the blood and converted into urea, a waste product found in urine . Urea cycle disorders are inherited disorders caused by the lack of an enzyme that removes ammonia from the bloodstream. Gene therapy is treatment given to change a gene so that it functions normally. Studying the treatment and metabolism of patients with urea cycle disorders may be helpful in developing new treatments for these disorders. PURPOSE: Two-part clinical trial to study the treatment and metabolism of patients who have urea cycle disorders.

Detailed description

PROTOCOL OUTLINE: This protocol involves 2 clinical studies. Part A is a metabolic study of glutamine conversion to urea at different levels of protein intake, while on and off medications. Part B is a dose escalation study of a first-generation adenoviral vector with an E1 deletion and an E3 deletion substitution (d1309) expressing ornithine transcarbamylase (OTC). In Part A, diet is controlled for protein and calories. Intravenous glutamine and urea are administered. Controls are given intravenous arginine, phenylacetate, and benzoate. In Part B, groups of 3 patients are given a single low, intermediate, or high dose of intravenous OTC vector. Allopurinol is administered every 12 hours for 12 days. As of 12/10/1999, Part B of the study is closed.

Conditions

• Amino Acid Metabolism, Inborn Errors

Interventions

Type	Name	Description
BEHAVIORAL	Protein and calorie controlled diet
GENETIC	Ornithine transcarbamylase vector

Timeline

• Start date: 1999-12-01
• First posted: 1999-10-19
• Last updated: 2005-06-24

Locations

1 site across 1 country: United States