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Home > "S" Clinical Trials Conditions > Study of Treatment and Metabolism in Patients With Urea Cycle Disorders  Study of Treatment and Metabolism in Patients With Urea Cycle Disorders
Study of Treatment and Metabolism in Patients With Urea Cycle Disorders
For Condition: Amino Acid Metabolism, Inborn Errors
Status: Recruiting
Sponsor(s): National Center for Research Resources (NCRR) , Baylor College of Medicine
Synopsis: 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.
Details: 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.
Eligibility:
Study Type: Interventional, Treatment
Minimum Age/Maximum Age: 6 Months/64 Years
Genders: Both
Protocol Entry Criteria: PROTOCOL ENTRY CRITERIA: Part A. Patients at least 6 months old with ornithine transcarbamylase deficiency (OTC), i.e.: Hemizygous OTC or homozygous autosomal recessive disorder with evidence of complete enzyme deficiency Hemizygous OTC male with late presentation and presumed evidence for residual enzyme activity OTC heterozygotes (molecular diagnosis) with severely symptomatic to asymptomatic disease Obligate heterozygotes for autosomal recessive disorder (parent or genotyped sibling) Normal adult volunteers and genotyped siblings entered as controls Part B. Metabolically stable heterozygous OTC females aged 18 to under 65 Orotic acid level at least 5 times normal on allopurinol Symptoms ranging from severe to asymptomatic acceptable No prior hospitalization for hyperammonemia Exclusion criteria (Parts A and B): Acute or chronic intercurrent illness Pregnancy Acute hyperammonemia
Total Enrollment: 66
Location and Contact Information:
Overall Study Official:
BrendanLee, Study Chair, Baylor College of Medicine
Baylor College of Medicine *Recruiting*
Houston, Texas, 77030
United States
Recruiting Brendan Lee 713-798-8835
Additional Information:
Study ID Numbers: NCRR-M01RR00188-0606; BCM-H4379
Study Start Date: December 1999
Record last reviewed: December 2003
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00004307
Other Amino Acid Metabolism, Inborn Errors Studies:
1. Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders
2. Study of Treatment and Metabolism in Patients With Urea Cycle Disorders
Related Studies:
Other Amino Acid Metabolism, Inborn Errors Clinical Trials
Other Texas Clinical Trials
Other Houston Clinical Trials
Study of Treatment and Metabolism in Patients With Urea Cycle Disorders
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