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Main
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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. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 February 2015 |
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Main ID: |
NCT00004307 |
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Date of registration:
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18/10/1999 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Study of Treatment and Metabolism in Patients With Urea Cycle Disorders
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Scientific title:
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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 |
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Date of first enrolment:
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December 1999 |
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Target sample size:
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66 |
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Recruitment status: |
Recruiting |
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URL:
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http://clinicaltrials.gov/show/NCT00004307 |
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Study type:
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Interventional |
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Study design:
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Primary Purpose: Treatment
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Phase:
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Phase 1
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Countries of recruitment
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United States
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Contacts
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Name:
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Brendan Lee |
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Address:
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Telephone:
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Email:
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Affiliation:
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Baylor College of Medicine |
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Name:
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Susan Carter |
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Address:
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Telephone:
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832-822-1630 |
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Email:
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scarter@bcm.tmc.edu |
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Affiliation:
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Key inclusion & exclusion criteria
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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
Age minimum:
6 Months
Age maximum:
64 Years
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Amino Acid Metabolism, Inborn Errors
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Intervention(s)
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Behavioral: Protein and calorie controlled diet
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Genetic: Ornithine transcarbamylase vector
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Secondary ID(s)
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BCM-H4379
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NCRR-M01RR00188-0606
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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