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Sponsors and Collaborators: |
FDA Office of Orphan Products Development University of Pennsylvania |
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Information provided by: | FDA Office of Orphan Products Development |
ClinicalTrials.gov Identifier: | NCT00004498 |
OBJECTIVES:
I. Determine the safety, feasibility, and potential efficacy of intravascular adenoviral vector mediated gene transfer in the liver in adults with partial ornithine transcarbamylase deficiency.
Condition | Intervention | Phase |
---|---|---|
Ornithine Transcarbamylase Deficiency Disease |
Gene Transfer: Adenoviral Vector-Mediated Gene Transfer |
Phase I |
Study Type: | Interventional |
Study Design: | Treatment, Safety Study |
Estimated Enrollment: | 21 |
Study Start Date: | July 1998 |
PROTOCOL OUTLINE: This is a dose escalation study. Patients undergo a femoral arterial placement of a hepatic intraarterial catheter. Patients then receive adenoviral vector mediated gene transfer intravascularly over 30 minutes.
Cohorts of 3 patients each receive escalating doses of adenoviral vector until the maximum tolerated dose is determined. Patients are followed at 3, 5, 7, 8, 15, and 29 days, at 2 months, and then every 3 months thereafter.
Ages Eligible for Study: | 18 Years to 69 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
--Prior/Concurrent Therapy--
--Patient Characteristics--
Study ID Numbers: | 199/14290, UPSM-FDR001529 |
Study First Received: | October 18, 1999 |
Last Updated: | June 23, 2005 |
ClinicalTrials.gov Identifier: | NCT00004498 History of Changes |
Health Authority: | United States: Federal Government |
genetic diseases and dysmorphic syndromes inborn errors of metabolism ornithine transcarbamylase deficiency rare disease urea cycle disorder |
Ornithine Metabolic Diseases Amino Acid Metabolism, Inborn Errors Rare Diseases Central Nervous System Diseases Brain Diseases Ornithine Carbamoyltransferase Deficiency Disease Metabolism, Inborn Errors Malnutrition |
Genetic Diseases, Inborn Inborn Amino Acid Metabolism Disorder Urea Cycle Disorders Nutrition Disorders Brain Diseases, Metabolic, Inborn Metabolic Disorder Ornithine Transcarbamylase Deficiency Deficiency Diseases Brain Diseases, Metabolic |
Metabolic Diseases Amino Acid Metabolism, Inborn Errors Nervous System Diseases Central Nervous System Diseases Brain Diseases Ornithine Carbamoyltransferase Deficiency Disease Metabolism, Inborn Errors |
Malnutrition Genetic Diseases, Inborn Nutrition Disorders Brain Diseases, Metabolic, Inborn Deficiency Diseases Brain Diseases, Metabolic |