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Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00004498
Recruitment Status : Terminated
First Posted : October 19, 1999
Last Update Posted : March 25, 2015
Information provided by:
FDA Office of Orphan Products Development

Brief Summary:


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 or disease Intervention/treatment Phase
Ornithine Transcarbamylase Deficiency Disease Genetic: Adenoviral Vector-Mediated Gene Transfer Phase 1

Detailed Description:

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.

Completion date provided represents the completion date of the grant per OOPD records

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Study Type : Interventional  (Clinical Trial)
Enrollment : 21 participants
Primary Purpose: Treatment
Study Start Date : July 1998
Study Completion Date : September 2000

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 69 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--Disease Characteristics--

  • Diagnosis of partial ornithine transcarbamylase deficiency Female heterozygote with abnormal allopurinol challenge or underlying defect in either N15 urea or N15 glutamine OR Male with childhood/adulthood onset OR Family history of 2 affected children
  • Stable for at least 1 month prior to study
  • Plasma ammonium levels less than 50 micromoles

--Prior/Concurrent Therapy--

  • Concurrent alternate pathway therapy to control hyperammonemia allowed

--Patient Characteristics--

  • Hepatic: No history of liver disease
  • Other: Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception No high level of neutralizing antibodies to the adenovirus

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00004498

Sponsors and Collaborators
University of Pennsylvania
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Study Chair: Steven E. Raper University of Pennsylvania
Layout table for additonal information Identifier: NCT00004498    
Other Study ID Numbers: 199/14290
First Posted: October 19, 1999    Key Record Dates
Last Update Posted: March 25, 2015
Last Verified: February 2000
Keywords provided by FDA Office of Orphan Products Development:
genetic diseases and dysmorphic syndromes
inborn errors of metabolism
ornithine transcarbamylase deficiency
rare disease
urea cycle disorder
Additional relevant MeSH terms:
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Ornithine Carbamoyltransferase Deficiency Disease
Deficiency Diseases
Nutrition Disorders
Urea Cycle Disorders, Inborn
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases