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

This study has been terminated.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00004498
First Posted: October 19, 1999
Last Update Posted: March 25, 2015
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.
Information provided by:
FDA Office of Orphan Products Development
  Purpose

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 Genetic: Adenoviral Vector-Mediated Gene Transfer Phase 1

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment: 21
Study Start Date: July 1998
Estimated Study Completion Date: September 2000
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

  Eligibility

Information from the National Library of Medicine

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

PROTOCOL ENTRY CRITERIA:

--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
  Contacts and Locations
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 ClinicalTrials.gov identifier (NCT number): NCT00004498


Sponsors and Collaborators
University of Pennsylvania
Investigators
Study Chair: Steven E. Raper University of Pennsylvania
  More Information

ClinicalTrials.gov Identifier: NCT00004498     History of Changes
Other Study ID Numbers: 199/14290
UPSM-FDR001529
First Submitted: October 18, 1999
First Posted: October 19, 1999
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:
Deficiency Diseases
Ornithine Carbamoyltransferase Deficiency Disease
Malnutrition
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