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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified February 2000 by FDA Office of Orphan Products Development.
Recruitment status was  Active, not recruiting
Sponsor:
Collaborator:
University of Pennsylvania
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
NCT00004498
First received: October 18, 1999
Last updated: June 23, 2005
Last verified: February 2000
History of Changes
  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: Endpoint Classification: Safety Study
Primary Purpose: Treatment

Resource links provided by NLM:

Drug Information available for: Ornithine
U.S. FDA Resources

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

Estimated Enrollment: 21
Study Start Date: July 1998
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.

  Eligibility

Ages Eligible for Study:   18 Years to 69 Years
Genders Eligible for Study:   Both
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00004498

Sponsors and Collaborators
FDA Office of Orphan Products Development
University of Pennsylvania
Investigators
Study Chair: Steven E. Raper University of Pennsylvania
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00004498     History of Changes
Other Study ID Numbers: 199/14290, UPSM-FDR001529
Study First Received: October 18, 1999
Last Updated: June 23, 2005
Health Authority: United States: Federal Government

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

ClinicalTrials.gov processed this record on May 16, 2013