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

Brief Title

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


Brief Summary

      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.
    

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
    

Study Phase

Phase 1

Study Type

Interventional




Condition

Ornithine Transcarbamylase Deficiency Disease

Intervention

Adenoviral Vector-Mediated Gene Transfer


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Genetic

Estimated Enrollment

21

Start Date

July 1998

Completion Date

September 2000


Eligibility 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
      

Gender

All

Ages

18 Years - 69 Years

Accepts Healthy Volunteers

No

Contacts

Steven E. Raper, , 



Administrative Informations


NCT ID

NCT00004498

Organization ID

199/14290

Secondary IDs

UPSM-FDR001529


Study Sponsor

University of Pennsylvania


Study Sponsor

Steven E. Raper, Study Chair, University of Pennsylvania


Verification Date

February 2000