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

Primary Purpose

Ornithine Transcarbamylase Deficiency Disease

Status
Terminated
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
Adenoviral Vector-Mediated Gene Transfer
Sponsored by
University of Pennsylvania
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Ornithine Transcarbamylase Deficiency Disease focused on measuring genetic diseases and dysmorphic syndromes, inborn errors of metabolism, ornithine transcarbamylase deficiency, rare disease, urea cycle disorder

Eligibility Criteria

18 Years - 69 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    October 18, 1999
    Last Updated
    March 24, 2015
    Sponsor
    University of Pennsylvania
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00004498
    Brief Title
    Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    February 2000
    Overall Recruitment Status
    Terminated
    Study Start Date
    July 1998 (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    September 2000 (undefined)

    3. Sponsor/Collaborators

    Name of the Sponsor
    University of Pennsylvania

    4. Oversight

    5. Study Description

    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

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Ornithine Transcarbamylase Deficiency Disease
    Keywords
    genetic diseases and dysmorphic syndromes, inborn errors of metabolism, ornithine transcarbamylase deficiency, rare disease, urea cycle disorder

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 1
    Enrollment
    21 (false)

    8. Arms, Groups, and Interventions

    Intervention Type
    Genetic
    Intervention Name(s)
    Adenoviral Vector-Mediated Gene Transfer

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Maximum Age & Unit of Time
    69 Years
    Accepts Healthy Volunteers
    No
    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
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Steven E. Raper
    Organizational Affiliation
    University of Pennsylvania
    Official's Role
    Study Chair

    12. IPD Sharing Statement

    Learn more about this trial

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

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