Clincosm LogoSign in Get a demo

Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency

Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (NIH)
Overall Status
Terminated
CT.gov ID
NCT00004386
Collaborator
Children's National Research Institute (Other)

Study Details

Study Description

Brief Summary

OBJECTIVES:

Evaluate the safety and feasibility of administering recombinant adenovirus containing the ornithine transcarbamylase gene to adults with partial ornithine transcarbamylase deficiency.

Condition or Disease Intervention/Treatment Phase
  • Genetic: recombinant adenovirus containing the ornithine transcarbamylase gene
 Phase 1

Detailed Description

PROTOCOL OUTLINE: This a dose escalation study to estimate the maximum tolerated dose of recombinant adenovirus encoded with the ornithine transcarbamylase gene.

Patients receive a single dose of virus infused into the liver under fluoroscopic guidance. Groups of 3 patients receive successively higher doses of virus; each cohort is observed for safety for 3 weeks before entry of the next group.

Patients are followed weekly for 1 month, then every 3 months until stable.

Study Design

Study Type:
Interventional
Primary Purpose:
Treatment
Study Start Date :
Oct 1, 1995

Outcome Measures

Primary Outcome Measures

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 65 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    PROTOCOL ENTRY CRITERIA:

    --Disease Characteristics-- Confirmed partial ornithine transcarbamylase deficiency Asymptomatic, i.e., at least 1 month since hyperammonemia --Prior/Concurrent Therapy-- Not specified --Patient Characteristics-- No pregnant or nursing women Negative pregnancy test required of fertile women

    Contacts and Locations

    Locations

    No locations specified.

    Sponsors and Collaborators

    • Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
    • Children's National Research Institute

    Investigators

    • Study Chair: Mark Batshaw, Children's National Research Institute

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    , ,
    ClinicalTrials.gov Identifier:
    NCT00004386
    Other Study ID Numbers:
    • 199/12054
    • CSH-3660
    First Posted:
    Oct 19, 1999
    Last Update Posted:
    Jun 24, 2005
    Last Verified:
    Jan 1, 2000
    Keywords provided by , ,
    inborn errors of metabolism
    rare disease
    urea cycle disorder
    Additional relevant MeSH terms:
    Ornithine Carbamoyltransferase Deficiency Disease
    Deficiency Diseases

    Study Results

    No Results Posted as of Jun 24, 2005