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Phase I Pilot Study of Gene Therapy for Cystic Fibrosis Using Cationic Liposome Mediated Gene Transfer

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
pGT-1 gene lipid complex
Sponsored by
University of Alabama at Birmingham
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis focused on measuring cardiovascular and respiratory diseases, cystic fibrosis, genetic diseases and dysmorphic syndromes, rare disease

Eligibility Criteria

16 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Cystic fibrosis patients Not pregnant Fertile patients must use effective contraception

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    October 18, 1999
    Last Updated
    March 28, 2011
    Sponsor
    University of Alabama at Birmingham
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00004471
    Brief Title
    Phase I Pilot Study of Gene Therapy for Cystic Fibrosis Using Cationic Liposome Mediated Gene Transfer
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    March 2011
    Overall Recruitment Status
    Completed
    Study Start Date
    August 1995 (undefined)
    Primary Completion Date
    May 2001 (Actual)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Name of the Sponsor
    University of Alabama at Birmingham

    4. Oversight

    5. Study Description

    Brief Summary
    OBJECTIVES: Determine whether copies of the cystic fibrosis gene (pGT-1) can be delivered to the cells lining the nose of cystic fibrosis patients using cationic liposome (DMRIE/DOPE) mediated gene transfer.
    Detailed Description
    PROTOCOL OUTLINE: Under direct visualization, patients receive lipid/DNA formulation (pGT-1 lipid complex) by syringe instillation over 30 minutes to the right inferior nasal turbinate. Patients are followed daily for 7 days, twice a week for 3 weeks, every 2-3 weeks for 10 weeks, then every 3 months thereafter.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Cystic Fibrosis
    Keywords
    cardiovascular and respiratory diseases, cystic fibrosis, genetic diseases and dysmorphic syndromes, rare disease

    7. Study Design

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

    8. Arms, Groups, and Interventions

    Intervention Type
    Genetic
    Intervention Name(s)
    pGT-1 gene lipid complex

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    16 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Cystic fibrosis patients Not pregnant Fertile patients must use effective contraception
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Eric J. Sorscher
    Organizational Affiliation
    University of Alabama at Birmingham
    Official's Role
    Study Chair

    12. IPD Sharing Statement

    Learn more about this trial

    Phase I Pilot Study of Gene Therapy for Cystic Fibrosis Using Cationic Liposome Mediated Gene Transfer

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