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Phase I Study of Liposome-Mediated Gene Transfer in Patients With Cystic Fibrosis

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
Cystic fibrosis transmembrane conductance regulator
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

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

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Cystic fibrosis (CF), i.e.: Sweat chloride greater than 60 mEq/L Clinical manifestations of CF Homozygous delta F508 mutation preferred Compound heterozygotes for F508 mutation with pancreatic insufficiency eligible FEV1 greater than 40% of predicted PO2 at least 60 mm Hg on room air No acute or recurrent sinusitis No obstructive nasal polyposis No pneumothorax or hemoptysis, e.g., more than 250 mL blood within 24-hour period, within past year No unstable lung disease with worsening pulmonary symptoms, arterial blood gas, or pulmonary function tests within 2 weeks prior to entry No pulmonary exacerbation within 4 weeks prior to entry --Prior/Concurrent Therapy-- At least 2 months since participation in any other clinical study At least 2 months since DNase At least 4 months since corticosteroids --Patient Characteristics-- Cardiac: No severe cardiac disease Other: No other severe organ system disease, e.g., juvenile-type diabetes mellitus No pregnant women

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    February 24, 2000
    Last Updated
    March 29, 2011
    Sponsor
    University of Alabama at Birmingham
    Collaborators
    National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00004806
    Brief Title
    Phase I Study of Liposome-Mediated Gene Transfer in Patients With Cystic Fibrosis
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    March 2011
    Overall Recruitment Status
    Completed
    Study Start Date
    June 1995 (undefined)
    Primary Completion Date
    November 2002 (Actual)
    Study Completion Date
    November 2002 (Actual)

    3. Sponsor/Collaborators

    Name of the Sponsor
    University of Alabama at Birmingham
    Collaborators
    National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

    4. Oversight

    Data Monitoring Committee
    No

    5. Study Description

    Brief Summary
    OBJECTIVES: Evaluate the efficacy and safety of lipid-mediated transfer of the cystic fibrosis transmembrane conductance regulator gene to nasal epithelium in patients with cystic fibrosis.
    Detailed Description
    PROTOCOL OUTLINE: Cystic fibrosis transmembrane conductance regulator (CFTR) gene complexed with lipid is administered intranasally to the right inferior turbinate. Lipid without CFTR is administered to the left nostril as a control. Cohorts of 3 patients are given successively lower doses of CFTR, each dose complexed with 500 micrograms of lipid. Patients are followed at days 2-12, 15, and 21, then every 3-4 days for 3 weeks, every 2-3 weeks for 10 weeks, and 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)
    Cystic fibrosis transmembrane conductance regulator

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Cystic fibrosis (CF), i.e.: Sweat chloride greater than 60 mEq/L Clinical manifestations of CF Homozygous delta F508 mutation preferred Compound heterozygotes for F508 mutation with pancreatic insufficiency eligible FEV1 greater than 40% of predicted PO2 at least 60 mm Hg on room air No acute or recurrent sinusitis No obstructive nasal polyposis No pneumothorax or hemoptysis, e.g., more than 250 mL blood within 24-hour period, within past year No unstable lung disease with worsening pulmonary symptoms, arterial blood gas, or pulmonary function tests within 2 weeks prior to entry No pulmonary exacerbation within 4 weeks prior to entry --Prior/Concurrent Therapy-- At least 2 months since participation in any other clinical study At least 2 months since DNase At least 4 months since corticosteroids --Patient Characteristics-- Cardiac: No severe cardiac disease Other: No other severe organ system disease, e.g., juvenile-type diabetes mellitus No pregnant women
    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 Study of Liposome-Mediated Gene Transfer in Patients With Cystic Fibrosis

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