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

This study has been completed.
Sponsor:
Collaborator:
Information provided by:
University of Alabama at Birmingham
ClinicalTrials.gov Identifier:
NCT00004806
First received: February 24, 2000
Last updated: March 29, 2011
Last verified: March 2011

February 24, 2000
March 29, 2011
June 1995
November 2002   (final data collection date for primary outcome measure)
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Complete list of historical versions of study NCT00004806 on ClinicalTrials.gov Archive Site
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Phase I Study of Liposome-Mediated Gene Transfer in Patients With Cystic Fibrosis
Not Provided

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.

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.

Interventional
Phase 1
Primary Purpose: Treatment
Cystic Fibrosis
Genetic: Cystic fibrosis transmembrane conductance regulator
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
9
November 2002
November 2002   (final data collection date for primary outcome measure)

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

Both
18 Years and older
No
Contact information is only displayed when the study is recruiting subjects
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NCT00004806
199/11983, UAB-11983
No
Not Provided
University of Alabama at Birmingham
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Study Chair: Eric J. Sorscher University of Alabama at Birmingham
University of Alabama at Birmingham
March 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP