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

NCT ID: NCT00004806

Last Updated: 2011-03-30

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 1995-06-30
• Study Completion Date: 2002-11-30

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.

Conditions

Cystic Fibrosis

Study Design

• Primary Study Purpose: TREATMENT

Interventions

Cystic fibrosis transmembrane conductance regulator

Intervention Type: GENETIC

Eligibility Criteria

Inclusion 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

NIH

Sponsor Role: collaborator

University of Alabama at Birmingham

OTHER

Sponsor Role: lead

Principal Investigators

Eric J. Sorscher

Role: STUDY_CHAIR

University of Alabama at Birmingham

Other Identifiers

UAB-11983

Identifier Type: -

Identifier Source: secondary_id

199/11983

Identifier Type: -

Identifier Source: org_study_id