Phase I Study of Liposome-Mediated Gene Transfer in Patients With Cystic Fibrosis
Phase 1
Completed
- Conditions
- Cystic Fibrosis
- Registration Number
- NCT00004806
- Lead Sponsor
- University of Alabama at Birmingham
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 9
Inclusion Criteria
Not provided
Exclusion Criteria
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method