Phase I Study of Liposome-Mediated Gene Transfer in Patients With Cystic Fibrosis
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.
Genetic: Cystic fibrosis transmembrane conductance regulator
|Study Design:||Primary Purpose: Treatment|
|Study Start Date:||June 1995|
|Study Completion Date:||November 2002|
|Primary Completion Date:||November 2002 (Final data collection date for primary outcome measure)|
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.
|Study Chair:||Eric J. Sorscher||University of Alabama at Birmingham|