Phase I Study of Liposome-Mediated Gene Transfer in Patients With Cystic Fibrosis
|ClinicalTrials.gov Identifier: NCT00004806|
Recruitment Status : Completed
First Posted : February 25, 2000
Last Update Posted : March 30, 2011
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.
|Condition or disease||Intervention/treatment||Phase|
|Cystic Fibrosis||Genetic: Cystic fibrosis transmembrane conductance regulator||Phase 1|
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 Type :||Interventional (Clinical Trial)|
|Enrollment :||9 participants|
|Study Start Date :||June 1995|
|Actual Primary Completion Date :||November 2002|
|Actual Study Completion Date :||November 2002|
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004806
|Study Chair:||Eric J. Sorscher||University of Alabama at Birmingham|