Gene Therapy for CF using Cationic Liposome Mediated Gene Transfer: Phase I Trial 
very slowly drip a salt-water solution, which I might taste in the back of my 
throat during the measurement. For part of the measurement, the solution may 
also contain small amounts of drugs which help detect the presence of the normal 
CF gene. These drugs include Amiloride and Isoproterenol, two drugs which are 
already approved for use in humans and have been given to cystic fibrosis patients 
and other patients in the past. The measurement will be made approximately 15 
times during the entire study. Each measurement requires about 10-15 minutes. 
The measurement can be made either while I am seated or lying down. 
5. Nasal brushes and biopsies. Nasal brushes and biopsies will be performed in 
order to evaluate whether administration of lipid or DNA caused any damage to 
the tissues lining the nose. A small nasal brush will be used to scrape cells lining 
the inside of my nose. These cells will be studied for the presence of the normal 
CF gene, and to look for evidence of inflammation resulting from administration 
of the gene. This procedure can be slightly painful, with pain lasting from one to 
several hours. Numbing medication (Lidocaine) or over-the-counter pain 
medications will be used to control the pain. In order to further study whether 
administration of the normal CF gene causes inflammation or other damage, and 
to help show that the normal CF gene is being utilized by the cells lining the 
nose, two nasal biopsies will be performed on one occasion during the study. A 
local anesthetic (Lidocaine) will be given to numb the area prior to taking the 
biopsies. The nasal biopsy procedure can be associated with some bleeding, 
discharge, and local pain. This biopsy will be performed by an ear, nose, and 
throat physician. 
6. Nasal Endoscopy 
Prior to administration of the normal CF gene or other studies of the tissue inside 
the nose, a small rubber tube (an endoscope) will be placed about 4" inside the 
nose so that the tissues can be evaluated more easily. This procedure usually takes 
less than 10 minutes, and can be associated with minor discomfort. Local 
anesthesia or other pain medicine will be given whenever possible to control the 
discomfort. 
With the exception of the nasal potential difference study, all of the tests and procedures 
described in this study are routinely performed at the UAB Hospitals. I understand that all of 
the test which I undergo will be only for the purpose of the CF Gene Transfer Study. 
RISKS AND DISCOMFORTS 
I am aware that there are several risks and discomforts associated with participation in 
this study. These include: 
a) Pain, bruising and possible infection at the site of blood drawing, and at the site 
of insertion of the needle in the forearm as part of the nasal potential difference 
measurements. 
b) Local pain and inflammation within the nose due to administration of the gene or 
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