CONTINUATION SHEET for either: 
NIH 2514-1, Consent to Participate In A Clinical Research Study 
NIH 2514-2, Minor Patient’s Assent to Participate In A Clinical Research Study 
STUDY NUMBER: 
_ CONTINUATION: page JL of _21 pages. 
All subjects should have reproducible airway epithelial potential difference 
to subcutaneous tissues of > -40 mV. Electrical potential difference is a 
measurement of the electric charge on one surface compared with another. The 
value of > -40 mV is expected for cystic fibrosis individuals. This technique 
is described in more detail below and in the protocol, section 5. 
Two criteria require the potential study participant to manifest some of the 
classical abnormalities associated with CF in their airway epithelial lining 
cells. These will be evaluated by obtaining from the prospective participant 
airway epithelial lining cells and evaluating them in the laboratory. The 
criteria are as follows: 
1. Airway epithelial cells not expressing the CFTR gene in a normal fash- 
ion, such that the CFTR protein (the product of the CFTR gene) , cannot 
be detected using established laboratory criteria. 
2. All subjects should have airway epithelial CFTR mRNA demonstrating the 
common "delta F508" CFTR mutation known to be associated with CF or the 
"delta F508" mutation along with what is referred to as a "null" muta- 
tion (a rare cause of CF) . This means that the airway cells recovered 
from prospective participants must show, by laboratory analysis, evi- 
dence of the abnormal gene sequences known to cause CF. The study is 
limited to those mutations for technical reasons associated with analy- 
sis of the cells in the lung following treatment. 
3 . As a secondary criteria (depending on the availability of the airway 
cells and technical aspects of carrying out the study in the laborato- 
ry) , the airway epithelial cells will be evaluated for their inability 
to secrete Cl~ (part of table salt) in a normal fashion in response to 
cAMP. A laboratory test will evaluate the airway lining cells removed 
by nasal brushing and by bronchial brushing. In airway cells removed 
from individuals with CF, stimulation of the cells with cAMP (a regula- 
tory molecule within cells) reveals abnormalities in the movement of 
salts and fluids (this includes Cl") in and out of the cells. 
All study subjects, to enter the experimental treatment part (part 3) where 
AdCFTR is administered, must be willing to remain at the NIH clinical center 
until they reach "outpatient criteria". These criteria are explained in the 
protocol in section 5.6.4. 
The AdCFTR virus has been designed so that it will not reproduce itself in 
cells lining the airways. This will be confirmed in your airway cells in the 
laboratory before you are administered AdCFTR. Studies in laboratory animals 
have demonstrated that AdCFTR can be detected in the nose, back of the mouth, 
PATIENT IDENTIFICATION 
CONTINUATION SHEET for either: 
r^r-i A 1 MO-fld) 
Recombinant DNA Research, Volume 16 
[ 779 ] 
