Study Groups 
Site 
Group 
Group 
Group 
Group 
Group 
Group 
Group 
Group 
1 
2 
3 
4 
5 
6 
7 
8 
Nasal 
lxlO 6 
lxlO 7 
3xl0 7 
1x108 
3xl0 8 
lxlO 9 
lxlO 9 
lxlO 9 
RLL 
— 
lxlO 7 
3x1 O' 7 
lxlO 8 
3xl0 8 
lxlO 9 
3xl0 9 
10 10 
Protocol. During the course of the 18 day hospital stay, you will be treated with 
intravenous antibiotics and chest physical therapy just as you are when you are treated for a 
pneumonia. Patients will undergo a battery of tests on the day of hospital admission, 
including blood-drawing, pulmonary function testing, chest X-ray, chest CT scanning, 
nasal transepithelial potential difference measurement (TEPD), nasal brushing, and 
fiberoptic bronchoscopy. At the end of the bronchoscopy, a small amount of salt-water 
solution will be administered through the bronchoscope. The entire battery of tests will be 
repeated four days later, but at that time, the actual gene transfer virus will be given to the 
lung and to the nose. Some of the tests will be repeated 3 days and 10 days after vector 
administration. These tests will indicate whether there has been any adverse effect from the 
gene transfer virus and will tell us if the virus is functioning to make the CFTR protein 
which is missing due to the disease. Discharge from the hospital is planned on the 14th 
day after virus administration. You will be followed up with a complete battery of tests at 
30 days, 60 days, 90 days, 180 days, 270 days, and 365 days as an outpatient. We will 
plan to see you every 3 months thereafter when you come for your regular CF clinic visits. 
Depending on the results of the laboratory assessments of how well the gene transfer virus 
is working, the later bronchoscopies may not need to be performed. There will also be 
follow-up appointments at 120, 150, 210 and 250 days after virus administration for 
check-ups with one of the study doctors. We plan to continue seeing you at least once a 
year thereafter as part of your regular CF clinic visits. 
Study Regimen 
Day 
-4 
0 
1 
3 
10 
14 
30 
60 
90 
180 
270 
365 
History 
* 
* 
* 
* 
* 
* 
* 
Physical Exam 
* 
* 
* 
* 
* 
* 
* 
* 
* 
* 
* 
* 
Nasal brush 
* 
* 
* 
* 
* 
* 
* 
* 
* 
* 
Bronchoscopy 
* 
* 
* 
* 
* 
* 
* 
* 
* 
Nasal Voltage 
* 
* 
* 
* 
* 
* 
* 
* 
* 
* 
Chest CT and 
X-ray 
* 
* 
* 
Lung Function 
♦ 
* 
* 
* 
* 
* 
* 
* 
* 
* 
* 
* 
Blood Studies 
* 
* 
* 
* 
* 
* 
* 
* 
* 
* 
Viral Cx. 
* 
* 
Admission 
* 
Vehicle Instilled 
* 
Vector Instilled 
* 
Discharge 
* 
[150] 
Recombinant DNA Research, Volume 20 
