Genzyme Corporation Ad2/CFTR-2 Gene Transfer Protocol: Single 
One Kendall Square, Cambridge, MA 02139 Aerosol Administration to the Lung of CF Patients 
PROTOCOL SCHEMATIC 
Day relative to virus administration: 
Pre-trea 
Outpatier 
-21 
tment 
it Days 
-7 
li 
1 
Viral 
ipatier 
2 
Udmin 
it Days 
3 
stratic 
4 
n & K 
Oi 
7 
Ionite 
jtpati 
14 
>ring 
ent Da 
21 
ys 
28 
History 
• 
• 
• 
Physical exam 
• 
• 
• 
Limited Hx & Px 
• 
• 
• 
• 
• 
• 
• 
Laboratory Analyses: Blood samples 
CBC 
• 
• 
Clinical chemistry 
• 
• 
• 
• 
• 
• 
t 
• 
t 
• 
PT/PTT 
• 
• 
• 
• 
• 
Antibody to adenovirus 
• 
Antibody to CFTR 
• 
• 
• 
• j 
CF Genotype 
• 
Urinalysis 
• 
• 
• 
• 
• 
• 
• 
• 
• 
• 
Ad2/CFTR-2 cultures on 293 and HeLa 
cells 
Sputum 
• 
• 
• 
• 
• 
• 
• 
• 
• 
• i 
Nasal swab 
• 
• 
• 
• 
• 
t 
• 
• 
• 
t 
Stool 
• 
• 
• 
• 
• 
• 
• 
• 
• 
• 
Urine 
• 
• 
• 
• 
• 
• 
• 
• 
• 
• 
Bacterial cultures 
Sputum 
• 
• 
• 
• 
Cytology & cell count 
Sputum 
• 
• 
• 
• 
Cytokines 
Sputum 
• 
• 
• 
• 
• 
• 
Pulmonary Function Tests 
Complete 
• 
• 
• 
Spirometry and Oximetry 
• 
• 
• 
• 
• 
Chest CT Scan 
High Resolution Thin Cut 
• 
• 
• | 
Chest X Ray 
PA and Lateral 
• 
Virus administration (aerosolized to the 
lung) 
• 
Bronchoscopy (with Brushing) 
• 
RS-PCR on cells from Brushing 
• 
\ 
[564] 
Recombinant DNA Research, Volume 20 
