These parameters will assess whether administration of the recombinant vector 
is associated with efficacy directly and clinically relevant to the patient. 
Some of these parameters are also used to monitor safety (see section 5.5.2). 
The clinical efficacy parameters include: 
General clinical 
Weight 
Dyspnea index 
Incidence of airway infections requiring antibiotic therapy 
Number and length of hospitalizations (other than as dictated by 
the protocol) for respiratory illness 
Roentgenographic 
Chest X-ray 
Chest CT scan 
Scintigraphic 
V/Q scan 
Lung function 
Spirometry, He dilution, DLCO, body box, ABG 
Sputum 
Volume, culture 
5.6 Clinical Protocol 
5.6.1 Overview 
The clinical study will include 10 individuals with cystic fibrosis, grouped 
into 5 groups based on the titer of vector that will be administered. In order 
to maintain a constant relative dose to the epithelium in the nose and 
airways, the volumes (0.2 ml to nose, 20 ml to large bronchus) are based on 
the estimates of surface area that will be exposed to the vector, 0.01 m 2 in 
the nose, 1.0 m 2 in the bronchi, a 100 -fold difference): 
group 1 : n=2 , 
group 2 : n=2 , 
group 3 : n=2 , 
group 4 : n=2 , 
group 5 : n=2 , 
titer of vector 10 6 
titer of vector 10 8 
titer of vector 10 9 
titer of vector 10 10 
titer of vector 10 11 
Each individual will serve as their own control, by comparing parameters in 
the initial baseline period and the vehicle control period to the AdCFTR 
experimental treatment period, as well as in the experimental treatment period 
by comparison of the untreated side (nose and lung) to the treated side (nose 
Recombinant DNA Research, Volume 16 
[749] 
