MEDICAL RECORD 
1 - 
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 U_ of Z±_ pages. 
are complete, study subjects will be divided sequentially into 5 groups based 
on the titer of AdCFTR that will be administered (titer is the amount of 
active virus contained in 1 milliliter of fluid, approximately one- fifth of a 
teaspoon) in part 3 . The individuals in each group will be evaluated in pairs 
(not more than 7 days apart) . 
group 
1: 
n - 2, 
titer 
of 
virus 
10 6 
group 
2: 
n = 2, 
titer 
of 
virus 
10 8 
group 
3: 
n - 2, 
titer 
of 
virus 
10 9 
group 
4: 
n = 2, 
titer 
of 
virus 
10 10 
group 
5: 
n - 2, 
titer 
of 
virus 
10 11 
The three parts of the protocol are: part 1 - baseline period, part 2 - 
vehicle control period, and part 3 - AdCFTR experimental treatment period. 
Part 1 (baseline) will last one month during which the study individual 
undergoes a variety of tests. This will be used to evaluate whether the 
individual is eligible for the remainder of the protocol i.e., meets all 
inclusion/exclusion criteria. The baseline period is also used to determine 
the "baseline" values for the safety and efficacy (potential good results) 
measurements to be used in parts 2 and 3 of the protocol. Details of the 
baseline period (part 1) can be found in section 5.6.2 of the protocol, and a 
schedule of all tests in the baseline period can be found in the protocol in 
Table 5. 6. 2. A. 
If, after completing part 1, the study individual wishes to continue and still 
meets all the inclusion and exclusion criteria he/she will next move to the 
vehicle control period (part 2). In this part they again undergo the same 
type of tests as in part 1 but with one difference. They will have adminis- 
tered to their nose and airway the "vehicle" in which the AdCFTR is to be 
carried. This vehicle consists of glycerol, salts and water. The vehicle 
carries AdCFTR to the site where it can deliver the normal CFTR gene to the 
airway cells. The components of the vehicle ensure that the virus remains 
effective while being transported. The purpose of administering the vehicle 
alone to study individuals is to check for any adverse reaction to the vehicle 
itself, and to determine the values for the safety and efficacy parameters to 
be used in part 3 of the protocol. 
The vehicle will first be administered to the nose through a nasal speculum, a 
device which is easily inserted into the opening of the nose. The vehicle 
will be administered through the nasal speculum directly onto the surface of 
the left nostril. The amount of vehicle will be 0.2 milliliter, about 4 
drops. The participant will then be observed for 24 hours to evaluate any 
reactions. If there are no adverse reactions to nasal administration of the 
vehicle the participant will then receive the vehicle onto the surface of 
PATIENT IDENTIFICATION 
CONTINUATION SHEET for either: 
[ 782 ] 
Recombinant DNA Research, Volume 16 
