Revised 8-26-94 
administered to their airways the "vehicle" in which the AdcvCFTR.10 is to be 
carried. This vehicle consists of glycerol, salts and water. The vehicle car- 
ries Ad^vCFTR.10 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 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 the 
AdcvCFTR.10 treatment period part of the protocol. 
The vehicle will be administered to the surface of the three major airways on 
the left or right side of the lung. This will be administered through a bron- 
choscope. The volume to each site will be 100 microliters, about 2 drops. The 
participant will then be observed for any reactions to this dose of vehicle or 
to the method of delivery. As in the baseline period, a variety of tests will 
be carried out. The vehicle control phase will require 2-3 days in the hospi- 
tal, and 1-2 visits to the outpatient clinic to be completed. Details of the 
vehicle control period can be found in section 6.2, of the protocol and the 
schedule of all tests in this period can be found in appendix A5 . 2 . 
If you wish to continue the protocol and still meet all the inclusion and 
exclusion requirements, you may move on to the AdcvCFTR.10 treatment period. 
Details of the AdcvCFTR.lO treatment period can be found in section 6.3 of the 
protocol, and the schedule of tests in this period can be found in appendix 
A5 . 3 . 
On day 1 of the AdcvCFTR.10 experimental treatment part of the protocol, you 
will receive AdcvCFTR.10, the modified adenovirus containing the normal CFTR 
gene . 
The AdcvCFTR.10 vector will then be administered (as was done for the vehicle) 
to three major airways of the left or right side of the lung through the bron- 
choscope in a volume of 100 nl (about 2 drops). To ensure that AdcvCFTR.10, or 
a combination of AdcvCFTR.10 and other viruses, does not spread to others af- 
ter administration of AdcvCFTR.10, you will be kept in isolation for 2 days. 
That means that while your room, no special precautions are needed but when 
going outside the room gloves and masks are required. Hospital staff and 
visitors when visiting you will be required to wear a gown, mask, and gloves 2 
days before you receive AdcvCFTR.10. You will undergo tests similar to those 
you have undergone in the previous two parts of the protocol (see Appendix 
A5.3). However, because one purpose of the study is to determine how long 
Ad^vCFTR-lO will function in your airway epithelial cells, several of those 
tests will be repeated more than once as detailed in the chart in Appendix 
A5 . 3 . 
Assuming that the function stops before 90 days, if you wish to continue the 
protocol, you will move on to part C of the protocol. This part is identical 
to the AdcvCFTR.lO treatment period in part A, except that the dose of 
AdcvCFTR.lO vector will be increased 10 -fold (but never greater than 1 . OxlO 9 
plaque forming units (a measure of virus dose) to each site. The test will 
then be repeated as in part A. At 180 days, you will again receive the 
Ad^vCFTR.10 treatment identical to that at 90 days. The tests will be repeated 
as after 90 days. A full description of the part C can be found in section 8.1 
and all of the tests are detailed in Appendix A5 . 7 . 
[234] 
Recombinant DNA Research, Volume 20 
