NOU 23 ’94 15=41 
FROM GENZYflE BIOTHERAP 
TO 9130149696,59 
PAGE . 007 
Page 2 
NEW ENGLAND MEDICAL CENTER 
CONSENT FORM 
PROJECT TITLE: Adenovirus -mediated gene transfer for Cystic 
Fibrosis: Safety of administration to the lung. 
II. Aerosol Administration 
PI: Henry L. Dorkin, M.D. 
Cl; Joseph Oren, M.D. 
which the U.S. Food and Drug Administration has allowed to be 
used only in research with a limited number of adults. 
Ad2 /CFTR-2 was well tolerated in preliminary studies of nasal 
administration in eight adults with CF. In some patients the 
gene transfer temporarily corrected the genetic defect in the 
nose tissue at the site of application. The purpose of the 
current study is to investigate the safety of administering 
Ad2 /CFTR-2 to the lung. You».will receive only one dose of 
Ad2 /CFTR-2. If gene transfer does occur, we would expect the 
effect to be temporary and to not produce any improvement in your 
CF. 
We will enroll a total of 40 patients who will be divided into 8 
groups. There will be 5 patients in each group, all of whom will 
receive the same dose of Ad2/CFTF.-2. The first 3 patients in 
each group will receive Ad2 /CFTR-2 through a bronchoscope, which 
is described below. The next two patients in the group will 
receive Ad2 /CFTR-2 by aerosol. The first group will start at a 
low dose and each subsequent group will receive a slightly higher 
dose, as long as no patient: develops a significant reaction to 
Ad2/CFTR-2. As a safety precaution, patients receiving aerosol 
administration will not receive their dose until 3 patients have 
safely received the same dose and 3 patients have safely received 
the next higher dose via bronchoscopy . 
This study will take twelve months to complete and will require 
four days hospitalization, plus twelve or more follow-up visits 
to the physician. 
You are being invited to participate in this research because you 
have CF and because your lung disease is of mild to moderate 
severity. However it is important for you to realize that it is 
unlikely that you will benefit directly from this study as it is 
expected that any correction of the CFTR gene defect will be 
temporary and not produce any improvement in your CF. 
PROCEDURES 
Screen i nc / Enrol lmenr. 
Please note that the procedures outlined in this section are 
described in detail in the following section. 
Recombinant DNA Research, Volume 20 
[567] 
