NO<J 23 '94 15:46 
FROn GENZYnE ElOTHERRP 
TO 913014969639 
PAGE. 019 
SHS Approval Stamp Patient ID Stamp 
Massachusetts General Hospital 
MEDICAL RESEARCH CONSENT FORM, Page Z 
Title of Project: Adenovirus-Mediated Gene Transfer for Cvstic 
Fibrosis-. Safety of Single Lcbar Administration in the Luna 
Principal Investigator: Allen Lapev. M.D. 
Ad2 /CFTR-2 has been shown to be able to enter the cells that form 
the lining of the airway and deposit the gene so that normal CFTR 
is produced by the cell. Ad2/CFTR-2 is an investigational agent 
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 pattents in each group, all of whom will 
receive the same dose of Ad2 /CFTR-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 
Recombinant DNA Research, Volume 20 
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