Revised 8-26-94 
Attachment to the Consent Form for the Protocol "Evaluation of Repeat Adminis- 
tration of a Replication Deficient, Recombinant Adenovirus Vector Containing 
the Normal Cystic Fibrosis Transmembrane Conductance Regulator cDNA to the 
Airways of Individuals with Cystic Fibrosis." 
Ronald G. Crystal, M.D., Principal Investigator 
Purpose and Background 
The purpose of this consent document is to explain to you the possible risks 
and benefits of an experimental gene therapy protocol for the treatment of 
cystic fibrosis. You will receive a copy of the protocol itself, which is re- 
ferred to throughout this consent document. We encourage you to read the pro- 
tocol and to ask as many questions as you feel are necessary to help you un- 
derstand and make an informed decision regarding participation in this clini- 
cal trial. 
It is important for you to know that this is an early trial that is not likely 
to cure your disease or relieve your symptoms. The study is designed to evalu- 
ate the possible side effects and desired changes in lung cells of placing 
fluid and a vector carrying the gene to be transferred into one part of your 
lungs. It is unlikely that this will benefit you personally, but knowledge may 
be gained from the study that will benefit others. Your participation in this 
study is voluntary. The trial will take a considerable amount of time, and in- 
volves risks that are important for you to understand. You are free to 
withdraw from the study without jeopardizing future care by the doctors carry- 
ing out this study. Any significant new findings will be made known to you. 
Your privacy will be protected and your name will not be mentioned in relation 
to this study without your written permission. 
Individuals with cystic fibrosis (CF) have abnormal lung function because they 
lack the presence of a normal functioning gene, called the "cystic fibrosis 
transmembrane conductance regulator" (CFTR) gene, in the cells lining the 
lung. Instead, such individuals contain in their lung cells alterations in the 
normal DNA sequence in the CFTR genes which they have inherited from both 
parents. These mutations cause CF because of the inability of the mutated gene 
to produce an effective CFTR protein. This protocol attempts to compensate for 
this abnormality by introducing a normal CFTR gene into the lung cells using a 
modified cold- like virus as a transporter. The safety, effectiveness, and 
practicality of this approach will be evaluated during the course of the 
protocol . 
The modified virus to be used is called AdcvCFTR.10. It is a modified adeno- 
virus, that is, a laboratory-altered virus that can infect cells in the lung 
like a "cold" virus, but unlike a "cold" virus, AdcvCFTR.10 cannot reproduce 
itself in human lung cells. AdcvCFTR.10 has had inserted into it the normal 
human CFTR gene, which directs the production of the CFTR protein essential 
for normal function of the lung. The Ad^vCFTR.lO virus is capable of carrying 
the normal human CFTR gene into the lung cells of animals where it produces 
the normal human CFTR protein. Also, in the laboratory, AcfjvCFTR.lO can trans- 
fer the normal CF gene to lung cells recovered from individuals with CF, and 
these cells then produce the normal human CFTR protein. If Ad^vCFTR-lO can 
achieve this in the lung cells of live animals , and in cells recovered from 
Recombinant DNA Research, Volume 20 
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