Children's Hospital Medical Center 
Consent to Participate in a Research Study 
A Phase I Study of Gene Therapy of Cystic Fibrosis Utilizing a Replication 
Deficient Recombinant Adenovirus Construct to Deliver the Cystic Fibrosis Transport 
Regulator Gene to the Airways 
Before agreeing that I will participate in this study, it is important that I read and 
understand the following explanation. It describes in words that can be understood by a 
lay person the purpose, the procedures, the benefits, risks, and discomforts of the study, 
and the precautions that will be taken. It also describes the alternatives available and 
the right to withdraw from the study at any time. It is important to understand that no 
guarantee or assurance can be made as to the results of this study. It is also understood 
that refusal to participate will not influence the availability of standard medical 
treatment. 
I, 
Address 
Patient 
of 
Street 
> 
consent 
City 
, voluntarily 
State Zip 
for myself , to participate 
''Name Age 
in a research study, the purpose of which is to evaluate the safety and feasibility of using 
gene therapy in cystic fibrosis with a replication deficient adenovirus to deliver the 
normal cystic fibrosis transmembrane conductance regulator (CFTR) gene to the upper 
and lower airways. I was selected to participate in this study because I am an adult with 
cystic fibrosis and mild to moderate lung disease. The cystic fibrosis transmembrane 
conductance regulator is the protein that is absent or deficient in people affected by 
cystic fibrosis. CFTR can now be manufactured in the laboratory as the gene for cystic 
fibrosis was identified in 1989. Adenovirus is a cold virus which has been altered in the 
laboratory so that it will not reproduce itself in my body and so that it can carry the 
normal version of the CFTR gene into the cells of my lungs and nose. 
The objective of this study is to determine whether the adenovirus-CFTR construct can 
be given into my nose and lower airways with incorporation in my cells resulting in 
manufacture of the normal CFTR by my body. This study will establish whether 
manufacture of the CFTR protein by these cells corrects the abnormal electrical voltages 
in the nose and airways of CF patients. This study will also evaluate the safety of this 
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Recombinant DNA Research, Volume 17 
