INFORMATION SUMMARY FOR 
INFORMED CONSENT 
Project Title: Adenovirus-mediated gene transfer for cystic fibrosis: 
Safety of repeat administration and clinical efficacy 
in the maxillary sinus 
Investigators: Michael J. Welsh, M.D. 
Joseph Zabner, M.D. 
Scott M. Graham, M.D. 
1. INTRODUCTION AND PROCEDURES : 
During the course of treatment of your disease, you have learned that cystic fibrosis is a 
serious genetic disease that causes the airways of the lung to fill with thick mucus. This 
situation leads to frequent infections which in turn cause destructive changes in the lung 
and increasing problems with breathing. The basic problem is a defect in a gene that is 
responsible for producing a protein called the cystic fibrosis transmembrane conductance 
regulator or CFTR. This protein regulates salt balance in the airways of the lungs; when 
the protein is not normal, excessive thick mucus is produced. Antibiotics have been 
helpful and are used to treat the infections that result from this problem. Various ways to 
clear the mucus, such as chest physical therapy, are also useful to treat cystic fibrosis. 
However, none of these treatments correct the basic problem. Thus, despite all efforts, the 
problem in the lungs usually worsens with time. 
la. This study involves research in gene therapy for cystic fibrosis. The ultimate purpose of 
the research is to find a better treatment for cystic fibrosis. In this study we will examine 
the safety and possibility of correcting the basic genetic defect and the clinical 
abnormalities by applying an altered virus onto the cells of the airway in people who have 
cystic fibrosis. 
lb. You are being invited to participate in this research because you have cystic fibrosis and 
because your disease is of mild to moderate severity. It is unlikely that participation in 
this study will be of any benefit to you. After enrolling into the study you will be 
monitored for a period of at least three weeks before the treatment. You will be admitted 
to the hospital for seven days before the first treatment, during which time you will be 
given antibiotics by vein and your maxillary sinuses will be washed three times a day 
with antibiotics. On the day after treatment you will be discharged from the hospital. 
You will need to return to the clinic for an evaluation 3, 7, 14, and 21 days after the 
treatment. The procedure will be repeated up to a maximum of six times (Tor a total of 
seven applications of the virus) . with about 4 weeks between each treatment. After each 
treatment you will need to stay in the hospital for one day and then return to the clinic at 
the same time intervals as you do after the first treatment. 
lc. If you agree to participate , these procedures will be followed. 
An extensive evaluation will be performed that includes a complete physical exam, 
computed tomographic X-rays of your sinuses, and pulmonary function tests. Blood will 
be drawn from your vein and artery (total of about 1 cup) for multiple studies. We will 
also determine whether or not you have immunity to adenovirus, so that we know that 
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