INFORMATION SUMMARY FOR 
INFORMED CONSENT 
TITLE : Adenovirus-mediated gene transfer for cystic fibrosis: 
Safety of dose and repeat administration in the nasal epithelium 
INVESTIGATORS : Michael J. Welsh, M.D. 
Joseph Zabner, M.D. 
Scott M. Graham, M.D. 
1. I NTRO DUCTIO N A ND PRO CEDURES: 
During the course of treatment of your disease, you have learned that cystic fibrosis is a 
serious generic 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 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 on the day of the treatment and discharged on the following day. You will 
need to return to the clinic for an evaluation 3, 7, 14, and 21 days after the treatment. The 
treatment will be repeated five times, with at least 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 chest 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 you 
already have protection against the virus. By collecting specimens of nasal secretions, 
stool, and urine, we will also determine that you do not already have the natural type of 
adenovirus in your body. The specific genes for cystic fibrosis which you carry in your 
chromosomes will be determined from a blood sample. We will also measure the voltage 
that normally exists across the cells that line your nose. You will be evaluated with these 
tests when you enter the study. You will then be clinically evaluated at about ten days 
Recombinant DNA Research, Volume 18 
[883] 
