Vll.TVie University of Michigan Medical Center 
INFORMED CONSENT FOR EXPERIMENTAL PROCEDURE 
(Version January 1992) 
1 . Title of research project 
Gene Therapy of Cystic Fibrosis Lung Disease Using El Deleted Adenoviruses: A Phase I 
Study 
2 . Names of the researchers 
James M. Wilson, M.D., Ph.D. 
Richard H Simon, M.D. 
John Engelhardt, Ph.D. 
Yiping Yang, M.D. 
Mariann Grossman, B.S. 
Francis S. Collins, M.D., Ph.D. 
James R. Baker, Jr., M.D. 
3 . Purpose of the research 
The purpose of this study is to determine if it is possible to correct the abnormality in the 
lungs of patients with cystic fibrosis. Patients with this disease have abnormalities in a 
gene that is normally responsible for transporting salt across cells. Abnormal salt 
transport in the lung leads to accumulations of thick sticky mucus that obstructs the 
airway passages. 
You have been asked to participate in this study because you have the disease, cystic 
fibrosis. We will attempt to correct the defective function in your lung cells by adding to 
your airways the gene that produces the normal cystic fibrosis protein. The gene will be 
added to a small area of your lung called a segment using a virus to transport the virus into 
the airway cells. This study will help us determine the feasibility and safety of this 
approach for treating lung disease in patients with cystic fibrosis. 
4 . Number of subjects included in the study 
Ten subjects will be recruited for the study from Cystic Fibrosis Centers in Michigan, 
Ohio, and Indiana. If more eligible subjects wish to participate than can be accommodated 
in the study, those who live closest to the University of Michigan Medical Center will be 
enrolled. 
5 . Description of the experiments 
1 . Pre-gene therapy evaluation. You will be evaluated to determine if you are a 
suitable subject to participate in the gene therapy study. This will involve your giving a 
detailed medical history and having a complete physical examination. Blood and urine tests 
will be done to detect any unsuspected problems that might complicate the investigation. 
Blood will be drawn by needle from an artery to measure blood gases and from a vein to 
check blood counts, liver function, kidney function, and other organ functions. Chest x- 
rays and high resolution computed tomography of the chest will be done to evaluate the 
condition of your lungs. You will perform pulmonary function tests as well. 
2. Administering the gene to the lung. Bronchoscopy will be used to administer the 
virus that contains the cystic fibrosis gene to one part of your lung. To prepare you for 
bronchoscopy, you will not eat or drink anything for 6 hours before the procedure. 
Through a needle in your vein, you will be given two medicines (glycopyrrolate and 
meperidine) to keep your heart beat at a steady rate, to relax you, and to temporarily 
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