' »y i 
UNC Hospitals 
Chapel Hill, North Carolina 
Study number 92-MED-290 
CONSENT TO PARTICIPATE IN A RESEARCH STUDY 
TITLE OF STUDY: Gene Therapy for Cystic Fibrosis using El Deleted 
Adenovirus: A Phase I Trial in the Nasal Cavity 
Principal Investigators: Michael R. Knowles, M.D., Richard C. Boucher, M.D. 
Co-Investigator: Larry G. Johnson, M.D. 
I have been asked to take part in a research study under the direction of 
Michael R. Knowles, M.D. and Richard C. Boucher, M.D. I understand that I will 
be one of approximately 9 subjects in this Phase I study, which is a pilot (first time) 
study. 
Purpose : I understand that this research study is designed to test whether it is 
possible to transfer safely a normal CF gene into the nasal cells of CF patients and 
correct the abnormal cell function that is associated with the genetic defect in CF. 
This method of treating patients with genetic diseases has been termed "gene 
therapy", and the ultimate goal of this initial study of gene therapy is to develop a 
better treatment for CF. The vehicle that will be used to carry the normal CF gene 
into the nasal cells will be a "cold" virus (adenovirus) that has been modified so that 
it should not multiply and spread throughout the body. This study will have two 
goals. The first goal is to determine if gene therapy can be accomplished safely in 
the nose, and thereby allow the possibility of later attempting treatment in the lungs. 
The second is to determine whether it is possible to correct the ion transport 
abnormalities in CF airway cells that are responsible for thick mucus. I understand 
that this study is not designed for treatment of the lungs, and that I am unlikely to 
get any medical benefit from this nose study of adenoviral gene therapy. 
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Recombinant DNA Research, Volume 17 
