J 
5) Blood will be drawn (about 2 tablespoonfuls) at the time of screening and 
through the hospitalization, and at follow-up visits. This will be performed 
approximately 10-12 times. The total volume of blood drawn during the study 
will be less than 1 unit (pint). 
6) A small brush or plastic instrument for "scrape biopsy" of nasal cells will be 
inserted into the nose to collect cells to test for evidence of inflammation or 
virus. This procedure may be slightly painful, but numbing medicine (lidocaine) 
can be used, and symptoms typically last for less than an hour. 
7) A nasal biopsy will be performed on one occasion by an ear, nose, and throat 
doctor to identify evidence of inflammation, and presence of the normal CF 
gene protein. This will be done using local anesthetic, and is usually associated 
with some local pain, nasal discharge, and/or minor bleeding. 
All of the procedures and tests will be solely for this research study. 
However, many of the procedures that are performed (throat swab, blood drawing, 
urine collection) are standard medical procedures that are performed at UNC 
Hospitals. 
Special considerations for the research subjects include : 
1) Gene therapy is a new form of treatment, and the long-term outcome is not 
known. My agreement to participate in this study means that I recognize the 
need for careful follow-up. This will involve evaluation for up to a year with a 
medical history, physical examination, chest X-ray, and routine blood tests. 
2) In the case of an unexpected death (such as from a car accident), a post-mortem 
evaluation would be valuable to search for any effects of gene therapy. My 
participation means that I would tend to favor an autopsy should such an 
occurrence take place. I will notify my family of my opinion, but I understand 
that this Consent Form does not bind my family to agree to an autopsy. 
3) I will not be identified in reports about this study, but the public interest in gene 
therapy may lead the media to attempt to identify who I am. The researchers 
will do everything possible to protect my privacy, but I may be identified by 
other means and subject to frequent requests for interviews and my privacy may 
be reduced. 
4) The study will involve three different doses of virus. The lower dose of virus 
will be completed in three patients before testing the mid-level dose, and the 
mid-level dose will be completed in three patients before testing the higher 
level dose (three patients). I understand that the chance of side-effects with 
each dose cannot be clearly predicted, although traditional concepts suggest 
that the higher doses may be more likely associated with side-effects. 
5) After addition of the virus into the nose, I will be required to remain in the 
hospital in respiratory isolation for approximately two (or up to four) weeks. In 
part, this is for the public welfare to make certain that the recombinant virus is 
not spread around to the general public. I understand that if I attempt to leave 
[520] 
Recombinant DNA Research, Volume 17 
_ 
