Duration : I understand that my most active participation in this study will last for 
approximately four months. I will be screened for this study at least four weeks 
before gene therapy is undertaken, and re-evaluated approximately two weeks 
before hospital admission. I will be in the hospital [General Clinical Research 
Center (GCRC)] for approximately 1 week before gene therapy, and for 
approximately two (possibly three or four) weeks after gene therapy. I will be 
monitored for several weeks after discharge from the hospital on a weekly basis, and 
then for two- to four-week intervals for a total of three months, and then at the end 
of a year. The initial (screening) outpatient visit will last most of a day, and the 
other outpatient visits will last several hours. 
Procedures : I understand that during this study, the following will occur: 
1) The study design will involve evaluation at least four weeks before gene therapy 
is initiated, and admission to a hospital room on the GCRC in respiratory 
isolation for one week for re-evaluation before gene therapy. Following gene 
therapy, I will remain in respiratory isolation in the GCRC for two (or up to 
four) weeks (see below), during which time the feasibility and safety of this 
method of gene therapy will be tested. 
2) The adenovirus containing the normal CF gene is an experimental medication. 
The virus will be administered into the nose by slow perfusion through a small 
tube for 30 minutes. This will be accomplished while I am lying on my side so 
that the solution can be placed into the nostril under direct vision. A control 
solution (not containing virus) will be placed into the opposite nostril to allow 
comparison of nasal cell function before and after virus, and as compared to the 
side not receiving gene therapy. A numbing medicine (lidocaine) will be 
applied inside the nose before putting in the virus. 
3) Nasal potential difference, i.e., "electrical activity" will be measured before and 
after virus to see if the abnormalities in CF nasal cells are corrected after gene 
therapy. This test is similar to the electrocardiogram, where electrical signals 
can be measured but not felt in the body. The electrical activity will be 
measured by inserting a needle under the skin of the forearm, and touching the 
inner surface of the nose with a small catheter. The electrical response to nasal 
addition of drugs will also be measured, including amiloride and isoproterenol 
(approved for human use). Amiloride is normally given for heart and renal 
disease. Isoproterenol is one of the original inhaled asthma drugs. This study 
will be performed approximately 10 times, beginning at the time of initial 
screening, and throughout the follow-up visits. 
4) Nasal washes and throat swabs will be performed to test for inflammatory cells 
and presence of the virus. The nasal wash is performed by instilling one 
teaspoonful of salt water into the nose, tilting the head back for approximately 
10 seconds, and collecting the expelled liquid when the head is tilted forward. 
The throat swab is performed with a cotton tipped swab applied to the back of 
the throat. These will be performed approximately 10-12 times from screening 
through the follow-up visits. 
Recombinant DNA Research, Volume 17 
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