Revised 8-26-94 
undergo the procedures for either personal reasons or for clinical reasons 
such as a tendency for easy bleeding, allergies to local anesthesia (numbing 
medicine) or difficulty tolerating the placement of the bronchoscopy tube in 
the airways . 
You should not be taking experimental medicines relevant to your cystic 
fibrosis for at least 4 weeks prior to entry into study. Such medicines in- 
clude alpha 1-antitrypsin or amiloride or other unproven therapies for cystic 
fibrosis. If you were taking these medicines, it would be difficult to under- 
stand whether changes or improvements were due to the gene therapy treatment 
in this protocol or to these other experimental medicines. 
You should be able to stay at the hospital for the period of time necessary to 
complete the baseline period (2-3 days + 1-3 outpatient visits), vehicle con- 
trol period (2-3 days + 1-3 outpatient visits), and vector administration 
period (a minimum of 2-3 days in the hospital once every month for as many as 
7-10 months, plus intermittent evaluations in the outpatient clinic). Addi- 
tionally, when the treatment is period is over, you will be asked to return to 
the hospital and/or outpatient clinic once a month for 5 months, then at 6 
month intervals over the next year, and then once yearly thereafter. This is 
in order to test for safety and effectiveness of the treatment during the ad- 
ministration part and follow-up. 
Assessment of your airway epithelium should show no evidence of adenovirus 
infection. This is in order to prevent, as far as possible, any contact 
between naturally occurring adenoviruses and the Ad^vCFTR-lO virus. Such con- 
tact could possibly enable the AdcvCFTR.lO virus to reproduce or give rise to 
a new virus by incorporating parts of the AdcvCFTR.lO virus with other 
viruses . 
You should have no history of allergy to glycerol which is part of the solu- 
tion used to carry the virus or to medicines used in the bronchoscopy 
procedures. This is to minimize any potential for allergic reactions. 
The AdcvCFTR-lO virus will be used to transfer and produce a functional CFTR 
protein to act in place of your abnormal CFTR protein. In animal studies, the 
AdcvCFTR.lO gene has produced higher levels of functional CFTR protein than 
the normal animal levels. In these instances where overproduction of the nor- 
mal protein has occurred, no adverse effects were noted. Therefore, it is our 
expectation that production or overproduction of the normal CFTR protein by 
AdcvCFTR.lO may compensate for your poorly functioning CFTR, and may be of 
benefit to you. However, until these studies are carried out in humans, we 
cannot totally exclude the potential for adverse side effects of the overpro- 
duction of the normal CFTR protein. 
The AdcvCFTR.lO virus has been designed so that it will not reproduce itself 
in cells lining the airways. Studies in laboratory animals have demonstrated 
that AcIgvCFTR. 10 can be detected in the nose, back of the mouth, the lining of 
the lung or in the rectum for a number of days after administration and up to 
15 days in a few instances. It is possible that this will be true for humans 
as well. In the animal studies, control animals that did not receive 
AdcvCFTR.10, yet were in the same environment as the animals treated with 
AdsvCFTR.10, did not get "infected" with AdcvCFTR.10. With regards to the p"- 
Recombinant DNA Research, Volume 20 
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