Revised 8-26-94 
the study subject. 
Finally, of the individuals receiving AdcvCFTR.lO to date, one (receiving a 
dose [total titer] of 2xl0 7 to the nose and, 1 day later, 2xl0 9 to the lung), 
developed a transient (1 week), clinical syndrome of fevers, (low blood 
pressure), shadows on the chest x-ray in the area the AdcvCFTR.10 was admin- 
istered, and decreased lung function. This clinical syndrome subsided with 
therapy (intravenous fluids, antibiotics and oxygen) and all clinical parame- 
ters were at the baseline values by the scheduled time of discharge from the 
hospital. Your doctors are not sure of the mechanisms of this clinical syn- 
drome but, until proven otherwise, it is being assumed that the dose admin- 
istered to the lung (2xl0 9 ) may represent the highest dose that can be given 
to individuals with CF. To minimize the chance that this syndrome will occur 
again, the total volume of fluid administered has been markedly decreased, to 
reduce the chance that it will reach the lower part of the lung, the site 
where this syndrome was primarily manifest. 
Potential Benefits: 
There is a possibility that administration of AdcvCFTR.10 to the airways will 
result in expression of normal CFTR protein in these areas with normalization 
of Cl' secretion, that is, a correction of the major biochemical abnormalities 
associated with CF. If these changes are extensive enough, they may result in 
normalization of the electrical charge on the airway surfaces. Will these bio- 
chemical changes result in any noticeable clinical change for you? This is 
very unlikely. Clinical improvement in the lung would likely depend on the 
percentage of corrected cells. As this protocol involves administration of 
AdcvCFTR.10 to a limited region of the airways, large scale correction will 
likely not occur. Furthermore, the correction may not last. Nevertheless, if 
biologic correction is found, it will provide motivation for further studies 
most likely with higher dosages and/or repetitive administrations of 
AdsvCFTR.10. Participation in this study does not mean automatic inclusion in 
subsequent studies. 
Time spent in the Hospital : 
You will be asked to spend a considerable amount of time in the hospital. If 
you feel unable to continue the protocol at any time during the protocol you 
are free to leave. Departure from the protocol after administration of 
AdcvCFTR.10 but before the "out-patient" part of the protocol is discouraged. 
As discussed above this is because there are a number of theoretical risks 
associated with administration of AdcvCFTR.10 not only to you but also to 
others. Because of this, you will be asked to give a commitment to fulfill all 
the planned assessments of the protocol if you wish to participate in part B 
or C of the protocol (the experimental treatment parts) . Followup to this 
protocol will be life-long; if you move, you should provide the investigators 
of this protocol your new address. 
Access to Records 
As discussed above, your privacy will be protected. Access to your medical 
records will be limited to hospital personnel of the Rockefeller University 
Hospital and The New York Hospital, The National Institutes of Health, The 
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