CONTINUATION SHEET for either: 
NIH 2514-1, Consent to Participate In A Clinical Research Study 
NIH 2514-2, Minor Patient’s Assent to Participate In A Clinical Research Study 
STUDY NUMBER: 
CONTINUATION: page Jt_of 21 _ pages. 
glycerol (a basic component of many natural fats in the body) . It ensures 
that the virus remains functional. The actual volumes of material to be 
administrated are 0.2 milliliters to the nose (about 4 drops) and 20 
milliliters to the airways (about 4 teaspoons). These volumes will not be 
uncomfortable for the patient. 
The protocol is designed to answer a number of important questions regarding 
gene therapy for CF. 
(1) Is it safe to administer the modified adenovirus, AdCFTR, to the airways 
of individuals with CF? 
(2) Will transfer of the normal CFTR gene to respiratory epithelial cells 
of individuals with CF enable these cells to produce a normal, 
functional CFTR protein? Ultimately, the question is: will this 
strategy normalize that part of the lung to which the normal gene has 
been transferred? 
(3) How long will this effect last? 
(4) Is biologic correction (production of a normal CFTR protein) sufficient 
to correct the abnormal electrical potential difference of the airway 
surface that occurs because of the underlying abnormalities in CF? 
(5) Is there improvement in respiratory clinical parameters (lung 
measurements and functions) relevant to the disease process? For 
example, does lung function improve? Is sputum production decreased? Is 
the amount of infection decreased? 
(6) Does the body develop immunity against AdCFTR that will prevent 
subsequent administrations, i.e., will the body react against the 
modified virus to prevent it from transferring the normal gene to 
the lung should repeated administrations be necessary? 
Inclusion and Exclusion Criteria: 
Based on the natural course of cystic fibrosis, the need to maximize safety 
while at the same time performing various tests to evaluate the effectiveness 
of the treatment, the following inclusion and exclusion criteria 
(requirements) for the study population have been defined. These criteria 
will be determined, to the extent possible, before entry into the protocol; 
some however will not be measurable until the baseline evaluation part of the 
protocol (see section 5.6 in the protocol). It is possible, therefore, that 
although started, at some point you will not be able to continue in the 
protocol if you do not meet these criteria. Likewise, there are possibilities 
as part of the natural history of your disease (i.e., unrelated to the 
PATIENT IDENTIFICATION 
CONTINUATION SHEET for either: 
Recombinant DNA Research, Volume 16 
i 
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