MEDICAL RECORD 
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 
1 STUDY NUMBER: 
CONTINUATION: pageJJ. 0 f 2I_ pages. 
studies using AdCFTR in animals at dosages similar to those intended for use 
in humans . 
While it is theoretically possible that the material produced by the CFTR gene 
in AdCFTR may be recognized as "foreign" by the immune system of the 
participant and result in an autoimmune type syndrome (the immune system 
attacking the body) there is no evidence to suggest that this will happen and 
participants have been chosen to include only those who do produce a CFTR 
protein (even though it is an abnormal one) so that the normal protein is less 
likely to be entirely "foreign" to the study subject. 
Potential Benefits: 
There is a possibility that administration of AdCFTR to the nose and airways 
will result in expression of normal CFTR protein in these areas with normal- 
ization 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 nasal and airway 
surfaces. Will these biochemical changes result in any noticeable clinical 
change for the study participant? This is possible but is by no means certain. 
Administration of AdCFTR to the nose is highly unlikely to result in any 
improvement in respiratory symptoms. Clinical improvement in the lung would 
likely depend on the percentage of corrected cells. As this protocol involves 
just one single dose of AdCFTR to the airways, large scale correction will 
likely not occur. Furthermore, as just a single dose is given to the nose and 
airways the correction may not last. Nevertheless, if biochemical correction 
is found, it will provide motivation for further studies most likely with 
higher dosages and/or repetitive administrations of AdCFTR. Participation in 
this study does not mean automatic inclusion in subsequent studies 
Time spent at the Clinical Center : 
All study participants will be asked to spend approximately 4 months at the 
NIH Clinical Center. If a study candidate feels unable to continue the proto- 
col at any time during parts 1 and 2 they are free to leave. Departure from 
the protocol after the administration of AdCFTR but before the "out-patient" 
criteria of the protocol are met is discouraged, as discussed above. This is 
because, as listed above, there are a number of theoretical risks associated 
with administration of AdCFTR not only to the individual receiving the treat- 
ment but also to others. Because of this, every individual who wishes to 
participate in part 3 of the protocol (the experimental treatment part) will 
be asked to give a commitment to fulfil all the "out-patient" criteria of the 
protocol prior to leaving the National Institutes of Health. 
PATIENT IDENTIFICATION 
CONTINUATION SHEET for either: 
Recombinant DNA Research, Volume 16 
[789] 
