Responses to Initial Review 
Appendix B 
RAC Application 
Kenneth L. Brigham, M.D. 
PRINCIPAL INVESTIGATOR: Kenneth L. Brigham, M.D, 
TITLE OF PROPOSAL: Expression of an Exogenously Delivered Human Aloha- 1 Antitrypsin Gene in Nasal 
Epithelium ; 
ITEM #3: The discomforts, inconveniences and/or risks that can reasonably be expected are: ^ 
! 
The procedure used to put the alpha 1 -antitrypsin gene into your nose may cause a temporary sense of , 
irritation in your nose and it may cause you to sneeze. Some persons experience increase tearing from their eyes ' 
for a brief period; this is also temporary. Although unlikely, you may develop sinusitis following administration 
of the material into your nose. If this should happen, routine medical care for sinusitis will be administered. We | 
know of no side effects of putting DNA or liposomes into your nose. It is possible that some of the DNA material ! 
will be absorbed and go to some other organ like the liver. Should this occur, we have no reason to believe that i 
it would be harmful. Given the nature of alpha 1 -antitrypsin deficiency, temporarily stopping your alpha 1- i 
antitrypsin replacement therapy or your nasal sprays should in no way affect your long term medical care or course. 
Pregnant women are excluded from this study. If you are a woman of reproductive age, a pregnancy test 
will be performed to document that you are not pregnant. If you are not pregnant and choose to participate in this 
study, contraception is required for one month prior to the study and for two months following the study. This i 
is strictly a safety precaution. Studies in animals given DNA/liposome mixtures have not detected any of the i 
delivered DNA in reproductive organs. | 
I 
NOTE: Immediate necessary care will be provided .without charge by Vanderbilt University If you are injured f 
because of participation in this research project. However, there is no provision for the costs of further medical > 
care or for the monetary compensation for such injury.'* j 
ITEM #4: The possible benefits to you from this study are: i 
I 
None ! 
ITEM #5: The benefits to science and to mankind that are sought by this study are: | 
The possibility of gene therapy is an entirely new area which may be beneficial in a large number of human 
diseases. This study is a first step in that direction and, if successful, will pave the way for application of this j 
method in patients with lung diseases like emphysema. I 
i 
ITEM #6:(a) Alternate procedures that might be advantageous; but which will not be used are: ■. 
There are no alternate procedures. I 
(b) The reasons for not using them are: 
N/A 
ITEM #7: Your rights of privacy will be maintained in the following manner: 
(1) AII data obtained on you during the course of this study will be kept confidential and accessible only to the ' 
principal investigator and his/her assistants on this project; except as stated elsewhere. 
I 
(2) Should the results of this project be published, you will be referred to only by number. 
NOTE: YOU ARE FREE TO WITHDRAW THIS CONSENT AND TO DISCONTINUE PARTICIPATION IN THIS 
STUDY OR ACTIVITY AT ANY TIME. 
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Recombinant DNA Research, Volume 19 
