Responses to Initial Review RAC Application Kenneth L. Brigham, M.D. 
Appendix B 
CONSENT FOR RESEARCH STUDY 
ADULT: (Patient) X CHILDREN: (Patient) PARENTS 
(Volunteer) (Volunteer) GUARDIANS 
PRINCIPAL INVESTIGATOR: Kenneth L. Brigham. M.D. 
TITLE OF PROPOSAL: Expression of an Exoeenouslv Delivered Human Alpha- 1 Antitrypsin Gene in the Lungs 
NAME OF RESEARCH VOLUNTEER: AGE: 
TO PERSONS WHO AGREE TO PARTICIPATE IN THIS STUDY: 
The following information is provided to inform you about the research project and your participation in it. 
Please read this form carefully. Any questions you may have about this study will be answered. Please feel free 
to ask any questions you may have about this study and/or about the information given below. 
Written form 
Oral form 
ITEM #1: Indicated below are the following: i 
(a) The purpose of this study 1 
(b) The procedures to be followed j 
(c) The approximate duration of this study < 
We want to see whether we can put a gene into the lungs in a way that the gene will produce a protein that | 
protects the lungs from injury. This has never been done in humans although we have shown that it works and is | 
safe in animals. We have used several different animals to put a gene into the lungs. We have used mice, rats, i 
rabbits, and sheep. In all the animals we have studied, no side effects or complications occurred due to putting the ; 
gene into them. The gene (or DNA) that we are going to put into your lung is called the alpha 1 -antitrypsin gene, j 
This gene normally exists in humans and helps protect the lungs from damage. In order for us to put this gene into | 
your lungs, small pieces of DNA from viruses and bacteria are included. These additional pieces of DNA are just | 
bits of DNA. They cannot cause a viral infection or a bacterial infection. There is no risk of you developing i 
cancer from this DNA. 
To get the gene material into the lung cells, we mix it with a solution of very small fat particles called | 
liposomes. Various types of liposomes have been used to deliver drugs to humans. The kind of liposome that we i 
are using have been used before to deliver DNA to individuals and no side effects or complications were seen. 
W'e chose to ask you to participate in this study because you are going to have one of your lungs removed 
as part of your medical care. Twenty four to forty eight hours before your surgeons are to remove that lung, we | 
want to do a brief bronchoscopy; a small tube will be passed through your nose and into a small part of the lung | 
to be removed. Bronchoscopy is a routine medical procedure done daily at Vanderbilt and other medical j 
institutions. The bronchoscope is a long flexible tube with a light source at one end and an eyepiece at the other ] 
end. The diameter of the tube is approximately the diameter of a normal sized writing pen. Bronchoscopy is simple 
and safe. First, your nose will be made numb as will the back of your throat with a type of medicine similar to i 
the numbing medicine used by dentists. The bronchoscope will be passed through your nose into the back of your 
throat. When the bronchoscope is advanced to your voice box, more numbing medicine will be put through the j 
bronchoscope to numb your larynx. Once your voice box is numb, the bronchoscope will be advanced through the 
voice box into your lung. Once the bronchoscope is in your lung, it will be placed securely into a area of your lung | 
that does not contain the cancer but within a part of the lung that the surgeon plans to remove. After the ’ 
bronchoscope is secure in place, sterile water will be rinsed into the lung and collected to be examined for various 
laboratory tests. 
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Recombinant DNA Research, Volume 19 
