Form C (Revised 6/93) 
CLINICAL INVESTIGATION CONSENT FORM 
The Johns Hopkins Medical Institutions 
(The Johns Hopkins Hospital 
The Francis Scott Key Medical Center, etc.) 
Title of Research Project: 
Phase I study of Administration of 
Adeno-associated Virus-CFTR Vector 
to Patients with Cystic Fibrosis 
Informed Consenl 
Patient I.D. Plate 
Explanation of Research Project to Subject: 
PURPOSE OF THE STUDY : You have a disease, cystic fibrosis (CF) , that is 
caused by a defect in a gene called CFTR. Genes are blueprints made of DNA 
that are present in each cell of your body. These blueprints are instructions 
that tell the cell how to make proteins, which carry out the work of each 
cell. In the case of CF, the CFTR gene has the wrong instructions and tells 
the lung cells to make a CFTR protein that does not work properly. We are 
doing a research study to find out if we can replace this defective gene with 
one that has the right instructions. The purpose of the study is to place an 
experimental gene into a CF patient's nose and lung to find out if there are 
any problems when the working gene is given to patients. The gene is put into 
the nose and lung using a virus called AAV. We wish to find out if giving the 
gene in this virus will cause the nose and lung cells to make CFTR protein. We 
do not think vour health will improve if vou join this study. This is the 
first time that the CFTR gene will be given to people using the AAV virus. You 
are being asked to join this gene transfer research study because you have CF . 
PROCEDURES : If you agree to join this study, the following procedures will be 
done. First, you will have a complete medical examination. This will include 
tests of your blood, urine, sputum, and your lung function. These are tests 
that are similar to those you have had in the past in the Cystic Fibrosis 
Clinic. If the results of the tests show that you are in good health and that 
your lungs are working reasonably well, you may continue with the study. 
l 
• 
The gene transfer part of the study will last about 18 days. During that time 
you will be admitted to the hospital and stay at the Pediatric Clinical 
Research Unit. When you are admitted to the hospital, blood will be drawn 
from a vein for laboratory studies. We will take about 2 tablespoons of blood. 
You will have a chest X-ray, a chest CT scan, and lung function tests 
performed. 
After the tests are done, you will then be taken to a special procedures room 
and put to sleep. This will be done by giving you drugs in a vein along with a 
drug to produce numbness in your throat. This will allow us to examine your 
nose and lungs. We will take samples of nasal mucous and will use a small 
brush to scrape cells from the lining of your nose for tests. We will measure 
the voltage difference across the lining of your nose. This is done by placing 
a small needle under the skin of your forearm and a small flexible plastic 
tube filled with salt water against the lining of the nose. After taking 
measurements, a few drops of a drug called amiloride will be dripped onto the 
lining of the nose and the test will be repeated. We have done this voltage 
measurements on many CF patients. Finally, we will place several drops of a 
salt solution in your nose. This will be done to help us compare what happens 
when we give salt solution and what happens when we give the experimental 
gene . 
After we examine your nose, we will slide a very thin flexible lighted tube, 
called a bronchoscope, through your mouth and throat. The tube will be put 
down into your right lung. We will use it to collect samples of your lung 
cells and lung fluids for studies. We will measure the voltage across the 
Recombinant DNA Research, Volume 20 
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