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 
STUDY NUMBER: CONTINUATION: page JL of JL pages. 
-6 and SCF during vector exposure in the laboratory. These are 
necessary to keep the stem cells alive and to increase the chance 
that the cells will take up the vector. These growth factors are 
experimental drugs, but they have been given to patients without 
serious side effects. Your cells will be washed thoroughly before 
giving them back to you, and we have not been able to detect any 
growth factors remaining in the solution containing the stem cells 
that will be reinfused. 
Stem Cell Reinfusion: After 3-4 days in culture with vector and 
growth factors, the cells will be collected, washed, and given back 
to you intravenously. You will receive three, daily infusions of 
cells on days 8, 9 and 10 after beginning G-CSF. The infusions 
will take 10-30 minutes, and you will be monitored closely for side 
effects during and after the infusions. We do not expect any side 
effects, but an allergic reaction is theoretically possible, and 
you would be treated for it occurred. It is also possible that the 
cells could become infected with bacteria or fungus during the 3 
day growth period in the laboratory. If there is any evidence for 
infection of the cells with unwanted agents, the cells will not be 
returned to you, but there is a remote chance that you could 
acquire a bacterial or fungal infection from the reinfused cells. 
After completion of the third infusion, you will be able to return 
home. You will return to NIH every 1-4 months for follow-up and to 
repeat the whole stem cell collection and gene transfer procedure 
for a total of four times. 
Follow-up: You will be followed as an outpatient approximately 
every month for at least one year. We will perform a number of 
blood tests to determine whether the gene is present in your blood 
cells, and whether it is working to produce the normal GO enzyme. 
We will also perform tests to make sure that the gene transfer 
procedure has been safe. The amount of blood drawn will be 
approximately two-four tablespoons every month. We may also 
request permission to perform a bone marrow aspiration tests up to 
six times over a two year period. X-rays or scans may be performed 
as necessary for the routine clinical care of your Gaucher disease, 
and to assess whether the gene transfer procedure has had any 
effect on your clinical condition. 
Since this study involves a very new approach to patients with 
Gaucher disease, it will be important to maintain contact with you 
for the rest of your life, so that we can monitor your overall 
progress and monitor for any possible long term side effects. If 
CONTINUATION SHEET for either: 
NIH-2514-1 (10-84) 
NIH ^ecoin$iKant DNA Research, Volume 0 !^ 0099 
PATIENT IDENTIFICATION 
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