CHILDREN'S ASSENT FORM 
Protocol: Treatment of Severe Combined Immunodeficiency Disease 
(SCID) Due to Adenosine Deaminase (ADA) Deficiency with 
Autologous Lymphocytes Transduced with a Human ADA Gene 
The NIH is a hospital where doctors and scientists study many 
diseases and try to learn about what causes them and what new 
treatments might help them. You have a disease called SCID. 
This disease makes it difficult for your body to fight 
infections, like pneumonia. The problem is that some of the blood 
cells of your body which help fight infection do not make a 
special protein that they need to function properly. 
Your doctor is trying to correct this problem by giving you 
injections of the medicine called PEG-ADA. You are doing fairly 
well on this therapy but your doctor thinks that maybe another 
type of therapy in combination with PEG-ADA might work better. 
This research study is like a scientific experiment to see if 
this new therapy is safe and whether it helps improve your body's 
ability to fight infections. You will come to the hospital about 
every month and stay overnight for about two days. Your parents 
will be able to stay with you while you are in the hospital. 
Many of the tests that the doctors want to do are the kind that 
you are used to having done. The doctors will take blood from 
your vein and analyze it about two times each month. They will 
also give you some vaccination shots in your arm or leg to see 
how well this therapy is working. This might hurt or bleed a 
little for a moment but that stops quickly. 
The new therapy that the doctors want to try is called "gene 
therapy." Some blood will be taken from your vein by a special 
procedure every month. This special procedure ("leukopheresis") 
requires that you lie on a bed and have a needle in each arm. 
You will be hooked up to a machine which will separate your blood 
into red cells and white cells. This procedure might make you 
feel a little uncomfortable since it requires that you lie still 
for about an hour or two and may make you a little dizzy. The 
red cells will be given back to you through the needle used to 
take out the blood. The white cells will be taken to the 
laboratory where doctors will try to make them grow and then add 
a gene that will allow your cells to make the special protein 
substance that is needed to help your body fight infection. Once 
these white cells are treated in the laboratory, they will be 
given back to you through a vein. During this time you will 
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