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 ^ of pages. 
the infusion of their own T-lymphocytes. If your child develops any side effects, he/she will be treated 
with medications to control these symptoms. The medications commonly used include: aspirin, Tylenol, 
antihistamines (such as Benadryl) or other pain relievers. If the symptoms persist or worsen despite 
medication, the infusion will be slowed or stopped until the symptoms have stopped. Part 1 will last 
approximately 6 months, or 6 infusions. 
In part 2, we will treat the gene-corrected T-lymphocytes in the laboratory to increase the amount of 
ADA they contain and repeat the monthly infusions as in part 1 . After 6 treatments with these high ADA 
producing cells, we will increase the number of cells to be given monthly to see if providing more 
ADA-corrected cells will be a more effective treatment for your child. The infusion will remain at 
approximately monthly intervals but the timing of infusions may vary depending upon the number of 
cells available. Infusions of ADA-corrected T-lymphocytes in part 2 will continue indefinitely as long 
as there is evidence of improved immunologic functioning. The interval between infusions may be 
lengthened depending on how your child responds to the treatment. 
During part 1 and 2, your child will be vaccinated with several different vaccines including tetanus, 
diphtheria, H. Influenzae B, and S. pneumonae to allow us to assess the effects of the ADA-corrected T- 
lymphocytes on your child’s Immune functioning. The most common side effects related to these 
vaccines are tenderness and swelling around the site of the injection. These symptoms are usually brief 
and easily controlled with Tyienol and ice applied locally. 
Your child will be evaluated for HIV infection (the cause of AIDS). Permission to do this testing requires 
your signing of an additional informed consent form. If found to be HIV positive, you will be told and 
your child will not be eligible for this study. 
Safety Issues 
The procedure carries several possible risks. Even though the vector used to transfer the ADA gene into 
the cells cannot grow and is considered harmless to humans, it is possible that events could occur 
within the cell that would permit the vector to grow and/or make the cells cancerous. In experiments 
with many mice and monkeys, we have seen no evidence of an altered vector or the development of 
cancerous cells after following the animals closely for more than four years since treatment. We began 
experiments in human patients in May, 1989, using a similar but not identical vector to insert a gene 
called NeoR into T-lymphocytes. NeoR is a bacterial g'^ne that was used as a cell marker in these adult 
patients with cancer. To date we have seen no side effects of any kind caused by the retroviral-mediated 
transfer of the gene in any of these patients. 
You should know that the vector we will insert into your child’s cells also contains the bacterial gene 
NeoR. The NeoR gene produces a protein that can inactivate certain antibiotics. These antibiotics are 
not commonly used to treat infections in people with ADA deficiency. There are many other antibiotics 
available that will not be inactivated by this protein and would be effective if your child develops a 
PATIENT IDENTIFICATION 
CONTINUATION SHEET for either: 
NIH-2514-1 (10-84) 
NIH-2514.2 (11^84) 
p.A : 09-25-0099 
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Recombinant DNA Research, Volume 14 
