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. 
This Gene Therapy Experimental Study 
Participation in this study will involve a committment for monthly treatments over a period of up to 2 
years. His/her present treatment with PEG-ADA will be continued unchanged during this time. 
Initially, each gene therapy treatment will be performed at the Clinical Center at the NIH. Once we 
better understand how your child responds to these treatments, we hope to be ^ble to arrange for your 
child’s physician to administer the infusions closer to your home. Initially each treatment will require 
a 2-3 day admission to the Clinical Center. Again, with the experience gained With the first infusions 
we hope to be able to carry out this treatment almost entirely on an outpatient basis. In order to 
evaluate the effectiveness of the ADA-corrected cells on improving your child’s immune functioning, we 
will need to obtain blood from a vein approximately one to two times each month. .We also plan to 
vaccinate your child and perform skin testing once or twice each year to further evaluate your childs 
immune system. If the immunologic testing reveals improvement, we will offer to continue the 
infusions indefinitely. The time between the infusions will be lengthened if there is no decrease in your 
child’s immune functioning with less frequent T-cell infusions. We hope that all patients who 
participate in this study will be willing to be followed for the rest of their lives to permit us to 
evaluate the long term effects of ADA gene transfer. 
\ 
If you decide to participate in this study, blood will be drawn from your child approximately every 4 
weeks. Through a procedure called lymphapheresis the white blood cells are separated from the red 
blood cells so that the red blood cells can be given back to your child through a vein the same day to 
minimize the amount of blood lost. Permission to do the apheresis requires that you sign an additional 
consent form. The maximum amount of blood drawn in this procedure to be used to grow T-lymphocytes 
will be 7 cc/kg of body weight/lymphaphoresis. T-lymphocytes will be grown from the blood white 
cells in the laboratory using interleukin-2 (IL-2). The cells may also be treated with 2 other 
experimental drugs to enrich for ADA-corrected cells. The 2 drugs and the IL-2 will be washed away 
prior to the infusion of the cells into your child. A normal human ADA gene will be transferred into the 
cells using retroviral-mediated gene transfer. The ADA-corrected cells will be grown in the 
laboratory until sufficient numbers of cells have grown and then the ADA-corrected cells will be 
infused intravenously into your child. We will then obtain blood samples to monitor the survival of the 
ADA-corrected cells and the effects of the ADA-corrected cells on your child’s immune system. The 
number and amount of blood tests will depend upon the age, weight and health of your child. We do not 
expect blood drawing to be greater than 2 times each month after the initial two months of treatment 
have passed. 
This study is divided into 2 parts. In part I, we will remove blood monthly and reinfuse cells monthly. 
The initial monthly infusions will consist of small numbers of ADA-corrected cells to minimize the 
possibility of developing side effects. Initially, the infusion of cells may be carried out in the pediatric 
intensive care unit at NIH to allow optimal monitoring of your child during the cell infusion. Each 
infusion will last from 10 minutes to 2 hours. Potential side effects might include chills, fever, nausea 
and/or body aches. Patients will occasionally develop temporary joint aches and a skin rash following 
PATIENT IDENTIFICATION 
CONTINUATION SHEET for either: 
NIH-2514-1 (10-84) 
NIH-2514-2 (1(^84) 
P.A ; 09-25-0099 
Recombinant DNA Research, Volume 14 
