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8.0 ADVERSE EXPERIENCES 
Appendix III is Adverse Experience Reporting (AERs) Guidelines 
Depending on the extent of the adverse experience, adverse events should be reported by each 
site investigator to their local Institutional Review Board (IRB), Institutional BioSafety 
Committee (IBC) and to Genetic Therapy, Inc. (sponsoring company) as specified in Appendix 
III. 
Adverse reactions or deaths requiring immediate reporting should be made by telephone to (301) 
590 2626 (available 24 hours a day, recorder after working hours). 
The sponsor, Genetic Therapy, Inc. will take the responsibility of informing and preparing 
reports for the Recombinant Advisory Committee (RAC) through the NIH Office of 
Recombinant DNA Activities (ORDA) and to the FDA. 
HHS Guidelines for Research Involving Recombinant DNA Molecules : It is the responsibility 
of the institution and those associated with it to adhere to the intent of the Guidelines as well as 
to their specifics. 
9.0 EVALUATION OF RESPONSE AND STUDY ENDPOINTS 
9.1 Response Criteria 
The study is designed primarily to determine if gene-altered marrow cells can be 
harvested and readministered to a patient without toxicity. Efficacy is measured in the 
percentage of various hematopoietic cell populations which contain the transferred gene 
and the extent to which the transferred gene is responsible for enzyme expression. The 
primary time point to be used for these determinations is six months after the last gene 
transfer treatment, although all time points will also be evaluated. 
No Response - Less than 0.1% of the circulating mononuclear cells contain the retroviral 
vector. No RNA signal from the retroviral vector is detected at any time during the 
study. 
Incomplete Response 
A. Less than 0.1% of the circulating mononuclear cells contain the retroviral vector. 
An RNA signal is detected from the retroviral vector at any time during the 
study. 
B. More than 0. 1 % of the circulating mononuclear cells contain the retroviral vector. 
No RNA signal is detected from the retroviral vector at any time during the 
study. 
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