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Partial Response - More than 0.1% but less than 2% of the circulating mononuclear cells 
contain the retroviral vector. An RNA signal is detected from the retroviral vector at 
any time during the study. • 
Complete Response - More than 2% of the circulating mononuclear cells contain the 
retroviral vector. An RNA signal is detected from the retroviral vector at any time 
during the study. 
Depending upon the results obtained from this study, alternative vectors and/or treatment 
schema will be used in future studies to try to increase the extent of glucocerebrosidase 
expression in Gaucher disease patients. 
Toxicity measurements will be analyzed in conjunction with the clinical stage of disease, 
and other study parameters. 
9.2 Study Endpoints 
• Development of high grade toxicities or intolerances. 
• Expression of GC enzyme levels equivalent to normal patients 
10.0 DOCUMENTATION, RECORD KEEPING, CRFs 
The investigator must maintain adequate records so that the conduct of the study can be fully 
documented and monitored. 
Copies of protocols, case report forms (CRFs), test result originals, all product accountability 
records, correspondence, patient informed consent, and any other documents relevant to the 
conduct of the study must be kept on file by the investigator for (five) years after all 
investigational use of product is discontinued and the FDA is so notified or until five years after 
a Product Licensing Application (PLA/ELA) is approved. Study documents should not be 
destroyed without prior written agreement between GTI and the study investigator. The Study 
Monitor should be notified if the investigator wishes to assign the study records to another party, 
or move them to another location. 
The investigator will supply the sponsor (GTI) on request with any required background data 
from all patient records and related documents. This may become necessary if the CRFs are 
illegible or when errors in data transcription are questionable. For FDA inspections, it is also 
necessary to have access to complete study patient records, provided that patient confidentially 
is maintained. 
The investigator should obtain a separate release of medical information form to be signed by 
the study patient in order to facilitate access to the patient’s medical records should the patient 
be hospitalized at an institution with which the study investigator is not associated. 
A record must be kept of all patients considered (and screened) for the study and subsequently 
deemed ineligible. The reason for ineligibility must be recorded. 
Recombinant DNA Research, Volume 17 
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