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11.0 DATA COLLECTION AND STUDY MONITORING 
11.1 Data Collection 
Case Report Forms (CRFs) will be available for each patient enrolled in the study. 
Study participants must NOT be identified by name on any study documents. Patients 
will be identified by a patient identification number (PIN). 
11.2 Study Monitoring 
It is understood that the GTI-designated person responsible for study monitoring of 
patient data will be permitted to contact and visit the investigator at regular intervals and 
will be allowed, on request, to review study documents such as consent forms, drug 
distribution forms, case report forms, and pertinent hospital or clinic records for 
confirmation of the study data provided that patient confidentiality is maintained in 
accord with local requirements. It will be the monitor’s responsibility to verify the 
adherence to the protocol and the completeness, consistency, and accuracy of the data 
being entered on the CRFs. If local regulations permit, the study monitor should have 
access to original laboratory test reports and other patient documents (source 
documentation) needed to verify data entered on the CRFs. The investigator will 
cooperate with the assigned study monitor to ensure that all problems are resolved. 
12.0 BIOSTATISTIC AL CONSIDERATIONS 
Summaries for the following parameters will be compiled at six months and one year: 
► Percentage of cells containing the GC vector (extent of engraftment) 
► Mean glucocerebrosidase levels in study participants 
► Safety 
13.0 ETHICAL CONSIDERATIONS 
The investigator will ensure that the study is conducted in full conformance with the FDA 
standards for human research. 
13.1 Informed Consent 
All study participants must sign an informed consent. The investigators will inform all 
subjects as to the nature, aims, duration, potential hazards, and procedures to be 
performed during the study and that his or her medical records may be reviewed by the 
sponsors or the FDA. This protocol must receive approval by the Institutional Review 
Board of all participating sites prior to implementation. The investigator must also 
explain that the patients are completely free to refuse to enter the study or to withdraw 
from it at any time. The protocol will be discussed in detail with all potentially eligible 
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Recombinant DNA Research, Volume 17 
