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6.3 Replacement Strategy for Discontinued Patients 
Discontinued patients that have received gene-modified cells will not be replaced in this 
study but the discontinued patient will still continue to be followed as previously 
described in Section 4.4. 
7.0 CRITERIA FOR DISCONTINUATION/WITHDRAWAL 
7.1 Criteria for Treatment Discontinuation 
Participation in this study can be discontinued for any of the following reasons listed 
below: 
o At the Patient’s request: Any patient who wishes to withdraw from the study may 
do so but will be counselled that long-term follow-up for the effects of gene 
therapy are strongly desired by the medical facility and federal regulatory groups. 
o A major, unexpected, or life-threatening toxicity requiring that treatment be 
discontinued 
o Generalized impairment or mental incompetence which would relinquish the 
patient unable to understand his/her participation in the study. 
o If, in the Investigator’s medical judgment, further participation would be injurious 
to the subject’s health or well-being. 
o Administrative reasons, such as subject non-compliance or a major protocol 
violation. 
o Pregnancy 
o In addition, if at any time during the study a patient is observed to have a level 
of glucocerebrosidase enzyme equivalent to that of a heterozygote (carrier) 
individual, the patient will not receive additional cycles of therapy and will be 
followed to determine the extent and duration of the observed enzyme levels. 
An explanation will be recorded for any patient that is taken off treatment. 
7.2 Criteria for Study Discontinuation 
Patients should understand that once they have completed at least one cycle of therapy, 
they will be asked to be followed long-term. They can stop study treatment at any time 
(see section 7.1), however they will still be considered on study until the study has been 
terminated. 
The study can be discontinued only with the understanding of the investigators, sponsor, 
RAC and the FDA. 
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