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3 . 3 Concomitant Treatment/Procedures : All allowed medications or treatments should be kept 
to a minimum and recorded. All questions regarding concomitant medications should be 
referred to the study chair or investigator. 
Use of Ceredase : Patients taking Ceredase prior to study entry will be permitted to 
participate in this trial. Patients will be allowed to continue Ceredase therapy if deemed 
clinically necessary. 
4.0 CLINICAL AND LABORATORY EVALUATIONS 
As patients may not receive significant therapeutic effects from the gene transfer, the monitoring 
will emphasize safety (if toxicity is found refer to section 6.0 and Appendices II and III). Study 
patients may also be on enzyme replacement therapy so the routine monitoring for this treatment 
may overlap with additional tests related to the gene transfer aspects. 
4. 1 Screening and Baseline Evaluations 
The following clinical and laboratory evaluations will be performed for all study 
participants within Study Days -30 to 0 unless otherwise noted. Screening evaluations, 
or evaluations that determine patient eligibility, are noted. These screening evaluations 
must be reviewed prior to initiation of study treatment. 
NOTE : Day 0 and 1 may be the same date or up to 24 hours apart, however Day 1 
begins with treatment initiation. Any baseline evaluations {Day 0) which are performed 
on the day treatment will begin (Day 1 of treatment) must be performed prior to 
treatment. 
4.11 Objective Signs and Symptoms : include vital signs (blood pressure, pulse, 
temperature and respirations). (Screening and repeat Day 0) 
4.12 History and Physical Examination : Complete history and comprehensive physical 
will be perfomed for screening. A targeted physical exam will done Day 0. 
4.13 Kamofskv Performance Status (see Appendix IV) (Screening) 
4.14 Chest X-Ray : Normal CXR within the last 30 days unless new symptoms suggest 
that this be repeated prior to dosing (Screening) 
4.15 Electrocardiogram (ECG): ECG must be normal within 30 days unless new 
symptoms suggest this be repeated prior to dosing (Screening) 
4.16 Urinalysis : Routine screening urinalysis including creatinine and creatinine 
clearance (Screening and repeat Day 0) 
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