GTI0101 (04/08/93) 
Page 2 of 35 
TABLE OF CONTENTS 
PAGE 
PROTOCOL ROSTER 4 
PROTOCOL SYNOPSIS 5 
SCHEMATIC 6, 7 
ABSTRACT 8 
1.0 BACKGROUND AND RATIONALE 9 
1.1 Background: Gaucher Disease 9 
1.2 Background: Treatment for Gaucher Disease 9 
1.3 Preclinical 11 
1.4 Clinical 12 
1.5 Rationale/Justification 13 
1.6 Objectives 14 
2.0 STUDY DESIGN 15 
2.1 Overview 15 
2.2 Number of Patients/Patient Management 15 
2.3 Source of Patients/Participating Sites 15 
2.4 Study Duration 15 
3.0 PATIENT SELECTION 16 
3.1 Inclusion Criteria 16 
3.2 Exclusion Criteria 18 
3.3 Concomitant Treatment/Procedures 18 
4.0 CLINICAL AND LABORATORY EVALUATIONS 18 
4.1 Screening and Baseline Evaluations 18 
4.2 Evaluations During Treatment 19 
4.3 Evaluations Between PBSC cycles 21 
4.4 Post-Treatment Evaluations 22 
4.5 Considerations/Evaluations Upon Premature Withdrawal 22 
Appendix IA AND IB are the Evaluation Tables 
5.0 STUDY TREATMENT 23 
5.1 Justification of Dosages 23 
5.2 Formulation, Packaging, and Storage 23 
5.3 Treatment Procedures 24 
5.4 Hazards and Discomforts 26 
Appendix V and VI contain additional treatment information 
6.0 TREATMENT MODIFICATION AND DISCONTINUATION 
ACCORDING TO LEVEL OF TOXICITY 27 
6. 1 Management of Toxicities (General) 27 
6.11 Management of Specific Toxicities 27 
6.2 Symptomatic Therapy for Toxicity 27 
6.3 Replacement Strategy for Discontinued Patients 28 
Appendix II is the Toxicity Grading Table 
Recombinant DNA Research, Volume 17 [787] 
