A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

NOTE: Other Phase 3 studies being conducted with eliglustat tartrate (Genz-112638) are
GZGD02507 (ENGAGE): NCT00891202 and GZGD02607 (ENCORE): NCT00943111

Inclusion Criteria:

- The patient is willing and able to provide signed informed consent prior to any
study-related procedures.

- The patient has a diagnosis of Gaucher disease type 1 confirmed by a documented
deficiency of acid β-glucosidase activity by enzyme assay.

- Female patients of childbearing potential must have a documented negative pregnancy
test prior to administration of the first dose of eliglustat tartrate (Genz-112638)
in this study. In addition, all female patients of childbearing potential must use a
medically accepted form of contraception throughout the study, i.e., either a barrier
method or hormonal contraceptive with norethindrone and ethinyl estradiol or similar
active components.

Exclusion Criteria:

- The patient has had a partial or total splenectomy within 3 years prior to
randomization.

- The patient received pharmacological chaperones or miglustat within 6 months prior to
administration of the first dose of eliglustat tartrate (Genz-112638) in this study.

- The patient has any clinically significant disease, other than Gaucher disease,
including cardiovascular, renal, hepatic, gastrointestinal, pulmonary, neurologic,
endocrine, metabolic (including hypokalaemia or hypomagnesemia), or psychiatric
disease, other medical conditions, or serious intercurrent illnesses that, in the
opinion of the Investigator, may preclude participation in the study.

- The patient is known to have tested positive for the human immunodeficiency virus
(HIV) antibody, Hepatitis C antibody, or Hepatitis B surface antigen.

- The patient has received an investigational product (other than eliglustat tartrate
(Genz-112638)) within 30 days prior to administration of the first dose of eliglustat
tartrate (Genz-112638) in this study.

- The patient is pregnant or lactating.