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



Status:Completed
Conditions:Metabolic
Therapuetic Areas:Pharmacology / Toxicology
Healthy:No
Age Range:18 - Any
Updated:4/21/2016
Start Date:June 2010
End Date:September 2015

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A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Once Daily Versus Twice Daily Dosing of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638

The primary objective of this study is to evaluate the efficacy and safety of once daily
(QD) versus twice daily (BID) dosing of eliglustat tartrate (Genz-112638) in patients with
Gaucher disease type 1 who have demonstrated clinical stability on BID dosing of eliglustat
tartrate (Genz-112638). The secondary objective is to evaluate the pharmacokinetics (PK) of
Genz-99067 when eliglustat tartrate (Genz-112638) is administered QD and BID in patients
with Gaucher disease type 1 who have demonstrated clinical stability on BID dosing of
eliglustat tartrate (Genz-112638).

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.
We found this trial at
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201 Presidents Circle
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