A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)



Status:Archived
Conditions:Metabolic
Therapuetic Areas:Pharmacology / Toxicology
Healthy:No
Age Range:Any
Updated:7/1/2011
Start Date:August 2009
End Date:February 2012

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A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy


This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate
(Genz-112638) in patients with Gaucher disease type 1 who have reached therapeutic goals
with enzyme replacement therapy.


Gaucher disease is characterized by lysosomal accumulation of glucosylceramide due to
impaired glucosylceramide hydrolysis. Gaucher disease type 1, which is the most common form,
accounts for >90% of cases and does not involve the CNS. Typical manifestations of Gaucher
disease type 1 include splenomegaly, hepatomegaly, thrombocytopenia, anemia, bone disease,
and decreased quality of life. The disease manifestations are caused by the accumulation of
glucosylceramide (storage material) in macrophages (called Gaucher cells) which have
infiltrated the spleen and liver as well as other tissues.

Eliglustat tartrate (Genz-112638) is a small molecule drug developed as an oral therapy
which acts to specifically inhibit production of this storage material in Gaucher cells.

This study is designed to determine the efficacy, safety, and pharmacokinetics (PK) of
eliglustat tartrate (Genz-112638) in adult patients with Gaucher disease type 1 who have
been stabilized on Cerezyme.


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185 Cambridge Street
Boston, Massachusetts 02114
617-724-5200
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Boston, MA
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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9300 Valley Children's Pl
Madera, California 93720
(559) 353-3000
Children's Hospital Central California The Children's Hospital Central California is a not-for-profit, state-of-the-art children’s hospital...
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300 Community Drive
Manhasset, New York 11030
(516) 562-0100
North Shore University Hospital North Shore-LIJ Health System includes 16 award-winning hospitals and nearly 400...
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Manhasset, NY
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2450 Riverside Avenue
Minneapolis, Minnesota 55455
(612) 273-3000
University of Minnesota Medical Center Improving patients' lives drives the innovation that makes University of...
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Minneapolis, MN
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1 Gustave L Levy Pl # 271
New York, New York 10029
 (212) 241-6500
Mount Sinai Med Ctr Founded in 1852, The Mount Sinai Hospital is a 1,171-bed, tertiary-care...
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Beverly Hills, California 90211
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Boulder, Colorado 80309
(303) 492-1411
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Brooklyn, New York 11203
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2301 Erwin Rd
Durham, North Carolina 27710
919-684-8111
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Durham, NC
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333 Cedar St
New Haven, Connecticut 06504
(203) 432-4771
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550 1st Ave
New York, New York 10016
(212) 263-7300
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200 Lothrop St
Pittsburgh, Pennsylvania 15213
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Rolling Meadows, Illinois 60007
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San Francisco, California 94115
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Springfield, Virginia 22152
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