Gaucher Disease, Type 1 Clinical Trial
Official title:
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 (ENCORE)
This Phase 3 study is designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in participants with Gaucher disease type 1 who have reached therapeutic goals with enzyme replacement therapy (ERT).
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 greater than (>) 90% of cases and does not involve the central nervous system
(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 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 in adult participants with Gaucher disease type 1 who have been
stabilized on ERT.
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Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
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