The purpose of this study is to compare and evaluate the efficacy and safety of ISU302, an investigational product, and Cerezyme®, comparator, for Type 1 Gaucher Disease patients
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Masking
DOUBLE
change in splenic volume compared to baseline
Primary endpoints to demonstrate non-inferiority of ISU302 against Cerezyme® for change in splenic volume compared to baseline.
Time frame: one year
change in organ parameters and biochemical value compared to baseline
* Change in splenic volume compared to baseline for all subjects enrolled * Change in hemoglobin concentration * Change in platelet counts * Change in liver volume compared to baseline * Change in range variation of liver function test (ALT/AST) * Change in skeletal status improvement level * Change in bone mineral density * Change in biomarkers (acid phosphatase, angiotensin-converting enzyme (ACE) and chitotriosidase) * After the 1st administration, to evaluate pharmacokinetic profile by glucocerebrosidase activity assay
Time frame: one year
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