The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher Disease

Study Overview
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Brief Summary
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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

Official Title
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A Multi-national Randomized Double Blinded Phase III Study to Evaluate the Safety and Efficacy of ISU302(Imiglucerase for Injection) or Cerezyme in Patient With Type I Gaucher Disease

Conditions
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Gaucher Disease

Intervention / Treatment
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* Drug: Cerezyme®
* Drug: ISU302


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: The subjects have a diagnosis of Type I Gaucher Disease Subjects between 2 years old and 75 years old Subjects documented with glucocerebrosidase deficiency Subjects with splenomegaly (as indicated by CT volumetric analysis as 5 times over than the standard size (0.2% of total body weight in kilograms)) A hemoglobin concentration level:Male > 12 years of age <12.0 g/dL Female>12 years of age<11.0 g/dL Child > 2 years of age and <12 years of age <10.5 g/dL Platelet count of < 120,000 / ㎣ Treatment-naive to enzyme replacement therapy (ERT) or treatment- experienced subjects who have not received ERT in the 12 months before screening and antibody test result is negative Treatment naive to substrate reduction therapy (SRT) or treatment- experienced subjects who have not received SRT in the 12 months before screening Subjects or their spouses who provide consent to use one of following contraception methods, or women in menopause. (In this case, menopause is defined as a period after 12 months from the last menstruation) Condoms, sponge, foams, jellies, diaphragm, or intrauterine device (Spouse or patient who had vasectomy or tubal ligation/ hysterectomy) Total abstinence from sexual intercourse: Female patient using oral contraceptives must use other contraception method (barrier method) also during the trial period and after the completion of trial as well as up to 90 days from the conclusion of trial. The subject or their legal representative has signed the informed consent. Exclusion Criteria: Treatment with any investigational product in 90 days before study entry Partial or total splenectomy Subjects who have a serious concurrent disease like infection or who abuse addictive drug and substances. Pregnant and/or breast-feeding women Presence of Hepatitis B surface antigen or Hepatitis C or the patients show positive reaction to human immunodeficiency virus (HIV) type1 Subjects with a history of allergic reaction to Imiglucerase Subjects with a history of severe pulmonary hypertension caused by Gaucher Disease Any subject whom the investigator or the sub investigator considers as inad equate for this trial

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Cerezyme®<br>60 U/kg infusion (every 2 weeks for 24 weeks) | Drug: Cerezyme®<br>* administered by IV infusion for a dose of 60 U/kg<br>|
| Experimental: ISU302<br>60 U/kg infusion (every 2 weeks for 24 weeks) | Drug: ISU302<br>* administered by IV infusion for a dose of 60 U/kg<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | one year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | one year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Type I Gaucher