A Switch-Over Study of the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease previously treated with Imiglucerase.

Official Title
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A Phase 2 Multi-Center, Open-Label, Switch-Over Trial to Evaluate the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase

Conditions
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Gaucher Disease

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patient diagnosed with type-1 Gaucher disease Patient who was stably treating Gaucher disease with Cerezyme® and who was maintaining the usage and dosage of Cerezyme® for at least 6 months prior to study drug administration Patient aged 2 years or higher Female patient with contraception during the study period (oral or injectable contraceptive hormones, intrauterine device, physical devices using condom, sponge form, jelly, and femidom, and abstinence) Patient who signed the informed consent form after hearing the detailed explanation about this study Definition of the stable treatment of type-1 Gaucher disease: No neurologic deficit Normal hemoglobin concentration, and platelet count that has increased to ≥100,000/㎣, or maintained to 100,000/㎣ Normal or no deteriorated bone mineral density Normal or no deteriorated splenomegaly or hepatomegaly Exclusion Criteria: Patient who participated in other clinical studies within 90 days before study drug administration Patient with unstable hemoglobin and platelet counts for at least 6 months before study drug administration Patient with hypersensitivity to Cerezyme® Patient positive to HIV antibody, hepatitis B antigen, and hepatitis C antibody Patient with Fe, folic acid, or vitamin B12-deficcient anemia Patient who received miglustat within 6 months before study drug administration Patient who received erythrocyte growth factor or chronic systemic corticosteroids within 6 months before study drug administration Patient who had clinically significant splenic obstruction within 12 months before study drug administration Pregnant or lactating patient Patient who had serious concurrent diseases such as infectious diseases or drug-addicted patient Patient who was considered inappropriate for this study by the investigators or sub-investigators

Ages Eligible for Study
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Minimum Age: 8 Years
Maximum Age: 29 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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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| ISU302<br>15 60U/kg (once every 2 weeks for 6 months) | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants with Adverse Events | To evaluate the safety of ISU302 in patients with Type 1 Gaucher disease previously treated with Cerezyme® after administering ISU302 alternative to Cerezyme® | From Screening to Week 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pharmacokinetics | Measurement of blood glucocerebrosidase activity for pharmacokinetic assessment after initial study drug administration | From Screening to Week 24 | 
| hemoglobin concentration | Change in hemoglobin concentration | From Screening to Week 24 | 
| platelet count | Change in platelet count | From Screening to Week 24 | 
| liver and spleen volumes and liver function | Changes in liver and spleen volumes and liver function | From Screening to Week 24 | 
| biomarker levels | Changes in biomarker levels (Acid Phosphatase, Angiotensin Converting Enzyme, and Chitotriosidase) | From Screening to Week 24 | 
| skeletal status and bone mineral density | Changes in skeletal status and bone mineral density | From Screening to Week 24 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ISU302, Imiglucerase, Type I Gaucher