NCT02053896已完成不适用
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
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 5
- 主要终点
- Number of participants with Adverse Events
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 8 Years 至 29 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
结局指标
主要结局
Number of participants with Adverse Events
时间窗: From Screening to Week 24
To evaluate the safety of ISU302 in patients with Type 1 Gaucher disease previously treated with Cerezyme® after administering ISU302 alternative to Cerezyme®
次要结局
- skeletal status and bone mineral density(From Screening to Week 24)
- platelet count(From Screening to Week 24)
- liver and spleen volumes and liver function(From Screening to Week 24)
- Pharmacokinetics(From Screening to Week 24)
- hemoglobin concentration(From Screening to Week 24)
- biomarker levels(From Screening to Week 24)
研究者
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