NCT01161914撤回3 期
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
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 主要终点
- change in splenic volume compared to baseline
研究概览
简要总结
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
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 2 Years 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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
研究组 & 干预措施
Cerezyme®
Active Comparator
60 U/kg infusion (every 2 weeks for 24 weeks)
干预措施: Cerezyme® (Drug)
ISU302
Experimental
60 U/kg infusion (every 2 weeks for 24 weeks)
干预措施: ISU302 (Drug)
结局指标
主要结局
change in splenic volume compared to baseline
时间窗: one year
Primary endpoints to demonstrate non-inferiority of ISU302 against Cerezyme® for change in splenic volume compared to baseline.
次要结局
- change in organ parameters and biochemical value compared to baseline(one year)
研究者
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