NCT01645189已完成3 期
To Evaluate the Safety and Efficacy of Hunterase(Idursulfase-beta) in Hunter Syndrome Patients < 6 Years of Age Receiving Idursulfase Enzyme Replacement Therapy
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Incidence of adverse events
研究概览
简要总结
The objective of this study is to determine the safety and efficacy of once weekly dosing of idursulfase-beta 0.5mg/kg administered by intravenous(IV) infusion for Hunter syndrome patients < 6 years old.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 5 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •The patient has a diagnosis of Hunter syndrome based upon biochemical criteria:
- •as measured in plasma, leukocytes, or fibroblasts,
- •a deficiency in iduronate-2-sulfatase (I2S) enzyme activity of ≤ 10 % of the lower limit of the normal range
- •That corresponds to one or more of the following:
- •a normal enzyme activity level of one other sulfatase
- •Confirmed as MPS2 by genetic test results
- •shows clinical symptoms/ visible signs of MPS2
- •< 6 years old and male
- •Patients who are able to comply with the study requirements
- •The patient's parent(s), or patient's legal guardian must have given voluntary written consent to participate in the study
排除标准
- •The patient has had a tracheostomy
- •The patient has known severe hypersensitivity or shock to any of the components of idursulfase
- •The patient has received treatment with another investigational therapy within 30 days prior to enrollment
- •History of a stem cell transplant
- •The patient has known severe hypersensitivity or shock to any of the components of test drug(excipient etc)
结局指标
主要结局
Incidence of adverse events
时间窗: One year
次要结局
- change of anti-idursulfase-beta antibody status(baseline and one year)
- Percent Change of Urine GAG(baseline to 53 weeks)
研究者
研究点 (1)
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