ISRCTN22324060已完成未知
Efficacy and safety of enzyme replacement therapy for MPS I with 100 I.U./Kg recombinant human a-L-iduronidase (ALDURAZYME™).
Erasmus Medical Centre (The Netherlands)0 个研究点目标入组 35 人开始时间: 2007年3月6日最近更新:
适应症
试验速览
- 阶段
- 未知
- 状态
- 已完成
- 发起方
- 入组人数
- 35
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •1. The patient must give written informed consent
- •2. If the patient is younger than 12 years, informed consent from his/her parents or his/her legal representative is necessary
- •3. If the patient is below 18 years, but older than 12 years, informed consent from the child is necessary if the patient is mentally and physically able to do so
- •4. The patients can be included in this protocol, and not in any of the two other MPS I treatment protocols
- •5. The patient must have a current diagnosis of MPS I, as documented by a decreased a-L-iduronidase activity in leukocytes or fibroblasts
- •6. Patients must be willing and able to comply with the study protocol
- •7. Female patients must have a negative pregnancy test, and must use a medically accepted method of contraception during the study
排除标准
- •1. Patient is unable or unwilling to comply with the study protocol
- •2. Parent(s) or legal representatives are unable or unwilling to comply with the evaluation program
- •3. Patient is pregnant or lactating
- •4. Life expectancy less than six months
- •5. Very severe neurological involvement as evidenced by:
- •a. total or subtotal absence of cortical activity (vegetative state)
- •b. untreatable seizures
- •c. loss of (almost) all abilities to communicate
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