EUCTR2007-001453-26-NL进行中（未招募）不适用
atural course, effects of enzyme therapy and health economic aspects in patients with mucopolysaccharidosis type I, II and VI. Long-term folloe-up of untreated patients and patients receiving commercially available Aldurazyme, Elaprase and Naglazyme.
未提供0 个研究点目标入组 50 人开始时间: 2009年11月13日最近更新:
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 50
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. The patient should have a biochemically confirmed deficiency of a-L-iduronidase (MPS I). iduronidate-2-sulfatase (MPS II), or N-acetylgalactosamine-4-sulfatase (MPS VI); or a confirmed mutation in the gene encoding for a-L-iduronidase (MPS I). iduronidate-2-sulfatase (MPS II, or N-acetylgalactosamine-4-sulfatase (MPS VI).
- •2. The patient has had least one evaluation through which the severity of the disease has been assessed and the urgency of enzyme therapy can be determined.
- •3. Written informed consent must be obtained from the patient and/or from the patient's parent/guardian if the patient is under 18 years of age.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. The patient (or parent/legal guardian) is unable or unwilling to comply with the study protocol.
- •2. The patient has severe neurological involvement as evidenced by:
- •* total or subtotal absence of cortical activity.
- •* untreatable seizures
- •* loss of (almost) all abilities to communicate.
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