NL-OMON35691招募中4 期
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. - Enzyme therapy in patients with MPS type I, II and VI
适应症
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 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
入排标准
- 年龄范围
- 2 至 99（—）
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.
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.
研究者
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