Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 26
- 试验地点
- 1
- 主要终点
- To demonstrate the efficacy of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I in decreasing neurodevelopmental deterioration
研究概览
简要总结
This protocol will examine whether the enzyme alpha-L-iduronidase (Laronidase), delivered into the spinal fluid of patients with Hurler syndrome at intervals before and after bone marrow transplant, is a safe and effective approach to slow the neurologic degeneration seen in Hurler patients undergoing transplantation.
详细描述
Subjects will receive an infusion of Laronidase into his/her spinal fluid approximately 12 weeks before, 2 weeks before, 100 days after and 6 months after transplant. This procedure is done by lumbar puncture (also called a "spinal tap").
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
- 6 Months 至 3 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with a diagnosis of MPS IH (Hurler syndrome) are candidates for this protocol if they are being considered for hematopoietic stem cell transplantation according the University of Minnesota guidelines.
排除标准
- •Patients are less than 6 months old, or older than 3 years of age.
- •There is a history of clinically-severe hypersensitivity to Laronidase.
- •There is a contraindication for repeated lumbar puncture.
- •The family is not willing to undergo the necessary procedures and evaluations inherent in the study.
- •Consent has not been signed for participation in the 2004-09 study of intravenous Laronidase administration.
研究组 & 干预措施
Intent-to-Treat
All patients who have received at least one dose of Laronidase.
干预措施: IRT Laronidase (Drug)
结局指标
主要结局
To demonstrate the efficacy of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I in decreasing neurodevelopmental deterioration
时间窗: 1 year
次要结局
- To determine the safety and toxicity of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I(1 year)
- To determine brain changes with magnetic resonance imaging(1 and 2 years)
- To determine neurocognitive changes present in patients with Hurler syndrome(6, 12, and 24 months)
- To determine cerebral spinal fluid levels of glycosaminoglycans, cytokines and antibodies to Laronidase at baseline and at each point CSF is obtained(through 1 year)