Pilot/Phase I Study of Intrathecal Laronidase for Spinal Cord Compression in Muco

鞘内注射 Laronidase 用于粘膜脊髓压迫的试点/第一阶段研究

• 批准号: 7628059
• 负责人: PATRICIA I DICKSON
• 金额: $ 22.94万
• 依托单位: LUNDQUIST INSTITUTE FOR BIOMEDICAL INNOVATION AT HARBOR-UCLA MEDICAL CENTER
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-06-01 至 2011-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7628059
• 关键词: Pilot; Phase; Study; Intrathecal; Laronidase

DESCRIPTION (provided by applicant): Enzyme replacement therapy with laronidase (Aldurazyme, recombinant human a-L-iduronidase, EC 3.2.1.76) has been developed for MPS I, a lysosomal storage disorder. Laronidase given weekly into the vein helps many physical problems due to the disease, but does not treat the brain and spinal cord. The investigators purpose is to test delivery of laronidase given into the spinal fluid in patients with MPS I. The first aim is a pilot study of laronidase administered once per month into the spinal fluid for four months. Up to 10 study participants with spinal cord compression due to MPS will be studied. In the second aim, the treatment is continued every one to three months for a one-year extension period. The goals are to measure the safety and effectiveness of laronidase given into the spinal fluid to treat or stabilize spinal cord compression in the Hurler-Scheie and Scheie forms of MPS I. If successful, delivery into the spinal fluid could represent a practical, straightforward method of treating central nervous system disease due to lysosomal storage.

描述（由申请人提供）： 已开发了用于MPS I（一种溶酶体储存障碍）的LARONIDASE（Aldurazyme，重组人A-L-二核酶，EC 3.2.1.76）的酶替代疗法。 每周进行洛烯酸酶有助于由于疾病而有助于许多身体问题，但不能治疗大脑和脊髓。 研究人员的目的是测试MPS I的LARONIDASE在脊髓液中的递送。第一个目的是对每月一次在脊髓液中施用的Laronidase的试验研究四个月。 将研究多达10名因MPS引起的脊髓压缩的研究参与者。 在第二个目标中，每一到三个月继续进行一年的延长期。 目标是衡量给予脊柱液中的Laronidase的安全性和有效性，以治疗或稳定MPS I的脊髓和Scheie形式中的脊髓压缩。如果成功的脊柱液体可以代表一种实际，直率的治疗溶酶体引起的中枢神经系统疾病的实用方法。