DRKS00011547已完成不适用
Expanded Access Program to provide Nusinersen to Patients with infantile-onset spinal muscular atrophy (SMA)
Klinik für Neuropädiatrie und MuskelerkrankungenZentrum für Kinder- und JugendmedizinUniversitätsklinikum Freiburg0 个研究点目标入组 61 人开始时间: 2017年1月11日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 61
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- one 至 18 Years（—）
- 性别
- All
入选标准
- •Patients must meet all of the following criteria to be eligible:
- •- Genetic documentation of 5q SMA homozygous gene deletion, homozygous mutation, or compound heterozygote
- •- Onset of clinical signs and symptoms at = 6 months (180 days) of age, consistent with infantile onset, Type I SMA
- •- Treatment with Nusinersen within the EAP
- •- Informed consent for the study
排除标准
- •- Patient qualifies to participate in an ongoing clinical trial with nusinersen
- •- Participation in a prior nusinersen study
- •- Previous exposure to nusinersen
- •- History of brain or spinal cord disease that would interfere with the LP procedures or CSF circulation
- •- Presence of implanted shunt for the drainage of CSF or implanted CNS catheter
- •- Previous or current participation in a clinical trial with an investigational gene therapy for SMA
- •- Participation in a study with an investigational therapy for SMA within 6 months or five half-lives of the investigational drug, whichever is the longer, prior to the first dose of nusinersen.
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