jRCT2071240053进行中（未招募）不适用
Phase I study of weekly infusion of JR-441 in patients with mucopolysaccharidosis type IIIA
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 3
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Single Arm Study
- 干预模型
- Single Assignment
- 主要目的
- Treatment Purpose
- 盲法
- Open(masking Not Used)
入排标准
- 年龄范围
- 1age old over 至 18age old not（—）
- 性别
- All
入选标准
- •Chronological age of 1 year or older and under 18 years at the time of signing ICF
- •A subject with a diagnosis of MPS IIIA at the time of ICF
- •A subject with a minimal body weight of 10 kg at the time of screening
排除标准
- •Prior experience to gene therapy or Hematopoietic stem cell transplantation with successful engraftment
- •Past use of another investigational drug or product in last 4 months or 5 half-lives (whichever is longer) before signing ICF
- •Current participation in a clinical trial or past participation (within 30 days of enrolment into this study) in a study involving invasive procedures
- •Past use of Genistein or Kineret (anakinra) within 4 months before signing ICF
- •Serious drug allergy or hypersensitivity
- •The above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.
研究者
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