NCT06488924招募中1 期
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 10
- 试验地点
- 3
- 主要终点
- To establish the safety and tolerability of JR-446 in MPSIIIB patients
研究概览
简要总结
A Phase I/ II, open-label study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIB patients.
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
- — 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Chronological age of <18 years
- •Confirmed diagnosis of MPS IIIB
排除标准
- •Prior experience to gene therapy or HSCT 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
- •Contraindication for lumbar puncture or MRI
- •History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following lumbar puncture
- •The above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.
研究组 & 干预措施
JR-446
Experimental
干预措施: JR-446 (Drug)
结局指标
主要结局
To establish the safety and tolerability of JR-446 in MPSIIIB patients
时间窗: up to 4 years (multiple visits)
To determine the pharmacodynamic effects of JR-446 in MPSIIIB patients
时间窗: up to 4 years (multiple visits)
次要结局
- To assess PK profile of JR-446 in MPSIIIB patients(up to 4 years (multiple visits))
- To explore the potential efficacy of JR-446 in the treatment of patients with MPS IIIB(up to 4 years (multiple visits))
研究者
研究点 (3)
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