NCT06095388进行中（未招募）1 期
Phase I/II Study of Weekly Infusions of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- To evaluate the tolerability of JR-441 in MPSIIIA patients
研究概览
简要总结
A Phase I/ II, open-label, randomized, 2-arm study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIA patients.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Chronological age of ≥1 year and ≤18 years.
- •Confirmed diagnosis of MPS IIIA.
- •Body weight ≥ 10 kg.
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-441 low dose
Experimental
干预措施: JR-441 (Drug)
JR-441 medium dose
Experimental
干预措施: JR-441 (Drug)
JR-441 high dose
Experimental
干预措施: JR-441 (Drug)
结局指标
主要结局
To evaluate the tolerability of JR-441 in MPSIIIA patients
时间窗: up to 5 years (multiple visits)
Adverse events will be reported and graded, laboratory tests will be conducted and vital signs will be monitored
To assess the safety of JR-441 in MPSIII-A patients
时间窗: up to 5 years (multiple visits)
Number and severity of infusion-associated reactions, including anaphylaxis
次要结局
- Plasma drug concentration(up to 5 years (multiple visits))
- Plasma PK parameters(up to 5 years (multiple visits))
- Change from baseline in heparan sulfate levels in cerebrospinal fluid (CSF), serum and urine(up to 5 years (multiple visits))
- Change from baseline in cognitive function(up to 5 years (multiple visits))
研究者
研究点 (1)
Loading locations...
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