NCT03568175已完成2 期
A Phase II/III Study of JR-141 in Patients With Mucopolysaccharidosis II
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 28
- 试验地点
- 19
- 主要终点
- Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid
研究概览
简要总结
A Phase II/ III multicenter, open-label, single-group, designed to evaluate the efficacy and safety of study drug for the treatment of the MPS II.
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
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Capable of providing written consent by himself (not mandatory for those aged under 20 years at the time of informed consent process, or those who is impossible to obtain consent from the patient himself due to intellectual disabilities associated with MPS II.)
- •In the case of a patient who is under the age of 20 years or from whom it is not possible to obtain consent due to intellectual disabilities associated with MPS II, he may be included if written consent can be provided by legal representative (however, written consent should be obtained from the patient himself too, whenever possible)
- •Males with confirmed diagnosis of MPS II, based on deficient activity of iduronate-2-sulfatase (IDS) in leucocytes, plasma or fibroblasts and/or pathogenic mutations identified in the IDS gene, etc.
- •Naïve patients or patients who are receiving stable enzyme replacement therapy with Elaprase for more than 8 weeks before the observational period starts.
排除标准
- •Previous engrafted HSCT, excluding those who need enzyme replacement therapy even after HSCT.
- •Judged by the investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking position for lumbar puncture due to joint contracture or those who are likely experience difficulty breathing during the lumbar puncture process.
- •Judged by the investigator or subinvestigator to be ineligible to participate in the study due to a history of serious drug allergy or sensitivity.
- •Patients who have received other investigational product within 4 months before enrollment in the study.
- •Otherwise judged by the investigator or subinvestigator to be ineligible to participate in the study.
研究组 & 干预措施
JR-141 2.0 mg/kg/week
Experimental
干预措施: JR-141 (Drug)
结局指标
主要结局
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid
时间窗: Baseline to 52 weeks
次要结局
- Change From Baseline in Urinary Dermatan Sulfate Levels.(Baseline, 25 weeks, 52 weeks)
- Change From Baseline in Spleen Volumes.(Baseline, 25 weeks, 52 weeks)
- Change From Baseline in Serum Heparan Sulfate Levels.(Baseline, 24-26 weeks, 50-52 weeks)
- Change From Baseline in Urinary Heparan Sulfate Levels.(Baseline, 25 weeks, 52 weeks)
- Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid.(Baseline to 25 weeks)
- Drug concentration in Cerebrospinal Fluid.(25, 52*weeks)
- Change From Baseline in Serum Dermatan Sulfate Levels.(Baseline, 24-26 weeks, 50-52 weeks)
- Change From Baseline in Liver Volumes.(Baseline, 25 weeks, 52 weeks)
- Change From Baseline in 6-minute Walk Test Distance.(Baseline, 25 weeks, 52 weeks)
- Change From Baseline in Joint Range of Motion.(Baseline, 25 weeks, 52 weeks)
- Change From Baseline in Cardiac Function.(Baseline, 25 weeks, 52 weeks)
- Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid.(Baseline, 25 weeks, 52 weeks)
- Change from Baseline in Neurocognitive Testing (Kyoto Scale of Psychological Development 2001)(25, 52 weeks)
- Change from Baseline in Adaptive Behavioral Testing ( Vineland Adaptive Behavior Scales Second Edition. )(25, 52 weeks)
研究者
研究点 (19)
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