NCT03708965进行中（未招募）2 期
An Extension Study of JR-141-BR21 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
- 进行中（未招募）
- 入组人数
- 19
- 试验地点
- 2
- 主要终点
- Number of participants with Adverse Events
研究概览
简要总结
A Phase II open-label, parallel group, 2 sites (Brazil), designed to evaluate the long term safety and efficacy 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
- 年龄范围
- 0 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Patients who will have completed clinical trial JR-141-BR
- •Capable of providing written consent by himself, unless the patient is under the age of 18 years at the time of informed consent process, or it is not possible to obtain consent from the patient himself due to his intellectual disabilities associated with MPS II.
- •In the case of a patient who is under the age of 18 years or from whom it is not possible to obtain consent due to his intellectual disabilities associated with MSP 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, wherever possible.
排除标准
- •Refusal to sign the informed consent form.
- •Unable to perform the study procedures, except for neurocognitive testing.
- •Previous engrafted BMT/HSCT.
- •Judged by the investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking a position for lumber puncture due to joint contracture or those who are likely to 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 a serious drug allergy or sensitivity.
- •Otherwise judged by the investigator or subinvestigator to be ineligible to participate in the study out of consideration for the subject safety.
研究组 & 干预措施
JR-141
Experimental
Subjects will be assigned to 1.0, 2.0 or 4.0 mg of JR-141 per kg of body weight once every week (the same dose taken during the previous study) in the beginning of the study.
During the study, the dose of all subjects will be switched to the selected one*.
* The dose was determined to be 2.0 mg/kg/week based on the safety and efficacy data of JR-141-BR21 study.
干预措施: JR-141 (Drug)
结局指标
主要结局
Number of participants with Adverse Events
时间窗: From screening up to the end of study, up to approximately 5 years
次要结局
- Urinary dermatan sulfate concentrations(through study completion, an average of 52 weeks, up to approximately 5 years)
- Liver and spleen volumes (MRI)(through study completion, an average of 52 weeks, up to approximately 5 years)
- Echocardiography(through study completion, an average of 52 weeks, up to approximately 5 years)
- Urinary heparan sulfate concentrations(through study completion, an average of 52 weeks, up to approximately 5 years)
- Serum heparan sulfate concentrations(through study completion, an average of 52 weeks, up to approximately 5 years)
- Serum dermatan sulfate concentrations(through study completion, an average of 52 weeks, up to approximately 5 years)
研究者
研究点 (2)
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