An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 27
- 试验地点
- 40
- 主要终点
- Time course of serum HS and DS concentrations from initial dosing in the preceding study
研究概览
简要总结
Multicenter, open-label, single-group, designed to evaluate the long term 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
- •A patient who participated in the preceding Study JR-141-301 and completed the Week 52 visit, and has no safety concerns to enter this study in the opinion of the investigator or subinvestigator.
- •A patient capable of providing written informed consent in person (However, this is not required for patients aged younger than 20 years at the time of consent or patients with intellectual disability associated with MPS II whose willingness cannot be verified.)
- •For patients aged younger than 20 years at the time of consent or patients with intellectual disability associated with MPS II whose willingness cannot be verified, written consent must be obtained from the legally acceptable representative. (Wherever possible, written consent of the patient should be obtained.)
- •Male patient whose partner is of child-bearing potential and agrees to use a medically accepted, highly effective method of contraception.
排除标准
- •A patient who used any concomitant medication or therapy that could affect study assessments in the opinion of the investigator or subinvestigator.
- •A patient with a history of serious drug allergy or hypersensitivity that precludes participation in this study in the opinion of the investigator or subinvestigator.
- •A patient judged to be ineligible by the investigator or subinvestigator for other reasons.
研究组 & 干预措施
JR-141
干预措施: JR-141 (Drug)
结局指标
主要结局
Time course of serum HS and DS concentrations from initial dosing in the preceding study
时间窗: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of uronic acid concentration from initial dosing in the preceding study
时间窗: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Vital signs (blood pressure)
时间窗: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
12-lead electrocardiogram
时间窗: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
The presence or absence of abnormal findings (if present, specific findings and whether or not they are reported as adverse events)
Time course of developmental assessment data (Vineland-II) from initial dosing in the preceding study
时间窗: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of CSF substrate (HS and DS) concentrations from initial dosing in the preceding study
时间窗: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of urinary DS concentration from initial dosing in the preceding study
时间窗: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of spleen volume (assessed by CT or MRI) from initial dosing in the preceding study
时间窗: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Occurrence of adverse events
时间窗: From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
Occurrence of adverse reactions
时间窗: From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
Laboratory tests (urinalysis)
时间窗: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Vital signs (pulse rate)
时间窗: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Vital signs (body temperature)
时间窗: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Antibody tests (anti-JR-141 antibodies)
时间窗: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
IAR
时间窗: From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
Time course of developmental assessment data (Kyoto Scale of Psychological Development 2001) from initial dosing in the preceding study
时间窗: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of urinary HS concentration from initial dosing in the preceding study
时间窗: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of 6-minute walk test distance from initial dosing in the preceding study
时间窗: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of developmental assessment data (Bayley-III or KABC-II) from initial dosing in the preceding study
时间窗: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of liver volume (assessed by CT or MRI) from initial dosing in the preceding study
时间窗: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of cardiac function (assessed by echocardiography) from initial dosing in the preceding study
时间窗: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of joint range of motion from initial dosing in the preceding study
时间窗: Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Incidence of abnormal vital signs
时间窗: Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Laboratory tests (hematology)
次要结局
未报告次要终点