An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Subjects
试验速览
- 阶段
- 3 期
- 状态
- Enrolling By Invitation
- 入组人数
- 80
- 试验地点
- 10
- 主要终点
- Change in heparan sulfate concentrations in cerebrospinal fluid from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study to each time point
研究概览
简要总结
An extension of Global Phase III open-label, multicenter 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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A subject who participated in the Parent Study (JR-141-GS31) and completed the assessments at Week 105 in Cohort A or Week 53 in Cohort B before being administered the study medication of that respective visit, and in the opinion of the principal investigator there are no safety concerns.
- •A subject from whom an IRB or IEC-approved written informed consent can be obtained, which is voluntarily signed. If the subject is aged under 18 years (aged under 16 years in the UK) at the time of enrollment or willingness to participate in the study cannot be confirmed due to MPS II-related intellectual disability, the subject's legally acceptable representative (e.g., his parents or guardians) may sign the ICF on behalf of the subject. Written informed assent should be obtained from the subject, wherever possible.
- •Female subject of child bearing potential or male subject whose female partner is of child-bearing potential, i.e., fertile, following menarche and until becoming post-menopausal unless permanently sterile, agrees to use a medically accepted, highly effective method of contraception from the time of signing the ICF. The method of contraception must be used during the study until 90 days for male subjects, and 30 days for female subjects after the final study drug administration.
排除标准
- •A subject who changed treatment from JR-141 to idursulfase during the treatment period in the Parent Study (JR-141-GS31).
- •A subject who is unable to comply with the protocol (e.g., is unable to return for safety evaluations or is otherwise unlikely to complete the study) as determined by the principal investigator or sub-investigator.
- •[Only in France] Persons deprived of their liberty by a judicial or administrative decision, according to article L. 1121-6 of the Public Health Code (Code de la santé publique, CSP) adults who are the subject of a measure of legal protection or unable to express their consent according to article L. 1121-8 of the CSP.
研究组 & 干预措施
JR-141 2.0 mg/kg/week
干预措施: JR-141 (Drug)
结局指标
主要结局
Change in heparan sulfate concentrations in cerebrospinal fluid from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study to each time point
时间窗: [A-1, A-2, A-3: Week 157, 209, 261] [A-4: Week 131, 183, 235, 287]
Assessment will be mandatory for the first two years after starting JR-141 administration through the parent study or the extension study.
Change in neurocognitive testing, calculated by the BSID-III or KABC-II, from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study
时间窗: [A-1: Week 157, 209, 261] [A-2: Week 131, 157, 183, 209, 261] [A-3: Week 131, 157, 209, 261 ] [A-4: Week 131, 157, 183, 235, 287]
Change in neurocognitive testing, calculated by the VABS-II from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study
时间窗: [A-1: Week 157, 209, 261] [A-2: Week 131, 157, 183, 209, 261] [A-3: Week 131, 157, 209, 261 ] [A-4: Week 131, 157, 183, 235, 287][B-1: Week 78, 105, 157, 209, 261] [B-2: Week 78, 105, 131, 157, 209, 261]
Change in neurocognitive testing, calculated by the Wechsler, from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study
时间窗: [B-1: Week 78, 105, 157, 209, 261] [B-2: Week 78, 105, 131, 157, 209, 261]
次要结局
- Change in liver and spleen volume from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study([A-1, A-3: Week 157, 209, 261] [A-2: Week 131, 157, 209, 261] [A-4: Week 131, 183, 235, 287] [B-1: Week 105, 157, 209, 261] [B-2: Week 78, 105, 157, 209, 261])
- Change in 6-minute walk distance from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study([B-1: Week 105, 157, 209, 261] [B-2: Week 78, 105, 157, 209, 261])