A Multicenter, Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection When Used in Routine Medical Practice in China
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Biogen
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
The primary objective of this study is to evaluate the safety of nusinersen sodium injection in the postmarketing setting in China.The secondary objectives are to collect data on the efficacy and the pharmacokinetics of nusinersen sodium injection in the post-marketing setting in China.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ability of the participant or his/her legally authorized representative (e.g., parent or legal guardian), as appropriate and applicable, to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information in accordance with national and local subject privacy regulations.
- •The decision to newly prescribe nusinersen sodium injection according to the local marketing authorization, including documentation of 5q SMA diagnosis, has been made by the Investigator and agreed to by the participant (or legally authorized representative)
排除标准
- •Hypersensitivity to the active substance or any of the excipients of nusinersen sodium injection.
- •Current or past administration of nusinersen sodium injection, in either a commercial or a clinical study setting.
- •Other unspecified reasons that, in the opinion of the Investigator or the Sponsor, make the participant unsuitable for enrollment.
- •NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
All Participants
Participants with 5q SMA and who were prescribed with nusinersen sodium injection in China according to the local marketing authorization.
干预措施: Nusinersen Sodium Injection (Drug)
结局指标
主要结局
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Day 0 up to End of Treatment (2 Years)
An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose results in death, places the participant at immediate risk of death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect, or is a medically important event.
次要结局
- Percentage of Participants who Achieved World Health Organization (WHO) Motor Milestone(Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years))
- Hammersmith Infant Neurological Examination (HINE) Section 2 Scores(Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years))
- Number of Participants with Ventilatory Support(Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years))
- Cerebrospinal Fluid (CSF) Concentrations of Nusinersen Sodium Injection(pre-dose on Day 0, Day 63, and Every 4 Months Up to End of Treatment (2 years))
- Plasma Concentrations of Nusinersen Sodium Injection(Pre-dose and multiple time points post-dose on Day 0, post-dose on Day 28 and pre-dose every 4 months up to end of treatment (2 years))