A Multicenter, Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium) When Used in Routine Medical Practice in Korea
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- Biogen
- 入组人数
- 74
- 试验地点
- 16
- 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 is to evaluate the safety of nusinersen sodium injection in the postmarketing setting in Korea. The secondary objective is to evaluate the effectiveness of nusinersen sodium injection in the postmarketing setting in Korea.
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
- •Currently receiving or about to initiate treatment with commercial Spinraza in the postmarketing setting
- •Genetic documentation of 5q-linked SMA
排除标准
- •Hypersensitivity to the active substance or any of the excipients of Spinraza
- •Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrollment in other interventional clinical trials for the treatment of SMA
- •Inability to comply with study requirements
- •NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
All Participants
Participants who were prescribed with nusinersen sodium injection in Korea according to 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: 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
- Number of Participants with Ventilatory Support(Up to End of Treatment (2 years))
- Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE)(Baseline, Day 63 and Approximately Every 4 Months Up to End of Treatment (2 years))
- Change from Baseline in Hammersmith Infant Neurological Examination (HINE) Section 2 Scores(Baseline, Day 63 and Approximately Every 4 Months Up to End of Treatment (2 years))
- Time to Death(Up to End of Treatment (2 years))