Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection
- Registration Number
- NCT04419233
- Lead Sponsor
- Biogen
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 50
- 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)
Key
- 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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description All Participants Nusinersen Sodium Injection Participants with 5q SMA and who were prescribed with nusinersen sodium injection in China according to the local marketing authorization.
- Primary Outcome Measures
Name Time Method Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) 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.
- Secondary Outcome Measures
Name Time Method 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) WHO Multicentre Growth Reference Study (MGRS), a component to assess gross motor development and is usually assessed in terms of age of achievement of motor milestones. Six distinct gross motor milestones criteria are as follows: sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone.
Hammersmith Infant Neurological Examination (HINE) Section 2 Scores Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years) Section 2 of the HINE is used to assess motor milestones of the participants. It is composed of 8 motor milestone categories: voluntary grasp, ability to kick in supine position, head control, rolling, sitting, crawling, standing, and walking.
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)
Trial Locations
- Locations (1)
Research Site
🇨🇳Hangzhou, Zhejiang, China