Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)
- Registration Number
- NCT04317794
- Lead Sponsor
- Biogen
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 145
- Currently receiving or about to initiate treatment with commercial Spinraza in the postmarketing setting
- Genetic documentation of 5q-linked SMA
Key
- 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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description All Participants Nusinersen Sodium Injection Participants who were prescribed with nusinersen sodium injection in Korea according to local marketing authorization.
- Primary Outcome Measures
Name Time Method Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) 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 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) The HFMSE is a tool used to assess motor function in children with SMA. The original 20 item Hammersmith Functional Motor Scale (HFMS) was expanded to include 13 additional items to improve sensitivity for the higher functioning ambulant population. Participants are asked to complete a specific movement and are then graded on the quality and execution of that movement. Higher scores indicate higher levels of motor ability where 0=unable, 1=performed with some assistance, and 2=unaided. The overall score is the sum of the scores for all activities.
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) 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.
Time to Death Up to End of Treatment (2 years)
Trial Locations
- Locations (16)
Pusan Natioanl University Hospital
🇰🇷Busan, Korea, Republic of
Samsung Changwon Hospital
🇰🇷ChangWon, Korea, Republic of
Chungbuk National University Hospital
🇰🇷Cheongju-si, Korea, Republic of
Kyungpook National University Hospital
🇰🇷Daegu, Korea, Republic of
Yeungnam University Hospital
🇰🇷Daegu, Korea, Republic of
Chungnam National University Hospital
🇰🇷Daejeon, Korea, Republic of
Chonbuk National University Hospital
🇰🇷Jeonju-si, Korea, Republic of
Chonnam National University Hospital
🇰🇷Gwangju, Korea, Republic of
Severance Hospital
🇰🇷Seoul, Korea, Republic of
Asan Medical Center
🇰🇷Seoul, Korea, Republic of
Korea university Anam Hospital
🇰🇷Seoul, Korea, Republic of
Korea University Guro Hospital
🇰🇷Seoul, Korea, Republic of
Samsung Medical Center
🇰🇷Seoul, Korea, Republic of
Seoul Natioanl University Hospital
🇰🇷Seoul, Korea, Republic of
Severance Hospital, Yonsei University Health System
🇰🇷Seoul, Korea, Republic of
Pusan National University Yangsan Hospital
🇰🇷Yangsan, Korea, Republic of