Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)

N/AActive Not RecruitingNCT04317794

Biogen74 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Muscular Atrophy, Spinal

Interventions

Nusinersen Sodium InjectionDRUG

Administered as specified in the treatment arm.

Eligibility

Sex: ALL

Medical Language ↔ Plain English

Key Inclusion Criteria: * Currently receiving or about to initiate treatment with commercial Spinraza in the postmarketing setting * Genetic documentation of 5q-linked SMA Key Exclusion Criteria: * 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.

Locations (16)

Pusan Natioanl University Hospital

Busan, South Korea

Samsung Changwon Hospital

Changwon, South Korea

Chungbuk National University Hospital

Cheongju-si, South Korea

Outcomes

Primary Outcomes

Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

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.

Time frame: Up to End of Treatment (2 Years)

Secondary Outcomes

Change from Baseline in Hammersmith Infant Neurological Examination (HINE) Section 2 Scores

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 frame: Baseline, Day 63 and Approximately Every 4 Months Up to End of Treatment (2 years)

Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE)

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.

Time frame: Baseline, Day 63 and Approximately Every 4 Months Up to End of Treatment (2 years)

Time to Death

Time frame: Up to End of Treatment (2 years)

Data from ClinicalTrials.gov

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