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

A Multicenter, Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium) When Used in Routine Medical Practice in Korea

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 Overview

• Status: Recruiting
• Conditions: Muscular Atrophy, Spinal
• Intervention / Treatment: Drug: Nusinersen Sodium Injection

• Study Type: Observational
• Enrollment (Anticipated): 145

Contacts and Locations

Study Locations

• Korea, Republic of
  • Busan, Korea, Republic of
    • Recruiting
    • Pusan Natioanl University Hospital
  • ChangWon, Korea, Republic of
    • Recruiting
    • Samsung Changwon Hospital
  • Cheongju-si, Korea, Republic of
    • Recruiting
    • Chungbuk National University Hospital
  • Daegu, Korea, Republic of
    • Recruiting
    • Kyungpook National University Hospital
  • Daegu, Korea, Republic of
    • Recruiting
    • Yeungnam University Hospital
  • Daejeon, Korea, Republic of
    • Recruiting
    • Chungnam National University Hospital
  • Gwangju, Korea, Republic of
    • Recruiting
    • Chonnam National University Hospital
  • Jeonju-si, Korea, Republic of
    • Recruiting
    • Chonbuk National University Hospital
  • Seoul, Korea, Republic of
    • Recruiting
    • Asan Medical Center
  • Seoul, Korea, Republic of
    • Recruiting
    • Samsung Medical Center
  • Seoul, Korea, Republic of
    • Recruiting
    • Korea University Anam Hospital
  • Seoul, Korea, Republic of
    • Recruiting
    • Korea University Guro Hospital
  • Seoul, Korea, Republic of
    • Recruiting
    • Severance Hospital, Yonsei University Health System
  • Seoul, Korea, Republic of
    • Recruiting
    • Seoul Natioanl University Hospital
  • Yangsan, Korea, Republic of
    • Recruiting
    • Pusan National University Yangsan Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Participants who are receiving or about to initiate treatment with commercial Spinraza in Korea according to the local marketing authorization (including documentation of SMA diagnosis), agree to the collection and use of data as specified in the protocol, and meet the eligibility criteria.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
All Participants; Participants who were prescribed with nusinersen sodium injection in Korea according to local marketing authorization.	Drug: Nusinersen Sodium Injection; Administered as specified in the treatment arm.; Other Names: ISIS 396443; Spinraza; BIIB058

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Up to End of Treatment (2 Years)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	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)
Number of Participants with Ventilatory Support		Up to End of Treatment (2 years)

Collaborators and Investigators

• Sponsor: Biogen

Study record dates

Study Major Dates

• Study Start (Actual): July 2, 2019
• Primary Completion (Anticipated): July 1, 2027
• Study Completion (Anticipated): July 1, 2027

Study Registration Dates

• First Submitted: March 5, 2020
• First Submitted That Met QC Criteria: March 20, 2020
• First Posted (Actual): March 23, 2020

Study Record Updates

• Last Update Posted (Actual): March 24, 2023
• Last Update Submitted That Met QC Criteria: March 23, 2023
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Neuromuscular Diseases; Neurodegenerative Diseases; Neuromuscular Manifestations; Pathological Conditions, Anatomical; Spinal Cord Diseases; Atrophy; Motor Neuron Disease; Muscular Atrophy; Muscular Atrophy, Spinal
• Other Study ID Numbers: 232SM403

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No