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

NCT04317794 | Active Not Recruiting DMC

• 1 other identifier: 232SM403
• Study Type: observational
• Target: 74
• Locations: 1 country
• Sites: 16

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.

Conditions

Muscular Atrophy, Spinal

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• 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)

• 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)

• Time to Death

  Up to End of Treatment (2 years)

• Number of Participants with Ventilatory Support

  Up to End of Treatment (2 years)

Study Arms (1)

All Participants

Participants who were prescribed with nusinersen sodium injection in Korea according to local marketing authorization.

Drug: Nusinersen Sodium Injection

Interventions

Nusinersen Sodium Injection DRUG

Administered as specified in the treatment arm.

Also known as: BIIB058, ISIS 396443, Spinraza

All Participants

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• Currently receiving or about to initiate treatment with commercial Spinraza in the postmarketing setting
• Genetic documentation of 5q-linked SMA

You may not qualify if:

• 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

Sponsors & Collaborators

• Biogen: lead

Study Sites (16)

Pusan Natioanl University Hospital

Busan, South Korea

Location

Samsung Changwon Hospital

Changwon, South Korea

Location

Chungbuk National University Hospital

Cheongju-si, South Korea

Location

Kyungpook National University Hospital

Daegu, South Korea

Location

Yeungnam University Hospital

Daegu, South Korea

Location

Chungnam National University Hospital

Daejeon, South Korea

Location

Chonnam National University Hospital

Gwangju, South Korea

Location

Chonbuk National University Hospital

Jeonju, South Korea

Location

Severance Hospital

Seoul, 03722, South Korea

Location

Asan Medical Center

Seoul, South Korea

Location

Korea university Anam Hospital

Seoul, South Korea

Location

Korea University Guro Hospital

Seoul, South Korea

Location

Samsung Medical Center

Seoul, South Korea

Location

Seoul Natioanl University Hospital

Seoul, South Korea

Location

Severance Hospital, Yonsei University Health System

Seoul, South Korea

Location

Pusan National University Yangsan Hospital

Yangsan, South Korea

Location

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Interventions

nusinersen

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases

Study Officials

• Medical Director

  Biogen

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 5, 2020
• First Posted: March 23, 2020
• Study Start: July 2, 2019
• Primary Completion (Estimated): February 5, 2027
• Study Completion (Estimated): February 5, 2027
• Last Updated: July 17, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will share

Locations

KR (16)