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NCT Number: NCT04317794

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

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Muscular Atrophy, Spinal Central Nervous System Diseases Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Pusan Natioanl University Hospital, Busan, South Korea

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Nusinersen Sodium Injection

Drug

Administered as specified in the treatment arm.

Other names: BIIB058, ISIS 396443, Spinraza

Primary outcomes

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

    Time frame: 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 outcomes

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

    Time frame: 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.

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

    Time frame: 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.

  3. Time to Death

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

  4. Number of Participants with Ventilatory Support

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

Sponsors and collaborators

Lead sponsor

Biogen

Industry

Registry information

Official study title

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

Acronym: STANDARD

Important dates

Study start
2019
Primary completion
2027
Study completion
2027
First posted
Mar 23, 2020
Registry last updated
Jul 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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