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

Sponsor
Biogen (Industry)
Overall Status
Recruiting
CT.gov ID
NCT04317794
Collaborator
(none)
145
Enrollment
14
Locations
96
Anticipated Duration (Months)
10.4
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Drug: Nusinersen Sodium Injection

Study Design

Study Type:
Observational
Anticipated Enrollment :
145 participants
Observational Model:
Case-Only
Time Perspective:
Prospective
Official Title:
A Multicenter, Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium) When Used in Routine Medical Practice in Korea
Actual Study Start Date :
Jul 2, 2019
Anticipated Primary Completion Date :
Jul 1, 2027
Anticipated Study Completion Date :
Jul 1, 2027

Arms and Interventions

Arm 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:
  • BIIB058
  • ISIS 396443
  • Spinraza

    		•

    Outcome Measures

    Primary Outcome Measures

    1. 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

    1. 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.

    2. 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.

    3. Time to Death [Up to End of Treatment (2 years)]

    4. Number of Participants with Ventilatory Support [Up to End of Treatment (2 years)]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Research Site Busan Korea, Republic of 49241
    2 Research Site ChangWon Korea, Republic of 51353
    3 Research Site Cheongju-si Korea, Republic of 35015
    4 Research Site Daegu Korea, Republic of 41944
    5 Research Site Daegu Korea, Republic of 42415
    6 Research Site Daejeon Korea, Republic of 35015
    7 Research site Jeonju Korea, Republic of 56171
    8 Research Site Seoul Korea, Republic of 03080
    9 Research Site Seoul Korea, Republic of 03722
    10 Research Site Seoul Korea, Republic of 08308
    11 Research site Seoul Korea, Republic of 2841
    12 Research site Seoul Korea, Republic of 5505
    13 Research site Seoul Korea, Republic of 6351
    14 Research site Yangsan Korea, Republic of 50612

    Sponsors and Collaborators

    • Biogen

    Investigators

    • Study Director: Medical Director, Biogen

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Biogen
    ClinicalTrials.gov Identifier:
    NCT04317794
    Other Study ID Numbers:
    • 232SM403
    First Posted:
    Mar 23, 2020
    Last Update Posted:
    Mar 24, 2021
    Last Verified:
    Mar 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Studies a U.S. FDA-regulated Drug Product:
    No
    Additional relevant MeSH terms:
    Muscular Atrophy
    Muscular Atrophy, Spinal

    Study Results

    No Results Posted as of Mar 24, 2021