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Pediatric SMA China Registry

Sponsor
Biogen (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05042921
Collaborator
(none)
300
Enrollment
30
Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) treatments among pediatric chinese participants with spinal muscular atrophy linked to chromosome 5q (5q-SMA).

Condition or Disease Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational [Patient Registry]
    Anticipated Enrollment :
    300 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    Pediatric Patients With Spinal Muscular Atrophy in China: A Nationwide Registry
    Anticipated Study Start Date :
    Dec 31, 2021
    Anticipated Primary Completion Date :
    Jun 30, 2024
    Anticipated Study Completion Date :
    Jun 30, 2024

    Arms and Interventions

    Arm Intervention/Treatment
    DMT Treated Participants

    Participants with SMA who received prior treatment with DMTs including nusinersen will be followed prospectively for up to 21 months and the available data is collected retrospectively.
    Untreated Participants

    Participants with SMA who received no treatment will be followed prospectively for up to 21 months.

    Outcome Measures

    Primary Outcome Measures

    1. Natural History and Utilization of Disease Modifying Therapy (DMT) Treatments Among Pediatric Chinese Participants With Spinal Muscular Atrophy Linked to Chromosome 5q (5q-SMA) [Up to 21 months]

    Other Outcome Measures

    1. Effectiveness of DMT Treatments [Up to 21 months]

      Effectiveness will be assessed using following measures: time to mortality, motor function, motor measures, pulmonary function, scoliosis, hospitalizations and comorbidities, clinical observations, patient reported outcomes, wheelchair use, nutrition and electrophysiology and biomarkers.

    2. Safety of DMT Treatments [Up to 21 months]

      Safety will be assessed by adverse events (AEs) and serious AEs.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A to 18 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Key Inclusion Criteria:

    • Ability of the participant and/or his/her legally authorized representative (e.g., parent or legal guardian), as appropriate and applicable, to understand the purpose and risks of the study, to provide informed consent, and to authorize the use of confidential health information in accordance with national and local privacy regulations.

    • Genetically confirmed 5q-SMA.

    • Age < 18 years at enrollment.

    Key Exclusion Criteria:

    • Unable or unwilling to provide informed consent.

    • Other types of SMA (non 5q-SMA).

    NOTE: Other protocol defined inclusion/ exclusion criteria may apply.

    Contacts and Locations

    Locations

    No locations specified.

    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:
    NCT05042921
    Other Study ID Numbers:
    • CN-SMG-11774
    First Posted:
    Sep 13, 2021
    Last Update Posted:
    Sep 13, 2021
    Last Verified:
    Aug 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Additional relevant MeSH terms:
    Muscular Atrophy
    Muscular Atrophy, Spinal
    Atrophy

    Study Results

    No Results Posted as of Sep 13, 2021