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Motor Function and Activity of Daily Life in Spinal Muscular Atrophy

Sponsor
National Taiwan University Hospital (Other)
Overall Status
Not yet recruiting
CT.gov ID
NCT05931211
Collaborator
(none)
100
Enrollment
1
Location
18.1
Anticipated Duration (Months)
5.5
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study will incorporate patients/ caregivers' perspectives to investigate the performance in daily activities of individuals with SMA and how it relates to their motor function abilities.

Condition or Disease Intervention/Treatment Phase

    Detailed Description

    Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease that causes progressive muscle weakness and atrophy and eventually leads to loss of function. Despite recent advances in disease-modifying treatments, SMA can still significantly impact an individual's functioning in daily living and quality of life. This study will incorporate patients/ caregivers' perspectives to investigate the performance in daily activities of individuals with SMA and how it relates to their motor function abilities.

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    100 participants
    Observational Model:
    Cohort
    Time Perspective:
    Cross-Sectional
    Official Title:
    Translation of Motor Function to Daily Activity Performance in Individuals With SMA
    Anticipated Study Start Date :
    Jun 30, 2023
    Anticipated Primary Completion Date :
    Jun 30, 2024
    Anticipated Study Completion Date :
    Dec 31, 2024

    Outcome Measures

    Primary Outcome Measures

    1. Revised Upper Limb Module [15-20 minutes]

      The physiotherapist will perform a serious of motor test to evaluate the patient's upper limb motor function. This test is to observe the manual ability on different tasks in sitting position, including drawing along the path, opening Ziploc container, or lifting weight. And the therapist will score for each item. The Revised Upper Limb Module (RULM) are consist of 19 items. It takes about 15-20 minutes for testing.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    3 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Having a diagnosis of SMA with genetics confirmed as 5q SMA.

    • Above 3 years of age (capable of performing motor assessments).

    Exclusion Criteria:

    • Having severe respiratory and other medical conditions precluding safe participation in standardized motor function tests.

    • Having any orthopedic surgery during the past 6 months.

    • Women who are pregnant.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 National Taiwan University Hospital Taipei Taiwan 100

    Sponsors and Collaborators

    • National Taiwan University Hospital

    Investigators

    • Principal Investigator: Hsi-Wen Huang, MSc, National Taiwan University Hospital

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    National Taiwan University Hospital
    ClinicalTrials.gov Identifier:
    NCT05931211
    Other Study ID Numbers:
    • 202305039RINB
    First Posted:
    Jul 5, 2023
    Last Update Posted:
    Jul 7, 2023
    Last Verified:
    May 1, 2023
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Muscular Atrophy
    Muscular Atrophy, Spinal
    Atrophy

    Study Results

    No Results Posted as of Jul 7, 2023