Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA

Study Identifier:
AAAV5804
ClinicalTrials.gov Identifier:
NCT06955897
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Columbia University
Recruiting

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
    Date
    Apr 2025 - Dec 2026
    Patient Requirements
    Sex: Female & Male
    Age: 12+ years
    Requirements Information
    Inclusion and Exclusion Criteria
    Inclusion Criteria
    • All types of 5qSMA (homozygous deletion or point mutation of the SMN1 gene and any number of SMN2 copies)
    • Receiving a stable dosing regimen of 12mg nusinersen for at least 6 months
    Exclusion Criteria
    • An injury or surgery within the previous 3 months that would impact their ability to perform in-clinic function and/or fatigability assessments
    • Enrolled in an ongoing clinical trial, or extension study, expanded access program, or long-term registry of an investigational or recently approved medication
    • Receiving adjuvant and/or dual therapy (e.g., muscle-targeted, NMJ, or symptomatic treatments)

    Protocol Summary

    The purpose of this project is to investigate the utility of the SMA EFFORT, an SMA-specific patient-reported outcome measure, to assess perceived physical fatigability that is anchored to intensity and duration of activities. We aim to characterize perceived physical fatigability (PPF) in a diverse cohort of people with SMA (pwSMA) and evaluate the change of PPF before and after nusinersen dosing.

    Trial Locations

    Location
    Status
    Location
    Columbia University Irving Medical Center
    New York, New York, United States, 10032
    Status
    Recruiting

    Contact Cure SMA

    User Information

    If you have general questions regarding clinical trials or would like to be added to the Cure SMA mailing list to receive communications regarding future registries, studies, or clinical trials in SMA, please fill out the form below. Fields marked with an asterisk (*) are mandatory.

    • Disability Advocate who does not have SMA
    • I am a healthcare provider
    • I am a researcher
    • I have no direct connection to SMA
    • I have SMA
    • I lost a child to SMA
    • My child has SMA
    • Someone close to me has/had SMA
    • Australia
    • Belgium
    • Brazil
    • Canada
    • Chile
    • China
    • Colombia
    • Croatia
    • Czech Republic
    • Denmark
    • Estonia
    • France
    • Germany
    • Greece
    • Hong Kong
    • Hungary
    • India
    • Ireland
    • Israel
    • Italy
    • Japan
    • Lebanon
    • Malaysia
    • Mexico
    • Netherlands
    • Norway
    • Poland
    • Portugal
    • Puerto Rico
    • Qatar
    • Republic of Korea
    • Romania
    • Russian Federation
    • Saudi Arabia
    • Serbia
    • Singapore
    • South Africa
    • Spain
    • Sweden
    • Switzerland
    • Taiwan, Province of China
    • Thailand
    • Ukraine
    • United Kingdom
    • United States
    • Vietnam
    This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.