Body Weight Support Harness System in Spinal Muscular Atrophy

Study Identifier:
IRB18-00484
ClinicalTrials.gov Identifier:
NCT05715749
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Nationwide Children's Hospital
Study Complete

Study Details

Medical Condition
  • Spinal Muscular Atrophy Type I
  • Spinal Muscular Atrophy Type II
Study Drug
    Date
    Sep 2018 - Jun 2020
    Patient Requirements
    Sex: Female & Male
    Age: N/A
    Requirements Information
    Inclusion and Exclusion Criteria
    Inclusion Criteria
    • Confirmed heterozygous mutation in SMN1 gene and 2 or 3 copies of SMN2 (historically, SMA Type 1 and Type 2)
    • Past or current treatment with nusinersen, onasemnogene abeparvovec, or risdiplam
    • Upright head control (defined as the ability to lift head from full forward flexion)
    • Weight under or 50lbs
    • Confirmed motor delay
    Exclusion Criteria
    • 4+ copies of SMN2 (historically, SMA Type 3 and 4)
    • Evidence of lower limb injury or recent fracture
    • In the opinion of the investigator, it was unsafe for the child to participate

    Protocol Summary

    The goal of this pilot interventional study is to learn about the use of an in-home harness system in children who have been treated for spinal muscular atrophy. The main questions it aims to answer are:

    1. Is the in-home body weight support harness system a feasible option for families to use?

    2. Is the in-home body weight support harness system a useful tool for children treated for spinal muscular atrophy?

    3. Is the in-home body weight support harness system a safe tool for children treated for spinal muscular atrophy?

    Participants will be given an in-home body weight support harness system and taught how to use it. Families will document how often and for how long they use the system over 6 months. Children will be given tests of motor function at the beginning, 3-months, and 6-months. At the end of the study, families will be asked to fill out a questionnaire about thier experience using the system.

    Trial Locations

    Location
    Status
    Location
    Nationwide Children's Hospital
    Columbus, Ohio, United States, 43205
    Status
    N/A

    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.