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Completed

NCT Number: NCT05715749

Body Weight Support Harness System in Spinal Muscular Atrophy

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.

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Key information

Conditions

Spinal Muscular Atrophy Type I Behavior Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Motor Activity Motor Neuron Disease Muscular Atrophy, Spinal Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases Spinal Muscular Atrophies of Childhood Spinal Muscular Atrophy Type II

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

In-home body weight support harness system

Other

A portable, adjustable body weight support harness system that occupies roughly 9 feet x 9 feet space in the home. The system allows 360 degrees of mobility anywhere within the footprint of the frame. The amount of body weight support is adjustable.

Other names: The Portable Mobility Aid for Children (PUMA)

Primary outcomes

  1. Caregiver survey

    Time frame: End of study visit (month 6)

    A five question likert-style survey asking families to rate their experience during the study. The scales asks the family to rate (1) the perceived impact of BWSS on motor and (2) non-motor skills, (3) the child's perceived enjoyment using the system, (4) the ease of equipment use, and (5) if the family believed that use of the BWSS was beneficial on a 5-point Likert scale; scores of 4 or 5 were considered favorable, scores of 3 were considered neutral, and scores of 1 or 2 were considered unfavorable. Higher scores indicate greater perceived benefit of participation.

  2. Change in The Neuromuscular Gross Motor Outcome (GRO) scale

    Time frame: Completed at baseline, 3 months, and 6 months

    A 50-item performance-based outcome developed to quantify motor function across the span of age and abilities in individuals with neuromuscular disorders including spinal muscular atrophy (SMA). Scores range from 0 - 100 points; higher scores reflect a greater level of function. An increase in score indicates a better outcome.

  3. Change in Hammersmith Functional Motor Scale Expanded (HFMSE)

    Time frame: Completed at baseline, 3 months, and 6 months

    A disease-specific and performance-based outcomes originally developed to measure motor skill in untreated individuals with SMA. Scores range from 0 - 66 points; higher scores reflect a greater level of function. An increase in score indicates a better outcome.

  4. Change in Revised Hammersmith Scale (RHS)

    Time frame: Completed at baseline, 3 months, and 6 months

    A disease-specific and performance-based outcomes originally developed to measure motor skill in untreated individuals with SMA, revised following Rasch analysis of HFMSE. Scores range from 0 - 69 points; higher scores reflect a greater level of function. An increase in score indicates a better outcome.

  5. Change in Bayley Scales of Infant and Toddler Development 3rd ed. (Bayley-III), Gross motor subtest

    Time frame: Completed at baseline, 3 months, and 6 months

    A norm-referenced developmental assessment of gross motor skill in children 16 days - 3.5 years of age. Raw scores range from 0 - 72. Higher scores indicate a greater level of funcion. An increase in score indicates a better outcome.

  6. Change in World Health Organization Motor Milestone Checklist

    Time frame: Completed at baseline, 3 months, and 6 months

    A checklist of key gross motor milestones expected during typical development. Scores can range from 0 - 6 milestones achieved. Achievement of a greater number of milestones indicates better outcomes.

Secondary outcomes

  1. Harness use log

    Time frame: Completed throughout 6-month study period

    A log to document frequency (each use) and duration (how long it was used at one time) of harness system use.

Sponsors and collaborators

Lead sponsor

Nationwide Children's Hospital

Other

Collaborators

  • Novartis

Registry information

Official study title

Exploring the Feasibility and Utility of In-home Body Weight Support Harness System Use in Children Treated for Spinal Muscular Atrophy: a Pilot Study

Important dates

Study start
2018
Primary completion
2020
Study completion
2024
First posted
Feb 8, 2023
Registry last updated
Jul 22, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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