Body Weight Support Harness System in Spinal Muscular Atrophy
- Conditions
- Spinal Muscular Atrophy Type ISpinal Muscular Atrophy Type II
- Interventions
- Other: In-home body weight support harness system
- Registration Number
- NCT05715749
- Lead Sponsor
- Nationwide Children's Hospital
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 33
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description In-home body weight support harness system In-home body weight support harness system All participants will be entered into treatment arm and receive an in-home body weight support harness system
- Primary Outcome Measures
Name Time Method Change in Revised Hammersmith Scale (RHS) 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.
Change in World Health Organization Motor Milestone Checklist 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.
Caregiver survey 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.
Change in Hammersmith Functional Motor Scale Expanded (HFMSE) 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.
Change in The Neuromuscular Gross Motor Outcome (GRO) scale 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.
Change in Bayley Scales of Infant and Toddler Development 3rd ed. (Bayley-III), Gross motor subtest 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.
- Secondary Outcome Measures
Name Time Method Harness use log 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.
Trial Locations
- Locations (1)
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States