Exploring the Feasibility and Utility of In-home Body Weight Support Harness System Use in Children Treated for Spinal Muscular Atrophy: a Pilot Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 33
- 试验地点
- 2
- 主要终点
- Change in Revised Hammersmith Scale (RHS)
研究概览
简要总结
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:
- Is the in-home body weight support harness system a feasible option for families to use?
- Is the in-home body weight support harness system a useful tool for children treated for spinal muscular atrophy?
- 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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
盲法说明
Outcomes assessors are masked to previous participant performance.
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
结局指标
主要结局
Change in Revised Hammersmith Scale (RHS)
时间窗: 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
时间窗: 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
时间窗: 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)
时间窗: 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
时间窗: 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
时间窗: 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.
次要结局
- Harness use log(Completed throughout 6-month study period)
研究者
Megan Iammarino
Research Physical Therapist
Nationwide Children's Hospital