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| Name | Class |
|---|---|
| Novartis | INDUSTRY |
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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:
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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| In-home body weight support harness system | Experimental | All participants will be entered into treatment arm and receive an in-home body weight support harness system |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Caregiver survey | 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. | End of study visit (month 6) |
| Change in The Neuromuscular Gross Motor Outcome (GRO) scale | 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. | Completed at baseline, 3 months, and 6 months |
| Change in Hammersmith Functional Motor Scale Expanded (HFMSE) | 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. | Completed at baseline, 3 months, and 6 months |
| Change in Revised Hammersmith Scale (RHS) | 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. | Completed at baseline, 3 months, and 6 months |
| Change in Bayley Scales of Infant and Toddler Development 3rd ed. (Bayley-III), Gross motor subtest |
| Measure | Description | Time Frame |
|---|---|---|
| Harness use log | A log to document frequency (each use) and duration (how long it was used at one time) of harness system use. | Completed throughout 6-month study period |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Megan A Iammarino, DPT | Nationwide Children's Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38502672 | Derived | Iammarino MA, Alfano LN, Reash NF, Sabo B, Conroy S, Noritz G, Wendland M, Lowes LP. Feasibility and utility of in-home body weight support harness system use in young children treated for spinal muscular atrophy: A single-arm prospective cohort study. PLoS One. 2024 Mar 19;19(3):e0300244. doi: 10.1371/journal.pone.0300244. eCollection 2024. |
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Participant data will be provided in summary form at manuscript submission. Certain individual participant data will be available in manuscript as well.
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| ID | Term |
|---|---|
| D014897 | Spinal Muscular Atrophies of Childhood |
| D009043 | Motor Activity |
| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| C553190 | bbc3 protein, zebrafish |
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Outcomes assessors are masked to previous participant performance.
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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. |
| Completed at baseline, 3 months, and 6 months |
| Change in World Health Organization Motor Milestone Checklist | 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. | Completed at baseline, 3 months, and 6 months |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D016472 | Motor Neuron Disease |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D001519 | Behavior |