CompletedNot Applicable

Body Weight Support Harness System in Spinal Muscular Atrophy

United States33 participantsStarted 2018-09-07

Plain-language 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.

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Caregiver survey

Timeframe: End of study visit (month 6)

Change in The Neuromuscular Gross Motor Outcome (GRO) scale

Timeframe: Completed at baseline, 3 months, and 6 months

Change in Hammersmith Functional Motor Scale Expanded (HFMSE)

Timeframe: Completed at baseline, 3 months, and 6 months

Change in Revised Hammersmith Scale (RHS)

Timeframe: Completed at baseline, 3 months, and 6 months

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

Timeframe: Completed at baseline, 3 months, and 6 months

Change in World Health Organization Motor Milestone Checklist

Timeframe: Completed at baseline, 3 months, and 6 months

Trial details

NCT IDNCT05715749

SponsorNationwide Children's Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2020-06-15

View on ClinicalTrials.gov More Spinal Muscular Atrophy Type I trials

Plain-language summary

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Caregiver survey

Timeframe: End of study visit (month 6)

Change in The Neuromuscular Gross Motor Outcome (GRO) scale

Timeframe: Completed at baseline, 3 months, and 6 months

Change in Hammersmith Functional Motor Scale Expanded (HFMSE)

Timeframe: Completed at baseline, 3 months, and 6 months

Change in Revised Hammersmith Scale (RHS)

Timeframe: Completed at baseline, 3 months, and 6 months

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

Timeframe: Completed at baseline, 3 months, and 6 months

Change in World Health Organization Motor Milestone Checklist

Timeframe: Completed at baseline, 3 months, and 6 months