Body Weight Support Harness System in Spinal Muscular Atrophy
Status
Conditions
Treatments
Details and patient eligibility
About
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.
Enrollment
Sex
Volunteers
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
Trial design
Primary purpose
Allocation
Interventional model
Masking
33 participants in 1 patient group
Trial contacts and locations
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Data sourced from clinicaltrials.gov
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