Clinical trial NCT05715749: Body Weight Support Harness System in Spinal Muscular Atrophy
Official title: Exploring the Feasibility and Utility of In-home Body Weight Support Harness System Use in Children Treated for Spinal Muscular Atrophy: a Pilot Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Spinal Muscular Atrophy Type I
Spinal Muscular Atrophy Type II
Keywords: exercise
physical therapy
Lead sponsor: Nationwide Children's Hospital
Sponsor class: OTHER
Collaborators: name: Novartis
First posted: 2023-02-08
Enrollment: 33
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Caregiver survey
timeFrame: End of study visit (month 6)
measure: Change in The Neuromuscular Gross Motor Outcome (GRO) scale
timeFrame: Completed at baseline, 3 months, and 6 months
measure: Change in Hammersmith Functional Motor Scale Expanded (HFMSE)
timeFrame: Completed at baseline, 3 months, and 6 months
measure: Change in Revised Hammersmith Scale (RHS)
timeFrame: Completed at baseline, 3 months, and 6 months
measure: 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
measure: Change in World Health Organization Motor Milestone Checklist
timeFrame: Completed at baseline, 3 months, and 6 months
Secondary outcomes:
measure: Harness use log
timeFrame: Completed throughout 6-month study period
Other outcomes:
Locations:
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
Study officials:
affiliation: Nationwide Children's Hospital
name: Megan A Iammarino, DPT
role: PRINCIPAL_INVESTIGATOR
References: