Spinal Muscular Atrophy Type INCT05715749
Body Weight Support Harness System in Spinal Muscular Atrophy
A study testing In-home body weight support harness system in people with Spinal Muscular Atrophy Type I and Spinal Muscular Atrophy Type II, run by Nationwide Children's Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
- What is being tested?
- In-home body weight support harness system
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Sep 2018to 15 Jul 2024
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
1 site in 1 country.
United States1 site
Nationwide Children's Hospital
Columbus, Ohio, 43205
About this study
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
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.
- Being tested
- In-home body weight support harness system
- Keywords
- exercise, physical therapy
- Medical subject headings
- Spinal Muscular Atrophies of Childhood, Motor Activity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
In-home body weight support harness systemOther
Also called The Portable Mobility Aid for Children (PUMA)
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.
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Other: In-home body weight support harness system |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Completed at baseline, 3 months, and 6 months
Change in Bayley Scales of Infant and Toddler Development 3rd ed. (Bayley-III), Gross motor subtest
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.
Measured: 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.
Measured: Completed at baseline, 3 months, and 6 months
Secondary outcomes (1)
Harness use log
A log to document frequency (each use) and duration (how long it was used at one time) of harness system use.
Measured: Completed throughout 6-month study period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Feb 2023
- Study start7 Sep 2018
- Primary completion15 Jun 2020
- Study completion15 Jul 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Spinal Muscular Atrophy Type IStudies of the same condition, open and under way.
- More trials run by Nationwide Children's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nationwide Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis
- Responsible party
- Principal investigator, Megan Iammarino, Nationwide Children's Hospital
- Investigators
- Megan A Iammarino, DPT, Principal investigator, Nationwide Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05715749
- Sponsor's protocol ID
- IRB18-00484
- Status last verified
- Jul 2024
- First submitted
- 20 Jan 2023
- Last updated
- 22 Jul 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Participant data will be provided in summary form at manuscript submission. Certain individual participant data will be available in manuscript as well.
Publications
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.
PubMed 38502672
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05715749
- Acronym / sponsor ID
- IRB18-00484
- Keywords
- exercise, physical therapy
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- Nationwide Children's HospitalUniversity, hospital or other
- Collaborators
- Novartis
- Study start
- 7 Sep 2018 (actual)
- Primary completion
- 15 Jun 2020 (actual)
- Study completion
- 15 Jul 2024 (actual)
- First posted
- 8 Feb 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| In-home body weight support harness system | Experimental | Other: In-home body weight support harness systemAll participants will be entered into treatment arm and receive an in-home body weight support harness system |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| In-home body weight support harness systemAlso: The Portable Mobility Aid for Children (PUMA)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. | Other | In-home body weight support harness system |
Primary outcome
Caregiver survey
Time frame: 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 The Neuromuscular Gross Motor Outcome (GRO) scale
Time frame: 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 Hammersmith Functional Motor Scale Expanded (HFMSE)
Time frame: 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 Revised Hammersmith Scale (RHS)
Time frame: 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 Bayley Scales of Infant and Toddler Development 3rd ed. (Bayley-III), Gross motor subtest
Time frame: 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.
Change in World Health Organization Motor Milestone Checklist
Time frame: 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.
1 secondary outcome
Harness use log
Time frame: Completed throughout 6-month study period
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jul 2024.
- Derived from this study: PubMed 38502672Iammarino 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.
- Individual participant dataSharing planned: No. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Megan A Iammarino, DPT principal investigator
Nationwide Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing In-home body weight support harness system in people with Spinal Muscular Atrophy Type I and Spinal Muscular Atrophy Type II, run by Nationwide Children's Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Caregiver survey (time frame: End of study visit (month 6)). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05715749, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jul 2024.