OsteopeniaNCT02428673
Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions
A study testing Assisted Standing Treatment Program in people with Osteopenia and Spinal Muscular Atrophy, run by Gillette Children's Specialty Healthcare at 1 site in United States.
Trial summary
Children with neuromuscular disabilities and limited ambulation are at significant risk for decreased bone mineral density (BMD) and increased incidence of fracture. This is caused, in part, by low levels of load experienced by the skeleton due to a child's functional limitations. Read more about this study
- Who is it for?
- Ages 3 to 14 years
- Conditions the study is for
- Osteopenia, Spinal Muscular Atrophy, Cerebral Palsy
- What is being tested?
- Assisted Standing Treatment Program
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 9 Dec 2015to 23 Mar 2018
- 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 3 to 14 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Must have a neuromuscular condition
- Must be between 3-14 years old
- Gross Motor Function Classification System (GMFCS) Level IV or V
- Must be on a standing treatment program
- Parent must be able to provide consent
Who cannot join
- 1. Currently taking bisphosphonates
Trial locations and contacts
1 site in 1 country.
United States1 site
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101
About this study
Children with neuromuscular disabilities and limited ambulation are at significant risk for decreased bone mineral density (BMD) and increased incidence of fracture. This is caused, in part, by low levels of load experienced by the skeleton due to a child's functional limitations. Low BMD has been shown to be predictive of fracture, and in fact, fractures usually occur without significant trauma in children with neuromuscular conditions. The discomfort and distress from fractures in this population are considerable, and the associated costs to the family and healthcare system are substantial. Numerous interventions have been devoted to improving BMD in these children. Stationary assisted standing devices are widely used and represent the standard-of-care. However, evidence supporting this approach is limited due to inadequate study designs with insufficient numbers of patients.
This study will use load-sensing platforms in patients with neuromuscular conditions. Successful completion of this pilot study will assist in the development of a future multicenter clinical trial to definitively determine relationships, if any, between passive standing and measures of BMD, fracture incidence, pulmonary function, and health-related quality-of-life measures in children with a variety of neuromuscular disabilities (e.g., spinal muscular atrophy, cerebral palsy, muscular dystrophy, spina bifida, Rett syndrome).
Hypothesis: Assisted standing treatment program will gradually increase their duration of standing by up to 75% after the baseline phase.
Read the detailed description
Children with neuromuscular disabilities and limited ambulation are at significant risk for decreased bone mineral density (BMD) and increased incidence of fracture. This is caused, in part, by low levels of load experienced by the skeleton due to a child's functional limitations. Low BMD has been shown to be predictive of fracture, and in fact, fractures usually occur without significant trauma in children with neuromuscular conditions such as cerebral palsy, spinal muscular atrophy, or other muscular dystrophies. The discomfort and distress from fractures in this population are considerable, and the associated costs to the family and healthcare system are substantial. Numerous interventions have been devoted to improving BMD in these children. Stationary assisted standing devices are widely used and represent the standard-of-care. However, evidence supporting this approach is limited due to inadequate study designs with insufficient numbers of patients.
Various modifications are added to assisted-standing devices to allow children with neuromuscular impairments to achieve standing postures. These customizations lead to load-sharing with the standing device, and consequently, a decrease in the amount of load passing through the lower extremities. Previous investigators developed custom-made load-measuring sensors to quantify the amount of load borne by the lower extremities while in passive standers, and found that the actual load varied from 23-102% of the child's body weight. These load-measuring sensors were specifically developed for restricted laboratory testing, rather than recording the load magnitude and duration of standing in any brand of standing device during daily use at home, school or therapy.
We previously developed load-sensing platforms that accurately measure loads experienced by the lower extremities of children with cerebral palsy in passive standers. These platforms can be incorporated into any stander design, are able to be used on a routine basis at a child's home, school or therapy, and are able to record the duration of weight-bearing to monitor compliance.
This study will use these load-sensing platforms in patients with neuromuscular conditions. Successful completion of this pilot study will assist in the development of a future multicenter clinical trial to definitively determine relationships, if any, between passive standing and measures of BMD, fracture incidence, pulmonary function, and health-related quality-of-life measures in children with a variety of neuromuscular disabilities (e.g., spinal muscular atrophy, cerebral palsy, muscular dystrophy, spina bifida, Rett syndrome).
- Conditions
- Osteopenia, Spinal Muscular Atrophy, Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Rett Syndrome
- Being tested
- Assisted Standing Treatment Program
- Keywords
- Osteopenia, spinal muscular atrophy, cerebral palsy, muscular dystrophy, spina bifida, Rett Syndrome
- Medical subject headings
- Bone Diseases, Metabolic, Muscular Atrophy, Spinal, Cerebral Palsy, Muscular Dystrophies, Spinal Dysraphism, Rett Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Assisted Standing Treatment ProgramOther
| Study group | Role | What they receive |
|---|---|---|
| Load-measuring platform | Other | A load-sensing platform will be placed under each foot of the subject to record the time course of load borne by each of the lower extremities during weight-bearing training in an assisted standing device. Receives: Other: Assisted Standing Treatment Program |
What is being measured
The primary outcome is the main question the study is designed to answer.
Bone Mineral Density (BMD) (measured by DXA)
Change from baseline in BMD, as , at 10 months.
Measured: 10 months
Bone Architecture (measured by pQCT)
Change from baseline in bone architecture, as measured by pQCT, at 10 months.
Measured: 10 months
Secondary outcomes (4)
Pediatric Quality of Life Inventory (PedsQL)
Change from baseline in PedsQL scores at 10 months.
Measured: 10 months
Neuromuscular Module of the PedsQL
Change from baseline in PedsQL neuromuscular module scores at 10 months.
Measured: 10 months
Change in pulmonary function test
Change from baseline in pulmonary function at 10 months.
Measured: 10 months
Change in Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD)
Change from baseline in CPCHILD score at 10 months.
Measured: 10 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Apr 2015 (estimated)
- Study start9 Dec 2015
- Primary completion23 Mar 2018
- Study completion23 Mar 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for OsteopeniaStudies of the same condition, open and under way.
- 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
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gillette Children's Specialty Healthcare
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Walter Truong, Gillette Children's Specialty Healthcare
- Investigators
- Walter Truong, MD, Principal investigator, Gillette Children's Specialty Healthcare
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02428673
- Sponsor's protocol ID
- Truong POSNA
- Status last verified
- Feb 2021
- First submitted
- 7 Jan 2015
- Last updated
- 21 Feb 2021
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 21 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions
- ClinicalTrials.gov ID
- NCT02428673
- Acronym / sponsor ID
- Truong POSNA
- Conditions
- Osteopenia, Spinal Muscular Atrophy, Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Rett Syndrome
- Keywords
- Osteopenia, spinal muscular atrophy, cerebral palsy, muscular dystrophy, spina bifida, Rett Syndrome
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Gillette Children's Specialty HealthcareUniversity, hospital or other
- Study start
- 9 Dec 2015 (actual)
- Primary completion
- 23 Mar 2018 (actual)
- Study completion
- 23 Mar 2018 (actual)
- First posted
- 29 Apr 2015 (estimated)
- 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.
Children with neuromuscular disabilities and limited ambulation are at significant risk for decreased bone mineral density (BMD) and increased incidence of fracture. This is caused, in part, by low levels of load experienced by the skeleton due to a child's functional limitations. Low BMD has been shown to be predictive of fracture, and in fact, fractures usually occur without significant trauma in children with neuromuscular conditions. The discomfort and distress from fractures in this population are considerable, and the associated costs to the family and healthcare system are substantial. Numerous interventions have been devoted to improving BMD in these children. Stationary assisted standing devices are widely used and represent the standard-of-care. However, evidence supporting this approach is limited due to inadequate study designs with insufficient numbers of patients.
This study will use load-sensing platforms in patients with neuromuscular conditions. Successful completion of this pilot study will assist in the development of a future multicenter clinical trial to definitively determine relationships, if any, between passive standing and measures of BMD, fracture incidence, pulmonary function, and health-related quality-of-life measures in children with a variety of neuromuscular disabilities (e.g., spinal muscular atrophy, cerebral palsy, muscular dystrophy, spina bifida, Rett syndrome).
Hypothesis: Assisted standing treatment program will gradually increase their duration of standing by up to 75% after the baseline phase.
Detailed description
Children with neuromuscular disabilities and limited ambulation are at significant risk for decreased bone mineral density (BMD) and increased incidence of fracture. This is caused, in part, by low levels of load experienced by the skeleton due to a child's functional limitations. Low BMD has been shown to be predictive of fracture, and in fact, fractures usually occur without significant trauma in children with neuromuscular conditions such as cerebral palsy, spinal muscular atrophy, or other muscular dystrophies. The discomfort and distress from fractures in this population are considerable, and the associated costs to the family and healthcare system are substantial. Numerous interventions have been devoted to improving BMD in these children. Stationary assisted standing devices are widely used and represent the standard-of-care. However, evidence supporting this approach is limited due to inadequate study designs with insufficient numbers of patients.
Various modifications are added to assisted-standing devices to allow children with neuromuscular impairments to achieve standing postures. These customizations lead to load-sharing with the standing device, and consequently, a decrease in the amount of load passing through the lower extremities. Previous investigators developed custom-made load-measuring sensors to quantify the amount of load borne by the lower extremities while in passive standers, and found that the actual load varied from 23-102% of the child's body weight. These load-measuring sensors were specifically developed for restricted laboratory testing, rather than recording the load magnitude and duration of standing in any brand of standing device during daily use at home, school or therapy.
We previously developed load-sensing platforms that accurately measure loads experienced by the lower extremities of children with cerebral palsy in passive standers. These platforms can be incorporated into any stander design, are able to be used on a routine basis at a child's home, school or therapy, and are able to record the duration of weight-bearing to monitor compliance.
This study will use these load-sensing platforms in patients with neuromuscular conditions. Successful completion of this pilot study will assist in the development of a future multicenter clinical trial to definitively determine relationships, if any, between passive standing and measures of BMD, fracture incidence, pulmonary function, and health-related quality-of-life measures in children with a variety of neuromuscular disabilities (e.g., spinal muscular atrophy, cerebral palsy, muscular dystrophy, spina bifida, Rett syndrome).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Load-measuring platform | Other | Other: Assisted Standing Treatment ProgramA load-sensing platform will be placed under each foot of the subject to record the time course of load borne by each of the lower extremities during weight-bearing training in an assisted standing device. |
Who may qualify
- Ages
- 3 to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Must have a neuromuscular condition
- Must be between 3-14 years old
- Gross Motor Function Classification System (GMFCS) Level IV or V
- Must be on a standing treatment program
- Parent must be able to provide consent
Key exclusion criteria (1 listed)
- 1. Currently taking bisphosphonates
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 |
|---|---|---|
| Assisted Standing Treatment Program | Other | Load-measuring platform |
Primary outcome
Bone Mineral Density (BMD) (measured by DXA)
Time frame: 10 months
Change from baseline in BMD, as , at 10 months.
Bone Architecture (measured by pQCT)
Time frame: 10 months
Change from baseline in bone architecture, as measured by pQCT, at 10 months.
4 secondary outcomes
Pediatric Quality of Life Inventory (PedsQL)
Time frame: 10 months
Neuromuscular Module of the PedsQL
Time frame: 10 months
Change in pulmonary function test
Time frame: 10 months
Change in Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD)
Time frame: 10 Months
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 21 Feb 2021.
Related trials for Osteopenia
Not yet recruitingPhase 2
Melatonin in Posterior Mandible Extraction Socket
Bone Loss
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
Active, not recruiting
AI-Supported Mobile High-Impact and Strengthening Exercise Program in Postmenopausal Women With Low Bone Density
Osteopenia
Active, not recruiting
L-PRF Block vs Composite Graft for Horizontal Ridge Augmentation
Alveolar Ridge Resorption After Extraction, Alveolar Bone Loss
RecruitingObservational study
Observational Study of Bone Complications in People With Post-menopausal Breast Cancer Who Have Stopped Treatment With Denosumab and Aromatase Inhibitors
Breast Cancer
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.
Walter Truong, MD principal investigator
Gillette Children's Specialty Healthcare
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Assisted Standing Treatment Program in people with Osteopenia and Spinal Muscular Atrophy, run by Gillette Children's Specialty Healthcare 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 1 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 21 Feb 2021. 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?
Bone Mineral Density (BMD) (measured by DXA) (time frame: 10 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 21 Feb 2021.