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Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

Descriptive Information
Brief Title † Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions
Official Title † Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions
Brief 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. 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 Phase N/A
Study Type † Interventional
Study Design † Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary Outcome Measure † Bone Mineral Density (BMD) (measured by DXA)
Secondary Outcome Measure † Pediatric Quality of Life Inventory (PedsQL)
Condition † Osteopenia Spinal Muscular Atrophy Cerebral Palsy Muscular Dystrophy Spina Bifida Rett Syndrome
Intervention † OtherAssisted Standing Treatment Program
Study Arms / Comparison Groups Load-measuring platform 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.
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Other
Estimated Enrollment † 5
Start Date † May 2015
Completion Date December 2016
Primary Completion Date December 2016
Eligibility Criteria † Inclusion Criteria: 1. Must have a neuromuscular condition 2. Must be between 3-14 years old 3. Gross Motor Function Classification System (GMFCS) Level IV or V 4. Must be on a standing treatment program 5. Parent must be able to provide consent Exclusion Criteria: 1. Currently taking bisphosphonates
Gender Both
Ages 3 Years - 14 Years
Accepts Healthy Volunteers No
Contacts †† Kari A Williams, MPH, 651-325-2316, kariawilliams@gillettechildrens.com
Location Countries †
Administrative Information
NCT ID † NCT02428673
Organization ID Truong POSNA
Secondary IDs ††
Responsible Party Principal Investigator
Study Sponsor † Gillette Children's Specialty Healthcare
Collaborators ††
Investigators † Principal Investigator: Walter Truong, MD, Gillette Children's Specialty Healthcare
Information Provided By
Verification Date April 2015
First Received Date † January 7, 2015
Last Updated Date April 28, 2015
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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