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Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

Primary Purpose

Osteopenia, Spinal Muscular Atrophy, Cerebral Palsy

Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Assisted Standing Treatment Program
Sponsored by
Gillette Children's Specialty Healthcare
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Osteopenia focused on measuring Osteopenia, spinal muscular atrophy, cerebral palsy, muscular dystrophy, spina bifida, Rett Syndrome

Eligibility Criteria

3 Years - 14 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Gillette Children's Specialty Healthcare

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Load-measuring platform

Arm Description

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.

Outcomes

Primary Outcome Measures

Bone Mineral Density (BMD) (measured by DXA)
Change from baseline in BMD, as , at 10 months.
Bone Architecture (measured by pQCT)
Change from baseline in bone architecture, as measured by pQCT, at 10 months.

Secondary Outcome Measures

Pediatric Quality of Life Inventory (PedsQL)
Change from baseline in PedsQL scores at 10 months.
Neuromuscular Module of the PedsQL
Change from baseline in PedsQL neuromuscular module scores at 10 months.
Change in pulmonary function test
Change from baseline in pulmonary function at 10 months.
Change in Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD)
Change from baseline in CPCHILD score at 10 months.

Full Information

First Posted
January 7, 2015
Last Updated
February 19, 2021
Sponsor
Gillette Children's Specialty Healthcare
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1. Study Identification

Unique Protocol Identification Number
NCT02428673
Brief Title
Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions
Official Title
Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions
Study Type
Interventional

2. Study Status

Record Verification Date
February 2021
Overall Recruitment Status
Completed
Study Start Date
December 9, 2015 (Actual)
Primary Completion Date
March 23, 2018 (Actual)
Study Completion Date
March 23, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Gillette Children's Specialty Healthcare

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Load-measuring platform
Arm Type
Other
Arm Description
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.
Intervention Type
Other
Intervention Name(s)
Assisted Standing Treatment Program
Primary Outcome Measure Information:
Title
Bone Mineral Density (BMD) (measured by DXA)
Description
Change from baseline in BMD, as , at 10 months.
Time Frame
10 months
Title
Bone Architecture (measured by pQCT)
Description
Change from baseline in bone architecture, as measured by pQCT, at 10 months.
Time Frame
10 months
Secondary Outcome Measure Information:
Title
Pediatric Quality of Life Inventory (PedsQL)
Description
Change from baseline in PedsQL scores at 10 months.
Time Frame
10 months
Title
Neuromuscular Module of the PedsQL
Description
Change from baseline in PedsQL neuromuscular module scores at 10 months.
Time Frame
10 months
Title
Change in pulmonary function test
Description
Change from baseline in pulmonary function at 10 months.
Time Frame
10 months
Title
Change in Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD)
Description
Change from baseline in CPCHILD score at 10 months.
Time Frame
10 Months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
14 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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 Exclusion Criteria: 1. Currently taking bisphosphonates
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Walter Truong, MD
Organizational Affiliation
Gillette Children's Specialty Healthcare
Official's Role
Principal Investigator
Facility Information:
Facility Name
Gillette Children's Specialty Healthcare
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55101
Country
United States

12. IPD Sharing Statement

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Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

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