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Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

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ClinicalTrials.gov Identifier: NCT02428673
Recruitment Status : Active, not recruiting
First Posted : April 29, 2015
Last Update Posted : July 10, 2018
Sponsor:
Information provided by (Responsible Party):
Walter Truong, Gillette Children's Specialty Healthcare

Tracking Information
First Submitted Date  ICMJE January 7, 2015
First Posted Date  ICMJE April 29, 2015
Last Update Posted Date July 10, 2018
Study Start Date  ICMJE December 2015
Estimated Primary Completion Date March 31, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 28, 2015)
  • 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.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02428673 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 28, 2015)
  • Pediatric Quality of Life Inventory (PedsQL) [ Time Frame: 10 months ]
    Change from baseline in PedsQL scores at 10 months.
  • Neuromuscular Module of the PedsQL [ Time Frame: 10 months ]
    Change from baseline in PedsQL neuromuscular module scores at 10 months.
  • Change in pulmonary function test [ Time Frame: 10 months ]
    Change from baseline in pulmonary function at 10 months.
  • Change in Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) [ Time Frame: 10 Months ]
    Change from baseline in CPCHILD score at 10 months.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions
Official Title  ICMJE 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 Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Osteopenia
  • Spinal Muscular Atrophy
  • Cerebral Palsy
  • Muscular Dystrophy
  • Spina Bifida
  • Rett Syndrome
Intervention  ICMJE Other: Assisted Standing Treatment Program
Study Arms  ICMJE 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.
Intervention: Other: Assisted Standing Treatment Program
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: April 28, 2015)
5
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 31, 2019
Estimated Primary Completion Date March 31, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Years to 14 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02428673
Other Study ID Numbers  ICMJE Truong POSNA
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Walter Truong, Gillette Children's Specialty Healthcare
Study Sponsor  ICMJE Gillette Children's Specialty Healthcare
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Walter Truong, MD Gillette Children's Specialty Healthcare
PRS Account Gillette Children's Specialty Healthcare
Verification Date July 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP