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Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02428673
First Posted: April 29, 2015
Last Update Posted: September 11, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Walter Truong, MD, Gillette Children's Specialty Healthcare
  Purpose

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.


Condition Intervention
Osteopenia Spinal Muscular Atrophy Cerebral Palsy Muscular Dystrophy Spina Bifida Rett Syndrome Other: Assisted Standing Treatment Program

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

Resource links provided by NLM:


Further study details as provided by Walter Truong, MD, Gillette Children's Specialty Healthcare:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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.


Estimated Enrollment: 5
Study Start Date: December 2015
Estimated Study Completion Date: March 31, 2018
Estimated Primary Completion Date: March 31, 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.
Other: Assisted Standing Treatment Program

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).

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 14 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02428673


Locations
United States, Minnesota
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States, 55101
Sponsors and Collaborators
Gillette Children's Specialty Healthcare
Investigators
Principal Investigator: Walter Truong, MD Gillette Children's Specialty Healthcare
  More Information

Responsible Party: Walter Truong, MD, Pediatric Orthopedic Surgeon, Gillette Children's Specialty Healthcare
ClinicalTrials.gov Identifier: NCT02428673     History of Changes
Other Study ID Numbers: Truong POSNA
First Submitted: January 7, 2015
First Posted: April 29, 2015
Last Update Posted: September 11, 2017
Last Verified: September 2017

Keywords provided by Walter Truong, MD, Gillette Children's Specialty Healthcare:
Osteopenia
spinal muscular atrophy
cerebral palsy
muscular dystrophy
spina bifida
Rett Syndrome

Additional relevant MeSH terms:
Atrophy
Cerebral Palsy
Muscular Dystrophies
Muscular Atrophy
Bone Diseases, Metabolic
Muscular Atrophy, Spinal
Rett Syndrome
Spinal Dysraphism
Pathological Conditions, Anatomical
Brain Damage, Chronic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Neuromuscular Manifestations
Neurologic Manifestations
Signs and Symptoms
Bone Diseases
Metabolic Diseases
Spinal Cord Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Genetic Diseases, X-Linked