Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

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.

Study Overview

• Status: Completed
• Conditions: Cerebral Palsy; Muscular Dystrophy; Osteopenia; Rett Syndrome; Spinal Muscular Atrophy; Spina Bifida
• Intervention / Treatment: 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).

• Study Type: Interventional
• Enrollment (Actual): 6
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Minnesota
    • Saint Paul, Minnesota, United States, 55101
      • Gillette Children's Specialty Healthcare

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 14 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: 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

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Gillette Children's Specialty Healthcare

Study record dates

Study Major Dates

• Study Start (Actual): December 9, 2015
• Primary Completion (Actual): March 23, 2018
• Study Completion (Actual): March 23, 2018

Study Registration Dates

• First Submitted: January 7, 2015
• First Submitted That Met QC Criteria: April 28, 2015
• First Posted (Estimate): April 29, 2015

Study Record Updates

• Last Update Posted (Actual): February 21, 2021
• Last Update Submitted That Met QC Criteria: February 19, 2021
• Last Verified: February 1, 2021

More Information

Terms related to this study

• Keywords: spinal muscular atrophy; cerebral palsy; Osteopenia; spina bifida; muscular dystrophy; Rett Syndrome
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Congenital Abnormalities; Brain Damage, Chronic; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Neuromuscular Manifestations; Pathological Conditions, Anatomical; Spinal Cord Diseases; Muscular Disorders, Atrophic; Bone Diseases; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Nervous System Malformations; Motor Neuron Disease; Neural Tube Defects; Cerebral Palsy; Muscular Dystrophies; Muscular Atrophy; Atrophy; Bone Diseases, Metabolic; Rett Syndrome; Muscular Atrophy, Spinal; Spinal Dysraphism
• Other Study ID Numbers: Truong POSNA