Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

Cerebral Palsy Clinical Trial

Official title:

Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02428673
• Other study ID #: Truong POSNA
• Status: Completed
• Phase: N/A
• Start date: December 9, 2015
• Est. completion date: March 23, 2018

Study information

• Verified date: February 2021
• Source: Gillette Children's Specialty Healthcare
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 6
• Est. completion date: March 23, 2018
• Est. primary completion date: March 23, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 14 Years

Eligibility

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

Related Conditions & MeSH terms

• Atrophy
• Bone Diseases, Metabolic
• Cerebral Palsy
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Muscular Dystrophies
• Muscular Dystrophy
• Osteopenia
• Rett Syndrome
• Spina Bifida
• Spinal Dysraphism
• Spinal Muscular Atrophy

Intervention

Other:
• Assisted Standing Treatment Program

Locations

Country	Name	City	State
United States	Gillette Children's Specialty Healthcare	Saint Paul	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Gillette Children's Specialty Healthcare

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Bone Mineral Density (BMD) (measured by DXA)	Change from baseline in BMD, as , at 10 months.	10 months
Primary	Bone Architecture (measured by pQCT)	Change from baseline in bone architecture, as measured by pQCT, at 10 months.	10 months
Secondary	Pediatric Quality of Life Inventory (PedsQL)	Change from baseline in PedsQL scores at 10 months.	10 months
Secondary	Neuromuscular Module of the PedsQL	Change from baseline in PedsQL neuromuscular module scores at 10 months.	10 months
Secondary	Change in pulmonary function test	Change from baseline in pulmonary function at 10 months.	10 months
Secondary	Change in Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD)	Change from baseline in CPCHILD score at 10 months.	10 Months