NCT02428673

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
6

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Dec 2015

Typical duration for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 7, 2015

Completed
4 months until next milestone

First Posted

Study publicly available on registry

April 29, 2015

Completed
7 months until next milestone

Study Start

First participant enrolled

December 9, 2015

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 23, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 23, 2018

Completed
Last Updated

February 21, 2021

Status Verified

February 1, 2021

Enrollment Period

2.3 years

First QC Date

January 7, 2015

Last Update Submit

February 19, 2021

Conditions

OsteopeniaSpinal Muscular AtrophyCerebral PalsyMuscular DystrophySpina BifidaRett Syndrome

Keywords

Osteopeniaspinal muscular atrophycerebral palsymuscular dystrophyspina bifidaRett Syndrome

Outcome Measures

Primary Outcomes (2)

  • 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 Outcomes (4)

  • Pediatric Quality of Life Inventory (PedsQL)

    10 months

  • Neuromuscular Module of the PedsQL

    10 months

  • Change in pulmonary function test

    10 months

  • Change in Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD)

    10 Months

Study Arms (1)

Load-measuring platform

OTHER

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

Interventions

Assisted Standing Treatment ProgramOTHER
Load-measuring platform

Eligibility Criteria

Age3 Years - 14 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • \. Currently taking bisphosphonates

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Gillette Children's Specialty Healthcare

Saint Paul, Minnesota, 55101, United States

Location

MeSH Terms

Conditions

Bone Diseases, MetabolicMuscular Atrophy, SpinalCerebral PalsyMuscular DystrophiesSpinal DysraphismRett Syndrome

Condition Hierarchy (Ancestors)

Bone DiseasesMusculoskeletal DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesSpinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular DiseasesBrain Damage, ChronicBrain DiseasesMuscular Disorders, AtrophicMuscular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNeural Tube DefectsNervous System MalformationsCongenital AbnormalitiesX-Linked Intellectual DisabilityIntellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsGenetic Diseases, X-LinkedHeredodegenerative Disorders, Nervous System

Study Officials

  • Walter Truong, MD

    Gillette Children's Specialty Healthcare

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Pediatric Orthopedic Surgeon

Study Record Dates

First Submitted

January 7, 2015

First Posted

April 29, 2015

Study Start

December 9, 2015

Primary Completion

March 23, 2018

Study Completion

March 23, 2018

Last Updated

February 21, 2021

Record last verified: 2021-02

Locations

US(1)