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Effects of Standing on Non-Ambulatory Children With Spinal Muscular Atrophy

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02341053
First Posted: January 19, 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.
Collaborator:
University of Minnesota, MN
Information provided by (Responsible Party):
Gillette Children's Specialty Healthcare
  Purpose

Non-ambulatory children with a neuromuscular disability such as spinal muscular atrophy (SMA) are at significant risk for poor bone health as defined by low bone mineral density (BMD) and increased propensity to fracture. Poor bone health is thought to be related, at least in part, to abnormally low levels of load experienced by the skeleton. A common physical approach for increasing bone density is to stimulate the musculoskeletal system by increasing the amount and duration of weight-bearing in the lower extremities. For non-ambulatory individuals, this takes the form of using an assisted standing device to enable the child to spend time in a standing position with some degree of weight placed on the lower limbs. Some of these physical interventions demonstrate variable improvement in BMD in children with neuromuscular conditions, and some do not. A serious limitation in the previous work in this area is a failure to objectively measure the magnitude and duration of the loading experienced by the lower extremities. Thus, a lack of change in BMD may be due to the extremities experiencing only a fraction of the body weight (due to load-sharing with the assistive device) for an inadequate duration of standing time.

In order to investigate the efficacy of standing treatment for increasing BMD, the investigators will develop a simple, portable and inexpensive transducer that will measure the magnitude and time course of the load experienced by the lower extremities of individuals with SMA who use a stationary assisted standing device. The specific goal of this proposed project will be to develop, validate and establish the initial feasibility of such a measurement device.


Condition Intervention
Spinal Muscular Dystrophy Neuromuscular Disability 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 Spinal Muscular Atrophy

Resource links provided by NLM:


Further study details as provided by Gillette Children's Specialty Healthcare:

Primary Outcome Measures:
  • Collection of load magnitude and duration data (Use load-sensing platforms to collect load magnitude and duration during standing treatment program) [ Time Frame: 12 months ]
    Use load-sensing platforms to collect load magnitude and duration during standing treatment program.


Secondary Outcome Measures:
  • Bone Mineral Density [ Time Frame: 12 months ]
    Determine whether an increase in the duration of standing leads to improvements in BMD, fracture incidence, pulmonary function and quality of life, and whether there is evidence that relationships exist between these outcome measures and load magnitude and duration.


Estimated Enrollment: 6
Study Start Date: September 2015
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Load-measuring platform

Load-sensing 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: Assisted Standing Treatment Program. Assisted standing treatment program will gradually increase their duration of standing by up to 75% after the baseline phase.

Other: Assisted Standing Treatment Program
Assisted Standing Treatment Program - Assisted standing treatment program will be gradually increase duration of standing by up to 75% after baseline.

Detailed Description:

Background: Non-ambulatory children with a neuromuscular disability such as spinal muscular atrophy (SMA) are at significant risk for poor bone health as defined by low bone mineral density (BMD) and increased propensity to fracture. Poor bone health is thought to be related, at least in part, to abnormally low levels of load experienced by the skeleton. A common physical approach for increasing bone density is to stimulate the musculoskeletal system by increasing the amount and duration of weight-bearing in the lower extremities. For non-ambulatory individuals, this takes the form of using an assisted standing device to enable the child to spend time in a standing position with some degree of weight placed on the lower limbs. Some of these physical interventions demonstrate variable improvement in BMD in children with neuromuscular conditions, and some do not. A serious limitation in the previous work in this area is a failure to objectively measure the magnitude and duration of the loading experienced by the lower extremities. Thus, a lack of change in BMD may be due to the extremities experiencing only a fraction of the body weight (due to load-sharing with the assistive device) for an inadequate duration of standing time.

Methods: In order to investigate the efficacy of standing treatment for increasing BMD, we will develop a simple, portable and inexpensive transducer that will measure the magnitude and time course of the load experienced by the lower extremities of individuals with SMA who use a stationary assisted standing device. This transducer will consist of an array of sensors embedded within a mat that will be placed under an individual's feet while they are in assisted standing. A prototype will be validated by comparing the output of this load-sensing mat against known loads that will be applied to the surface of the mat statically. The specific characteristics of the loading in the lower extremities of subjects can then be correlated with changes in their BMD to obtain a true outcome assessment. As a demonstration of this approach, we will recruit 3 children with SMA from Gillette Children's Specialty Healthcare who are currently participating in a standing program. Once enrolled and informed consent is obtained, the participants will use the mat provided to them to collect load and duration data during a baseline period where they will continue their standing program, and during a subsequent intervention period where they will increase the duration of their standing. The specific goal of this proposed project will be to develop, validate and establish the initial feasibility of such a measurement device.

Implications: Successful completion of this proposed project will provide the necessary groundwork to move forward with a multicenter clinical trial on a large number of subjects with a variety of neuromuscular conditions. The measurement device will allow us to correlate change in BMD with the specific load history for each participant, thereby allowing us to determine whether physical interventions can lead to increased BMD in individuals with neuromuscular disabilities. Analysis of the data will help define a dose-dependent relationship between load, duration and changes in BMD, thereby guiding providers and therapists in prescribing standing interventions.

  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. Age 3-14 years old
  2. Diagnosed with Spinal Muscular Atrophy (SMA)
  3. Participating in a standing program using a stationary assisted standing device.
  4. Gross Motor Function Classification System (GMFCS) Level IV or V
  5. Parent must be able to provide consent.

Exclusion Criteria:

1. Currently on 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): NCT02341053


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

Responsible Party: Gillette Children's Specialty Healthcare
ClinicalTrials.gov Identifier: NCT02341053     History of Changes
Other Study ID Numbers: 1411M55281
First Submitted: January 2, 2015
First Posted: January 19, 2015
Last Update Posted: September 11, 2017
Last Verified: September 2017

Keywords provided by Gillette Children's Specialty Healthcare:
SMA
Neuromuscular disability

Additional relevant MeSH terms:
Atrophy
Muscular Dystrophies
Muscular Atrophy
Muscular Atrophy, Spinal
Pathological Conditions, Anatomical
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Neuromuscular Manifestations
Neurologic Manifestations
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases