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

NCT02341053 | Completed DMC

• 1 other identifier: 1411M55281
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Spinal Muscular Dystrophy; Neuromuscular Disability

Keywords

SMA; Neuromuscular disability

Outcome Measures

Primary Outcomes (1)

• Collection of load magnitude and duration data (Use load-sensing platforms to collect load magnitude and duration during standing treatment program)

  Use load-sensing platforms to collect load magnitude and duration during standing treatment program.

  12 months

Secondary Outcomes (1)

• Bone Mineral Density

  12 months

Study Arms (1)

Load-measuring platform

OTHER

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

Interventions

Assisted Standing Treatment Program OTHER

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

Load-measuring platform

Eligibility Criteria

• Age: 3 Years - 14 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

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

You may not qualify if:

• \. Currently on bisphosphonates

Sponsors & Collaborators

• Gillette Children's Specialty Healthcare: lead
• University of Minnesota: collaborator

Study Sites (1)

Gillette Children's Specialty Healthcare

Saint Paul, Minnesota, 55101, United States

Location

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: SPONSOR

Study Record Dates

• First Submitted: January 2, 2015
• First Posted: January 19, 2015
• Study Start: September 8, 2016
• Primary Completion: July 25, 2018
• Study Completion: July 25, 2018
• Last Updated: February 17, 2021

Record last verified: 2021-02

Locations

US (1)