Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy

May 18, 2015 updated by: Michael D. Sussman, MD, Shriners Hospitals for Children
The purpose of this research study is to understand the walking patterns, strength and function changes of boys with Duchenne muscular dystrophy on/off corticosteroids to determine the best timing and treatment options to maintain walking for as long as possible.

Study Overview

Status

Completed

Conditions

Detailed Description

Duchenne muscular dystrophy (DMD) is an X-linked recessive disease of muscle characterized by a progressive loss of functional muscle mass, which is replaced with fibrofatty tissue. Historically, boys with DMD lose the ability to walk between the ages of 8-12 years, due to progressive weakness of the quadriceps coupled with the development of contractures at the hip, knee and ankle. This progressive loss in function necessitates individuals with DMD to spend less time walking and more time in wheelchairs, leading to the development of spinal deformities. Recently, corticosteroids have been shown to reduce the expected loss of muscle strength, extend the time that ambulation and standing are maintained, and minimize or eliminate spinal deformity in individuals with DMD; yet, the side effects of such treatment preclude use in some patients. To date, differences in gait patterns and other markers of disease progression between boys on corticosteroids and those not utilizing such treatment have not been objectively quantified. This lack of knowledge is a major obstacle to determining the most effective treatment for subsets of boys with DMD.

Study Type

Observational

Enrollment (Actual)

85

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90095
        • UCLA Department of Orthopaedic Surgery
      • Sacramento, California, United States, 95817
        • Shriners Hospitals for Children
    • Oregon
      • Portland, Oregon, United States, 97239
        • Shriners Hospitals for Children

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

4 years to 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Sampling Method

Non-Probability Sample

Study Population

Boys with DMD who are ambulatory starting at the age of 4 until ambulation ceases

Description

Inclusion Criteria:

  • Confirmed diagnosis of DMD
  • Male.
  • Four years of age or older.
  • Ability to walk independently for five minutes to 10 minutes at self-selected speed.
  • Ability to cognitively understand directions for testing procedures.

Exclusion Criteria:

  • Female
  • Nonambulatory

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Boys taking steroids
Boys who are taking prednisone or deflazacort
Boys who are steroid naive
Boys who are not taking steroids for a variety of reasons

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Gait pattern
Time Frame: every six months (2x/year)
computerized assessment of walking
every six months (2x/year)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
muscle strength
Time Frame: every six months (2x/year)
quantitative assessment of strength with a Biodex
every six months (2x/year)
energy cost of walking
Time Frame: every six months (2x/year)
assessment of how much energy it takes to walk, assessed with a Cosmed K4b2
every six months (2x/year)
gross motor functional skills
Time Frame: every six months (2x/year)
assessment of gross motor skills, ie getting up off the floor, ascending/descending stairs
every six months (2x/year)
Step activity Monitor-participation
Time Frame: one week every six months
measurement of the number of steps taken in the community/home environment during weekdays and weekends
one week every six months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Shriners Hospitals for Children

Investigators

  • Principal Investigator: Michael D Sussman, MD, Shriners Hospitals for Children

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2006

Primary Completion (Actual)

December 1, 2014

Study Completion (Actual)

March 1, 2015

Study Registration Dates

First Submitted

April 5, 2006

First Submitted That Met QC Criteria

April 5, 2006

First Posted (Estimate)

April 7, 2006

Study Record Updates

Last Update Posted (Estimate)

May 19, 2015

Last Update Submitted That Met QC Criteria

May 18, 2015

Last Verified

May 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SHC-DMD-79115
  • SHC-79115 (Other Grant/Funding Number: Shriners Hospitals for Children)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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