Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy

Conditions
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Duchenne Muscular Dystrophy

Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 4 Years
Maximum Age: 21 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Boys taking steroids<br>Boys who are taking prednisone or deflazacort | |
| Boys who are steroid naive<br>Boys who are not taking steroids for a variety of reasons | |

What is the study measuring?
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Primary Outcome Measures

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

Secondary Outcome Measures

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

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Walking, Muscle Strength, Energy Cost, Quality of Life