Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy

Completed

• Conditions: Duchenne Muscular Dystrophy

• Registration Number: NCT00312247
• Lead Sponsor: Shriners Hospitals for Children

Brief Summary

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

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 Timeline

• Study Start: 2006-04
• Study First Submitted: 2006-04-05
• Study First Submitted QC: 2006-04-05
• Study First Posted: 2006-04-07
• Primary Completion: 2014-12
• Study Completion: 2015-03
• Status Verified: 2015-05
• Last Update Submitted: 2015-05-18
• Last Update Posted: 2015-05-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 85

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Gait pattern	every six months (2x/year)	computerized assessment of walking

Secondary Outcome Measures

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

Trial Locations

Locations (2)

UCLA Department of Orthopaedic Surgery; 🇺🇸 Los Angeles, California, United States

Shriners Hospitals for Children; 🇺🇸 Portland, Oregon, United States