Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy Clinical Trial

Official title:

Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00312247
• Other study ID #: SHC-DMD-79115
• Secondary ID: SHC-79115
• Status: Completed
• Phase: N/A
• First received: April 5, 2006
• Last updated: May 18, 2015
• Start date: April 2006
• Est. completion date: March 2015

Study information

• Verified date: May 2015
• Source: Shriners Hospitals for Children
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 85
• Est. completion date: March 2015
• Est. primary completion date: December 2014
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 4 Years to 21 Years

Eligibility

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

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Locations

Country	Name	City	State
United States	UCLA Department of Orthopaedic Surgery	Los Angeles	California
United States	Shriners Hospitals for Children	Portland	Oregon
United States	Shriners Hospitals for Children	Sacramento	California

Sponsors (1)

Lead Sponsor	Collaborator
Shriners Hospitals for Children

Country where clinical trial is conducted

United States, 

References & Publications (2)

McDonald CM, McDonald DA, Bagley A, Sienko Thomas S, Buckon CE, Henricson E, Nicorici A, Sussman MD. Relationship between clinical outcome measures and parent proxy reports of health-related quality of life in ambulatory children with Duchenne muscular dy — View Citation

Sienko Thomas S, Buckon CE, Nicorici A, Bagley A, McDonald CM, Sussman MD. Classification of the gait patterns of boys with Duchenne muscular dystrophy and their relationship to function. J Child Neurol. 2010 Sep;25(9):1103-9. doi: 10.1177/088307381037100 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Gait pattern	computerized assessment of walking	every six months (2x/year)	No
Secondary	muscle strength	quantitative assessment of strength with a Biodex	every six months (2x/year)	No
Secondary	energy cost of walking	assessment of how much energy it takes to walk, assessed with a Cosmed K4b2	every six months (2x/year)	No
Secondary	gross motor functional skills	assessment of gross motor skills, ie getting up off the floor, ascending/descending stairs	every six months (2x/year)	No
Secondary	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	No

External Links

•   Shriners Hospitals for Children