Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy

NCT ID: NCT00312247

Last Updated: 2015-05-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

85 participants

Study Classification

OBSERVATIONAL

Study Start Date

2006-04-30

Study Completion Date

2015-03-31

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.

Conditions

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Duchenne Muscular Dystrophy

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Boys taking steroids

Boys who are taking prednisone or deflazacort

No interventions assigned to this group

Boys who are steroid naive

Boys who are not taking steroids for a variety of reasons

No interventions assigned to this group

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
Minimum Eligible Age

4 Years

Maximum Eligible Age

21 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Shriners Hospitals for Children

OTHER

Sponsor Role lead

Responsible Party

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Michael D. Sussman, MD

Principal Investigator

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Michael D Sussman, MD

Role: PRINCIPAL_INVESTIGATOR

Shriners Hospitals for Children

Locations

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UCLA Department of Orthopaedic Surgery

Los Angeles, California, United States

Site Status

Shriners Hospitals for Children

Sacramento, California, United States

Site Status

Shriners Hospitals for Children

Portland, Oregon, United States

Site Status

Countries

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United States

References

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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: 10.1177/0883073810371002. Epub 2010 Jun 29.

Reference Type RESULT
PMID: 20587736 (View on PubMed)

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 dystrophy. J Child Neurol. 2010 Sep;25(9):1130-44. doi: 10.1177/0883073810371509. Epub 2010 Jun 17.

Reference Type RESULT
PMID: 20558672 (View on PubMed)

Related Links

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http://www.Shrinershq.org

Shriners Hospitals for Children

Other Identifiers

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SHC-79115

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

SHC-DMD-79115

Identifier Type: -

Identifier Source: org_study_id

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