Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Michael D. Sussman, MD, Shriners Hospitals for Children
ClinicalTrials.gov Identifier:
NCT00312247
First received: April 5, 2006
Last updated: April 28, 2014
Last verified: April 2014
  Purpose

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.


Condition
Duchenne Muscular Dystrophy

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy

Resource links provided by NLM:


Further study details as provided by Shriners Hospitals for Children:

Primary Outcome Measures:
  • Gait pattern [ Time Frame: every six months (2x/year) ] [ Designated as safety issue: No ]
    computerized assessment of walking


Secondary Outcome Measures:
  • muscle strength [ Time Frame: every six months (2x/year) ] [ Designated as safety issue: No ]
    quantitative assessment of strength with a Biodex

  • energy cost of walking [ Time Frame: every six months (2x/year) ] [ Designated as safety issue: No ]
    assessment of how much energy it takes to walk, assessed with a Cosmed K4b2

  • gross motor functional skills [ Time Frame: every six months (2x/year) ] [ Designated as safety issue: No ]
    assessment of gross motor skills, ie getting up off the floor, ascending/descending stairs

  • Step activity Monitor-participation [ Time Frame: one week every six months ] [ Designated as safety issue: No ]
    measurement of the number of steps taken in the community/home environment during weekdays and weekends


Estimated Enrollment: 85
Study Start Date: April 2006
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Groups/Cohorts
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

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.

  Eligibility

Ages Eligible for Study:   4 Years to 21 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

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

Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00312247

Locations
United States, California
UCLA Department of Orthopaedic Surgery
Los Angeles, California, United States, 90095
Shriners Hospitals for Children
Sacramento, California, United States, 95817
United States, Oregon
Shriners Hospitals for Children
Portland, Oregon, United States, 97239
Sponsors and Collaborators
Shriners Hospitals for Children
Investigators
Principal Investigator: Michael D Sussman, MD Shriners Hospitals for Children
  More Information

Additional Information:
Publications:
Responsible Party: Michael D. Sussman, MD, Principal Investigator, Shriners Hospitals for Children
ClinicalTrials.gov Identifier: NCT00312247     History of Changes
Other Study ID Numbers: SHC-DMD-79115, SHC-79115
Study First Received: April 5, 2006
Last Updated: April 28, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by Shriners Hospitals for Children:
Walking
Muscle Strength
Energy Cost
Quality of Life

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on August 26, 2014