Clinical trial NCT00312247: Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy
Official title: Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Duchenne Muscular Dystrophy
Keywords: Walking
Muscle Strength
Energy Cost
Quality of Life
Lead sponsor: Shriners Hospitals for Children
Sponsor class: OTHER
First posted: 2006-04-07
Enrollment: 85
Sex: MALE
Minimum age (years): 4
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Boys with DMD who are ambulatory starting at the age of 4 until ambulation ceases

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.

Eligibility 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

Primary outcomes:
measure: Gait pattern
timeFrame: every six months (2x/year)
Secondary outcomes:
measure: muscle strength
timeFrame: every six months (2x/year)
measure: energy cost of walking
timeFrame: every six months (2x/year)
measure: gross motor functional skills
timeFrame: every six months (2x/year)
measure: Step activity Monitor-participation
timeFrame: one week every six months
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: UCLA Department of Orthopaedic Surgery
state: California
city: Sacramento
country: United States
facility: Shriners Hospitals for Children
state: California
city: Portland
country: United States
facility: Shriners Hospitals for Children
state: Oregon
Study officials:
affiliation: Shriners Hospitals for Children
name: Michael D Sussman, MD
role: PRINCIPAL_INVESTIGATOR
References:
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: 10.1177/0883073810371002. Epub 2010 Jun 29.
citation: 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.