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| ID | Type | Description | Link |
|---|---|---|---|
| SHC-79115 | Other Grant/Funding Number | Shriners Hospitals for Children |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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 |
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| Measure | Description | Time Frame |
|---|---|---|
| Gait pattern | computerized assessment of walking | every six months (2x/year) |
| Measure | Description | Time Frame |
|---|---|---|
| muscle strength | quantitative assessment of strength with a Biodex | every six months (2x/year) |
| energy cost of walking | assessment of how much energy it takes to walk, assessed with a Cosmed K4b2 |
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Inclusion Criteria:
Exclusion Criteria:
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Boys with DMD who are ambulatory starting at the age of 4 until ambulation ceases
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| Name | Affiliation | Role |
|---|---|---|
| Michael D Sussman, MD | Shriners Hospitals for Children | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCLA Department of Orthopaedic Surgery | Los Angeles | California | 90095 | United States | ||
| Shriners Hospitals for Children |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 20587736 | Result | 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. | |
| 20558672 | Result | 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. |
| Label | URL |
|---|---|
| Shriners Hospitals for Children | View source |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| every six months (2x/year) |
| gross motor functional skills | assessment of gross motor skills, ie getting up off the floor, ascending/descending stairs | every six months (2x/year) |
| 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 |
| Sacramento |
| California |
| 95817 |
| United States |
| Shriners Hospitals for Children | Portland | Oregon | 97239 | United States |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |