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临床试验/NCT00312247
NCT00312247已完成不适用

Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy

Shriners Hospitals for Children2 个研究点 分布在 1 个国家目标入组 85 人开始时间: 2006年4月最近更新:
适应症

试验速览

阶段
不适用
状态
已完成
入组人数
85
试验地点
2
主要终点
Gait pattern

研究概览

简要总结

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.

详细描述

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.

研究设计

研究类型
Observational
观察模型
Cohort
时间视角
Prospective

入排标准

年龄范围
4 Years 至 21 Years(Child, Adult)
性别
Male
接受健康志愿者

入选标准

  • Confirmed diagnosis of DMD
  • 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.

排除标准

  • Nonambulatory

结局指标

主要结局

Gait pattern

时间窗: every six months (2x/year)

computerized assessment of walking

次要结局

  • muscle strength(every six months (2x/year))
  • gross motor functional skills(every six months (2x/year))
  • energy cost of walking(every six months (2x/year))
  • Step activity Monitor-participation(one week every six months)

研究者

申办方类型
Other
责任方
Principal Investigator
主要研究者

Michael D. Sussman, MD

Principal Investigator

Shriners Hospitals for Children

研究点 (2)

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