This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy

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

50 participants

Study Classification

OBSERVATIONAL

Study Start Date

2010-03-31

Study Completion Date

2013-11-30

Brief Summary

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Many therapeutic trials in DMD exclude non-ambulatory boys and men. Rate of progression in these non-ambulatory patients has been studied but consensus has not been reached for what measures are most reliable and reproducible. Furthermore, any treatment trial would be expected to demonstrate improved function and improvement in quality of life. Therefore, function, strength, and quality of life must be understood and standardized. While the goal of this proposal is to standardize clinical outcomes for therapeutic trials, careful understanding of the progression of DMD in non ambulatory boys may also lead to better medical treatment.

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Detailed Description

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For Non-ambulatory study, boys must be unable to walk without assistive devices for greater than or equal to one year.

Age at onset of study for non-ambulatory boys and men: greater than or equal to 7 years through age 22 years.

Genetic or biopsy confirmation of dystrophinopathy or clinical diagnosis of DMD with biopsy/genetic confirmation in a primary relative.

Conditions

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Duchenne Muscular Dystrophy

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

1. For Non-ambulatory study, boys must be unable to walk without assistive devices for greater than or equal to one year.
2. Age at onset of study for non-ambulatory boys and men: greater than or equal to 7 years through age 22 years.
3. Genetic or biopsy confirmation of dystrophinopathy or clinical diagnosis of DMD with biopsy/genetic confirmation in a primary relative.