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

Completed

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT01098708

Run by

Washington University School of Medicine

People needed

50

Starts

2010-03-01

Expected to finish

2013-11-01

Last updated by the study team

2017-01-18

Who can join

Age: 7 and older, up to 22. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• For Non-ambulatory study, the inability to understand and cooperate with the testing would exclude a subject. -

Where it is running

• Washington University — St Louis, Missouri, United States

Full record on ClinicalTrials.gov

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