An observational study in Duchenne Muscular Dystrophy, sponsored by Washington University School of Medicine. Completed at 1 site in United States. Open to male participants aged 7 Years to 22 Years. Per ClinicalTrials.gov, last updated 2017-01-18.
Sponsored by Washington University School of Medicine · Observational
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.
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.
548 studies on the registry are indexed under Muscular Dystrophies; 89 are open to participants now.
This study's enrollment of 50 is below the median of 69 across 179 observational studies indexed under Muscular Dystrophies.
Browse Muscular Dystrophies studies →Washington University School of Medicine is the lead sponsor of 1,765 studies on the registry; 271 are open to participants now.
Of its 324 completed or terminated interventional studies of FDA-regulated products, 212 (65%) have results posted.
Counted across the registry records on this site, refreshed daily.
Non ambulatory boys and men with Duchenne muscular dystrophy
Exclusion Criteria:
Dystrophin gene mutations that predispose to early onset cardiomyopathy
Time frame: 3 years
Plan to share: No
No publications or documents are linked to this record.
This study is completed, as verified in Jan 2017. You cannot join it, but the record below documents what was studied.
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Washington University School of Medicine