This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy (DMD)
Recruitment status was Recruiting
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.
Duchenne Muscular Dystrophy
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||Clinical Outcomes Validation in Non Ambulatory and Young Boys/Men With Duchenne Muscular Dystrophy (DMD)|
- Dystrophin gene mutations that predispose to early onset cardiomyopathy [ Time Frame: 3 years ] [ Designated as safety issue: No ]
|Study Start Date:||March 2010|
|Estimated Study Completion Date:||October 2012|
|Estimated Primary Completion Date:||October 2012 (Final data collection date for primary outcome measure)|
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.
|Contact: Pallavi Anandfirstname.lastname@example.org|
|Contact: Charlie Wulfemail@example.com|
|United States, Missouri|
|St. Louis, Missouri, United States, 63110|
|Contact: Pallavi Anand 314-362-2490 firstname.lastname@example.org|
|Contact: Charlie Wulf 314-362-6980 email@example.com|
|Principal Investigator: Anne M Connolly, M.D.|
|Principal Investigator:||Anne Connolly, M.D.||Washington University School of Medicine|