A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
|Study Design:||Observational Model: Case-Only
Time Perspective: Prospective
|Official Title:||A Prospective Natural History Study of Progression of Physical Impairment, Activity Limitation and Quality of Life in Duchenne Muscular Dystrophy.|
- 6 minute walk distance [ Time Frame: Change from visit 1 walking distance ] [ Designated as safety issue: No ]Participants are asked to walk at their own preferred speed on a fixed distance for 6 minutes. Subjects are warned of the time and that they may stop earlier if they feel unable to continue. Total distance walked within 6 minutes (or until stopping) is recorded.
Biospecimen Retention: Samples Without DNA
|Study Start Date:||September 2012|
|Estimated Study Completion Date:||December 2017|
|Estimated Primary Completion Date:||December 2016 (Final data collection date for primary outcome measure)|
All participants will follow the same protocol, including muscle strength and function testing, and blood and urine collection, for a maximum of 7 visits over 3 years.
Other: Observational study
There is no medication or device tested in this study. This is an obversational study on the progression of the disease.
This is a prospective study. All DMD patients that fulfil the inclusion/exclusion criteria are eligible although the study is weighted towards ambulant subjects aged 3 years or older. There will be 7 study visits and subjects will be in the study for a maximum of 3 years. Visits will occur every 6 months (+/- 1 month).
Up to 250 DMD subjects planned in the following categories :
- 75 % ambulant subjects aged between 3 and 18 years at study entry
- 25% non-ambulant subjects with a maximum age of 18 years at study entry
Subjects will be asked to perform muscle testing assessment with a clinical evaluator, such as walking for 6 minutes, climb stairs, breathe in a tube, see how they can move their arms and legs. They will be asked questions about how they feel overall and perform daily activities. These measurements will be assessed every 6 months.
Urine and blood samples will be collected once a year to measure biomarkers that will allow to have a better overview of DMD.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01753804
|United States, California|
|UC Davis Health System|
|Sacramento, California, United States, 95817|
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229|
|Nationwide Children's Hospital|
|Columbus, Ohio, United States, 43205|
|Hospital de Pediatria Prof Dr Juan P Garrahan|
|Buenos Aires, Argentina|
|Universitair Ziekenhuis Leuven|
|Hospital das Clinicas da Faculdade de Medicina da USP|
|Sao Paulo, Brazil|
|CHU Hopital des enfants|
|Azienda Ospedaliera Universitaria Policlinico G. Martino|
|Policlinico Univsersitario Agostino Gemelli|
|Leids Universitair Medisch Centrum|
|UMC St. Radboud|
|Drottning Silvias Barn- ochungdomssjukhus|
|Hacettepe University Medical Faculty|
|Principal Investigator:||Nathalie Goemans, MD||UZ Leuven, Belgium|