A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
To characterize the natural history and progression of Duchenne Muscular Dystrophy (DMD) to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible.
|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
Blood sampling at 4 time points (first visit then once a year). Urinalysis sampling at 4 time points (first visit then once a year).
|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|