A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
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|ClinicalTrials.gov Identifier: NCT01753804|
Recruitment Status : Terminated
First Posted : December 20, 2012
Last Update Posted : December 8, 2017
|Condition or disease||Intervention/treatment|
|Duchenne Muscular Dystrophy||Other: Observational study|
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.
|Study Type :||Observational|
|Actual Enrollment :||269 participants|
|Official Title:||A Prospective Natural History Study of Progression of Physical Impairment, Activity Limitation and Quality of Life in Duchenne Muscular Dystrophy.|
|Actual Study Start Date :||September 1, 2012|
|Actual Primary Completion Date :||October 1, 2016|
|Actual Study Completion Date :||October 1, 2016|
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.
- 6 minute walk distance [ Time Frame: Change from visit 1 walking distance ]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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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|