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.
|Contact: Laurence Callendretfirstname.lastname@example.org|
|United States, California|
|UC Davis Health System||Not yet recruiting|
|Sacramento, California, United States, 95817|
|Contact: Erica Goude 916-734-0968 email@example.com|
|Principal Investigator: Erik Henricson, MPH|
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center||Recruiting|
|Cincinnati, Ohio, United States, 45229|
|Contact: Lauren Miller, BA 513-636-3202 firstname.lastname@example.org|
|Contact: Paula Morehart +1-513-636-8967 email@example.com|
|Principal Investigator: Brenda Wong, MD|
|Nationwide Children's Hospital||Not yet recruiting|
|Columbus, Ohio, United States, 43205|
|Contact: Susan Gailey 614-355-2897 firstname.lastname@example.org|
|Principal Investigator: Kevin Flanigan, MD|
|Contact: Lien Everaert +3293321954 Lien.Everaert@uzgent.be|
|Principal Investigator: Nicolas DeConinck, MD|
|Universitair Ziekenhuis Leuven||Recruiting|
|Contact: Corine Antonis +3216343845 email@example.com|
|Principal Investigator: Nathalie Goemans, MD|
|Hospital das Clinicas da Faculdade de Medicina da USP||Not yet recruiting|
|Sao Paulo, Brazil|
|Principal Investigator: Maria Bernadete Dutra Resende, MD|
|CHU Hopital des enfants||Not yet recruiting|
|Principal Investigator: Claude Cances, MD|
|Contact: Barbel Leiendecker firstname.lastname@example.org|
|Principal Investigator: Ulrike Schara, MD|
|Contact: Gudrun Kohlmeier +4976127044740 email@example.com|
|Principal Investigator: Jan-Bernd Kirschner, MD|
|Sub-Investigator: Petra Boettler, MD|
|Azienda Ospedaliera Universitaria Policlinico G. Martino||Recruiting|
|Contact: Sonia Messina, MD +390902217178 firstname.lastname@example.org|
|Principal Investigator: Giuseppe Vita, MD|
|Policlinico Univsersitario Agostino Gemelli||Recruiting|
|Contact: Marika Pane email@example.com|
|Principal Investigator: Eugenio Mercuri, MD|
|Leids Universitair Medisch Centrum||Not yet recruiting|
|Principal Investigator: Erik Niks, MD|
|UMC St. Radboud||Not yet recruiting|
|Principal Investigator: Lilian Sie, MD|
|Drottning Silvias Barn- ochungdomssjukhus||Recruiting|
|Contact: Ulrika Sterky +46 31 3438207 firstname.lastname@example.org|
|Principal Investigator: Mar Tulinius, MD|
|Hacettepe University Medical Faculty||Not yet recruiting|
|Contact: Ayse Karaduman email@example.com|
|Principal Investigator: Haluk Topaloglu, MD|
|Principal Investigator:||Nathalie Goemans, MD||UZ Leuven, Belgium|