A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
NCT ID: NCT01753804
Last Updated: 2017-12-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
269 participants
OBSERVATIONAL
2012-09-01
2016-10-01
Brief Summary
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Detailed Description
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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.
Conditions
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Keywords
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Study Design
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CASE_ONLY
PROSPECTIVE
Study Groups
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Study participants
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.
Observational study
There is no medication or device tested in this study. This is an obversational study on the progression of the disease.
Interventions
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Observational study
There is no medication or device tested in this study. This is an obversational study on the progression of the disease.
Eligibility Criteria
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Inclusion Criteria
* Age 3 - 18 years
* Willing and able to comply with protocol requirements
* Life expectancy of at least 3 years
* Able to give informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations)
Exclusion Criteria
* Participation within the previous 1 month in a clinical study with an IMP
3 Years
18 Years
MALE
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Principal Investigators
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Nathalie Goemans, MD
Role: PRINCIPAL_INVESTIGATOR
UZ Leuven, Belgium
Locations
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UC Davis Health System
Sacramento, California, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Hospital de Pediatria Prof Dr Juan P Garrahan
Buenos Aires, , Argentina
Universitair Ziekenhuis
Ghent, , Belgium
Universitair Ziekenhuis Leuven
Leuven, , Belgium
Hospital das Clinicas da Faculdade de Medicina da USP
São Paulo, , Brazil
CHU Hopital des enfants
Toulouse, , France
Universitaetsklinikum Essen
Essen, , Germany
Universitaetsklinikum Freiburg
Freiburg im Breisgau, , Germany
Azienda Ospedaliera Universitaria Policlinico G. Martino
Messina, , Italy
Policlinico Univsersitario Agostino Gemelli
Rome, , Italy
Leids Universitair Medisch Centrum
Leiden, , Netherlands
UMC St. Radboud
Nijmegen, , Netherlands
Drottning Silvias Barn- ochungdomssjukhus
Gothenburg, , Sweden
Hacettepe University Medical Faculty
Ankara, , Turkey (Türkiye)
Countries
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Related Links
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BioMarin website
Other Identifiers
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PRO-DMD-01
Identifier Type: -
Identifier Source: org_study_id