A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.

Stopped early

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT01753804

Run by

BioMarin Pharmaceutical

People needed

269

Starts

2012-09-01

Expected to finish

2016-10-01

Last updated by the study team

2017-12-08

Who can join

Age: 3 and older, up to 18. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of DMD resulting from a mutation in the DMD gene confirmed by a state of the art DNA diagnostic technique covering all DMD gene exons.
• 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)

You may not qualify if…

• Current participation in a clinical study with an Investigational Medicinal Product (IMP)
• Participation within the previous 1 month in a clinical study with an IMP

Where it is running

• 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)

Full record on ClinicalTrials.gov

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