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

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Trial Status Terminated

Trial Identifier

NCT01753804

Condition

Duchenne Muscular Dystrophy

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial Summary

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.

Eligibility Criteria

Birth Sex

Male

Age

3 Years to 18 Years

Healthy Volunteers

No

Drug/Treatment:

Observational study

Study Type:

Observational

Number of Participants:

269

Study Started:

2012-09-01

Study Updated:

2017-12-08

Trial Locations

  • 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

    Gent, , Belgium

  • Universitair Ziekenhuis Leuven

    Leuven, , Belgium

  • Hospital das Clinicas da Faculdade de Medicina da USP

    Sao Paulo, , Brazil

  • CHU Hopital des enfants

    Toulouse, , France

  • Universitaetsklinikum Essen

    Essen, , Germany

  • Universitaetsklinikum Freiburg

    Freiburg, , 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

    Goteborg, , Sweden

  • Hacettepe University Medical Faculty

    Ankara, , Turkey

Inclusion Criteria

  • 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)

Exclusion Criteria

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

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