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

Terminated

• Conditions: Duchenne Muscular Dystrophy

• Registration Number: NCT01753804
• Lead Sponsor: BioMarin Pharmaceutical

Brief 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.

Detailed Description

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 Timeline

• Study Start: 2012-09-01
• Study First Submitted: 2012-12-13
• Study First Submitted QC: 2012-12-17
• Study First Posted: 2012-12-20
• Primary Completion: 2016-10-01
• Study Completion: 2016-10-01
• Status Verified: 2017-12
• Last Update Submitted: 2017-12-06
• Last Update Posted: 2017-12-08

Recruitment & Eligibility

• Status: TERMINATED
• Sex: Male
• Target Recruitment: 269

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 IMP

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
6 minute walk distance	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.

Trial Locations

Locations (16)

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

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