NCT01753804

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

A Prospective Natural History Study of Progression of Physical Impairment, Activity Limitation and Quality of Life in Duchenne Muscular Dystrophy.

• Status: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 269 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: Male
• Age: 3 Years – 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
OTHER	Observational study	There is no medication or device tested in this study. This is an obversational study on the progression of the disease.

Timeline

• Start date: 2012-09-01
• Primary completion: 2016-10-01
• Completion: 2016-10-01
• First posted: 2012-12-20
• Last updated: 2017-12-08

Locations

16 sites across 10 countries: United States, Argentina, Belgium, Brazil, France, Germany, Italy, Netherlands, Sweden, Turkey (Türkiye)