Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2025-12-24 @ 9:08 PM
Ignite Modification Date:
2025-12-24 @ 9:08 PM
NCT ID:
NCT01753804
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:
NCT01753804
Study Brief:
Protocol Section:
NCT01753804