A Prospective Natural History Study of the Progression of Physical Impairment, Activity Limitation and Quality of Life in Duchenne Muscular Dystrophy (DMD)
- Conditions
- 1002839610028302Duchenne Muscular Dystrophy (DMD)muscular dystrophy
- Registration Number
- NL-OMON39538
- Lead Sponsor
- Prosensa Therapeutics B.V.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- Not specified
- Target Recruitment
- 30
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 invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>This is an exploratory observational study with no formal statistical<br /><br>hypotheses. All data will be summarised at each time point. In general,<br /><br>categorical data will be presented using counts and percentages, whilst<br /><br>continual variable will be presented using the mean, standard deviation,<br /><br>median, minimum, maximum and number of patients.<br /><br>Interim analyses will be conducted at yearly intervals during this study. </p><br>
- Secondary Outcome Measures
Name Time Method <p>N/A</p><br>