A nationwide natural history study in Becker muscular dystrophy: Modeling a slowly progressive neuromuscular disease to prepare for clinical trials.
- Conditions
- Becker muscular dystrofie / BMD10029317
- Registration Number
- NL-OMON52088
- Lead Sponsor
- eids Universitair Medisch Centrum
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 75
BMD patient cohort:
• Male
• Confirmation of diagnosis of BMD by DNA analysis
• Age >=5 years
In order to be eligible to partake in the muscle biopsy part of this study, the
following additional inclusion criteria apply:
• Age >= 18 years
BMD trial participants cohort:
• Male
• Confirmation of diagnosis of BMD by DNA analysis
• Age >=5 years
• Inclusion in the phase II study of EDG-5506 in BMD (P22-064)
Healthy controls:
• Male
• Age >=5 years
In order to be eligible to partake in the muscle biopsy part of this study, the
following additional inclusion criteria apply:
• Age >= 18 years
Patients will not undergo MRI or muscle biopsy if they have contraindication
for the MRI (such as a metal implant) or muscle biopsy (such as use of
anticoagulation) respectively. If this is the case, it is still possible to
partake in the other parts of this study.
Study & Design
- Study Type
- Observational invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The main study endpoint is a model to predict the disease course of individual<br /><br>BMD patients based on the following parameters: clinical parameters (e.g. loss<br /><br>of ambulation), functional parameters (e.g. North Star Ambulatory assessment,<br /><br>strength measures), pulmonary parameters (e.g. forced vital capacity), cardiac<br /><br>parameters (e.g. left ventricular ejection fraction), muscle MRI<br /><br>characteristics (e.g. fat fraction) and biomarkers in serum (e.g.<br /><br>creatine/creatinine ratio).</p><br>
- Secondary Outcome Measures
Name Time Method