Limb Girdle Muscular Dystrophy (LGMD) Natural History
Completed
- Conditions
- Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)
- Registration Number
- NCT01783509
- Lead Sponsor
- Wake Forest University Health Sciences
- Brief Summary
The purpose of this study is to understand more about limb-girdle muscular dystrophy. Therefore, the investigators would like to track the following information collected once a year from patients with GENETICALLY CONFIRMED LGMD: quality of life questionnaires, muscle strength, motor function, routine examination, assessment of patient (or parent) understanding of LGMD, and serum (blood) for growth factors, cytokines, and biomarkers (all parts of your blood). By tracking this information, we hope to be able to understand more about the diagnosis, progression and natural history of this disorder.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 13
Inclusion Criteria
- You have a GENETICALLY CONFIRMED diagnosis of Limb Girdle Muscular Dystrophy
- You must be at least 6 years of age or older (if under 18 you will need Parental consent)
- You must be able to travel to the study site
- You must be able to provide a DNA/Gene testing report that confirms a diagnosis of LGMD
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Exclusion Criteria
- You or your child do not have a diagnosis of LGMD
- Your child is under age 6
- You or your child are not able to travel to the study site
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Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Comprehensive clinical evaluation of individuals with GENETICALLY CONFIRMED LGMD, according to the study protocol, in order to evaluate disease progression yearly up to 10 years
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Carolinas Medical Center - Pediatrics
🇺🇸Charlotte, North Carolina, United States