CompletedNot Applicable

Limb Girdle Muscular Dystrophy (LGMD) Natural History

United States13 participantsStarted 2011-11

Plain-language 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.

Who can participate

Age range6 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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 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

What they're measuring

Comprehensive clinical evaluation of individuals with GENETICALLY CONFIRMED LGMD, according to the study protocol, in order to evaluate disease progression

Timeframe: yearly up to 10 years

Trial details

NCT IDNCT01783509

SponsorWake Forest University Health Sciences

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2017-07

View on ClinicalTrials.gov More Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED) trials