Clinical Trial: Limb Girdle Muscular Dystrophy (LGMD) Natural History

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational [Patient Registry]

Official Title: Longitudinal Assessment and Genetic Understanding of Limb-Girdle Muscular Dystrophy

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 Summary:
Sponsor: Carolinas Healthcare System

Current Primary Outcome: Comprehensive clinical evaluation of individuals with GENETICALLY CONFIRMED LGMD, according to the study protocol, in order to evaluate disease progression [ Time Frame: yearly up to 10 years ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Carolinas Healthcare System

Dates:
Date Received: January 18, 2013
Date Started: November 2011
Date Completion: November 2017
Last Updated: February 2, 2015
Last Verified: February 2015