Limb Girdle Muscular Dystrophy (LGMD) Natural History

Overview

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.

Full Title of Study: “Longitudinal Assessment and Genetic Understanding of Limb-Girdle Muscular Dystrophy”

Study Type

  • Study Type: Observational [Patient Registry]
  • Study Design
    • Time Perspective: Prospective
  • Study Primary Completion Date: July 2017

Arms, Groups and Cohorts

  • LGMD
    • Patients with GENETICALLY CONFIRMED limb girdle muscular dystrophy

Clinical Trial Outcome Measures

Primary Measures

  • 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

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 6 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Atrium Health
  • Collaborator
    • Muscular Dystrophy Association
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Ibrahim Binalsheikh, MD, Principal Investigator, Carolinas Medical Center – Pediatrics

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