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Completed

NCT Number: NCT01783509

Limb Girdle Muscular Dystrophy (LGMD) Natural History

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.

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Key information

Conditions

Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED) Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

6 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Carolinas Medical Center - Pediatrics

Charlotte, North Carolina, 28203, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. 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

Sponsors and collaborators

Lead sponsor

Wake Forest University Health Sciences

Other

Collaborators

  • Muscular Dystrophy Association

Registry information

Official study title

Longitudinal Assessment and Genetic Understanding of Limb-Girdle Muscular Dystrophy

Important dates

Study start
2011
Primary completion
2017
Study completion
2017
First posted
Feb 5, 2013
Registry last updated
Apr 28, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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