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Limb Girdle Muscular Dystrophy (LGMD) Natural History

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01783509
Recruitment Status : Completed
First Posted : February 5, 2013
Last Update Posted : April 28, 2022
Muscular Dystrophy Association
Information provided by (Responsible Party):
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.

Condition or disease
Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)

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Study Type : Observational [Patient Registry]
Actual Enrollment : 13 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 20 Years
Official Title: Longitudinal Assessment and Genetic Understanding of Limb-Girdle Muscular Dystrophy
Study Start Date : November 2011
Actual Primary Completion Date : July 2017
Actual Study Completion Date : July 2017

Patients with GENETICALLY CONFIRMED limb girdle muscular dystrophy

Primary Outcome Measures :
  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 ]

Biospecimen Retention:   Samples Without DNA

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with a diagnosis of GENETICALLY CONFIRMED LGMD

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01783509

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United States, North Carolina
Carolinas Medical Center - Pediatrics
Charlotte, North Carolina, United States, 28203
Sponsors and Collaborators
Wake Forest University Health Sciences
Muscular Dystrophy Association
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Principal Investigator: Ibrahim Binalsheikh, MD Carolinas Medical Center - Pediatrics
Additional Information:

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Responsible Party: Wake Forest University Health Sciences Identifier: NCT01783509    
Other Study ID Numbers: LGMD Nat Hx
First Posted: February 5, 2013    Key Record Dates
Last Update Posted: April 28, 2022
Last Verified: July 2017
Keywords provided by Wake Forest University Health Sciences:
GENETICALLY CONFIRMED limb girdle muscular dystrophy
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophies, Limb-Girdle
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn