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Limb Girdle Muscular Dystrophy (LGMD) Natural History

Sponsor
Wake Forest University Health Sciences (Other)
Overall Status
Completed
CT.gov ID
NCT01783509
Collaborator
Muscular Dystrophy Association (Other)
13
Enrollment
1
Location
68
Duration (Months)
0.2
Patients Per Site Per Month

Study Details

Study Description

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 Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational [Patient Registry]
    Actual Enrollment :
    13 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    Longitudinal Assessment and Genetic Understanding of Limb-Girdle Muscular Dystrophy
    Study Start Date :
    Nov 1, 2011
    Actual Primary Completion Date :
    Jul 1, 2017
    Actual Study Completion Date :
    Jul 1, 2017

    Arms and Interventions

    Arm Intervention/Treatment
    LGMD

    Patients with GENETICALLY CONFIRMED limb girdle muscular dystrophy

    Outcome Measures

    Primary Outcome Measures

    1. Comprehensive clinical evaluation of individuals with GENETICALLY CONFIRMED LGMD, according to the study protocol, in order to evaluate disease progression [yearly up to 10 years]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    6 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Carolinas Medical Center - Pediatrics Charlotte North Carolina United States 28203

    Sponsors and Collaborators

    • Wake Forest University Health Sciences
    • Muscular Dystrophy Association

    Investigators

    • Principal Investigator: Ibrahim Binalsheikh, MD, Carolinas Medical Center - Pediatrics

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Wake Forest University Health Sciences
    ClinicalTrials.gov Identifier:
    NCT01783509
    Other Study ID Numbers:
    • LGMD Nat Hx
    First Posted:
    Feb 5, 2013
    Last Update Posted:
    Apr 28, 2022
    Last Verified:
    Jul 1, 2017
    Keywords provided by Wake Forest University Health Sciences
    GENETICALLY CONFIRMED limb girdle muscular dystrophy
    Additional relevant MeSH terms:
    Muscular Dystrophies
    Muscular Dystrophies, Limb-Girdle

    Study Results

    No Results Posted as of Apr 28, 2022