Limb Girdle Muscular Dystrophy (LGMD) Natural History

NCT ID: NCT01783509

Last Updated: 2022-04-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

13 participants

Study Classification

OBSERVATIONAL

Study Start Date

2011-11-30

Study Completion Date

2017-07-31

Brief Summary

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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 Description

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Conditions

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Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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LGMD

Patients with GENETICALLY CONFIRMED limb girdle muscular dystrophy

No interventions assigned to this group

Eligibility Criteria

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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
Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Muscular Dystrophy Association

OTHER

Sponsor Role collaborator

Wake Forest University Health Sciences

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Ibrahim Binalsheikh, MD

Role: PRINCIPAL_INVESTIGATOR

Carolinas Medical Center - Pediatrics

Locations

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Carolinas Medical Center - Pediatrics

Charlotte, North Carolina, United States

Site Status

Countries

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United States

Other Identifiers

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LGMD Nat Hx

Identifier Type: -

Identifier Source: org_study_id

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