Limb Girdle Muscular Dystrophy (LGMD) Natural History

April 20, 2022 updated by: Wake Forest University Health Sciences

Longitudinal Assessment and Genetic Understanding of Limb-Girdle Muscular Dystrophy

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.

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

13

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • North Carolina
      • Charlotte, North Carolina, United States, 28203
        • Carolinas Medical Center - Pediatrics

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with a diagnosis of GENETICALLY CONFIRMED LGMD

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
LGMD
Patients with GENETICALLY CONFIRMED limb girdle muscular dystrophy

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
yearly up to 10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Wake Forest University Health Sciences

Collaborators

Muscular Dystrophy Association

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2011

Primary Completion (Actual)

July 1, 2017

Study Completion (Actual)

July 1, 2017

Study Registration Dates

First Submitted

January 18, 2013

First Submitted That Met QC Criteria

February 1, 2013

First Posted (Estimate)

February 5, 2013

Study Record Updates

Last Update Posted (Actual)

April 28, 2022

Last Update Submitted That Met QC Criteria

April 20, 2022

Last Verified

July 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • LGMD Nat Hx

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)

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