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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.
 
ClinicalTrials.gov Identifier: NCT01783509
Recruitment Status : Completed
First Posted : February 5, 2013
Last Update Posted : July 19, 2017
Sponsor:
Collaborator:
Muscular Dystrophy Association
Information provided by (Responsible Party):
Atrium Health

Tracking Information
First Submitted Date January 18, 2013
First Posted Date February 5, 2013
Last Update Posted Date July 19, 2017
Study Start Date November 2011
Actual Primary Completion Date July 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: February 1, 2013)
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 ]
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Limb Girdle Muscular Dystrophy (LGMD) Natural History
Official Title Longitudinal Assessment and Genetic Understanding of Limb-Girdle Muscular Dystrophy
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.
Detailed Description Not Provided
Study Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration 20 Years
Biospecimen Retention:   Samples Without DNA
Description:
Blood
Sampling Method Non-Probability Sample
Study Population Patients with a diagnosis of GENETICALLY CONFIRMED LGMD
Condition Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)
Intervention Not Provided
Study Groups/Cohorts LGMD
Patients with GENETICALLY CONFIRMED limb girdle muscular dystrophy
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: July 18, 2017)
13
Original Estimated Enrollment
 (submitted: February 1, 2013)
100
Actual Study Completion Date July 2017
Actual Primary Completion Date July 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria

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
Sex/Gender
Sexes Eligible for Study: All
Ages 6 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT01783509
Other Study ID Numbers LGMD Nat Hx
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Atrium Health
Study Sponsor Atrium Health
Collaborators Muscular Dystrophy Association
Investigators
Principal Investigator: Ibrahim Binalsheikh, MD Carolinas Medical Center - Pediatrics
PRS Account Atrium Health
Verification Date July 2017