Limb Girdle Muscular Dystrophy (LGMD) Natural History
This study is currently recruiting participants.
(see Contacts and Locations)
Verified February 2015 by Carolinas Healthcare System
Sponsor:
Carolinas Healthcare System
Collaborator:
Muscular Dystrophy Association
Information provided by (Responsible Party):
Amy D. Harper, Carolinas Healthcare System
ClinicalTrials.gov Identifier:
NCT01783509
First received: January 18, 2013
Last updated: February 2, 2015
Last verified: February 2015
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Purpose
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 |
|---|
|
Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED) |
| Study Type: | Observational [Patient Registry] |
| Study Design: | Observational Model: Cohort Time Perspective: Prospective |
| Target Follow-Up Duration: | 20 Years |
| Official Title: | Longitudinal Assessment and Genetic Understanding of Limb-Girdle Muscular Dystrophy |
Resource links provided by NLM:
Genetics Home Reference related topics:
limb-girdle muscular dystrophy
MedlinePlus related topics:
Muscular Dystrophy
Genetic and Rare Diseases Information Center resources:
Muscular Dystrophy
Limb-girdle Muscular Dystrophy
U.S. FDA Resources
Further study details as provided by Carolinas Healthcare System:
Primary Outcome Measures:
- 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 ] [ Designated as safety issue: No ]
Biospecimen Retention: Samples Without DNA
Blood
| Estimated Enrollment: | 100 |
| Study Start Date: | November 2011 |
| Estimated Study Completion Date: | November 2017 |
| Estimated Primary Completion Date: | November 2017 (Final data collection date for primary outcome measure) |
| Groups/Cohorts |
|---|
|
LGMD
Patients with GENETICALLY CONFIRMED limb girdle muscular dystrophy
|
Eligibility| Ages Eligible for Study: | 6 Years and older (Child, Adult, Senior) |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
Study Population
Patients with a diagnosis of GENETICALLY CONFIRMED LGMD
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
Contacts and Locations
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, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01783509
Please refer to this study by its ClinicalTrials.gov identifier: NCT01783509
Contacts
| Contact: Jacqueline Sykes | 704-333-3967 | Jacqueline.Sykes@carolinashealthcare.org |
Locations
| United States, North Carolina | |
| Carolinas Medical Center - Pediatrics | Recruiting |
| Charlotte, North Carolina, United States, 28203 | |
| Contact: Jacqueline Sykes 704-333-3967 jacqueline.sykes@carolinashealthcare.org | |
| Principal Investigator: Susan Sparks, MD | |
| Sub-Investigator: Amy Harper, MD | |
| Sub-Investigator: Elena Bravver, MD | |
Sponsors and Collaborators
Carolinas Healthcare System
Muscular Dystrophy Association
Investigators
| Principal Investigator: | Susan Sparks, MD | Carolinas Medical Center - Pediatrics |
More Information
| Responsible Party: | Amy D. Harper, Principal Investigator, Carolinas Healthcare System |
| ClinicalTrials.gov Identifier: | NCT01783509 History of Changes |
| Other Study ID Numbers: | LGMD Nat Hx |
| Study First Received: | January 18, 2013 |
| Last Updated: | February 2, 2015 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Carolinas Healthcare System:
|
GENETICALLY CONFIRMED limb girdle muscular dystrophy |
Additional relevant MeSH terms:
|
Muscular Dystrophies Muscular Dystrophies, Limb-Girdle Muscular Disorders, Atrophic Muscular Diseases |
Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn |
ClinicalTrials.gov processed this record on September 29, 2016