Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED) Clinical Trial
Official title:
Longitudinal Assessment and Genetic Understanding of Limb-Girdle Muscular Dystrophy
| NCT number | NCT01783509 |
| Other study ID # | LGMD Nat Hx |
| Secondary ID | |
| Status | Completed |
| Phase | |
| First received | |
| Last updated | |
| Start date | November 2011 |
| Est. completion date | July 2017 |
| Verified date | July 2017 |
| Source | Wake Forest University Health Sciences |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Observational [Patient Registry] |
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.
| Status | Completed |
| Enrollment | 13 |
| Est. completion date | July 2017 |
| Est. primary completion date | July 2017 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 6 Years and older |
| Eligibility | 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 |
| Country | Name | City | State |
|---|---|---|---|
| United States | Carolinas Medical Center - Pediatrics | Charlotte | North Carolina |
| Lead Sponsor | Collaborator |
|---|---|
| Wake Forest University Health Sciences | Muscular Dystrophy Association |
United States,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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 |