Limb Girdle Muscular Dystrophy (LGMD) Natural History

Wake Forest University (WFU) logo

Wake Forest University (WFU)

Status

Completed

Conditions

Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)

Study type

Observational

Funder types

Other

Identifiers

NCT01783509
LGMD Nat Hx

Details and patient eligibility

About

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.

Enrollment

13 patients

Sex

All

Ages

6+ years old

Volunteers

No Healthy Volunteers

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

Trial design

13 participants in 1 patient group

LGMD
Description:
Patients with GENETICALLY CONFIRMED limb girdle muscular dystrophy

Trial contacts and locations

1

Loading...

Data sourced from clinicaltrials.gov

Clinical trials

Find clinical trialsTrials by location
© Copyright 2024 Veeva Systems