Limb-Girdle Muscular Dystrophy Clinical Trial
Official title:
Genetic Characterization of Individuals With Limb Girdle Muscular Dystrophy
The objective of this study is to identify and maintain a registry of well-characterized limb-girdle muscular dystrophy (LGMD) patients. Patients seen as part of this study may be candidates for future treatment trials based on their defined genetic classification of LGMD. In the course of this study, the investigators will perform a muscle biopsy and DNA testing in an unlimited number of patients with clinically diagnosed LGMD. The genetic testing will be extended to the family of the study subject in order to better understand true genetic defect.
n/a
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT01403402 -
Congenital Muscle Disease Study of Patient and Family Reported Medical Information
|
||
| Terminated |
NCT03783923 -
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
|
Phase 3 | |
| Completed |
NCT02635321 -
MRI and Muscle Involvement in Patients With Mutations in GMPPB
|
N/A | |
| Completed |
NCT00873782 -
Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy
|
Phase 1 | |
| Completed |
NCT04054375 -
Weekly Steroids in Muscular Dystrophy
|
Phase 2 | |
| Recruiting |
NCT00390104 -
Molecular Analysis of Patients With Neuromuscular Disease
|
||
| Recruiting |
NCT06246513 -
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4 (EMERGENE)
|
Phase 3 | |
| Completed |
NCT00104078 -
Study Evaluating MYO-029 in Adult Muscular Dystrophy
|
Phase 1/Phase 2 | |
| Recruiting |
NCT04475926 -
A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
|
||
| Completed |
NCT02759302 -
MRI on Persons With Mutations in POMT2 Gene (LGMD2N)
|
N/A |