Clinical trials: LGMD2A
LGMD1B, LGMD1C
Long-Term Development of Muscular Dystrophy Outcome Assessments
This is a 24-month, observational study of up to 1000 participants with Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset Pompe disease (LOPD).
- Conditions
- LGMD1B, LGMD1C, LGMD1D, LGMD1E
- Trial name
- GRASP-01-005 (NCT05989620)
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Virginia Commonwealth University
Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A, Limb Girdle Muscular Dystrophy
LGMD R1 Natural History Study
This is a 24-month, observational study of 100 participants with Limb Girdle Muscular Dystrophy type R1, also known as CAPN3.
- Conditions
- Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A, Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type R1, LGMD2A
- Trial name
- GRASP-01-003 (NCT05618080)
- Where
- 9 sites in United States
- Volunteers
- 100 people
- Sponsor
- Virginia Commonwealth University
Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A
Clinical and Biochemical Features for the Identification of Dominant Calpainopathies
Mutations in the CAPN3 gene cause muscular dystrophies with dysfunction in calpain-3. Calpainopathies are usually inherited in an autosomal recessive manner but in some families they can occur in a dominant inheritance.
- Trial name
- DOM-CAL (NCT05956132)
- Testing
- retrospective study
- Where
- 2 sites in Italy
- Volunteers
- 50 people
- Sponsor
- IRCCS San Camillo, Venezia, Italy
Muscular Dystrophies, Muscular Dystrophy, Duchenne
User-centred Assistive System for Arm Functions in Neuromuscular Subjects
Restore a lost function is a special experience for people affected by neuromuscular evolutive diseases. "From the patient's point of view improvement is measured by regaining lost abilities,-by being able to do something…
- Conditions
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Dystrophy, Becker, Muscular Dystrophy, Limb-Girdle Type 2
- Trial name
- USEFUL (NCT03127241)
- Testing
- Assistive device (Armon Ayura) test, Assistive device (Jaeco Wrex) test
- Where
- 2 sites in Italy
- Volunteers
- 38 people
- Sponsor
- Politecnico di Milano
Limb-girdle Muscular Dystrophy
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
This study will follow participants who are screened and confirmed with a genetic diagnosis of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D (LGMD2D/R3), Limb-girdle muscular dystrophy…
- Trial name
- NCT04475926
- Where
- 26 sites in United States, Belgium and 8 more
- Volunteers
- 205 people
- Sponsor
- Sarepta Therapeutics, Inc.
Limb-Girdle Muscular Dystrophy Type 2A, Limb-Girdle Muscular Dystrophy, Type 2E
Natural History of Limb Girdle Muscular Dystrophy Type 2A and Type 2E
This is an observational study, no drug (marketed or investigational) will be provided as part of the study, and the study procedures will have no impact on the medical care delivered to patients participating in this study.
- Trial name
- NCT03488784
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Lindsay Alfano
LGMD, LGMDR2
A Multicenter Phenotype-Genotype Analysis of LGMD Patients in China
Limb-girdle muscular dystrophies (LGMD) are a series of rare progressive genetic disorders that are characterized by wasting and weakness of the voluntary proximal muscles.
- Conditions
- LGMD, LGMDR2, LGMDR1
- Trial name
- NCT04989751
- Testing
- Electromyography, IDEAL MRI
- Where
- China
- Volunteers
- 450 people
- Sponsor
- Huashan Hospital