Clinical trials: Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A
Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency)
Evaluation of Limb-Girdle Muscular Dystrophy
The purpose of this study is to understand the biochemistry of different types of Limb-Girdle Muscular Dystrophy (LGMD) and to determine appropriate outcome measures for future clinical treatment trials for LGMD.
- Conditions
- Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency), Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency), Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)
- Trial name
- NCT00893334
- Where
- 2 sites in United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Cooperative International Neuromuscular Research Group
Limb Girdle Muscular Dystrophy
Evolution of the Functional and Muscular State of Patients With Muscular Dystrophy 2A Belts
Limb girdle muscular dystrophies were originally defined as a postnatal progressive muscle disease, which begins and primarily affects the pelvic and scapular muscles.
- Trial name
- CALNATHIS (NCT06390566)
- Testing
- calpaïnopathies
- Where
- France
- Volunteers
- 25 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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
Becker Muscular Dystrophy, McArdle Disease
Fast Troponin as a Biomarker to Assess Exercise-induced Muscle Damage in Muscle Diseases
The purpose of the study is to explore the biomarker Fast Troponins response to exercise in patients with Becker muscular dystrophy, Limb-girdle muscular dystrophy and McArdle disease
- Conditions
- Becker Muscular Dystrophy, McArdle Disease, Limb-Girdle Muscular Dystrophy Type 2
- Trial name
- NCT04349566
- Where
- Denmark
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Mads Peter Godtfeldt Stemmerik
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 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
Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).
- Conditions
- Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy
- Trial name
- NCT05230459
- Testing
- AB-1003 dose level 1, AB-1003 dose level 2
- Where
- 6 sites in United States
- Volunteers
- 9 people
- Sponsor
- AskBio Inc