Clinical trials: LGMD2B
Dysferlinopathy
rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies
The proposed clinical trial is a double-blind, randomized controlled study with direct intramuscular injection of rAAVrh.74.MHCK7.DYSF.DV gene vector to the extensor digitorum brevis muscle (EDB).
- Trial name
- NCT02710500
- Testing
- rAAVrh74.MHCK7.DYSF.DV
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Sarepta Therapeutics, Inc.
Dysferlinopathy
Proteasomal Inhibition for Patients With Mis-sense Mutated Dysferlin
Dysferlin is a protein with an important role in the repair of muscle surface membranes. Mutations in dysferlin cause different forms of muscular dystrophies.
- Trial name
- Dysferlin (NCT01863004)
- Testing
- Bortezomib
- Where
- Switzerland
- Volunteers
- 3 people
- Sponsor
- University Hospital, Basel, Switzerland
Dysferlinopathy, Miyoshi Myopathy
Clinical, Immunological, Morphological and Genetic Characteristics of Patients With Dysferlinopathy (LGMD R2) in the RF
To evaluate specific characteristics of phenotype, immune status, molecular and genetic as well as morphological characteristics of adult patients with limb-girdle muscular dystrophy R2 in various regions of the Russian…
- Conditions
- Dysferlinopathy, Miyoshi Myopathy, LGMDR2, DMAT
- Trial name
- DYSF-RUS (NCT04824040)
- Where
- Russia
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Artgen Biotech
Limb Girdle Muscular Dystrophy
A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)
The primary purpose of this study is to evaluate the safety of SRP-6004 administered by intravenous (IV) infusion in ambulatory participants with LGMD2B/R2 (DYSF related).
- Trial name
- NCT05906251
- Testing
- SRP-6004
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Sarepta Therapeutics, Inc.
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
Muscular Dystrophies, Limb-Girdle Muscular Dystrophy Type 2B
Dysferlinopathy Protein in Peripheral Blood Monocytes.
The objective of the study is to answer the following important questions. Deficiency of the dysferlin protein is the cause of a very rare limb-girdle muscular dystrophy (LGMD-2B) that leads to significant disability.
- Conditions
- Muscular Dystrophies, Limb-Girdle Muscular Dystrophy Type 2B, Miyoshi Myopathy, Distal Myopathy With Anterior Tibial Onset
- Trial name
- NCT06507215
- Testing
- Protein analysis
- Where
- Spain
- Volunteers
- 149 people, healthy volunteers welcome
- Sponsor
- Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
Dysferlinopathy, LGMD2B Now Classified as LGMDR2
Clinical Outcome Study for Dysferlinopathy
The "Clinical Outcome Study for Dysferlinopathy" is being performed in centres in Europe (UK- Newcastle; Spain- Barcelona, Sevilla; San Sebastian;Denmark, Copenhagen, Italy- Padova; France- Paris,), USA (Charlotte, NC; Columbus…
- Conditions
- Dysferlinopathy, LGMD2B Now Classified as LGMDR2, Miyoshi Myopathy
- Trial name
- Jain COS (NCT01676077)
- Where
- 16 sites in United States, Chile and 7 more
- Volunteers
- 200 people
- Sponsor
- Newcastle-upon-Tyne Hospitals NHS Trust
LGMD2B, Miyoshi Myopathy
Deflazacort in Dysferlinopathies
The present study is designed to assess the natural history in a one year pre-phase of the trial and evaluate therapeutic efficacy and side effects of deflazacort in LGMD2B/MM patients in a placebo-controlled trial.
- Conditions
- LGMD2B, Miyoshi Myopathy, Dysferlinopathy
- Trial name
- NCT00527228
- Testing
- deflazacort
- Where
- Germany
- Volunteers
- 25 people
- Sponsor
- Ludwig-Maximilians - University of Munich
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
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 Dystrophies, Facioscapulohumeral Muscular Dystrophy
Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD)
The purpose of this study is to assess the safety and biological activity of ATYR1940 in participants with LGMD2B and FSHD.
- Trial name
- FSHD (NCT02579239)
- Testing
- ATYR1940
- Where
- 6 sites in United States, Denmark and 1 more
- Volunteers
- 18 people
- Sponsor
- aTyr Pharma, Inc.