Clinical trials: Limb Girdle Muscular Dystrophy
Type 2 Diabetes
Influence of Endothelial Function on Central and Peripheral Causes Of Exercise Impairment in Type 2 Diabetes
This study will evaluate the effects of impaired blood flow regulation on exercise. It will also determine whether the effects are more important in the heart or in the skeletal muscle tissue during exercise.
- Trial name
- InsITE (NCT00786019)
- Testing
- Exercise program, Ascorbic Acid (Vitamin C)
- Where
- United States
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- University of Colorado, Denver
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
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.
LGMD2E
Clinical Determinants of Disease Progression in Patients With Limb Girdle Muscular Distrophy Type 2E
A retrospective observational study that will enable us to collect retrospective data from the clinical records of LGMD 2E patients in order to highlight any possible correlation between: clinical variables and patient age…
- Trial name
- NeuroLGMD2E (NCT04509609)
- Testing
- Exon 3 truncating mutation, Any other mutation in SGBC gene
- Where
- Italy
- Volunteers
- 33 people
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Limb-girdle Muscular Dystrophy
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
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).
- Trial name
- EMERGENE (NCT06246513)
- Testing
- SRP-9003, Glucocorticoid
- Where
- 10 sites in United States, Belgium and 4 more
- Volunteers
- 17 people
- Sponsor
- Sarepta Therapeutics, Inc.
Hypertension, Pulmonary
Prospective, Non-interventional, Multi-center, Post-authorization Safety Study of Riociguat for Pulmonary Arterial Hypertension (PAH)
The primary objective in this study is to collect post-marketing information on Riociguat safety.Thus, the information on adverse events (AEs) and adverse drug reactions (ADRs) that occur within the first 12 months and in the…
- Trial name
- JPMS-PAH (NCT02428985)
- Testing
- Riociguat (ADEMPAS, BAY63-2521)
- Where
- Japan
- Volunteers
- 882 people
- Sponsor
- Bayer
Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy)
Congenital Muscle Disease Study of Patient and Family Reported Medical Information
The Congenital Muscle Disease Patient and Proxy Reported Outcome Study (CMDPROS) is a longitudinal 10 year study to identify and trend care parameters, adverse events in the congenital muscle diseases using the Congenital Muscle…
- Conditions
- Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan)
- Trial name
- CMDPROS (NCT01403402)
- Where
- United States
- Volunteers
- 4,000 people
- Sponsor
- Cure CMD
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
LGMD2I
Study of BBP-418 in Patients With LGMD2I
BBP-418 is being developed for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I). This is an open label study to determine the safety and tolerability of ascending dose levels of BBP-418 in the…
- Trial name
- NCT04800874
- Testing
- BBP-418
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- ML Bio Solutions, Inc.
Limb-Girdle Muscular Dystrophy
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.
- Trial name
- NCT00457912
- Where
- United States
- Volunteers
- 277 people
- Sponsor
- Nationwide Children's Hospital
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
Gamma-sarcoglycanopathy
Natural History Study in Patients With LGMDR5/2c
Prospective, longitudinal, interventional, multicenter natural history study to better understand the LGMDR5/2c disease physiopathology. The duration of participation for each patient will be up to 24 months.
- Trial name
- NCT06210672
- Where
- 3 sites in France, Tunisia
- Sponsor
- Atamyo Therapeutics
Changes in Peripheral Blood Population Numbers and Type.
Stem Cell Quantification In Peripheral Blood After Hyperbaric Oxygen Therapy Treatments
This is an observational research study whose purposes are to see the effects of repeated hyperbaric oxygen therapy treatments on the levels of stem/progenitor cells in peripheral circulating blood following five days of…
- Trial name
- NCT01740908
- Testing
- Hyperbaric Oxygen Treatment
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Restorix Research Institute, LLLP
Limb Girdle Muscular Dystrophy
Stem Cell Therapy in Limb Girdle Muscular Dystrophy
The purpose of this study was to study the effect of stem cell therapy on the course of the disease in patients with Limb Girdle Muscular Dystrophy.
- Trial name
- NCT02050776
- Testing
- Autologous bone marrow mononuclear cell transplantation
- Where
- India
- Sponsor
- Neurogen Brain and Spine Institute
Facioscapulohumeral Muscular Dystrophy, Limb Girdle Muscular Dystrophy
Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)
ATYR1940-C-006 is a multi-national, multicenter study being conducted at centers in the United States (US) and Europe who participated in Study ATYR1940-C-003 (Stage 1 only) or Study ATYR1940-C-004 (that is, the parent studies).
- Trial name
- FSHD (NCT02836418)
- Testing
- ATYR1940
- Where
- 5 sites in United States, Denmark and 1 more
- Volunteers
- 8 people
- Sponsor
- aTyr Pharma, Inc.
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
Limb-Girdle Muscular Dystrophy
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
This study is designed to evaluate the safety and efficacy of deflazacort in participants with LGMD2I. Most participants enrolled will have a screening visit and 3 additional visits (after 1, 13, and 26 weeks of treatment).
- Trial name
- NCT03783923
- Testing
- Deflazacort
- Where
- 18 sites in United States, Canada and 6 more
- Volunteers
- 11 people
- Sponsor
- PTC Therapeutics
Inclusion Body Myositis, Myotonic Dystrophy
Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
Currently, there are no standardised fall risk scores or guidelines on when to use appropriate assistive gait devices (AGDs) for people with neuromuscular disorders (NMDs).
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy, Limb-girdle and Facioscapulohumeral Muscular Dystrophies, Pompe Disease
- Trial name
- FBIndex (NCT06605612)
- Testing
- FBIndex
- Where
- Germany
- Volunteers
- 108 people
- Sponsor
- LMU Klinikum
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