Clinical trials: Limb-girdle Muscular Dystrophy, open to volunteers
Limb Girdle Muscular Dystrophy Type 2D/R3
BBM-L101 Gene Therapy in Participants With LGMD2D/R3
This phase 1 study will evaluate a single intravenous infusion of BBM-L101, an investigational AAV-based gene therapy designed to deliver a functional SGCA gene, in ambulatory children aged 4 to <13 years with genetically…
- Trial name
- NCT07874516
- Testing
- BBM-L101 Injection
- Where
- China
- Volunteers
- 8 people
- Sponsor
- Peking University First Hospital
Neuromuscular; Disorder, Hereditary, Duchenne/Becker Muscular Dystrophy
Molecular Analysis of Patients With Neuromuscular Disease
The purpose of this study is to identify new genes responsible for neuromuscular disorders and study muscle tissue of patient with known neuromuscular disease, as well as their family members.
- Conditions
- Neuromuscular; Disorder, Hereditary, Duchenne/Becker Muscular Dystrophy, Limb-girdle Muscular Dystrophy
- Trial name
- NCT00390104
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Boston Children's Hospital
Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth
Natural History Study for Charcot Marie Tooth Disease
The goal of this Natural History Study for Charcot-Marie-Tooth is to acquire, record, and analyze patient-reported data and associated genetic reports, Electronic Health Records (EHRs) and clinical notes to identify the burden…
- Conditions
- Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth, Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A
- Trial name
- NCT05902351
- Where
- United States
- Volunteers
- 10,000 people
- Sponsor
- Hereditary Neuropathy Foundation
Inclusion Body Myositis, Myotonic Dystrophy 1
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
The planned project is an intervention study to assess the risk of falling after adaptation of an assistive gait devices in patients with the following neuromuscular diseases: Inclusion body myositis, myotonic dystrophy, limb…
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Facio-Scapulo-Humeral Dystrophy
- Trial name
- Risk of fall (NCT07072676)
- Testing
- Assistive gait devices combined with physiotherapy
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- LMU Klinikum
Muscular Dystrophy
Clinical Trial Readiness for the Dystroglycanopathies
The purpose of the study is to describe the early signs and symptoms of the dystroglycanopathies, and to gather information that will be required for future clinical trials.
- Trial name
- NCT00313677
- Where
- United States
- Volunteers
- 190 people
- Sponsor
- Katherine Mathews
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
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
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
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, Social Competence
Telecare Psychosocial and Cognitive Intervention for Children and Adolescents With Limb-Girdle Muscular Dystrophy
This clinical trial aims to evaluate the efficacy of a telecare-mediated psychosocial and cognitive intervention for children and adolescents aged 7 to 17 with limb-girdle muscular dystrophy.
- Conditions
- Limb-Girdle Muscular Dystrophy, Social Competence, Self Esteem, Health Related Quality of Life
- Trial name
- TPCLGMD (NCT07711730)
- Testing
- Neuro-psychosocial teleassistance program
- Where
- Spain
- Volunteers
- 30 people
- Sponsor
- University of Deusto
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
Type and Grade of Toxicities, Demographic and Background Characteristics, Advanced, Metastatic, Recurrent Adenocarcinoma and Malignant Solid Neoplasm
Labyrinthin Vaccination in Veterans With Lung Adenocarcinoma
This phase 2 trial tests the safety and effectiveness of a cancer vaccine called LabVax 3(22)-23 and GM-CSF in combination with pembrolizumab in treating adenocarcinoma that has spread to other places in the body (advanced…
- Trial name
- NCT07859592
- Testing
- LabVax 3(22)-23 and GM-CSF (adjuvant) in combination with pembrolizumab
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- VA Office of Research and Development