Clinical trials: Limb Girdle Muscular Dystrophy
Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
Open-Label Extension Study of BBP-418 (Ribitol) for LGMD2I/R9
This is an open-label extension (rollover) study designed to evaluate the long-term safety and efficacy of BBP-418 (ribitol) in participants with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) who have previously…
- Trial name
- NCT07678775
- Testing
- BBP-418 (ribitol)
- Where
- 20 sites in United States, Australia and 6 more
- Volunteers
- 107 people
- Sponsor
- ML Bio Solutions, Inc.
Limb Girdle Muscular Dystrophy, Muscular Dystrophies
Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)
Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles.
- Trial name
- GRASP-01-001 (NCT03981289)
- Where
- 10 sites in United States, United Kingdom
- Volunteers
- 116 people
- Sponsor
- Virginia Commonwealth University
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 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
MRI on Persons With Mutations in POMT2 Gene (LGMD2N)
POMT2 mutation is known to cause Walker Warburg Syndrome and Muscle-Brain-Eye syndrome. Recently it has been connected to limb girdle muscular dystrophy (LGMD), a disorder characterized by muscle weakness and atrophy of the…
- Trial name
- NCT02759302
- Where
- Denmark
- Volunteers
- 12 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Muscle Diseases
Acceptance and Commitment Therapy for Muscle Disease
In adults, muscle diseases are usually chronic long-term conditions that do not have a definitive cure. Supportive care has been shown to reduce complications from muscle disease and improved survival in some cases.
- Trial name
- ACTMuS (NCT02810028)
- Testing
- Acceptance and Commitment Therapy (ACT)
- Where
- United Kingdom
- Volunteers
- 155 people
- Sponsor
- King's College Hospital NHS Trust
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
Limb-girdle Muscular Dystrophy
MRI and Muscle Involvement in Patients With Mutations in GMPPB
Limb girdle muscular dystrophies (LGMD) are a very heterogeneous group of muscle disorders characterized by muscle weakness and atrophy of the proximal muscles of the shoulder and pelvic girdles.
- Trial name
- NCT02635321
- Where
- Denmark
- Volunteers
- 4 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Limb-Girdle Muscular Dystrophy, Type 2E
Limb Girdle Muscular Dystrophy Type 2E Recruitment Study
This study is to recruit and establish baseline measurements for potential subjects that may be eligible for a gene therapy trial.
- Trial name
- NCT03492346
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Nationwide Children's Hospital
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
Limb Girdle Muscular Dystrophies
Motor Parameters in Patients With Limb Girdle Muscular Dystrophy
The primary objective of the study is to perform 2-year follow up with motor parameters evolution using instrumental assessments in patients with limb girdle muscular dystrophie, and to identify which motor parameters are…
- Trial name
- EIDY (NCT04772027)
- Where
- France
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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
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, Type 2E
Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.
- Trial name
- NCT03652259
- Testing
- SRP-9003
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Sarepta Therapeutics, Inc.
Muscular Dystrophies, Limb Girdle Muscular Dystrophy
Rehabilitation in Muscular Dystrophies From the Hospital Facility to the Home: Pilot Project [RIMUDI]
Until twenty years ago physical exercise in muscular dystrophies was considered harmful to the muscle cells, inducing an acceleration of cell necrosis.
- Conditions
- Muscular Dystrophies, Limb Girdle Muscular Dystrophy, Facio-Scapulo-Humeral Dystrophy, Becker Muscular Dystrophy
- Trial name
- RIMUDI (NCT06378203)
- Testing
- tele-rehabilitation system with virtual reality Home Kit - Virtual Reality Rehabilitation System (VRRS KHYMEIA)
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- IRCCS Eugenio Medea
Type 2 Diabetes
What Makes Exercise Feel More Difficult to Women With and Without Type 2 Diabetes
Given that sedentary behavior is associated with T2DM, the purpose of this study is to evaluate whether subjects with T2DM have a significant disincentive to performing exercise (due to greater perceived effort) .
- Trial name
- ExPRESS (NCT00785005)
- Where
- United States
- Volunteers
- 99 people, healthy volunteers welcome
- Sponsor
- University of Colorado, Denver
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
Migraine
Safety and Tolerability of Frovatriptan to Prevention of Menstrually Associated Migraine (MAM) Headaches
In this open-label, multi-center study, patients were treated with frovatriptan for a maximum of 12 perimenstrual periods (PMPs).
- Trial name
- MAM (NCT01035983)
- Testing
- Frovatriptan 2.5 mg
- Where
- 5 sites in Germany, Hungary and 3 more
- Volunteers
- 550 people
- Sponsor
- Endo USA Inc., a Keenova Therapeutics Company
Lymphoma, Large B-Cell, Diffuse
Efficacy and Safety Study of Lenalidomide Plus R-CHOP Chemotherapy Versus Placebo Plus R-CHOP Chemotherapy in Untreated ABC Type Diffuse Large B-cell Lymphoma
To evaluate the efficacy and safety of lenalidomide, rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R2-CHOP) chemotherapy versus placebo, rituximab, cyclophosphamide, doxorubicin, vincristine, and…
- Trial name
- ROBUST (NCT02285062)
- Testing
- lenalidomide, Rituximab, Cyclophosphamide
- Where
- 158 sites in United States, Australia and 19 more
- Volunteers
- 570 people
- Sponsor
- Celgene
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.