Clinical trials: Limb Girdle Muscular Dystrophy
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
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
LGMD
Evaluating Safety and Efficacy of Autologous Gene-edited Muscle Stem Cells (GenPHSats-bASKet)
This study is an investigator initiated first-in-human interventional open label phase 1/2a clinical trial investigating an ATMP in the orphan disease LGMD to evaluate safety and efficacy.
- Trial name
- NCT05588401
- Testing
- GenPHSat injection (Safety), GenPHSat injection (Efficacy), Muscle Biopsy (Safety)
- Volunteers
- 6 people
- Sponsor
- Simone Spuler, MD
LGMD2I
Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I
Prospective, longitudinal, interventional, single-group, multicenter natural history study to better know the LGMD2I disease physiopathology. The duration of participation for each patient will be up to 24 months.
- Trial name
- NCT03842878
- Where
- 3 sites in Denmark, France and 1 more
- Volunteers
- 52 people
- Sponsor
- Genethon
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.
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
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.
LGMDR9
ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9
Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up
- Trial name
- NCT05224505
- Testing
- ATA-100 (AAV9 encoding FKRP gene)
- Where
- 3 sites in Denmark, France and 1 more
- Volunteers
- 6 people
- Sponsor
- Atamyo Therapeutics
Limb Girdle Muscular Dystrophy
A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)
The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).
- Trial name
- NCT05876780
- Testing
- SRP-9003
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Cardiac Outcome Measures in Children With Muscular Dystrophy
The purpose of the research study is to evaluate different cardiac measures that are obtained by echocardiographic tests in patients with muscular dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Trial name
- NCT01066455
- Where
- 5 sites in United States
- Volunteers
- 48 people
- Sponsor
- Cooperative International Neuromuscular Research Group
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
Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type 2D/R3
Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States
The primary objective of this study is to evaluate the safety of SRP-9004.
- Trial name
- DISCOVERY (NCT06747273)
- Testing
- SRP-9004
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Sarepta Therapeutics, Inc.
Limb Girdle Muscular Dystrophy Type 2C, Gamma-sarcoglycanopathy
Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C
The purpose of this trial is to study the evaluation of clinical safety and feasibility of gene therapy in patients with limb girdle muscular dystrophy type 2C (gamma-sarcoglycanopathy).
- Trial name
- NCT01344798
- Testing
- AAV1-gamma-sarcoglycan vector injection
- Where
- France
- Volunteers
- 9 people
- Sponsor
- Genethon
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
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
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Cardiac Magnetic Resonance in Children With Muscular Dystrophy
This protocol will exploit novel state of the art cardiovascular magnetic resonance techniques to examine important changes in the heart in children with muscular dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Trial name
- NCT01081080
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Cooperative International Neuromuscular Research Group
LGMD2I, LGMDR9
Global FKRP Registry
Mutations in the Fukutin Related Protein (FKRP) gene cause the condition Limb Girdle Muscular Dystrophy type R9 (LGMDR9) also known as LGMD2I, and the rarer conditions Congenital Muscular Dystrophy (MDC1C), Muscle Eye Brain…
- Conditions
- LGMD2I, LGMDR9, Limb Girdle Muscular Dystrophy, Congenital Muscular Dystrophy
- Trial name
- NCT04001595
- Testing
- Patient Registry
- Where
- United Kingdom
- Volunteers
- 1,000 people
- Sponsor
- Newcastle University
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
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.