Clinical trials: Limb-Girdle Muscular Dystrophy, Type 2E
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 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.
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
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
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
Limb-girdle Muscular Dystrophy
A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
This study will follow participants who are screened and confirmed with a genetic diagnosis of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D (LGMD2D/R3), Limb-girdle muscular dystrophy…
- Trial name
- NCT04475926
- Where
- 26 sites in United States, Belgium and 8 more
- Volunteers
- 205 people
- Sponsor
- Sarepta Therapeutics, Inc.
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
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.
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
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