Gene Therapy for Limb-Girdle Muscular Dystrophy
(EMERGENE Trial)
Trial Summary
Do I need to stop my current medications to join the trial?
The trial protocol does not specify if you need to stop your current medications. However, if you are on active immunosuppressant treatment for an autoimmune disease, you may be excluded from the trial.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. However, if you are on active immunosuppressant treatment for an autoimmune disease, you may not be eligible to participate.
What data supports the idea that Gene Therapy for Limb-Girdle Muscular Dystrophy (also known as: Bidridistrogene Xeboparvovec (SRP-9003), SRP-9003) is an effective treatment?
The available research shows that gene therapy using AAV9.BVES significantly improved muscle mass, strength, and exercise performance in a mouse model of Limb-Girdle Muscular Dystrophy type R25. It also normalized heart rate under stress and showed no obvious toxicity. This suggests that gene therapy can effectively improve symptoms of muscular dystrophy. While the research primarily focuses on animal models, these results provide promising evidence for the potential effectiveness of gene therapy in treating this condition.12345
What data supports the effectiveness of the treatment Bidridistrogene Xeboparvovec (SRP-9003) for Limb-Girdle Muscular Dystrophy?
What safety data exists for gene therapy in treating Limb-Girdle Muscular Dystrophy?
The safety data for delandistrogene moxeparvovec (SRP-9001) gene therapy, primarily studied for Duchenne muscular dystrophy, indicates that the most common treatment-related adverse events were vomiting, decreased appetite, and nausea, which mostly occurred within the first 90 days and resolved. Long-term safety and functional outcomes have been evaluated, showing robust expression of the therapeutic protein and stabilization of motor function up to 2 years post-treatment. No obvious toxicity was detected in related studies, suggesting a favorable safety profile.12367
Is the gene therapy SRP-9003 safe for humans?
Is the treatment Bidridistrogene Xeboparvovec (SRP-9003) a promising treatment for Limb-Girdle Muscular Dystrophy?
The treatment Bidridistrogene Xeboparvovec (SRP-9003) shows promise for Limb-Girdle Muscular Dystrophy because similar gene therapies have been successful in improving muscle function and strength in other types of muscular dystrophy. These therapies use a virus to deliver a helpful gene to muscle cells, which can lead to better muscle performance and overall health.12467
How is the treatment Bidridistrogene Xeboparvovec (SRP-9003) unique for limb-girdle muscular dystrophy?
Bidridistrogene Xeboparvovec (SRP-9003) is a gene therapy that uses an adeno-associated virus (AAV) vector to deliver a gene encoding a micro-dystrophin protein, which is a shortened version of the dystrophin protein. This approach is unique because it targets the underlying genetic cause of the disease, potentially offering a long-term solution by restoring the function of the dystrophin protein in muscle cells.12467
What is the purpose of this trial?
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). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).
Research Team
Medical Director
Principal Investigator
Sarepta Therapeutics, Inc.
Eligibility Criteria
This trial is for people with a muscle condition called Limb Girdle Muscular Dystrophy 2E/R4. It's open to those who can move around on their own (Cohort 1) and those who cannot (Cohort 2). Specific eligibility details are not provided, but typically participants must meet certain health criteria.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single intravenous (IV) infusion of SRP-9003
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Bidridistrogene Xeboparvovec (SRP-9003)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sarepta Therapeutics, Inc.
Lead Sponsor