Gene Transfer with SRP-6004 for Limb-Girdle Muscular Dystrophy
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial is testing the safety of a medicine called SRP-6004, given through a vein, in people who can walk but have a specific type of muscular dystrophy (LGMD2B/R2).
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. However, if you require chronic drug treatment that the investigator believes poses a risk for gene transfer, it might be a concern.
Is SRP-6004 gene therapy safe for humans?
Research on similar gene therapies using adeno-associated virus (AAV) vectors, like those for other muscular dystrophies, shows they are generally well-tolerated with no significant toxicity observed in animal studies. However, there are concerns about immune responses, so safety measures are important to minimize risks.12345
How does the treatment SRP-6004 differ from other treatments for limb-girdle muscular dystrophy?
SRP-6004 is unique because it uses gene transfer therapy to deliver a functional version of the γ-sarcoglycan gene directly into muscle cells, aiming to correct the underlying genetic defect causing limb-girdle muscular dystrophy. This approach is different from traditional treatments as it targets the root cause of the disease rather than just managing symptoms.46789
What data supports the effectiveness of the treatment SRP-6004 for Limb-Girdle Muscular Dystrophy?
Who Is on the Research Team?
Medical Director
Principal Investigator
Sarepta Therapeutics, Inc.
Are You a Good Fit for This Trial?
This trial is for ambulatory individuals with Limb Girdle Muscular Dystrophy, Type 2B/R2. Participants must have specific genetic mutations related to the condition, be able to perform motor tests, and not have high levels of certain antibodies. They can't join if they've had recent gene therapy or other investigational treatments, significant health issues, or need chronic drug treatment that could pose risks.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single IV infusion of SRP-6004 on Day 1
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- SRP-6004
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sarepta Therapeutics, Inc.
Lead Sponsor