Your session is about to expire
← Back to Search
Gene Therapy
Gene Therapy for Limb-Girdle Muscular Dystrophy
Phase 1
Waitlist Available
Research Sponsored by Sarepta Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
Summary
This trial is testing a drug for safety and to measure its effects on LGMD2E/R4 patients, both ambulatory and non-ambulatory.
Who is the study for?
This trial is for individuals aged 4-50 with Limb Girdle Muscular Dystrophy, type 2E/R4 who are either non-ambulatory (can't walk) or ambulatory (can walk). They must have specific genetic mutations and be able to undergo muscle testing. Those with significant other illnesses, exposure to gene therapy, or conditions affecting protocol compliance cannot participate.
What is being tested?
The study tests the safety and effectiveness of SRP-9003, a gene transfer treatment aimed at expressing β-SG in skeletal muscles of LGMD2E/R4 patients. It includes two groups: one that can walk (Cohort 1) and one that cannot (Cohort 2), assessing how well the treatment works in each.
What are the potential side effects?
While not explicitly listed here, potential side effects may include reactions related to immune response against the new gene or vector used for transfer, inflammation at injection sites, general discomforts like headaches or fever associated with gene therapies.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
Treatment Details
Trial Design
1Treatment groups
Experimental Treatment
Group I: SRP-9003Experimental Treatment1 Intervention
Participants will receive single IV infusion of SRP-9003 on Day 1.
Find a Location
Who is running the clinical trial?
Sarepta Therapeutics, Inc.Lead Sponsor
49 Previous Clinical Trials
33,530 Total Patients Enrolled
Medical DirectorStudy DirectorSarepta Therapeutics, Inc.
2,834 Previous Clinical Trials
8,079,774 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am between 4 and 50 years old and cannot walk.I have specific genetic mutations in the β-sarcoglycan gene.I can participate in muscle strength tests.I haven't had gene therapy or experimental drugs within the trial's time limits.I cannot take corticosteroids due to health reasons.I can walk and move around without help.
Research Study Groups:
This trial has the following groups:- Group 1: SRP-9003
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger