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Gene Therapy

Gene Therapy for Limb-Girdle Muscular Dystrophy

Phase 3
Recruiting
Research Sponsored by Sarepta Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Cohort 1, only ambulatory participants: Able to walk without assistive aid, 10-meter walk test (10MWT) <30 seconds, NSAD ≥25
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
Pivotal Trial
No Placebo-Only Group

Summary

This trial will investigate the impact of a single dose of SRP-9003 on beta-sarcoglycan gene expression in individuals with limb-girdle muscular dystrophy type 2E/R

Who is the study for?
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.
What is being tested?
The study tests an experimental gene therapy named Bidridistrogene Xeboparvovec (SRP-9003), which aims to correct the genetic defect causing the disease. Participants will receive one dose of this therapy, and its effects on gene expression will be monitored.
What are the potential side effects?
Potential side effects aren't listed here, but gene therapies like SRP-9003 could cause immune reactions, swelling at the injection site, headache, fever or chills. The exact side effects would be explained by the research team.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I can walk without help and do so quickly.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: SRP-9003Experimental Treatment2 Interventions
Participants will receive a single intravenous (IV) infusion of SRP-9003.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Glucocorticoid
2019
Completed Phase 2
~340

Find a Location

Who is running the clinical trial?

Sarepta Therapeutics, Inc.Lead Sponsor
52 Previous Clinical Trials
33,953 Total Patients Enrolled
Medical DirectorStudy DirectorSarepta Therapeutics, Inc.
2,905 Previous Clinical Trials
8,091,454 Total Patients Enrolled
~1 spots leftby Jan 2025
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