← Back to Search

Gene Therapy

Gene Therapy for Limb-Girdle Muscular Dystrophy

Phase 1 & 2
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
A 100 meter walk/run (MWR) test result of ≥40% of predicted for age-, height-, gender-, and weight-matched healthy controls at the screening visit
Presence of β-SG deoxyribonucleic acid (DNA) gene mutations at both alleles
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline up to 7 years
Awards & highlights

Study Summary

This trial will test a new gene delivery treatment for participants with a form of muscular dystrophy called LGMD2E.

Who is the study for?
This trial is for males and females with Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E), who have specific gene mutations and experience difficulty in physical activities like running or climbing stairs. Participants must be able to complete a 100 meter walk/run test at a minimum of 40% efficiency compared to healthy individuals.Check my eligibility
What is being tested?
The study tests SRP-9003 (bidridistrogene xeboparvovec), which is a new gene therapy delivered once to patients with LGMD2E. It's an open-label trial, meaning both the researchers and participants know what treatment is being given.See study design
What are the potential side effects?
Potential side effects are not explicitly listed but may include typical risks associated with gene therapies such as immune reactions, mild to severe infusion-related responses, and possible unintended effects on other genes or systems.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I can walk or run 100 meters at least 40% as well as others my age and size.
Select...
I have mutations in both copies of my β-SG gene.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to 7 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to 7 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
Secondary outcome measures
Change From Baseline in Quantity of Beta-Sarcoglycan (β-SG) Protein Expression at Day 60, as Measured by Western Blot
Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunofluorescence
Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunohistochemistry Percent B-SG Positive Fibers

Trial Design

2Treatment groups
Experimental Treatment
Group I: Cohort 2: SRP-9003Experimental Treatment1 Intervention
Participants will receive a single IV infusion of SRP-9003. Dose will be determined based on the findings from Cohort 1.
Group II: Cohort 1: SRP-9003Experimental Treatment1 Intervention
Participants will receive a single intravenous (IV) infusion of SRP-9003 at a prespecified dose.

Find a Location

Who is running the clinical trial?

Sarepta Therapeutics, Inc.Lead Sponsor
49 Previous Clinical Trials
33,533 Total Patients Enrolled
Medical DirectorStudy DirectorSarepta Therapeutics, Inc.
2,777 Previous Clinical Trials
8,063,474 Total Patients Enrolled

Media Library

SRP-9003 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03652259 — Phase 1 & 2
Limb-Girdle Muscular Dystrophy Research Study Groups: Cohort 1: SRP-9003, Cohort 2: SRP-9003
Limb-Girdle Muscular Dystrophy Clinical Trial 2023: SRP-9003 Highlights & Side Effects. Trial Name: NCT03652259 — Phase 1 & 2
SRP-9003 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03652259 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

To what extent does this experiment restrict subject participation?

"This study is looking for 6 young patients, aged 4 to 15 years old, that have muscular dystrophy. To be eligible, participants must meet the following conditions: Be male or female of any ethnicity, have β-SG deoxyribonucleic acid (DNA) gene mutations at both alleles, demonstrate weakness through a history of difficulty with running, jumping and climbing stairs, and finally score ≥40% on a 100 meter walk/run (MWR) test in comparison to age-, height-, gender-, and weight-matched healthy controls."

Answered by AI

Are there any positions available for participants in this trial?

"The aforementioned clinical trial is not presently looking for patients, as stated on the website. However, this could change in the future as the study was last updated recently on September 28th, 2022. Additionally, there are 87 other trials that are currently recruiting patients."

Answered by AI

Are elderly patients being recruited for this study?

"This particular trial is designed for children aged 4 to 15 years old. Out of the total of 111 studies, 61 are catered to patients under 18 and 50 focus on those over 65."

Answered by AI

Who else is applying?

How old are they?
< 18
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
2018. "Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03652259.
All > Muscular Dystrophy
~2 spots leftby Feb 2027
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top