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Gene Therapy

SRP-9003 for Limb-Girdle Muscular Dystrophy

Q: Are there any available vacancies for potential participants in this research study?

Indeed, as per the details available on clinicaltrials.gov, this medical research study is actively in search of eligible participants. The trial was initially listed on January 15th, 2024 and underwent its latest update on January 30th, 2024. A total of 15 individuals are being sought from a single designated site for enrollment.

Q: What are the safety implications of SRP-9003 for individuals undergoing treatment?

Our team at Power has evaluated the safety of SRP-9003 to be a level 3 on our scale due to its Phase III trial status, indicating existing efficacy data and robust safety records.

Q: What is the overall patient enrollment count for this particular research investigation?

Indeed, information from clinicaltrials.gov highlights that this particular research study is actively in search of participants. Initially uploaded on January 15, 2024, and subsequently revised on January 30, 2024, the trial aims to enroll a total of 15 patients spread over one designated site.

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 baseline through month 60

Awards & highlights

Study 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.Check my eligibility

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.See study design

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline through month 60

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline through month 60

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline through month 60 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers

Secondary outcome measures

Change From Baseline Through Month 60 in Creatine Kinase Level

Change From Baseline Through Month 60 in North Star Assessment for Dysferlinopathy (NSAD) Total Score

Change From Baseline Through Month 60 in Performance of Upper Limb Version 2.0 (PUL 2.0) Total Score

+9 more

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

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

Sarepta Therapeutics, Inc.Lead Sponsor

49 Previous Clinical Trials

33,524 Total Patients Enrolled

Medical DirectorStudy DirectorSarepta Therapeutics, Inc.

2,777 Previous Clinical Trials

8,064,204 Total Patients Enrolled

Frequently Asked Questions

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

Are there any available vacancies for potential participants in this research study?

"Indeed, as per the details available on clinicaltrials.gov, this medical research study is actively in search of eligible participants. The trial was initially listed on January 15th, 2024 and underwent its latest update on January 30th, 2024. A total of 15 individuals are being sought from a single designated site for enrollment."

Answered by AI

What are the safety implications of SRP-9003 for individuals undergoing treatment?

"Our team at Power has evaluated the safety of SRP-9003 to be a level 3 on our scale due to its Phase III trial status, indicating existing efficacy data and robust safety records."

Answered by AI

What is the overall patient enrollment count for this particular research investigation?

"Indeed, information from clinicaltrials.gov highlights that this particular research study is actively in search of participants. Initially uploaded on January 15, 2024, and subsequently revised on January 30, 2024, the trial aims to enroll a total of 15 patients spread over one designated site."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4 (EMERGENE)

2024. "A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4 (EMERGENE)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06246513.

All > Muscular Dystrophy > Experimental