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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
No Placebo-Only Group

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 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

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 Treatment1 Intervention
Participants will receive single IV infusion of SRP-9003 on Day 1.

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Who is running the clinical trial?

Sarepta Therapeutics, Inc.Lead Sponsor
52 Previous Clinical Trials
33,962 Total Patients Enrolled
Medical DirectorStudy DirectorSarepta Therapeutics, Inc.
2,885 Previous Clinical Trials
8,088,779 Total Patients Enrolled

Media Library

SRP-9003 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05876780 — Phase 1
Muscular dystrophy Research Study Groups: SRP-9003
Muscular dystrophy Clinical Trial 2023: SRP-9003 Highlights & Side Effects. Trial Name: NCT05876780 — Phase 1
SRP-9003 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05876780 — Phase 1
~4 spots leftby Aug 2028
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