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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 baseline, month 60

Awards & highlights

Study 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.Check my eligibility

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.See study design

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 ~ baseline, month 60

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, month 60

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, 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 Protein Expression Quantity Assessed by IF Percent Protein Fibers (PPF) at Day 60

Change from Baseline in β-SG Protein Expression Quantity Assessed by Immunofluorescence (IF) Fiber Intensity at Day 60

Change from Baseline in β-SG Protein Expression Quantity Assessed by Western Blot at Day 60

+1 more

Secondary outcome measures

Change From Baseline in Time to Rise From the Floor, Time to Complete 100 and 10 meter Walk/Run, and the Timed Stair Ascend 4 Steps Test

Change from Baseline in Ability Captured Through Interactive Video Evaluation (ACTIVE) - Seated Workspace Volume Task at Month 60

Change from Baseline in Angular Wrist Velocity at Month 60

+11 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: SRP-9003Experimental Treatment1 Intervention

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

Do I qualify?Apply below to find out

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

8,063,882 Total Patients Enrolled

Media Library

SRP-9003 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05876780 — Phase 1

Limb-Girdle Muscular Dystrophy Research Study Groups: SRP-9003

Limb-Girdle 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

Frequently Asked Questions

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

Is it possible for me to become a participant in this clinical experiment?

"Candidates between the ages of 4 and 50 with a diagnosis of limb-girdle muscular dystrophy may be eligible for this clinical trial. A maximum of 5 patients are being enrolled in total."

Answered by AI

Has SRP-9003 been granted authorization from the FDA?

"Due to the fact that SRP-9003 is still in its initial trial phase, our team at Power assessed a score of 1 for safety due to minimal data suggesting efficacy and security."

Answered by AI

Is enrollment open to adults aged eighteen and over for this investigation?

"The parameters of this trial necessitate that candidates should be aged between 4 and 50. Moreover, there are 61 studies for minors and 53 for seniors."

Answered by AI

Are there any openings available to participate in this clinical exploration?

"According to clinicaltrials.gov, the recruitment of participants for this medical trial has been suspended since May 17th 2023. Initially posted on December 19th 2022, no further enrolment is being accepted at present; however, there are 89 other trials that are still actively recruiting patients."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)

2022. "A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05876780.

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