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Gene Therapy

Gene Transfer with SRP-6004 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
Possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic DYSF Deoxyribonucleic acid (DNA) gene mutations as documented prior to screening visits.
Has accessible and intact lower and upper extremity musculature for biopsy.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing the safety of a medicine called SRP-6004, given through a vein, in people who can walk but have a specific type of muscular dystrophy (LGMD2B/R2).

Who is the study for?
This trial is for ambulatory individuals with Limb Girdle Muscular Dystrophy, Type 2B/R2. Participants must have specific genetic mutations related to the condition, be able to perform motor tests, and not have high levels of certain antibodies. They can't join if they've had recent gene therapy or other investigational treatments, significant health issues, or need chronic drug treatment that could pose risks.
What is being tested?
The study is testing SRP-6004's safety when given through an IV infusion to people who can walk but have LGMD2B/R2. It aims to understand how well participants tolerate this potential new treatment and its effectiveness in treating their muscular dystrophy.
What are the potential side effects?
While the trial primarily focuses on safety and tolerability of SRP-6004 for muscular dystrophy patients, potential side effects are not detailed here. Generally, such interventions may cause immune reactions or muscle-related symptoms.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have specific genetic mutations in the DYSF gene.
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My arm and leg muscles are suitable for biopsy.
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I can participate in tests that measure my muscle movements.
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I can walk on my own without help.
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My test shows low levels of rAAVrh74 antibodies.

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-6004Experimental Treatment1 Intervention
Participants will receive single IV infusion of SRP-6004 on Day 1.

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Limb-Girdle Muscular Dystrophy (LGMD) include gene therapy and exon skipping, which aim to correct or bypass genetic mutations responsible for the disease. SRP-6004, for example, is a gene therapy targeting dysferlin deficiency in LGMD2B/R2. This approach involves delivering a functional copy of the dysferlin gene to muscle cells, thereby restoring the production of the dysferlin protein, which is essential for muscle membrane repair. These treatments are significant for LGMD patients as they target the root cause of the disease, offering the potential for more effective and durable therapeutic outcomes compared to symptomatic treatments.
Limb-Girdle Muscular Dystrophy 2B and Miyoshi Presentations of Dysferlinopathy.Mutations in Czech LGMD2A patients revealed by analysis of calpain3 mRNA and their phenotypic outcome.

Find a Location

Who is running the clinical trial?

Sarepta Therapeutics, Inc.Lead Sponsor
52 Previous Clinical Trials
33,966 Total Patients Enrolled
Medical DirectorStudy DirectorSarepta Therapeutics, Inc.
2,905 Previous Clinical Trials
8,091,467 Total Patients Enrolled

Media Library

SRP-6004 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05906251 — Phase 1
Muscular dystrophy Research Study Groups: SRP-6004
Muscular dystrophy Clinical Trial 2023: SRP-6004 Highlights & Side Effects. Trial Name: NCT05906251 — Phase 1
SRP-6004 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05906251 — Phase 1
~1 spots leftby Aug 2028
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