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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 baseline up to month 60

Awards & highlights

Study Summary

This trial tests if a drug is safe for people with a muscle-wasting disorder called 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.Check my eligibility

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.See study design

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline up to month 60

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline up to month 60

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to 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

Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)

Secondary outcome measures

Change from Baseline in Percent of Normal DYSF Protein Expression as Assessed by IF Percent DYSF Positive Fibers (PPF: DYSF)

Change from Baseline in Percent of Normal DYSF Protein Expression as Measured by Immunofluorescence (IF) Fiber Intensity

Change from Baseline in Percent of Normal DYSF Protein Expression as Measured by Western Blot

Trial Design

1Treatment groups

Experimental Treatment

Group I: SRP-6004Experimental Treatment1 Intervention

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

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

Sarepta Therapeutics, Inc.Lead Sponsor

49 Previous Clinical Trials

33,537 Total Patients Enrolled

Medical DirectorStudy DirectorSarepta Therapeutics, Inc.

2,777 Previous Clinical Trials

8,063,478 Total Patients Enrolled

Media Library

SRP-6004 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05906251 — Phase 1

Limb-Girdle Muscular Dystrophy Research Study Groups: SRP-6004

Limb-Girdle 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

Frequently Asked Questions

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

Is there any way for me to participate in this experiment?

"Those hoping to take part in this clinical trial must be diagnosed with limb-girdle muscular dystrophy, aged between 18 and 50. Only a maximum of 2 patients can join the study."

Answered by AI

Are there vacancies for volunteers in this investigation?

"Clinicaltrials.gov shows that this study, posted on May 22nd 2023 and last refreshed June 7th 2023, is not currently open for enrollment. Nonetheless, there are 93 other clinical trials actively seeking participants."

Answered by AI

Is the age restriction for this trial above or below thirty years of age?

"As outlined by the trial's ineligibility parameters, only those aged 18 and above yet below 50 can enroll."

Answered by AI

Has SRP-6004 obtained regulatory approval from the Food and Drug Administration?

"SRP-6004 has limited clinical data to support its safety and efficacy, thus earning it a 1 on this scale."

Answered by AI

Who else is applying?

What portion of applicants met pre-screening criteria?

Did not meet criteria

Met criteria

What site did they apply to?

Nationwide Children's Hospital

Why did patients apply to this trial?

I want to cure my disease. To be healthy and self sufficient again. I have though of trying an experimental drug for a while now.

PatientReceived 1 prior treatment

Arms muscles legs muscle thigs sholder weekness I m not welking sciting. I, ve tried different treatments... Ayurvedic, homeopathy, etc... As in medical science there is no treatment for this... So now I'm willing to explore this therapy to improve my condition.

PatientReceived 2+ prior treatments

Recent research and studies

clinicaltrials.govStudy

A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)

2023. "A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05906251.

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