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Bidridistrogene Xeboparvovec

Bidridistrogene Xeboparvovec, also known as SRP-9003, is an investigational gene therapy being studied for the treatment of Limb-Girdle Muscular Dystrophy Type 2E/R4 (LGMD2E/R4). This article discusses the ongoing clinical trials evaluating the safety and effectiveness of this promising therapy for patients with this rare genetic muscle disorder.

Table of Contents

What is BIDRIDISTROGENE XEBOPARVOVEC?

BIDRIDISTROGENE XEBOPARVOVEC, also known as SRP-9003, is an innovative gene therapy medication designed to treat a specific type of muscular dystrophy[1]. This therapy is currently being studied in clinical trials to evaluate its safety and effectiveness in patients with Limb-Girdle Muscular Dystrophy Type 2E/R4 (LGMD2E/R4).

The medication is classified as an advanced therapy medicinal product, specifically a gene therapy[1]. It uses a modified virus called adeno-associated virus serotype rh74 (AAVrh74) to deliver a healthy copy of the β-sarcoglycan gene to muscle cells[1].

Target Condition: Limb-Girdle Muscular Dystrophy Type 2E/R4

BIDRIDISTROGENE XEBOPARVOVEC is designed to treat Limb-Girdle Muscular Dystrophy Type 2E/R4 (LGMD2E/R4), also known as β-sarcoglycanopathy[1]. This is a rare genetic disorder that causes progressive muscle weakness, primarily affecting the shoulders, upper arms, pelvic area, and thighs. The condition is caused by mutations in the gene that produces a protein called β-sarcoglycan, which is important for maintaining muscle fiber strength and stability.

How Does BIDRIDISTROGENE XEBOPARVOVEC Work?

BIDRIDISTROGENE XEBOPARVOVEC works by introducing a functional copy of the β-sarcoglycan gene into the patient’s muscle cells[1]. Here’s a simplified explanation of the process:

  1. The medication contains a modified virus (AAVrh74) that carries a healthy copy of the β-sarcoglycan gene.
  2. This virus is engineered to be harmless and serves only as a delivery vehicle for the gene.
  3. The medication is administered to the patient through intravenous infusion (directly into the bloodstream).
  4. The virus travels through the bloodstream and enters muscle cells.
  5. Once inside the cells, the healthy gene is released, allowing the cells to produce the β-sarcoglycan protein.
  6. The presence of this protein helps strengthen and stabilize muscle fibers, potentially slowing or halting the progression of the disease.

Current Clinical Trial

BIDRIDISTROGENE XEBOPARVOVEC is currently being studied in a Phase 3 clinical trial[1]. This trial is designed to evaluate the safety and efficacy of the treatment in patients with LGMD2E/R4. The study is multinational and open-label, meaning that both the researchers and participants know which treatment is being administered.

The trial includes two groups of participants:

  • Cohort 1: Ambulatory subjects (able to walk without assistance)
  • Cohort 2: Non-ambulatory subjects (unable to walk or requiring significant assistance)

Who Can Participate in the Trial?

The trial has specific criteria for who can participate. Some key eligibility requirements include[1]:

  • Age 4 years or older
  • Confirmed genetic diagnosis of LGMD2E/R4
  • For Cohort 1 (ambulatory): Able to walk without assistance and complete certain physical tests
  • For Cohort 2 (non-ambulatory): Unable to walk or requiring significant assistance, but able to perform certain upper limb functions
  • No recent infections or major surgeries
  • No previous gene therapy treatments
  • No significant heart or lung problems

What Are the Trial Objectives?

The main goals of the clinical trial include[1]:

  • Evaluating the effect of SRP-9003 on β-sarcoglycan expression in muscle tissue
  • Assessing improvements in physical function over time
  • Monitoring the safety of the treatment
  • Measuring changes in muscle enzyme levels (creatine kinase)
  • Tracking disease milestones, such as loss of ambulation (ability to walk)

Safety Considerations

As with any experimental treatment, there are potential risks and safety considerations[1]. The clinical trial closely monitors participants for:

  • Side effects related to the treatment
  • Immune reactions to the viral vector
  • Changes in heart and lung function
  • Abnormal laboratory values

To help manage potential immune reactions, participants receive a course of glucocorticoids (a type of steroid) starting shortly before the treatment[1].

Aspect Details
Study Type Phase 3, multinational, open-label gene delivery study
Condition Limb-Girdle Muscular Dystrophy Type 2E/R4 (LGMD2E/R4)
Intervention Bidridistrogene Xeboparvovec (SRP-9003)
Primary Objective Evaluate β-sarcoglycan expression at Day 60 post-dose
Key Secondary Objectives Assess physical function, safety, and disease progression
Eligibility Patients ≥4 years old with confirmed LGMD2E/R4 mutations
Study Duration 60 months (5 years) follow-up after treatment
Key Assessments Muscle biopsies, functional tests, safety monitoring

FAQ

  • What is Bidridistrogene Xeboparvovec? Bidridistrogene Xeboparvovec, also known as SRP-9003, is a gene therapy designed to treat Limb-Girdle Muscular Dystrophy Type 2E/R4. It uses a modified virus to deliver a healthy copy of the β-sarcoglycan gene to muscle cells.
  • What is the main goal of the clinical trial? The main goal is to evaluate the effect of SRP-9003 on β-sarcoglycan expression in muscle cells 60 days after treatment, measured by the percentage of muscle fibers expressing the protein.
  • Who can participate in the trial? The trial includes both ambulatory (able to walk) and non-ambulatory patients aged 4 years and older with confirmed LGMD2E/R4 genetic mutations. Participants must meet specific criteria related to their physical abilities and medical history.
  • How is the treatment administered? SRP-9003 is administered through intravenous infusion. Patients receive a single dose of the gene therapy along with glucocorticoids to help manage the immune response to the treatment.
  • How long does the trial last? The trial includes a screening period, a lead-in period, treatment administration, and a 60-month (5-year) follow-up period to assess long-term safety and effectiveness of the therapy.

Ongoing Clinical Trials on Bidridistrogene Xeboparvovec

  • Study on the Safety and Effectiveness of SRP-9003 and Prednisone for Patients with Limb-Girdle Muscular Dystrophy Type 2E/R4

    Not recruiting

    3 1 1 1
    Investigated drugs:
    Belgium Germany Italy Spain

Glossary

  • Limb-Girdle Muscular Dystrophy Type 2E/R4 (LGMD2E/R4): A rare genetic disorder causing progressive muscle weakness, primarily affecting the shoulders, upper arms, pelvic area, and thighs.
  • β-sarcoglycan (β-SG): A protein important for muscle function, which is deficient in patients with LGMD2E/R4.
  • Gene therapy: A treatment approach that involves introducing genetic material into cells to compensate for abnormal genes or to produce a beneficial protein.
  • Adeno-associated virus (AAV): A small virus used as a vehicle to deliver genetic material in gene therapy.
  • Immunofluorescence (IF): A laboratory technique used to detect specific proteins in cells or tissues using fluorescent antibodies.
  • Western blot: A laboratory method used to detect and analyze specific proteins in a mixture of proteins.
  • Creatine kinase (CK): An enzyme found in various tissues, especially muscles. Elevated levels can indicate muscle damage or disease.
  • North Star Assessment for Dysferlinopathy (NSAD): A standardized test used to evaluate motor function in patients with certain types of muscular dystrophy.
  • Performance of Upper Limb (PUL) 2.0: A test designed to assess upper limb function in patients with neuromuscular disorders.
  • 10-meter walk test (10MWT): A timed test measuring how long it takes a person to walk 10 meters, used to assess walking ability and speed.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-srp-9003-and-prednisone-for-patients-with-limb-girdle-muscular-dystrophy-type-2e-r4/