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Study on the Safety and Effectiveness of Delandistrogene Moxeparvovec for Patients with Duchenne Muscular Dystrophy

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle weakness and degeneration. The study will evaluate a treatment called Delandistrogene Moxeparvovec, also known by its code name SRP-9001. This treatment is a type of gene therapy, which involves introducing new genetic material into the body to help treat the disease. The trial will also use a placebo, which is a substance with no active therapeutic effect, to compare the results.

The purpose of the study is to assess the safety and effectiveness of Delandistrogene Moxeparvovec in both non-ambulatory (those who cannot walk) and ambulatory (those who can walk) participants with Duchenne Muscular Dystrophy. Participants will receive the treatment through an intravenous infusion, which means the solution is delivered directly into the bloodstream. The study will monitor changes in muscle function and other health indicators over a period of time, with key assessments occurring at 72 weeks after the start of the trial.

Throughout the study, researchers will track various health outcomes, including muscle strength and respiratory function, to determine the impact of the treatment. The trial will also measure the amount of dystrophin, a protein that is typically lacking in individuals with Duchenne Muscular Dystrophy, produced by the body after receiving the gene therapy. Participants will be closely monitored for any side effects or adverse reactions to ensure their safety during the trial.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, eligibility is confirmed based on specific criteria, including a diagnosis of Duchenne Muscular Dystrophy (DMD) and age requirements for certain groups.

    Participants must have stable doses of oral corticosteroids and meet other health criteria.

  2. Step 2

    Randomization and treatment assignment

    Participants are randomly assigned to receive either the gene therapy delandistrogene moxeparvovec or a placebo, which is a saline solution.

    The treatment is administered through an intravenous infusion.

  3. Step 3

    Treatment administration

    The gene therapy or placebo is given as a single dose through an intravenous infusion.

    The infusion is a solution specifically prepared for this study.

  4. Step 4

    Monitoring and follow-up

    Participants are monitored for changes in muscle function and other health indicators over a period of 72 weeks.

    Regular assessments include tests of upper limb performance and lung function, as well as measurements of dystrophin protein levels.

  5. Step 5

    Safety and efficacy evaluation

    The study evaluates the safety of the treatment by tracking any adverse events or side effects.

    Efficacy is assessed by measuring improvements in muscle function and other health outcomes.

  6. Step 6

    Completion of the study

    The study is expected to conclude in September 2027.

    Final assessments will determine the overall impact of the treatment on participants' health and quality of life.

Who can join the trial?

7 criteria

  • The patient must have a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD), which is a genetic disorder that affects muscles, based on clinical findings and previous genetic tests.
  • For Cohort 1, the patient must be non-ambulatory, meaning they cannot walk, according to specific criteria in the study.
  • For Cohort 2, the patient must be able to walk and be between 8 and 18 years old at the time of screening.
  • The patient must be able to cooperate with tests that assess their ability to move.
  • The patient must have been taking a stable daily dose of oral corticosteroids (a type of medication) for at least 12 weeks before the screening, and the dose should remain the same during the study, except for changes due to weight adjustments.
  • The patient should not have elevated levels of antibodies against Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74), which is a type of virus used in gene therapy, as specified by the study requirements.
  • The patient must have a specific type of genetic mutation called a pathogenic frameshift mutation or a premature stop codon between exons 18 and 79. These are changes in the DNA that affect how proteins are made in the body.

Who cannot join the trial?

4 criteria

  • Only males can participate in the study. Females are not eligible.
  • Participants must have Duchenne Muscular Dystrophy, a specific muscle condition.
  • The study is for a specific age group, so participants must be within the required age range.
  • Participants should not belong to any vulnerable population that might be at risk during the study.
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Investigated drugs

SRP-9001 is a gene transfer therapy being studied for its potential to treat Duchenne Muscular Dystrophy. This therapy involves delivering a specific gene to the body to help produce a protein that is missing or not working properly in patients with this condition. The goal is to improve muscle function and slow the progression of the disease.

What is already known about the treatment

SRP-9001 – This medication is administered through systemic gene transfer therapy, typically via intravenous infusion. It is currently in Phase 3 clinical trials, focusing on its safety and effectiveness for treating Duchenne Muscular Dystrophy, a genetic disorder characterized by progressive muscle degeneration. The main therapeutic indication is to improve muscle function in both non-ambulatory and ambulatory patients. At the molecular level, SRP-9001 works by delivering a functional copy of the dystrophin gene to muscle cells, aiming to restore the production of dystrophin protein, which is deficient in patients with this condition. It is classified as a gene therapy product, representing a novel approach in the treatment of genetic muscular disorders.

Investigated diseases

Duchenne Muscular Dystrophy – Duchenne Muscular Dystrophy is a genetic disorder characterized by progressive muscle weakness and degeneration. It primarily affects boys and is caused by mutations in the dystrophin gene, which is crucial for maintaining muscle cell structure. Symptoms usually begin in early childhood, with affected individuals experiencing difficulty in walking, running, and jumping. As the disease progresses, muscle weakness spreads to the arms, neck, and other areas, leading to increased difficulty in performing daily activities. Over time, individuals may lose the ability to walk and require assistance with mobility. The progression of muscle weakness can also impact respiratory and cardiac functions.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-512626-28-00Protocol codeSRP-9001-303Estimated enrolment114 patientsSponsorSarepta Therapeutics Inc.

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