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Study on the Safety and Effects of Delandistrogene Moxeparvovec in Children Under 4 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, a genetic disorder characterized by progressive muscle weakness and degeneration. The treatment being tested is called delandistrogene moxeparvovec, also known by its code name SRP-9001. This treatment is a solution for injection or infusion, designed to deliver a specific gene to help produce a protein called dystrophin, which is typically lacking in individuals with this condition.

The purpose of the study is to evaluate the safety of delandistrogene moxeparvovec in young children under the age of four who have been diagnosed with Duchenne Muscular Dystrophy. Participants will receive the treatment through an intravenous method, which means it will be administered directly into a vein. The study will monitor the participants for any side effects or changes in their health, including any serious adverse events or significant changes in vital signs and laboratory assessments.

The study will also measure the amount of dystrophin protein produced in the body after receiving the treatment, specifically looking at changes from the start of the study to 12 weeks later. This will help researchers understand how well the treatment works in increasing dystrophin levels, which is crucial for improving muscle function in individuals with Duchenne Muscular Dystrophy.

The research process

The trial runs in 5 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, the patient must have a signed informed consent form. If applicable, a signed assent form is also required, depending on the patient's age and local standards.

    The patient must be male and meet specific age requirements for the cohort they are assigned to. For example, Cohort A requires the patient to be 3 years old, while Cohort D requires the patient to be 6 months old or younger.

    A definitive diagnosis of Duchenne Muscular Dystrophy (DMD) is necessary, confirmed by genetic testing showing specific mutations in the DMD gene.

  2. Step 2

    Initial assessment

    The patient undergoes an initial assessment to confirm eligibility, including the ability to cooperate with motor assessment testing.

    The patient's parent or legal guardian must be able to understand and comply with the study visit schedule and protocol requirements.

  3. Step 3

    Treatment administration

    The patient receives the study medication, delandistrogene moxeparvovec-rokl, which is a solution for injection or infusion.

    The medication is administered intravenously, meaning it is given directly into a vein.

  4. Step 4

    Monitoring and follow-up

    The patient is monitored for any treatment-emergent adverse events, serious adverse events, and adverse events of special interest.

    Regular assessments are conducted to check for clinically significant changes in vital signs, physical examination findings, safety laboratory assessments, ECGs, and ECHOs.

    The study also measures changes in the quantity of SRP-9001-dystrophin protein expression from baseline to Week 12 using a method called Western blot.

  5. Step 5

    Study duration

    The study is estimated to continue until November 30, 2032.

    The recruitment for the study began on July 1, 2023.

Who can join the trial?

10 criteria

  • Must have a signed Informed Consent Form. This is a document that explains the study and confirms that you agree to participate.
  • If appropriate, based on age and local standards, must have a signed Assent Form. This is similar to the consent form but is for younger participants.
  • Must be male at birth.
  • Must meet specific age requirements at the time of receiving the study drug:
    • Cohort A: 3 years old
    • Cohort B: 2 years old
    • Cohort C: Older than 6 months but younger than 2 years
    • Cohort D: 6 months old or younger
    • Must have a confirmed diagnosis of **Duchenne Muscular Dystrophy (DMD)** before the screening. This means having medical records showing clinical findings and genetic testing results. The genetic test must show specific changes in the **DMD gene** between exons 18 to 79 that lead to no **dystrophin protein**. Changes in exons 1-17 or other types of genetic changes are not eligible.
    • Must be able to participate in motor assessment tests suitable for their age, as judged by the study doctor.
    • Must have a parent or legal guardian who can understand and follow the study visit schedule and all study requirements.

Who cannot join the trial?

10 criteria

  • Only males can participate, so females are excluded.
  • Participants must be within a specific age range, so those outside this range cannot join.
  • Individuals with certain health conditions that might interfere with the study are not allowed to participate.
  • Participants must not have any other medical conditions that could affect the study results.
  • Those who have taken certain medications recently may be excluded.
  • Participants must not have had any recent surgeries that could impact the study.
  • Individuals who are unable to follow the study procedures or attend all required visits are excluded.
  • Participants must not have any allergies or reactions to the study medication or its ingredients.
  • Those who have participated in another clinical trial recently may not be eligible.
  • Individuals with a history of substance abuse or alcohol dependency might be excluded.
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Investigated drugs

RO7494222 (SRP-9001) is a gene therapy being studied for its potential to treat Duchenne Muscular Dystrophy in young children. This therapy involves delivering a new gene into 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

Delandistrogene Moxeparvovec – This medication is administered through systemic gene delivery, which involves introducing the treatment into the bloodstream to reach affected tissues. It is currently being studied in clinical trials, particularly for its safety and expression in young patients with Duchenne Muscular Dystrophy. The main therapeutic indication is to address Duchenne Muscular Dystrophy, a genetic disorder characterized by progressive muscle degeneration. At the molecular level, the medication 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, a type of treatment that involves modifying or manipulating genes to treat or prevent disease.

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 movement. Over time, individuals may lose the ability to walk and require assistance with daily activities. The progression of muscle weakness can also affect the heart and respiratory muscles.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IITrial ID2023-509901-57-00Protocol codeBN43881Estimated enrolment13 patientsSponsorF. Hoffmann-La Roche AG

sourced from the EU Clinical Trials Register and site verification

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).