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Long-term Safety Study of Delandistrogene Moxeparvovec for Patients with Duchenne Muscular Dystrophy Who Previously Received SRP-9001

Verified siteInvestigationalNo placebo
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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 evaluated is called Delandistrogene Moxeparvovec, also known by its code name SRP-9001. This treatment is a solution for injection or infusion, designed to help manage the symptoms of Duchenne Muscular Dystrophy.

The purpose of this study is to assess the long-term safety of Delandistrogene Moxeparvovec in participants who have previously received this treatment in an earlier clinical study. Participants will be monitored over a period of time to observe any potential side effects or adverse events that may occur after receiving the treatment. The study will also track changes in participants' physical abilities, such as their ability to walk, run, and perform upper limb tasks, as well as their lung function and heart health.

Throughout the study, participants will undergo various assessments, including the North Star Ambulatory Assessment and Magnetic Resonance Imaging (MRI), to evaluate changes in their condition from the time before they received the treatment to five years after. This long-term follow-up is crucial to understanding the overall impact of Delandistrogene Moxeparvovec on individuals with Duchenne Muscular Dystrophy.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying that the participant has previously received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a prior clinical study.

    The participant or their legal caregiver must demonstrate the ability to understand and comply with the study visit schedule and protocol requirements.

  2. Step 2

    Treatment administration

    The study involves the administration of delandistrogene moxeparvovec, which is a solution for injection or infusion given intravenously.

    This step is a follow-up to the initial treatment received in a previous study and does not involve new administration during this trial.

  3. Step 3

    Monitoring and follow-up

    Participants are monitored for any treatment-emergent adverse events, serious adverse events, and adverse events of special interest.

    The study aims to evaluate the long-term safety of delandistrogene moxeparvovec over a period of up to five years post-infusion.

  4. Step 4

    Assessment of physical abilities

    Changes in physical abilities are assessed using various measures, including the North Star Ambulatory Assessment, time to rise from the floor, and time for a 10-meter walk or run.

    These assessments are conducted from the pre-infusion baseline to five years post-infusion.

  5. Step 5

    Evaluation of respiratory and cardiac function

    Changes in respiratory function are evaluated through measures such as forced vital capacity and peak expiratory flow percent predicted.

    Cardiac function is assessed using cardiac magnetic resonance imaging (MRI) findings.

  6. Step 6

    Musculoskeletal evaluation

    Musculoskeletal MRI findings are evaluated to assess any changes from the pre-infusion baseline to five years post-infusion.

  7. Step 7

    Completion of study

    The study is estimated to conclude by November 15, 2030, with all assessments and evaluations completed by this date.

Who can join the trial?

3 criteria

  • The patient must have received **delandistrogene moxeparvovec** for **Duchenne Muscular Dystrophy** in a previous clinical study. This means they have already been treated with a specific medication for a muscle condition.
  • The patient must have a parent or legal caregiver, or be at least 18 years old and able to understand and follow the study visit schedule and all other study requirements. This means they need to be able to attend appointments and follow instructions.
  • The study is only for **male** patients. This means only boys or men can participate.

Who cannot join the trial?

4 criteria

  • Only males can participate in the study. Females are not eligible.
  • Participants must have a specific condition called Duchenne Muscular Dystrophy.
  • Participants must fall within certain age ranges, which are not specified here.
  • There are no specific exclusion criteria listed, but participants must meet the general requirements mentioned above.
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Investigated drugs

SRP-9001 is a gene therapy being studied for its long-term safety and effectiveness. It is designed to help treat certain genetic conditions by delivering a specific gene to the body. This therapy aims to address the underlying cause of the condition by providing the body with the necessary genetic material to produce a missing or faulty protein.

What is already known about the treatment

Delandistrogene Moxeparvovec – This medication is administered through an intravenous infusion, allowing it to be delivered directly into the bloodstream. It is currently being studied in clinical trials, with a focus on its long-term safety and effectiveness. The primary therapeutic indication for this medication is Duchenne Muscular Dystrophy, a genetic disorder characterized by progressive muscle degeneration. At the molecular level, delandistrogene moxeparvovec 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, representing a novel approach in the treatment of genetic 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 loss of mobility. Over time, complications can arise in the heart and respiratory muscles, further impacting physical abilities. The progression of muscle deterioration varies among individuals, but it generally leads to significant physical disability.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-505043-39-00Protocol codeSRP-9001-305Estimated enrolment393 patientsSponsorSarepta Therapeutics Inc.

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