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A study to evaluate the long-term safety and effectiveness of BMN 351 in patients with Duchenne muscular dystrophy amenable to exon 51 skipping.

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What is this trial about?

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

This study focuses on Duchenne Muscular Dystrophy, a condition that causes progressive muscle weakness and loss of muscle function. The investigation aims to evaluate the long-term safety and effectiveness of a medication known as BMN 351. This drug is a type of exon skipping therapy, which is a method used to help the body produce a more functional version of a protein that is missing or broken due to genetic errors. Specifically, it is designed for individuals whose genetic makeup allows for this specific treatment approach.

The treatment involves intravenous infusion, meaning the medication is delivered directly into a vein through a tube. During this study, participants receive these infusions on a weekly basis. The process is designed to monitor how the body reacts to the medication over an extended period of time to ensure it can be used safely over the long term.

The research process

The trial runs in 3 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Weekly medication administration

    The treatment involves receiving an intravenous infusion, which is a method where medicine is delivered directly into a vein through a small tube, of the test substance bmn 351.

    The dose for this medication is 12 mg/kg, meaning 12 milligrams of the medicine for every kilogram of body weight.

    This infusion is administered on a weekly basis.

  2. Step 2

    Regular medical monitoring

    Safety is monitored through physical examinations and laboratory tests to check how the body is functioning.

    The heart is monitored using an ecg (an electrical test of heart activity) and an echocardiogram (an ultrasound to look at the structure of the heart).

    Side effects and any unexpected medical events are recorded to evaluate how well the body tolerates the medicine.

  3. Step 3

    Periodic functional assessments

    Every 24 weeks, certain physical abilities are measured using specific scales and tests, such as the nsaa (a scale used to check muscle strength and movement) and the timed 4sc (a test of physical function).

    Other assessments like sv95c, pul 2.0, and specific timed walking tests are performed to track changes in physical performance over time.

Who can join the trial?

4 criteria

  • Participants must have previously finished the 351-201 or 351-203 studies without stopping the investigational medicinal product, which is the experimental medicine being tested, or leaving the study early.
  • Participants must currently be taking oral corticosteroids, which are a type of medicine used to reduce inflammation, and must stay on a steady, unchanging dose throughout the study, unless the dose needs to change because of changes in the person's weight.
  • Sexually active males must be willing to use contraception, which is a method used to prevent pregnancy, during the study and for 90 days after taking the last dose.
  • The study is specifically for male patients.

Who cannot join the trial?

3 criteria

  • You cannot participate if you have a known coagulation disorder, which is a condition that affects your body's ability to form blood clots to stop bleeding.
  • The study is only for males, so females are not eligible.
  • The study is designed for a specific age range, so if you are outside the required age group, you cannot join.
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Investigated drugs

BMN 351 is a therapy given through an intravenous infusion (a liquid delivered directly into a vein) that is designed to help the body skip a specific part of a gene called exon 51. This process aims to help the body produce a more functional version of a protein that is missing in people with Duchenne Muscular Dystrophy.

What is already known about the treatment

BMN 351 - This medication is an experimental treatment given as a concentrated liquid through an intravenous infusion into a vein once a week. It is currently being studied in clinical trials to see how well it works for people with Duchenne Muscular Dystrophy who have a specific genetic mutation. This drug belongs to a class of medicines called antisense oligonucleotides, which work by binding to specific parts of the genetic code to skip over a faulty section called exon 51. By doing this, the drug helps the body produce a shorter but still functional version of a protein that is essential for healthy muscle function.

Investigated diseases

Duchenne Muscular Dystrophy - This is a genetic condition that primarily affects the muscles. It is caused by a lack of a protein called dystrophin, which helps keep muscle cells intact. Over time, the muscles become progressively weaker. This weakness typically starts in the muscles closer to the center of the body, such as the hips and legs. As the condition continues, the muscle tissue is gradually replaced by fat and fibrous tissue.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-511656-41-00Protocol code351-202Estimated enrolment13 patientsSponsorBiomarin Pharmaceutical Inc.

sourced from the EU Clinical Trials Register and site verification

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