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Study on the Safety and Effectiveness of Emicizumab for Patients with Type 3 Von Willebrand Disease

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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 the effects of a medication called Emicizumab in patients with a rare bleeding disorder known as Type 3 Von Willebrand Disease. This condition is characterized by a severe deficiency of a protein called von Willebrand factor, which is essential for blood clotting. The study aims to evaluate how well Emicizumab works in preventing bleeding episodes when used regularly, compared to the usual treatment that is given only when bleeding occurs.

Participants in the study will receive Emicizumab as a preventive treatment, which means it will be administered on a regular schedule to help prevent bleeding episodes. The study will monitor the number of bleeding events over time, as well as any side effects or reactions to the medication. The trial will also assess the overall safety of Emicizumab and its impact on the participants' health, including any changes in their physical condition and laboratory test results.

The study will take place over several years, allowing researchers to gather comprehensive data on the long-term effects of Emicizumab in managing Type 3 Von Willebrand Disease. This research is important for understanding how this medication can improve the quality of life for individuals with this challenging condition by reducing the frequency and severity of bleeding episodes.

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, you will be introduced to the trial procedures and objectives. You will receive detailed information about the study, including the purpose, duration, and what is expected from you as a participant.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to evaluate your current health status. This may include a physical examination, blood tests, and a review of your medical history to confirm your eligibility for the study.

  3. Step 3

    Medication administration

    You will receive the study medication, emicizumab, which is administered as a solution for injection. The medication is given subcutaneously, meaning it is injected under the skin. The dosage and frequency will be determined by the study protocol and your specific needs.

  4. Step 4

    Regular follow-up visits

    You will attend regular follow-up visits to monitor your response to the medication and any side effects. These visits will include physical examinations, blood tests, and discussions about any symptoms or concerns you may have.

  5. Step 5

    Monitoring and reporting

    Throughout the study, you will be asked to report any bleeding episodes, side effects, or other health changes. This information is crucial for assessing the safety and effectiveness of the medication.

  6. Step 6

    End of study evaluation

    At the end of the study, a final evaluation will be conducted. This will include a comprehensive assessment of your health and a review of your experience during the trial. The results will contribute to the overall findings of the study.

Who can join the trial?

6 criteria

  • Must be at least 2 years old at the time of signing the consent form.
  • Must have adequate hematological function, which means the blood is working well enough for the study.
  • Must have a history of using both on-demand (as needed) and prophylactic (preventive) standard care treatments.
  • If able to have children, must agree to follow the contraception requirements to prevent pregnancy during the study.
  • Must have a confirmed diagnosis of Type 3 Von Willebrand Disease (VWD), based on specific blood tests that measure VWF and FVIII levels.
  • Must have information about the status of VWF inhibitor, which is a substance that can affect how the treatment works.

Who cannot join the trial?

8 criteria

  • Patients with any other bleeding disorders besides Type 3 Von Willebrand Disease cannot participate.
  • Patients who have had a recent major surgery or are planning to have surgery during the study period are not eligible.
  • Patients who are currently participating in another clinical trial are excluded.
  • Patients with a known allergy or severe reaction to emicizumab or any of its ingredients cannot join the study. Emicizumab is a medication used to prevent bleeding episodes.
  • Patients with severe liver or kidney disease are not eligible to participate.
  • Pregnant or breastfeeding women cannot take part in the study.
  • Patients who are unable to comply with the study procedures or follow-up visits are excluded.
  • Patients with any condition that the study doctors believe would make participation unsafe or affect the study results are not eligible.
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Investigated drugs

Emicizumab is a medication used in this clinical trial to help prevent bleeding in patients with Type 3 Von Willebrand Disease. It is given on a regular schedule to reduce the number of bleeding episodes. Emicizumab works by helping the blood to clot more effectively, which is particularly important for people who have a bleeding disorder like Von Willebrand Disease. This medication is being tested to see how well it works compared to the usual treatment that is given only when bleeding occurs.

What is already known about the treatment

Emicizumab – Emicizumab is administered as an injection under the skin, typically on a regular schedule to prevent bleeding episodes. It is currently being studied in clinical trials for its effectiveness and safety in treating Type 3 Von Willebrand Disease, a rare bleeding disorder. The main therapeutic use of Emicizumab in this context is to prevent bleeding in patients with this condition. At the molecular level, Emicizumab works by mimicking a protein that helps blood to clot, thereby reducing the risk of bleeding. It is classified as a monoclonal antibody, which is a type of medication designed to target specific proteins in the body.

Investigated diseases

Type 3 Von Willebrand Disease – This is the most severe form of Von Willebrand Disease, a genetic bleeding disorder caused by a deficiency or dysfunction of the von Willebrand factor, a protein crucial for blood clotting. Individuals with this condition have very low or undetectable levels of von Willebrand factor and often have low levels of factor VIII, another protein important for clotting. The disease is characterized by frequent and severe bleeding episodes, which can occur spontaneously or after minor injuries. Bleeding can affect various parts of the body, including the skin, mucous membranes, joints, and muscles. Women with this condition may experience heavy menstrual bleeding. The progression of the disease involves recurrent bleeding episodes that can lead to complications if not managed properly.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-515622-80-00Protocol codeWP45338Estimated enrolment79 patientsSponsorF. Hoffmann-La Roche AG

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