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Study on Preventing Bleeding in Children with Severe von Willebrand Disease Using Vonicog Alfa and Octocog Alfa

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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 effectiveness and safety of a treatment for children with a severe form of von Willebrand disease (VWD). VWD is a genetic disorder that affects the blood's ability to clot properly, leading to excessive bleeding. The treatment being tested is called rVWF, which stands for recombinant von Willebrand factor. This is a lab-made version of a protein that helps blood clot. The study aims to see how well this treatment can prevent bleeding episodes in children with severe VWD.

Participants in the study will receive the treatment rVWF regularly to prevent bleeding. The study will compare the number of bleeding episodes they experience during the study to the number they had before joining the study. This will help determine if the treatment is effective in reducing bleeding. The study will also monitor for any side effects or reactions to the treatment, such as allergic reactions or changes in vital signs.

The study will last for up to 12 months, during which time participants will receive the treatment through an injection into a vein, known as intravenous use. The goal is to gather information on how well the treatment works and how safe it is for children with severe von Willebrand disease. The results will help doctors understand if this treatment can be a reliable option for managing this condition in children.

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 will be informed about the trial's purpose and procedures. The patient or their legal representative will provide consent to participate.

    The patient will undergo a screening process to confirm eligibility, which includes verifying the diagnosis of severe von Willebrand disease and reviewing medical history.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to establish baseline health status. This includes recording the patient's history of bleeding episodes and any previous treatments received.

    The patient's body mass index (BMI) and other relevant health metrics will be measured.

  3. Step 3

    Treatment phase

    The patient will begin prophylactic treatment with recombinant von Willebrand factor (rVWF). This involves regular intravenous injections to prevent bleeding episodes.

    The specific dosage and frequency of rVWF will be determined by the healthcare provider based on the patient's needs.

  4. Step 4

    Monitoring and follow-up

    Throughout the study, the patient will attend regular follow-up visits to monitor the effectiveness and safety of the treatment.

    Healthcare providers will assess the annualized bleeding rate (ABR) and any side effects or adverse reactions to the treatment.

  5. Step 5

    Completion of the study

    At the end of the study period, a final assessment will be conducted to evaluate the overall efficacy and safety of the prophylactic treatment.

    The patient will receive a summary of their participation and any relevant findings from the study.

Who can join the trial?

8 criteria

  • The patient must have a confirmed diagnosis of **severe von Willebrand Disease (VWD)**, which is a condition that affects blood clotting. This includes having a specific test result (VWF:RCo <20 IU/dL) and a history of needing treatment to control bleeding. The diagnosis can be confirmed by genetic testing or other specific tests.
  • The patient must be under 18 years old at the time of joining the study.
  • The patient must have been receiving treatment for bleeding episodes for at least 12 months if they are 2 years or older, or for at least 6 months if they are younger than 2 years. This treatment could be either as needed (On Demand) or regular preventive treatment (prophylaxis).
  • The patient must have medical records that show details about their bleeding episodes, such as type, frequency, and severity, for at least 12 months if they are 2 years or older, or for at least 6 months if they are younger than 2 years.
  • If the patient is 12 years or older, they must have a **Body Mass Index (BMI)** between 15 and 40 kg/m². If they are between 2 and 12 years old, their BMI should be between the 5th and 95th percentile according to growth charts. For those under 2 years old, their weight should also be between the 5th and 95th percentile based on gender-specific growth charts.
  • Female patients who have started menstruating must have a negative pregnancy test and agree to use effective birth control during the study.
  • The patient must agree to participate in the study, and their parent or guardian must give permission.
  • The patient and their parent or guardian must be willing and able to follow the study's requirements, as confirmed by a preliminary evaluation.

Who cannot join the trial?

8 criteria

  • Patients with a history of severe allergic reactions to the study medication.
  • Patients who have received another investigational drug within the last 30 days.
  • Patients with a medical condition that the study doctor believes would make it unsafe for them to participate.
  • Patients who are pregnant or breastfeeding.
  • Patients who are unable to comply with the study procedures.
  • Patients with a history of drug or alcohol abuse that could interfere with the study.
  • Patients with a known history of poor adherence to medical treatments.
  • Patients with any other condition that the study doctor thinks would make it difficult for them to participate.
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Investigated drugs

rVWF is a medication used in this clinical trial to help prevent bleeding in children with severe von Willebrand disease. It is a recombinant form of von Willebrand factor, which is a protein that helps blood to clot. The trial aims to evaluate how effective and safe this medication is when used regularly to prevent bleeding episodes.

What is already known about the treatment

rVWF – This medication is administered intravenously and is used as a prophylactic treatment for children with severe von Willebrand Disease. It is currently being studied in a Phase 3 clinical trial to assess its efficacy and safety. The main therapeutic indication is to reduce the annualized bleeding rate in patients. At the molecular level, rVWF works by replacing the deficient or defective von Willebrand factor, which is crucial for blood clotting. It is classified as a hemostatic agent.

Investigated diseases

Severe von Willebrand Disease – This is a genetic disorder that affects the blood's ability to clot properly. It is caused by a deficiency or dysfunction of von Willebrand factor, a protein that is crucial for blood clotting. Individuals with this condition may experience frequent and prolonged bleeding episodes, which can occur spontaneously or as a result of injury. The severity of bleeding can vary, with some individuals experiencing mild symptoms and others having more severe bleeding issues. Common symptoms include nosebleeds, easy bruising, and excessive bleeding during surgical procedures or after injuries. In women, it may also cause heavy menstrual bleeding.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IIITrial ID2023-509877-22-00Protocol codeTAK-577-3001Estimated enrolment28 patientsSponsorTakeda Development Center Americas Inc.

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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).