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Study on the Effectiveness and Safety of Vonicog Alfa with or without Octocog Alfa for Children with Severe von Willebrand Disease Experiencing Bleeding or Undergoing Surgery

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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 a condition called Severe von Willebrand Disease in children. This is a hereditary bleeding disorder where the blood does not clot properly, leading to excessive bleeding. The study is testing the effectiveness, safety, and tolerability of a treatment called Recombinant Von Willebrand Factor (rVWF), which may be used with or without another medication called Advate. These treatments are given as injections to help control bleeding episodes and to assist during surgeries, whether they are planned or emergencies.

The purpose of the study is to evaluate how well rVWF works in managing bleeding events in children under 18 years old who have severe von Willebrand Disease. The study will also look at how safe the treatment is and how well children tolerate it. Participants in the study will receive the treatment over a period of time, and their response to the treatment will be monitored. Some children may receive a placebo, which is a substance with no active medication, to compare the effects of the treatment.

Throughout the study, researchers will collect information on how effective the treatment is in stopping bleeding, how many doses are needed, and any side effects that may occur. They will also study how the body processes the treatment, which is known as pharmacokinetics. This includes understanding how long the treatment stays in the body and how it is broken down. The study aims to provide valuable information that could improve the management of severe von Willebrand Disease in children.

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, the patient is diagnosed with severe von Willebrand Disease, a condition affecting blood clotting.

    The patient is under 18 years of age and has provided necessary consent to participate.

  2. Step 2

    Treatment initiation

    The patient begins treatment with recombinant Von Willebrand Factor (rVWF), which may be administered with or without ADVATE.

    The treatment is designed to manage and control bleeding episodes.

  3. Step 3

    Medication administration

    The medications are provided as a solution for injection and are administered through intravenous use.

    The specific products used include ADVATE 1000 IU, VEYVONDI 1300 IU, VEYVONDI 650 IU, and ADVATE 500 IU.

  4. Step 4

    Monitoring and evaluation

    The effectiveness of the treatment is evaluated based on the control of bleeding episodes, using a 4-point scale to measure success.

    The safety of the treatment is monitored by assessing any adverse events or reactions.

  5. Step 5

    Surgery considerations

    For patients undergoing surgery, the treatment's effectiveness is assessed 24 hours after the last infusion.

    The evaluation is conducted by a hematologist using a 4-point scale.

  6. Step 6

    Pharmacokinetics study

    The study includes an analysis of how the medication is processed in the body, measuring factors like concentration over time and clearance rates.

    This analysis helps understand the medication's behavior in different age groups.

Who can join the trial?

8 criteria

  • The child must have a diagnosis of **severe von Willebrand Disease (VWD)**. This means their blood has very low levels of a protein called von Willebrand factor (VWF), which helps with blood clotting.
  • The child must be younger than 18 years old at the time of joining the study.
  • The child, if old enough, must agree to participate, and their parent or guardian must give permission for them to join the study.
  • If the child is a girl who can become pregnant, she must have a negative pregnancy test before joining the study.
  • If the child is a girl who can become pregnant, she must agree to use birth control during the study.
  • The child and their parent or guardian must be willing and able to follow the study rules and requirements.
  • If the child has been treated before, they must have had at least one bleeding event in the past year that needed treatment with a VWF product, and they must have used VWF treatment on at least three different days in the past.
  • If the child has not been treated before, they must not have received any VWF treatment in the past.

Who cannot join the trial?

7 criteria

  • Children who do not have a diagnosis of **severe hereditary von Willebrand Disease** cannot participate. This is a genetic condition that affects blood clotting.
  • Children who are 18 years or older are not eligible to join the study.
  • Children who have any other medical conditions that might interfere with the study's treatment or results may be excluded.
  • Children who are unable to follow the study procedures or attend required visits may not be able to participate.
  • Children who have had a recent major surgery or are planning to have surgery soon might not be eligible.
  • Children who are currently participating in another clinical trial may not be allowed to join this study.
  • Children who have allergies or reactions to the study medications or similar treatments may be excluded.
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Investigated drugs

  • rVWF

    is a medication used in this clinical trial to help manage bleeding episodes in children with severe von Willebrand Disease. It works by replacing the von Willebrand factor, a protein that is missing or not functioning properly in these patients, which is essential for blood clotting.

  • ADVATE

    is another medication used in combination with rVWF in this trial. It is a form of factor VIII, a protein that helps blood to clot. When used together with rVWF, it aims to improve the control of bleeding episodes in patients with von Willebrand Disease.

What is already known about the treatment

rVWF – This medication is administered intravenously and is currently being studied in clinical trials for its effectiveness and safety in treating severe hereditary von Willebrand Disease in children. It is used to manage and control bleeding episodes, as well as during surgeries. The main therapeutic indication is to aid in blood clotting for patients with this genetic disorder. At the molecular level, rVWF works by replacing the deficient von Willebrand factor, which is crucial for platelet adhesion and aggregation, essential steps in the blood clotting process. It is classified as a hemostatic agent.

Investigated diseases

Hereditary 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 experience frequent and prolonged bleeding episodes, which can occur spontaneously or after an injury. The disease is present from birth and can vary in severity, with some individuals experiencing mild symptoms and others having severe bleeding issues. In children, the condition can lead to challenges in managing bleeding during everyday activities and minor injuries. The progression of the disease involves ongoing bleeding risks, requiring careful management to prevent complications.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IIITrial ID2023-509769-18-00Protocol code071102Estimated enrolment31 patientsSponsorBaxalta Innovations GmbH

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.

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