A Phase 3, Prospective, Open-label, Uncontrolled, Multicenter Study onEfficacy and Safety of Prophylaxis with rVWF in Children Diagnosed With Severe von Willebrand disease

Study on Preventive Treatment with Investigational Medication for Severe von Willebrand Disease in Children

K
Kerry Hege, MD

Primary Investigator

Enrolling By Invitation
17 years or below
All
Phase 3
1 participants needed
1 Location

Brief description of study

What is the purpose of this study?
The main aim of the study is to evaluate the effectiveness of prophylaxis with recombinant von Willebrand factor (rVWF) in children. This study will enroll those participants who have been previously treated with VWF product or with a plasma-derived VWF (pdVWF) product. 

 

Detailed description of study

What will happen during the study?
  • In this study, participants will be treated with rVWF for 12 months.
  • During the study, participants will visit the study clinic 6 times after treatment initiation.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Von Willebrand Disease, VWD, Riley
  • Age: 17 years or below
  • Gender: All

Inclusion Criteria:
  •  Prescreening treatment requirements:
  • The participant has been receiving OD therapy with VWF products for at least 12 months (for participants >=2 years of age) prior to screening, has experienced at least 1 VWF-treated bleeding event during (excluding menorrhagia/heavy menstrual bleeding [HMB], as applicable) in the last 12 months, and prophylactic treatment is recommended by the investigator (Prior OD participants); or
  •        The participant has been receiving prophylactic treatment with pdVWF products for at least 12 months prior to screening (for participants >=2 years of age) and switching to prophylaxis with rVWF is recommended by the investigator (Switch participants).
  •        For participants <2 years of age, the required duration for prior OD therapy with VWF products or for prior prophylactic treatment with pdVWF products is at least 6 months. Prior OD participants <2 years of age should have experienced at least 1 VWF-treated bleeding event during the last 6 months based on medical records and be recommended to receive prophylactic treatment by the investigator.
  •    For participants >=2 years of age, the participant has available records that reliably evaluate type, frequency, severity, and treatment of BEs for at least 12 months preceding enrollment. For participants <2 years of age, the participant has available records that reliably evaluate type, frequency, severity and treatment of BEs for at least 6 months preceding enrollment.
  •    If >=12 years old at the time of screening, the participant has a body mass index (BMI) >=15 but <40 kilogram per square meter (kg/m^2). If >=2 to <12 years old at the time of screening, the participant has a BMI of >=5th and <95th percentile (per Centers for Disease Control and Prevention [CDC] clinical charts). For younger participants who are <2 years old, the "weight-for-age" clinical charts (5th to 95th percentile) provided by the CDC should be utilized to ensure the participant has a body weight of >=5th and <95th percentile based on gender (for clinical charts provided by CDC, refer to: https://www.cdc.gov/growthcharts/clinical_charts.htm).
  •    Female participants of childbearing potential (that is, had onset of menses/reached puberty) must have a negative blood/urine pregnancy test result at screening and agree to employ highly effective birth control measures for the duration of their participation in the study.
  •    The participant has voluntarily provided assent (if appropriate) and the legally authorized representative(s) has provided informed consent.
  •    The participant and/or legally authorized representative is willing and able to comply with the requirements of the protocol, which should also be confirmed based on a prescreening evaluation held between the investigator and the sponsor to ensure no eminent risk is present that could challenge the participant's compliance with the study requirements.


Exclusion Criteria:
 

  •     The participant has been diagnosed with pseudo VWD or another hereditary or acquired coagulation disorder other than VWD (example, qualitative and quantitative platelet disorders or elevated prothrombin time/international normalized ratio 1.4).
  •    The participant has a history or presence of a VWF inhibitor at screening.
  •    The participant has a history or presence of an factor VIII (FVIII) inhibitor with a titer >=0.4 Bethesda units (BU) (by the Nijmegen-modified Bethesda assay) or >=0.6 BU (by the Bethesda assay).
  •    The participant has a known hypersensitivity to any of the components of the study drugs, such as mouse or hamster proteins.
  •    The participant has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies, or animal allergies.
  •    The participant has a medical history of a thromboembolic event.
  •    The participant is human immunodeficiency virus (HIV)-positive with an absolute helper T cell (CD4) count <200 per cubic millimeter or microliter (/mm^3).
  •    The participant has been diagnosed with significant liver disease per the investigator's medical assessment of the participant's current condition or medical history or as evidenced by, but not limited to, any of the following: serum alanine aminotransferase (ALT) greater than 5 times the upper limit of normal (ULN), hypoalbuminemia, portal vein hypertension (example, presence of otherwise unexplained splenomegaly, history of esophageal varices), or liver cirrhosis classified as Child-Pugh class B or C.
  •    The participant has been diagnosed with renal disease, with a serum creatinine level >=2.5 milligram per deciliter (mg/dL).
  •    The participant has a platelet count <100,000 per milliliter (/mL) at screening (because participants with type 2B VWD are considered eligible for this study, for participants with type 2B VWD, platelet count[s] at screening will be evaluated in consultation with the sponsor, taking into consideration historical trends in platelet counts and the investigator's medical assessment of the participants condition).
  •    The participant has been treated with an immunomodulatory drug, excluding topical treatment (example, ointments, nasal sprays), within 30 days prior to signing the informed consent (or assent, if appropriate).
  •    The participant is pregnant or lactating at the time of enrollment.

Updated on 30 Jun 2025. Study ID: PHO-TAKEDA-TAK-577-3001, 24614

This study investigates the use of prophylaxis with an investigational medication in children diagnosed with severe von Willebrand disease. Von Willebrand disease is a bleeding disorder caused by low levels or dysfunction of von Willebrand factor, a protein that helps blood clot. This study aims to evaluate the effectiveness of this prophylaxis in children who have been previously treated with von Willebrand factor products.

Participants in this study will receive the investigational medication for 12 months. They will need to visit the study clinic for monitoring and assessments six times during the course of the study. The study will observe the safety and efficacy of the treatment in managing the condition.

  • Who can participate: Children aged 2 years and older who have been receiving treatment with von Willebrand factor products for at least 12 months and have experienced at least one bleeding event are eligible. Participants must have a BMI within specified ranges based on age and meet additional health criteria.
  • Study details: Participants will receive the investigational medication for one year. The study will not involve any changes to their existing treatment regimen beyond the introduction of the investigational medication.
  • Study timelines and visits: The study will last 12 months. The study requires 6 visits.
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Interested in the study?

This study is accepting only persons who receive care at a certain clinic or doctor or who are part of an invited group. Questions about this study can be directed to the study team listed in the description or contact your doctor to see if you are eligible.

Accepting Referrals by Invitation Only