Clinical trial Recruiting at 19 sites Phase 3 Interventional Ages Any age to 17

Evaluating vonicog alfa for children with severe Von Willebrand Disease

Official title: A Phase 3, Prospective, Open-label, Uncontrolled, Multicenter Study on Efficacy and Safety of Prophylaxis With Vonicog Alfa (rVWF) in Children Diagnosed With Severe Von Willebrand Disease

Run by Takeda · NCT05582993 · Data updated 2026-11-12

This study is testing a new treatment called vonicog alfa to see if it can help children with severe Von Willebrand Disease manage their bleeding better.

More Von Willebrand Disease (VWD) trials

Quick facts

PhasePhase 3
Study typeInterventional
AgesAny age to 17 Years
SexAll
Enrollment24 (estimated)
SponsorTakeda
Industry-sponsored
Locations23 sites (Birmingham, Alabama and 22 other locations)
StatusRecruiting
StartedNovember 6, 2024
Main results expectedApril 11, 2030
Study endsApril 11, 2030
Registry record updatedSeptember 9, 2026
Trial IDNCT05582993 on ClinicalTrials.gov

Is this trial a fit for you?

Likely a good fit

Ideal candidates are children under 18 with a documented diagnosis of severe Von Willebrand Disease and a history of treatment with VWF products.

Probably not a fit

Patients who do not have severe Von Willebrand Disease or those who have not previously received VWF treatment may not benefit from this study.

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study aims to assess the effectiveness of vonicog alfa, a recombinant von Willebrand factor, in providing prophylactic treatment for children diagnosed with severe Von Willebrand Disease (vWD). Participants, aged under 18, will receive treatment for 12 months and will visit the clinic five times during this period for monitoring. Eligible candidates must have a history of treatment with VWF products and a documented diagnosis of severe vWD. The study will help determine if this new treatment can effectively manage bleeding episodes in affected children.

Why it matters

Potential benefit: If successful, this treatment could significantly improve bleeding management in children with severe Von Willebrand Disease.

How similar studies have gone: Other studies have shown promise in using recombinant therapies for bleeding disorders, suggesting potential success for this approach.

Who can join: the full rules

Show full inclusion / exclusion criteria
1. The participant has a documented diagnosis of severe VWD (baseline von Willebrand factor ristocetin cofactor activity \[VWF:RCo\] \<20 international units per deciliter \[IU/dL\]) with a history of replacement therapy with VWF concentrate required to control bleeding and a diagnosis of VWD type 1, type 2 (2A, 2B, 2M, 2N), or type 3. Diagnosis is confirmed, as applicable, by genetic testing and/or by multimer analysis, which may be documented in participant's history or at screening.
2. The participant is \<18 years of age at the time of screening.
3. Prescreening treatment requirements:

   1. 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
   2. 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 vonicog alfa (rVWF) is recommended by the investigator (Switch participants).
   3. 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.
4. 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.
5. 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).
6. 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.
7. The participant has voluntarily provided assent (if appropriate) and the legally authorized representative(s) has provided informed consent.
8. 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:

1. 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).
2. The participant has a history or presence of a VWF inhibitor at screening.
3. The participant has a history or presence of an factor VIII (FVIII) inhibitor with a titer \>=0.6 Bethesda units per milliliter (/mL).
4. The participant has a known hypersensitivity to any of the components of the study drugs, such as mouse or hamster proteins.
5. The participant has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies, or animal allergies.
6. The participant has a medical history of a thromboembolic event.
7. The participant is human immunodeficiency virus (HIV)-positive with an absolute helper T cell (CD4) count \<200 per cubic millimeter or microliter (/mm\^3).
8. 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.
9. The participant has been diagnosed with renal disease, with a serum creatinine level \>=2.5 milligram per deciliter (mg/dL).
10. The participant has a platelet count \<100,000/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).
11. 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).
12. The participant is pregnant or lactating at the time of enrollment.
13. The participant has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia).
14. The participant has participated in another clinical study involving another IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
15. The participant has not received OD or prophylactic treatment with a VWF product prior to this study.
16. The participant has a progressive fatal disease and/or life expectancy of less than 15 months.
17. The participant is unable to complete screening procedures and/or comply with the requirements of the protocol in the opinion of the investigator, based on the joint prescreening evaluation held between the investigator and the sponsor.
18. The participant has a mental condition rendering him/her unable to understand the nature, scope, and possible consequences of the study and/or evidence of an uncooperative attitude.
19. The participant is member of the study team or in a dependent relationship with one of the study team members, which includes close relatives (that is, children, partner/spouse, siblings, and parents) as well as employees.

Where it's running

  • University of Alabama at Birmingham
    Birmingham, Alabama, United States
    Recruiting
  • Bleeding and Clotting Disorders Institute
    Peoria, Illinois, United States
    Recruiting
  • Riley Hospital for Children Indiana University Health
    Indianapolis, Indiana, United States
    Recruiting
  • University of Iowa Hospitals & Clinics PARENT
    Iowa City, Iowa, United States
    Recruiting
  • Childrens Hospital of Michigan
    Detroit, Michigan, United States
    Recruiting
  • Children's Health Care d/b/a Children's Minnesota
    Minneapolis, Minnesota, United States
    Recruiting
Show 17 more locations
  • Cure 4 the Kids
    Las Vegas, Nevada, United States
    Recruiting
  • Rutgers - Robert Wood Johnson Medical School
    New Brunswick, New Jersey, United States
    Recruiting
  • New York - Presbyterian/Weill Cornell Medical Center
    New York, New York, United States
    Recruiting
  • Medical University of South Carolina (MUSC)
    Charleston, South Carolina, United States
    Recruiting
  • Hemostase Clinique - Institut Cœur-Poumons (4eme étage aile est) Bureau 419
    Lille, France
    Recruiting
  • Hopital Edouard Herriot - CHU Lyon
    Lyon, France
    Recruiting
  • Children's Health Ireland
    Dublin, Ireland
    Recruiting
  • Japanese Red Cross Narita Hospital
    Narita, Chiba, Japan
    Recruiting
  • Nara Medical University Hospital
    Kashihara, Nara, Japan
    Recruiting
  • NHO Beppu Medical Center
    Beppu, Oita Prefecture, Japan
    Recruiting
  • Nagasaki University Hospital
    Nagasaki, Japan
    Recruiting
  • Haemophilia Comprehensive Care Centre, Great Ormond Street Hospital for Children NHS Foundation Trust
    London, United Kingdom
    Recruiting
  • Oxford University Hospitals NHS Foundation Trust
    Oxford, United Kingdom
    Recruiting
  • Arnold Palmer Childrens Hosptial
    Orlando, Florida, United States
    Not yet recruiting
  • Michigan State University Center for Bleeding Disorders & Clotting Disorders
    East Lansing, Michigan, United States
    Not yet recruiting
  • Azienda Ospedaliera Pediatrica Santobono Pausillipon
    Naples, Italy
    Completed
  • Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
    Turin (Torino), Italy
    Completed

Study contacts

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.