A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)
NCT ID: NCT05582993
Last Updated: 2025-10-15
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
24 participants
INTERVENTIONAL
2024-11-06
2030-04-11
Brief Summary
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During the study, participants will visit the study clinic 5 times after treatment initiation.
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Cohort 1: Participants With Age >=12 to <18 years
Participants with age greater than or equal to (\>=) 12 to less than (\<) 18 years who have received on-demand (OD) therapy or prophylactic treatment with a pdVWF product will receive vonicog alfa (rVWF) with an initial dose selected within the range of 40 to 60 international units per kilogram (IU/kg) vonicog alfa (rVWF), intravenous infusions, twice-weekly for 12 months. Participants may receive vonicog alfa (rVWF) with or without ADVATE intravenous infusions, when indicated (as deemed necessary for breakthrough bleeding episode treatment and perioperative bleeding management).
Vonicog Alfa
Vonicog Alfa administered by intravenous injection.
ADVATE
ADVATE administered by intravenous injection.
Cohort 2: Participants With Age >=6 to <12 years
Participants with age \>=6 to \<12 years who have received OD therapy of VWF product or prophylactic treatment with a pdVWF product will receive vonicog alfa (rVWF) with an initial dose selected within the range of 40 to 60 IU/kg vonicog alfa (rVWF), intravenous infusions, twice-weekly for 12 months. Participants may receive vonicog alfa (rVWF) with or without ADVATE intravenous infusions, when indicated (as deemed necessary for breakthrough bleeding episode treatment and perioperative bleeding management).
Vonicog Alfa
Vonicog Alfa administered by intravenous injection.
ADVATE
ADVATE administered by intravenous injection.
Cohort 3: Participants With Age <6 years
Participants with age \<6 years who have received OD therapy of VWF product or prophylactic treatment with a pdVWF product will receive vonicog alfa (rVWF) with an initial dose selected within the range of 40 to 60 IU/kg vonicog alfa (rVWF), intravenous infusions, twice-weekly for 12 months. Participants may receive vonicog alfa (rVWF) with or without ADVATE intravenous infusions, when indicated (as deemed necessary for breakthrough bleeding episode treatment and perioperative bleeding management).
Vonicog Alfa
Vonicog Alfa administered by intravenous injection.
ADVATE
ADVATE administered by intravenous injection.
Interventions
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Vonicog Alfa
Vonicog Alfa administered by intravenous injection.
ADVATE
ADVATE administered by intravenous injection.
Other Intervention Names
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Eligibility Criteria
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Exclusion Criteria
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.
17 Years
ALL
No
Sponsors
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Takeda
INDUSTRY
Responsible Party
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Principal Investigators
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Study Director
Role: STUDY_DIRECTOR
Takeda
Locations
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University of Alabama at Birmingham
Birmingham, Alabama, United States
Bleeding and Clotting Disorders Institute
Peoria, Illinois, United States
Riley Hospital for Children Indiana University Health
Indianapolis, Indiana, United States
University of Iowa Hospitals & Clinics PARENT
Iowa City, Iowa, United States
Childrens Hospital of Michigan
Detroit, Michigan, United States
Children's Health Care d/b/a Children's Minnesota
Minneapolis, Minnesota, United States
Rutgers - Robert Wood Johnson Medical School
New Brunswick, New Jersey, United States
New York - Presbyterian/Weill Cornell Medical Center
New York, New York, United States
Medical University of South Carolina (MUSC)
Charleston, South Carolina, United States
Hemostase Clinique - Institut Cœur-Poumons (4eme étage aile est) Bureau 419
Lille, , France
Hopital Edouard Herriot - CHU Lyon
Lyon, , France
Children's Health Ireland
Dublin, , Ireland
Azienda Ospedaliera Pediatrica Santobono Pausillipon
Napoli, , Italy
Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
Turin (Torino), , Italy
Nara Medical University Hospital
Kashihara, , Japan
Countries
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Central Contacts
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Facility Contacts
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Related Links
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To obtain more information on the study, click here/on this link
Other Identifiers
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2023-509877-22-00
Identifier Type: CTIS
Identifier Source: secondary_id
TAK-577-3001
Identifier Type: -
Identifier Source: org_study_id
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