A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)
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
2 other identifiers
interventional
24
5 countries
15
Brief Summary
The main aim of the study is to evaluate the effectiveness of prophylaxis with vonicog alfa (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. In this study, participants will be treated with vonicog alfa (rVWF) for 12 months. During the study, participants will visit the study clinic 5 times after treatment initiation.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Nov 2024
Longer than P75 for phase_3
15 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 14, 2022
CompletedFirst Posted
Study publicly available on registry
October 17, 2022
CompletedStudy Start
First participant enrolled
November 6, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 11, 2030
ExpectedStudy Completion
Last participant's last visit for all outcomes
April 11, 2030
October 15, 2025
October 1, 2025
5.4 years
October 14, 2022
October 14, 2025
Conditions
Outcome Measures
Primary Outcomes (1)
Annualized Bleeding Rate (ABR) for Spontaneous or Traumatic Bleeding Episodes as Assessed by Investigator During Prophylactic Treatment With Vonicog Alfa (rVWF)
ABR during the study compared to historical ABR for each participant for both spontaneous and traumatic bleeding episodes as classified by the investigator during prophylactic treatment with vonicog alfa (rVWF) will be reported.
12 months
Secondary Outcomes (38)
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
12 months
Number of Participants With TEAEs by Severity
12 months
Number of Participants With TEAEs and SAEs by Causality
12 months
Number of Participants With Thromboembolic Events, Hypersensitivity Reactions and Infusion-Related Reactions (IRR)
12 months
Number of Participants Who Develop Neutralizing Antibodies to VWF and Factor VIII (FVIII)
12 months
- +33 more secondary outcomes
Study Arms (3)
Cohort 1: Participants With Age >=12 to <18 years
EXPERIMENTALParticipants 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).
Cohort 2: Participants With Age >=6 to <12 years
EXPERIMENTALParticipants 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).
Cohort 3: Participants With Age <6 years
EXPERIMENTALParticipants 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).
Interventions
Vonicog Alfa administered by intravenous injection.
ADVATE administered by intravenous injection.
Eligibility Criteria
You may not qualify if:
- 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 \>=0.6 Bethesda units per milliliter (/mL).
- 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/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.
- The participant has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia).
- 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.
- The participant has not received OD or prophylactic treatment with a VWF product prior to this study.
- +4 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Takedalead
Study Sites (15)
University of Alabama at Birmingham
Birmingham, Alabama, 35223, United States
Bleeding and Clotting Disorders Institute
Peoria, Illinois, 61614, United States
Riley Hospital for Children Indiana University Health
Indianapolis, Indiana, 46202, United States
University of Iowa Hospitals & Clinics PARENT
Iowa City, Iowa, 52242, United States
Childrens Hospital of Michigan
Detroit, Michigan, 48201, United States
Children's Health Care d/b/a Children's Minnesota
Minneapolis, Minnesota, 55404, United States
Rutgers - Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901, United States
New York - Presbyterian/Weill Cornell Medical Center
New York, New York, 10021, United States
Medical University of South Carolina (MUSC)
Charleston, South Carolina, 29425, United States
Hemostase Clinique - Institut Cœur-Poumons (4eme étage aile est) Bureau 419
Lille, 59037, France
Hopital Edouard Herriot - CHU Lyon
Lyon, 69677, France
Children's Health Ireland
Dublin, D12N512, Ireland
Azienda Ospedaliera Pediatrica Santobono Pausillipon
Napoli, 80123, Italy
Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
Turin (Torino), 10126, Italy
Nara Medical University Hospital
Kashihara, 634-8522, Japan
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 14, 2022
First Posted
October 17, 2022
Study Start
November 6, 2024
Primary Completion (Estimated)
April 11, 2030
Study Completion (Estimated)
April 11, 2030
Last Updated
October 15, 2025
Record last verified: 2025-10
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, ICF, CSR
- Access Criteria
- IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/ For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.