A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)

Recruiting now · Phase 3

Conditions studied: Von Willebrand Disease (VWD)

In brief

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.

Key facts

Study ID

NCT05582993

Run by

Takeda

People needed

24

Starts

2024-11-06

Expected to finish

2030-04-11

Last updated by the study team

2026-05-27

Who can join

Age: any, up to 17. Sex: any. Healthy volunteers: not accepted.

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.
• The participant has a progressive fatal disease and/or life expectancy of less than 15 months.
• 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.
• 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.
• 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 is running

• Haemophilia Comprehensive Care Centre, Great Ormond Street Hospital for Children NHS Foundation Trust — London, United Kingdom (enrolling)
• Bleeding and Clotting Disorders Institute — Peoria, Illinois, United States (enrolling)
• Riley Hospital for Children Indiana University Health — Indianapolis, Indiana, United States (enrolling)
• University of Iowa Hospitals & Clinics PARENT — Iowa City, Iowa, United States (enrolling)
• Childrens Hospital of Michigan — Detroit, Michigan, United States (enrolling)
• Michigan State University Center for Bleeding Disorders & Clotting Disorders — East Lansing, Michigan, United States (enrolling)
• Children's Health Care d/b/a Children's Minnesota — Minneapolis, Minnesota, United States (enrolling)
• Cure 4 the Kids — Las Vegas, Nevada, United States (enrolling)
• Rutgers - Robert Wood Johnson Medical School — New Brunswick, New Jersey, United States (enrolling)
• New York - Presbyterian/Weill Cornell Medical Center — New York, New York, United States (enrolling)
• Medical University of South Carolina (MUSC) — Charleston, South Carolina, United States (enrolling)
• Hemostase Clinique - Institut Cœur-Poumons (4eme étage aile est) Bureau 419 — Lille, France (enrolling)
• Hopital Edouard Herriot - CHU Lyon — Lyon, France (enrolling)
• Children's Health Ireland — Dublin, Ireland (enrolling)
• Nagasaki University Hospital — Nagasaki, Japan (enrolling)
• University of Alabama at Birmingham — Birmingham, Alabama, United States (enrolling)
• Japanese Red Cross Narita Hospital — Narita, Chiba, Japan (enrolling)
• Nara Medical University Hospital — Kashihara, Nara, Japan (enrolling)
• NHO Beppu Medical Center — Beppu, Oita Prefecture, Japan (enrolling)
• Azienda Ospedaliera Pediatrica Santobono Pausillipon — Naples, Italy
• Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino — Turin (Torino), Italy

Full record on ClinicalTrials.gov

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