Actively Recruiting
A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)
Led by Takeda · Updated on 2025-10-15
24
Participants Needed
15
Research Sites
283 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Documented diagnosis of severe Von Willebrand Disease (VWD) with baseline VWF:RCo activity less than 20 IU/dL and history of replacement therapy to control bleeding
- Participant is younger than 18 years at screening
- Prior treatment with VWF products: at least 12 months for those 2 years or older, or at least 6 months for those under 2 years
- For prior on-demand therapy, experienced at least one VWF-treated bleeding event in the last 12 months (or 6 months if under 2 years)
- Reliable records of bleeding events for at least 12 months (or 6 months if under 2 years) before enrollment
- Appropriate body mass index or weight-for-age percentile based on age and CDC charts
- Female participants of childbearing potential must have a negative pregnancy test and agree to use effective birth control
- Provided assent and informed consent
- Willing and able to comply with study requirements as confirmed by prescreening evaluation
You will not qualify if you...
- Diagnosis of pseudo VWD or other coagulation disorders besides VWD
- History or presence of VWF or factor VIII inhibitors
- Known hypersensitivity to study drug components
- Medical history of immunological disorders (except mild allergies), thromboembolic events, significant liver or kidney disease
- HIV-positive with low CD4 count
- Platelet count under 100,000/mL at screening (with exceptions for type 2B VWD under consultation)
- Recent treatment with immunomodulatory drugs (within 30 days)
- Pregnant or lactating at enrollment
- Cervical or uterine conditions causing abnormal bleeding
- Participation in another investigational study within 30 days prior or planned during this study
- No prior on-demand or prophylactic VWF treatment
- Progressive fatal disease or life expectancy under 15 months
- Unable to complete screening or comply with protocol
- Mental conditions impacting understanding or cooperation
- Related to or employed by study team members
AI-Screening
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Trial Site Locations
Total: 15 locations
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35223
Actively Recruiting
2
Bleeding and Clotting Disorders Institute
Peoria, Illinois, United States, 61614
Actively Recruiting
3
Riley Hospital for Children Indiana University Health
Indianapolis, Indiana, United States, 46202
Actively Recruiting
4
University of Iowa Hospitals & Clinics PARENT
Iowa City, Iowa, United States, 52242
Actively Recruiting
5
Childrens Hospital of Michigan
Detroit, Michigan, United States, 48201
Actively Recruiting
6
Children's Health Care d/b/a Children's Minnesota
Minneapolis, Minnesota, United States, 55404
Actively Recruiting
7
Rutgers - Robert Wood Johnson Medical School
New Brunswick, New Jersey, United States, 08901
Actively Recruiting
8
New York - Presbyterian/Weill Cornell Medical Center
New York, New York, United States, 10021
Actively Recruiting
9
Medical University of South Carolina (MUSC)
Charleston, South Carolina, United States, 29425
Actively Recruiting
10
Hemostase Clinique - Institut Cœur-Poumons (4eme étage aile est) Bureau 419
Lille, France, 59037
Actively Recruiting
11
Hopital Edouard Herriot - CHU Lyon
Lyon, France, 69677
Actively Recruiting
12
Children's Health Ireland
Dublin, Ireland, D12N512
Actively Recruiting
13
Azienda Ospedaliera Pediatrica Santobono Pausillipon
Naples, Italy, 80123
Actively Recruiting
14
Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
Turin (Torino), Italy, 10126
Actively Recruiting
15
Nara Medical University Hospital
Kashihara, Japan, 634-8522
Actively Recruiting
Research Team
T
Takeda Contact
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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