Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease (VWD) Patients… (NCT04953884) | Clinical Trial Compass
CompletedPhase 3
Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease (VWD) Patients <6 Years of Age
United States, Czechia, Germany12 participantsStarted 2021-09-22
Plain-language summary
The WIL-33 study aimed to determine the efficacy, pharmacokinetics, immunogenicity and safety of wilate as routine prophylaxis in up to 12 paediatric patients (eight evaluable) with severe von Willebrand Disease VWD (defined as screening von Willebrand factor ristocetin cofactor activity \[VWF:RCo\] \<20%) under the age of 6 years, over a period of 12 months.
Who can participate
Age range
5 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Patients aged \<6 years at the time of screening
. Type 3 (at least four patients), severe type 2 (except 2N) or severe type 1 VWD (any of which with VWF:RCo \<20%) according to medical history, requiring substitution therapy with a VWF-containing product
. Minimum BW 12.5 kg at the time of screening (for Moldova and Czech Republic, minimum BW 11.0 kg at the time of screening)
. Voluntarily given, fully informed written and signed consent obtained before any study-related procedures are conducted (obtained from the patient's parent(s) / legal guardian(s))
Exclusion criteria
. History or current suspicion of VWF or FVIII inhibitors
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Total Annualised Bleeding Rate (TABR) During Prophylactic Treatment With Wilate.
Timeframe: During 12 months of prophylactic treatment
. Injection of 1-deamino-8-D-arginine vasopressin (DDAVP) or VWF-containing product within 72 hours prior to inclusion
. Medical history of a thromboembolic event
. Platelet count \<100,000/µL at screening (except for VWD type 2B)
. Patients receiving, or scheduled to receive, immunosuppressant drugs (other than antiretroviral chemotherapy), such as prednisone (equivalent to \>10 mg/day), or similar drugs
. Treatment with any investigational medicinal product (IMP) in another interventional clinical study currently or within four weeks before enrolment
. Other coagulation disorders or bleeding disorders
. Known hypersensitivity to any of the components of the study drug