Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease (VWD) Patients <6 Years of Age

Completed · Phase 3

Conditions studied: Von Willebrand Disease

In brief

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.

Key facts

Study ID

NCT04953884

Run by

Octapharma

People needed

12

Starts

2021-09-22

Expected to finish

2024-12-16

Last updated by the study team

2026-02-20

Who can join

Age: any, up to 5. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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))

You may not qualify if…

• History or current suspicion of VWF or FVIII inhibitors
• 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

Where it is running

• Tulane University — New Orleans, Louisiana, United States
• University Hospital Ostrava Department for Pediatric Medicine — Ostrava, Czechia
• University Hospital Motol, Department of Paediatric Haematology and Oncology — Prague, Czechia
• Gerinnungszentrum Rhein-Ruhr Ambulanz und Fachlaboratorium für Gerinnungserkrankungen/Hämophilie — Duisburg, Germany
• IMSP Mother and Child Institute — Chisinau, Moldova
• PHI University Clinic for Child Diseases — Skopje, North Macedonia
• FSBI National Research Medical Center of Pediatric Hematology, Oncology and Immunology — Moscow, Russia
• Morozovskaya Children's Hospital — Moscow, Russia
• Danylo Halytsky Lviv National Medical University, Communal Institution of Lviv Regional Council "Western Ukrainian Specialized Children's Medical Centre" — Lviv, Ukraine

Full record on ClinicalTrials.gov

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