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

NCT04953884 | Completed Phase 3 Results FDA Drug

• 1 other identifier: WIL-33
• Study Type: interventional
• Target: 12
• Locations: 7 countries
• Sites: 9

Brief 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.

Conditions

Von Willebrand Disease

Outcome Measures

Primary Outcomes (1)

• Total Annualised Bleeding Rate (TABR) During Prophylactic Treatment With Wilate.

  TABR is defined as the total number of bleeding episodes (BEs) including spontaneous, traumatic, and other bleeds, occurring during the period from the first prophylactic dose of wilate to the study completion visit, divided by the duration (in years) between these two time points. Bleeding episodes that occurred during surgery periods were excluded from the calculation of TABR. Total BEs refers to all bleeding episodes that occurred during the study period, regardless of whether they were treated with wilate or not. Treated BEs are a subset of total BEs comprising of bleeding episodes that were treated with wilate.

  During 12 months of prophylactic treatment

Secondary Outcomes (26)

• Area Under the Curve (AUC) of Wilate for VWF:Ac (VWF:RCo) Over Time

  At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate

• Area Under the Curve (AUC) of Wilate for FVIII (OS) Over Time

  At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate

• AUC Normalised for the Administered Dose (AUCnorm) of Wilate for VWF:Ac (VWF:RCo) Over Time

  At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate

• AUC Normalised for the Administered Dose (AUCnorm) of Wilate for FVIII:C (OS) Over Time

  At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate

• Clearance (CL) of Wilate for VWF:Ac (VWF:RCo) Over Time

  At baseline, 15 minutes, 3, 9, 24, 48 and 72 hours after dosing of 80 IU/kg BW wilate

Study Arms (1)

Wilate treatment

EXPERIMENTAL

PK: Single dose of 80 IU/kg body weight (BW). Prophylactic treatment: 30-50 IU/kg BW administered 2-3 times per week at recommended dose over 12 months. Minor haemorrhage: loading dose 30-50 IU/kg BW followed by a maintenance dose of 30-40 IU/kg BW every 12-24 hours to achieve von Willebrand factor activity (VWF:Ac) and Factor VIII coagulant activity (FVIII:C) trough levels of \>30%. Major haemorrhage: loading dose 50-80 IU/kg BW then maintenance dose of 30-50 IU/kg BW every 12-24 hours to achieve VWF:Ac and FVIII:C trough levels of \>50%. Minor surgery: loading dose of 40-60 IU/kg BW then maintenance dose of 20-30 IU/kg BW every 12-24 hours for up to 3 days, to achieve VWF:Ac peak levels of 50% after loading dose and trough levels \>30% during maintenance. Major surgery: loading dose of 60-80 IU/kg BW then a maintenance dose of 30-40 IU/kg BW every 12-24 hours for up to 6 days or longer, to achieve VWF:Ac peak levels of 100% after loading dose and trough levels \>50% during maintenance

Drug: Wilate

Interventions

Wilate DRUG

Wilate is a plasma-derived, stable, highly purified, double virus inactivated concentrate of freeze-dried active VWF and factor VIII (FVIII) prepared from cryoprecipitate and intended for the treatment of patients with VWD and/or haemophilia A

Wilate treatment

Eligibility Criteria

• Age: Up to 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

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
• 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

Sponsors & Collaborators

• Octapharma: lead

Study Sites (9)

Tulane University

New Orleans, Louisiana, 70118, United States

Location

University Hospital Ostrava Department for Pediatric Medicine

Ostrava, Czechia

Location

University Hospital Motol, Department of Paediatric Haematology and Oncology

Prague, Czechia

Location

Gerinnungszentrum Rhein-Ruhr Ambulanz und Fachlaboratorium für Gerinnungserkrankungen/Hämophilie

Duisburg, Germany

Location

IMSP Mother and Child Institute

Chisinau, Moldova

Location

PHI University Clinic for Child Diseases

Skopje, North Macedonia

Location

FSBI National Research Medical Center of Pediatric Hematology, Oncology and Immunology

Moscow, Russia

Location

Morozovskaya Children's Hospital

Moscow, Russia

Location

Danylo Halytsky Lviv National Medical University, Communal Institution of Lviv Regional Council "Western Ukrainian Specialized Children's Medical Centre"

Lviv, Ukraine

Location

MeSH Terms

Conditions

von Willebrand Diseases

Interventions

von Willebrand Factor

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Biological Factors

Results Point of Contact

• Title: Sigurd Knaub
• Organization: Octapharma AG

Sigurd.Knaub@octapharma.com

+41 55 451 21 89

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 18, 2021
• First Posted: July 8, 2021
• Study Start: September 22, 2021
• Primary Completion: December 16, 2024
• Study Completion: December 16, 2024
• Last Updated: February 20, 2026
• Results First Posted: February 20, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

MO (1); UA (1); NO (1); RU (2); DE (1); CZ (2); US (1)