NCT04953884

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

Clinical Study to Investigate the Efficacy, Pharmacokinetics, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease Patients Under the Age of 6 Years

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Octapharma · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
DRUG	Wilate	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

Timeline

• Start date: 2021-09-22
• Primary completion: 2024-12-16
• Completion: 2024-12-16
• First posted: 2021-07-08
• Last updated: 2026-02-20
• Results posted: 2026-02-20

Locations

9 sites across 7 countries: United States, Czechia, Germany, Moldova, North Macedonia, Russia, Ukraine

Regulatory

• FDA-regulated drug study