NCT03879135

A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)

A Phase 3b, Prospective, Open-Label, Uncontrolled, Multicenter Study on Long-Term Safety and Efficacy of rVWF in Pediatric and Adult Subjects With Severe Von Willebrand Disease (VWD)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 38 (actual)
• Sponsor: Baxalta now part of Shire · Industry
• Sex: All
• Age: 0 Years
• Healthy volunteers: Not accepted

Summary

The main aim of the study is to check effectiveness of rVWF (vonicog alfa) prophylaxis based on the annualized bleeding rate (ABR) of spontaneous (not related to trauma) bleeding episodes in pediatric and adult participants during the first 12 months on study treatment. The participants will be treated with rVWF for a maximum of 3 years. Their von Willebrand Disease will be treated according to Investigational product (IP) dosing directions.

Conditions

• Von Willebrand Disease (VWD)

Interventions

Type	Name	Description
BIOLOGICAL	rVWF	Recombinant von Willebrand factor
BIOLOGICAL	rFVIII	Recombinant Factor VIII

Timeline

• Start date: 2019-04-01
• Primary completion: 2025-01-30
• Completion: 2025-01-30
• First posted: 2019-03-18
• Last updated: 2025-09-03
• Results posted: 2025-09-03

Locations

33 sites across 9 countries: United States, Austria, France, Germany, Italy, Netherlands, Russia, Spain, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study