Phase 3 Study Evaluating Safety and Effectiveness of Vonicog Alfa rVWF Treatment in Children With Severe Von Willebrand Disease

What this Trial Is About

Researchers are studying the effectiveness of prophylactic treatment with vonicog alfa, a recombinant von Willebrand factor rVWF, in children diagnosed with severe Von Willebrand Disease VWD. This Phase 3, open-label study aims to evaluate how well vonicog alfa works in preventing bleeding episodes in participants who have previously been treated with VWF or plasma-derived VWF products. The focus is on children under 18 years of age with severe VWD who require ongoing replacement therapy to control bleeding. Participants will receive intravenous infusions of vonicog alfa twice weekly for 12 months. The initial dose will range from 40 to 60 international units per kilogram, adjusted by age groups under 6 years, 6 to under 12 years, and 12 to under 18 years. Some participants may also receive ADVATE, another intravenous treatment, as needed to manage breakthrough bleeding episodes or bleeding related to surgery. Treatment is personalized and monitored throughout the study. During the 12-month treatment period, participants will visit the study clinic five times to assess their response and safety. Researchers will monitor the annualized bleeding rate ABR for spontaneous or traumatic bleeding events and record any adverse events. Blood samples will be taken to measure vonicog alfa levels and antibody development. Other assessments include vital signs, laboratory tests, and evaluation of breakthrough bleeding treatment efficacy. The study also collects data on infusion frequency and the amount of vonicog alfa used.

A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)