Phase III Study of Emicizumab Injections to Treat Patients With Type 3 Von Willebrand Disease

What this Trial Is About

Researchers are conducting a Phase III, multicenter, open-label clinical study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and older diagnosed with Type 3 von Willebrand disease VWD. The study compares emicizumab prophylaxis with standard of care SOC on-demand therapy in participants previously on on-demand treatment, and assesses emicizumab prophylaxis in those already on SOC prophylactic therapy by comparing with prior data from a non-interventional study. Participants are divided into three groups Arm A receives emicizumab subcutaneous injections weekly for 4 weeks followed by doses every 2 weeks as prophylaxis Arm B continues their current SOC on-demand therapy for 24 weeks and Arm C, previously on SOC prophylaxis, receives emicizumab prophylaxis. After completing 24 weeks of treatment, participants benefiting from emicizumab in Arms A and C may continue the drug in an extension phase. Participants in Arm B may switch to emicizumab prophylaxis after 24 weeks. During the study, participants will have their bleeding rates measured and compared between treatments, along with monitoring for adverse events, thromboembolic events, injection-site reactions, and laboratory abnormalities. Various health parameters including vital signs, electrocardiograms, and patient-reported outcomes like pain and fatigue will be assessed regularly. The study duration may extend up to nearly four years from first dose through follow-up and extension periods.

A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease