A Phase III, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Prophylaxis in Patients With Type 3 Von Willebrand Disease

What this Trial Is About

Researchers are evaluating emicizumab prophylaxis in people aged 1 month and older diagnosed with Type 3 von Willebrand disease (VWD). This Phase III, multicenter, open-label clinical study aims to assess the efficacy, safety, and how the body processes and responds to emicizumab. Participants previously receiving standard of care (SOC) on-demand therapy will be compared in a randomized manner, while those already on SOC prophylactic therapy will be evaluated based on prior study data. Participants are divided into three groups: those switching from on-demand SOC to emicizumab prophylaxis (Arm A), those continuing on-demand SOC therapy (Arm B), and those switching from SOC prophylaxis to emicizumab prophylaxis (Arm C). Emicizumab is given as weekly subcutaneous injections of 3 mg/kg for 4 weeks, followed by maintenance doses every two weeks. After 24 weeks, participants benefiting from emicizumab may continue it during an extension period, while those on SOC on-demand can switch to emicizumab in the extension. During the study, participants will undergo regular assessments measuring bleeding rates, safety events, and laboratory tests including blood levels of emicizumab and immune responses. Vital signs and questionnaires on pain and fatigue will be collected at scheduled intervals. Safety monitoring will continue up to nearly four years after the last dose, with detailed tracking of adverse events and heart and lung function. The study participation may last up to several years, including the treatment and extension phases.

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