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

This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and above, who have been diagnosed with Type 3 von Willebrand disease (VWD). Participants on prior standard of care (SOC) on-demand therapy will be assessed via a randomized comparison (Arm A - emicizumab prophylaxis and Arm B - continuation of SOC on-demand therapy), while participants on prior SOC prophylactic therapy (Arm C - emicizumab prophylaxis) will be assessed via intra-participant analysis with data obtained from the preceding non-interventional study (NIS), WP45335 (NCT06883240).

NCT Number	NCT06998524
Lead Sponsor	Hoffmann-La Roche
Conditions	Von Willebrand Disease, Type 3
Enrollment	75 participants
Start Date	2025-06-27
Primary Completion	2027-04-30 (estimated)
Study Completion	2029-04-30 (estimated)
Updated on ClinicalTrials.gov	2026-08-10