NCT06998524

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

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

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 75 (estimated)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 1 Month
• Healthy volunteers: Not accepted

Summary

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).

Conditions

• Von Willebrand Disease, Type 3

Interventions

Type	Name	Description
DRUG	Emicizumab	Participants will receive emicizumab 3 milligrams per kilogram (mg/kg) subcutaneous (SC) injections every week (QW) for the first 4 weeks as loading doses, followed by maintenance doses of emicizumab 3 mg/kg SC once every 2 weeks (Q2W). During the extension period, participants may remain on maintenance dose of emicizumab 3 mg/kg Q2W, or change their emicizumab maintenance regimen to 1.5 mg/kg once every week (QW) or 6 mg/kg once every 4 weeks (Q4W), if they prefer and if agreed by the investigators.
DRUG	von Willebrand Factor (VWF) Concentrates	Used according to local labeling or local treatment guidelines.
DRUG	Factor VIII (FVIII) Concentrates	Used according to local labeling or local treatment guidelines.
DRUG	von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates	Used according to local labeling or local treatment guidelines.
DRUG	Bypassing Agents	Used according to local labeling or local treatment guidelines.

Timeline

• Start date: 2025-06-27
• Primary completion: 2027-03-30
• Completion: 2029-03-30
• First posted: 2025-05-31
• Last updated: 2026-03-19

Locations

27 sites across 14 countries: United States, Belgium, Canada, Colombia, France, Germany, Italy, Japan, Netherlands, Poland, South Africa, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study