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

Hoffmann-La Roche

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).

Eligibility

Age range: 0+ years
Sex: All
Healthy volunteers: No

Detailed criteria

Inclusion Criteria: * Confirmed diagnosis of Type 3 von Willebrand disease (VWD), based on medical records * Preexisting medical record verifying the status of von Willebrand factor (VWF) inhibitor (positive or negative, including titer if available) * Adequate hematologic, hepatic, and renal function * For participants of childbearing potential: agreement to remain abstinent or adhere to the contraception requirements Additional Inclusion Criteria for Arms A and B: * Age ≥1 month at the time of signing Informed Consent/Assent Form * Documented previous use of on-demand therapy with intermi…

Interventions

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.

Locations (29)

UC Davis
Sacramento, California
University of Florida
Gainesville, Florida
University of Kentucky Children's Hospital
Lexington, Kentucky
University of Minnesota Medical Center
Minneapolis, Minnesota
Washington University School of Medicine
St Louis, Missouri
Virginia Commonwealth University
Richmond, Virginia