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Sponsor: Hoffmann-La Roche
Conditions: Von Willebrand Disease, Type 3
Interventions: Emicizumab, von Willebrand Factor (VWF) Concentrates, Factor VIII (FVIII) Concentrates, von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates, Bypassing Agents
Countries: United States, Belgium, Canada, Colombia, France, Germany, Italy, Japan
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).
Sex: ALL
Age: 1 Month to —
Healthy volunteers: No
Study type: INTERVENTIONAL
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 intermittent (less than once a week) on-demand SOC therapy for VWD * Having ≥2 treated bleeds (except menstrual bleeds) with factor concentrate within 24 weeks prior to enrollment Additional Inclusion Criteria for Arm C: * Age ≥2 years at the time of signing Informed Consent/Assent Form * Documented and confirmed previous use of SOC prophylactic therapy for VWD (1-3 times weekly, as per prescribed dose) as described in the eligibility of Study WP45335 * Have completed all study requirements as defined in the WP45335 protocol for at least 24 weeks Exclusion Criteria: * Inherited or acquired bleeding disorder other than Congenital Type 3 VWD * History of gastrointestinal bleeding within 18 months prior to enrollment, or any previous diagnosis of angiodysplasia * History of intracranial hemorrhage * Previous or current treatment for thromboembolic disease or signs of thromboembolic disease * Other conditions (e.g., certain autoimmune diseases) that may increase risk of bleeding or thrombosis * History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection * Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use during the study, with the exception of anti-retroviral therapy
- Sacramento, California, United States
- Gainesville, Florida, United States
- Minneapolis, Minnesota, United States
- St Louis, Missouri, United States
- Richmond, Virginia, United States
- Leuven, Belgium
- Toronto, Ontario, Canada
- Montreal, Quebec, Canada
- Medellín, Colombia
- Lille, France
- Paris, France
- Bonn, Germany
- Duisburg, Germany
- Frankfurt/M., Germany
- Rome, Lazio, Italy
- Milan, Lombardy, Italy
- Florence, Tuscany, Italy
- Fukuoka, Japan
- Nagoya, Japan
- Rotterdam, Netherlands
- Warsaw, Poland
- Johannesburg, South Africa
- Madrid, Spain
- Seville, Spain
- Gothenburg, Sweden
- London, United Kingdom
- London, United Kingdom
- Manchester, United Kingdom