At a glance
ClinicalIndex Comparison Record- ✓Severe von Willebrand disease confirmed by genetic testing and multimer analysis with baseline VWF:RCo <20 IU/dL and history of requiring VWF substitute therapy
- ✓On-demand group: ≥3 documented spontaneous bleeds (excluding menorrhagia) requiring VWF treatment in past 12 months
- ✓pdVWF switch group: receiving prophylactic pdVWF products for ≥12 months prior to screening
- ✓Age ≥18 years with BMI ≥15 but <40 kg/m²
- ✕Type 2N VWD, pseudo-VWD, or other hereditary/acquired coagulation disorders (e.g., platelet disorders, PT/INR >1.4)
- ✕History or presence of VWF inhibitor at screening
- ✕FVIII inhibitor with titer ≥0.4 BU (Nijmegen) or ≥0.6 BU (Bethesda assay)
- ✕Known hypersensitivity to study drug components including mouse or hamster proteins
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A PROSPECTIVE, PHASE 3, OPEN-LABEL, INTERNATIONAL MULTICENTER STUDY ON EFFICACY AND SAFETY OF PROPHYLAXIS WITH rVWF IN SEVERE VON WILLEBRAND DISEASE
In Brief
A Phase 3 clinical trial evaluating von Willebrand factor (Recombinant) and Antihemophilic Factor (Recombinant) for Von Willebrand Disease. Completed, enrolled 29 participants across 35 sites in 9 countries.
Detailed Summary
The purpose of this phase 3 study is to investigate the efficacy and safety, including immunogenicity, thrombogenicity and hypersensitivity reactions, as well as pharmacokinetics (PK), health related quality of life (HRQoL) and pharmacoeconomics of prophylactic treatment with recombinant von Willebrand factor (rVWF) (vonicog alfa) in adult participants with severe von Willebrand disease (VWD).
Study Details
Timeline
Interventions
OD participants will receive intravenous (IV) rVWF:RCo at an initial prophylactic dose of 50 +/- 10 International Unit per Kilogram (IU/kg) twice (two infusions) a week for at least 12 months up to 15 months and may be increased up to 80 IU/kg. pdVWF switch cohort participants will receive rVWF:RCo equivalent (± 10%) to the weekly VWF dose received during prophylactic treatment with pdVWF.
During prophylaxis period any bleeding episodes requiring substitution therapy with VWF concentrate to control bleeding will be treated with rVWF with or without ADVATE. Participants will receive rFVIII IV if necessary for OD treatment of breakthrough bleeds or for peri-operative. The dose will be according to the bleeding type and severity and it will be adjusted to the clinical response.