A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF With or Without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed With Severe Von Willebrand Disease
The main aim of the study is to check effectiveness, side effects, and tolerability of vonicog alfa (recombinant von Willebrand factor \[rVWF\]), with or without ADVATE, in the treatment and control of nonsurgical bleeding events in pediatric participants (less than (\<)18 years of age) with severe hereditary von Willebrand disease (VWD). The participants will be treated with vonicog alfa for 12-18 months. Their von Willebrand Disease will be treated by their doctor according to their doctor's usual clinical practice. During the study, participants will be followed up at clinics or over telephone calls.
• Diagnosis of severe von Willebrand disease (VWD) (defined as von Willebrand factor: ristocetin cofactor \[VWF:RCo\] less than \[\<\] 20 percent \[%\]):
‣ Type 1 (VWF:RCo \<20 International Units per deciliter \[IU/dL\]); or
⁃ Type 2A (VWF:RCo \<20 IU/dL), Type 2B (as diagnosed by genotype), Type 2N (Factor VIII coagulation activity \[FVIII:C\] \<10 % and historically documented genetics), Type 2M; or
⁃ Type 3 (VWF:Ag less than or equal to \[=\<\] 3 IU/dL).
• Age 0 to \<18 years at the time of Screening.
• The participant has provided assent (if appropriate) and legally authorized representative(s) has provided informed consent.
• If female of childbearing potential, participant presents with a negative serum pregnancy test.
• If applicable, participant agrees to employ adequate birth control measures for the duration of the study.
• The participant and/or the legally authorized representative are willing and able to comply with the requirements of the protocol, which should also be confirmed based on a pre-screening evaluation held between the Investigator and the Sponsor, to ensure no eminent risk is present that could challenge the participants compliance with the study requirements.
∙ Additional inclusion criteria for both previously treated participants and participants undergoing surgery are as follows:
• Unable to tolerate are inadequately responsive to, or not a good candidate for 1-deamino-8-D-arginine vasopressin (DDAVP). Examples of participants who are not good candidates for DDAVP include participants with type 2B or type 3 VWD.
• The participant has had a minimum of 1 documented bleed requiring VWF coagulation factor replacement therapy (i.e. treatment with a VWF product) during the previous 12 months prior to enrollment and overall historically 3 or more exposure days (EDs) to VWF replacement therapy.
∙ Additional inclusion criterion for previously untreated participants are as follows:
∙ \- The participant has not received prior VWF coagulation factor replacement therapy.