A Clinical Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in the Treatment of Bleeding Episodes in Chinese Subjects Diagnosed With Von Willebrand Disease
The main aim of this study is to find out if VONVENDI is safe for adult Chinese participants with VWD. The study will also check how well VONVENDI helps control bleeding with or without product ADVATE in the participants who may need elective surgery or dental procedures. In addition, the study will also examine how VONVENDI is processed by the body (known as pharmacokinetic \[PK\]) and how the drug helps the body respond or improve a condition (pharmacodynamic \[PD\]). Participants will receive an initial dose of VONVENDI of 40 to 80 international units per kilogram (IU/kg) of body weight. If a participant's baseline factor VIII (FVIII) level is not high enough to help stop bleeding, VONVENDI will be given along with 30 to 45 IU/kg of ADVATE rFVIII. Participants will be in the study for approximately 14 months. During the study, participants will be followed up at clinics or over telephone calls.
• Participant must voluntarily sign an institutional review board (IRB)/independent ethics committee-approved written informed consent form after all relevant aspects of the study have been explained and discussed with the participant.
• Participant has a documented diagnosis of severe VWD (baseline VWF:RCo less than \[\<\]20 international units \[IU\]deciliter \[dL\]) with a diagnosis of VWD type verified per the following recommended criteria:
‣ Type 1 (von Willebrand factor:Ristocetin cofactor activity \[VWF:RCo\] \<20 IU/dL and by VWF activity/VWF:antigen \[Ag\] ratio) or,
⁃ Type 2A or type 2B (by VWF activity/VWF:Ag ratio and multimer pattern, with genetics if necessary), type 2N (FVIII:C \<10% and genetics), type 2M (by VWF activity/VWF:Ag ratio and multimer pattern) or
⁃ Type 3 (VWF:Ag \<=3 IU/dL). Diagnosis is confirmed, when applicable, by genetic testing and/or by multimer analysis.
• Participant is at least 18 years of age at screening.
• Participant is ethnic Chinese and lives in China, including those from Taiwan, Hong Kong, and Macao.
• If female of childbearing potential, participant presents with a negative pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
• Participant is willing and able to comply with the requirements of the protocol.
• Participant has had a minimum of 3 documented bleeds that indicated the need for VWF coagulation factor replacement therapies during the previous 12 months prior to enrollment.