A Study of Recombinant Von Willebrand Factor (rVWF) in Chinese Participants With Von Willebrand Disease (vWD)

Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorTakeda

About this trial

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.

Eligibility criteria

Qualifiers

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.

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.

Disqualifiers

Participant has been diagnosed with pseudo VWD or another hereditary or acquired coagulation disorder other than VWD (example [eg], qualitative and quantitative platelet disorders or elevated prothrombin time [PT]/international normalized ratio >1.4).

Participant has a history or presence of a VWF inhibitor at screening.

Participant has a documented history of a VWF:RCo half-life of <6 hours.

Participant has a history or presence of a FVIII inhibitor with a titer greater than or equal to [>=] 0.6 Bethesda units per milliliter [BU/mL] (by Bethesda assay or Bethesda method with Nijmegen modification).

Trial design

Treatments tested in this trial

  • VONVENDI
  • ADVATE

Treatment groups

20 Participants
are divided into 1 treatment group

Sponsors and collaborators