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

January 7, 2026 updated by: Takeda

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
20

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • China
      • Beijing, China, 100006
        • Recruiting
        • Peking Union Medical College Hospital
        • Contact:
        • Principal Investigator:
          • Tienan Zhu
      • Guangzhou, China, 510515
        • Recruiting
        • Nanfang Hospital Southern Medical University
        • Principal Investigator:
          • Jing Sun
        • Contact:
      • Jihan, China, 250013
        • Recruiting
        • Jinan Central Hospital
        • Contact:
        • Principal Investigator:
          • Yun Chen
      • Shanghai, China, 201801
        • Recruiting
        • Ruijin Hospital Shanghai Jiaotong University School of Medicine
        • Principal Investigator:
          • Xuefeng Wang
        • Contact:
      • Suzhou, China, 215005
        • Recruiting
        • The First Affiliated Hospital of Soochow University
        • Principal Investigator:
          • Ziqiang Yu
        • Contact:
      • Tianjin, China, 300052
        • Recruiting
        • Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences
        • Principal Investigator:
          • Feng Xue
        • Contact:
      • Wuhan, China, 430032
        • Recruiting
        • Tongji Hospital Tongji Medical College Huazhong University of Science and Technology
        • Contact:
        • Principal Investigator:
          • Dengju Li

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria

  1. 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.

  2. 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.
  3. Participant is at least 18 years of age at screening.
  4. Participant is ethnic Chinese and lives in China, including those from Taiwan, Hong Kong, and Macao.
  5. 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.
  6. Participant is willing and able to comply with the requirements of the protocol.
  7. 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.

Exclusion Criteria

  1. 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).
  2. Participant has a history or presence of a VWF inhibitor at screening.
  3. Participant has a documented history of a VWF:RCo half-life of <6 hours.
  4. 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).
  5. Participant has a known hypersensitivity to any of the components of the study drugs, such as to mouse or hamster proteins.
  6. Participant has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies or animal allergies.
  7. Participant has a medical history of a thromboembolic event.
  8. Participant is human immunodeficiency virus (HIV) positive with an absolute cluster of differentiation 4 (CD4) count <200/cubic millimeter (mm^3).
  9. Participant has been treated with an immunomodulatory drug, other than antiretroviral chemotherapy eg, α-interferon, or corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 milligram (mg)/day (excluding topical treatment [eg, ointments, nasal sprays]), within 30 days prior to signing the informed consent (or assent, if appropriate).
  10. Participant is pregnant or lactating at the time informed consent is obtained.
  11. Participant has participated in another clinical study involving an IP, or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
  12. Participant is diagnosed with progressive fatal disease and/or has a life expectancy of less than 15 months.
  13. Participant is member of the study team conducting this study or in a dependent relationship with one of the study team members. Dependent relationships include close relatives (that is, children, partner/spouse, siblings, parents) as well as employees.
  14. Participant has an acute illness (eg, influenza, flu-like syndrome, allergic rhinitis/conjunctivitis, nonseasonal asthma) at screening.
  15. Participant is diagnosed with significant liver disease, as evidenced by, but not limited to, any of the following: serum alanine aminotransferase (ALT) greater than 5 times the upper limit of normal; hypoalbuminemia; portal vein hypertension (eg, presence of otherwise unexplained splenomegaly, history of esophageal varices) or liver cirrhosis classified as Child-Pugh class B or C.
  16. Participant has been diagnosed with renal disease, with a serum creatinine level >=2.5 milligram per deciliter (mg/dL).
  17. Participant has a platelet count <100,000/ milliliter (mL) at screening (except for participants with type 2B VWD, whose platelet count[s] at screening will be evaluated taking into consideration historical trends in platelet counts and the investigator's medical assessment of the participant's condition).
  18. Participant has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia).
  19. In the judgment of the investigator, the participant has another clinically significant concomitant condition (eg, uncontrolled hypertension) that may pose additional risks for the participant.
  20. Participant is identified by the investigator as being unable or unwilling to cooperate with study procedures.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: All Participants With VWD
Participants will receive a single intravenous (IV) dose of VONVENDI at baseline PK assessment. During the 12-month on-demand (OD) treatment period, any bleeding episodes requiring replacement therapy with VWF will be treated with VONVENDI with or without ADVATE. Participants may also receive VONVENDI with or without ADVATE intravenous infusions, when indicated deemed necessary for perioperative bleeding management [major, minor and oral surgery]. Participants will receive initial dose of VONVENDI of 40 to 80 IU/kg of body weight.
Biological: VONVENDI
VONVENDI is administered by intravenous injection.
Other Names:
  • rVWF
  • TAK-577
  • vonicog alfa
Biological: ADVATE
ADVATE is administered by intravenous injection.
Other Names:
  • rFVIII
  • Octocog alfa

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame: Up to 14 months
A TEAE is defined as an adverse event with an onset that occurs after receiving study drug. An SAE is any untoward medical occurrence that at any dose: results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent, significant disability/incapacity, a congenital abnormality/birth defect, an important medical event in the opinion of the healthcare provider, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition above. TEAEs consist of both serious and non-serious adverse events.
Up to 14 months
Number of Participants With TEAEs by Severity
Time Frame: Up to 14 months
Number of participants with severity of TEAE will be reported.
Up to 14 months
Number of Participants With TEAEs and SAEs by Causality
Time Frame: Up to 14 months
Number of participants with causality related TEAEs and SAEs will be reported.
Up to 14 months
Number of Participants With Thromboembolic Events and Severe Hypersensitivity Reactions
Time Frame: Up to 14 months
Number of participants with thromboembolic events and severe hypersensitivity reactions will be reported.
Up to 14 months
Number of Participants Who Develop Neutralizing (Inhibitory) Antibodies to VWF and FVIII
Time Frame: Up to 14 months
Number of participants who develop neutralizing antibodies to VWF and FVIII will be reported.
Up to 14 months
Number of Participants Who Develop Binding Antibodies to VWF and FVIII
Time Frame: Up to 14 months
Number of participants who develop total binding antibodies to VWF and FVIII will be reported.
Up to 14 months
Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters
Time Frame: Up to 14 months
Number of participants with clinically significant abnormalities from baseline values in laboratory parameters per investigator assessment will be reported.
Up to 14 months
Number of Participants With Clinically Significant Abnormalities in 12-lead Electrocardiogram (ECG)
Time Frame: Up to 14 months
Number of participants with clinically significant abnormalities from baseline values in ECG per investigator assessment will be reported.
Up to 14 months
Number of Participants With Clinically Significant Abnormalities in Vital Signs Parameters
Time Frame: Up to 14 months
Number of participants with clinically significant abnormalities from baseline values in vital sign parameters per investigator assessment will be reported.
Up to 14 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Number of Infusions of VONVENDI With or Without ADVATE per Bleeding Episode
Time Frame: Up to 12 months
Number of infusions of VONVENDI with or without ADVATE per bleeding episode will be reported.
Up to 12 months
Number of Infusions of ADVATE per Bleeding Episode
Time Frame: Up to 12 months
Number of infusions of ADVATE per bleeding episode will be reported.
Up to 12 months
Weight-adjusted Consumption of VONVENDI and ADVATE per Bleeding Episode
Time Frame: Up to 12 months
Weight-adjusted consumption of VONVENDI and ADVATE per bleeding episode will be reported.
Up to 12 months
Time to Resolution of Bleeding Episodes
Time Frame: Up to 12 months
Time to resolution of the bleeding episodes will be calculated as the difference between the date/time of the first IP infusion for the bleeding episode to the date/time of the bleeding episode resolution.
Up to 12 months
Area Under the Plasma Concentration/Time Curve From Time 0 to Infinity (AUC0-inf) for VWF: Ristocetin Cofactor (VWF:Rco), VWF: Antigen (VWF:Ag) and VWF: Collagen Binding Capacity (VWF:CB)
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
AUC0-inf parameter at the baseline PK assessment will be calculated using noncompartmental analysis (NCA) for VWF:RCo, VWF:Ag, and VWF:CB.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Dose Normalized AUC (0-inf) for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Dose normalized AUC0-inf parameter at the baseline PK assessment will be calculated using NCA for VWF:RCo, VWF:Ag, and VWF:CB.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
AUC From Time 0 to 96 Hours (AUC0-96h) for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
AUC0-96h parameter at the baseline PK assessment will be calculated using NCA for VWF:RCo, VWF:Ag, and VWF:CB.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Dose Normalized AUC(0-96h) for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Dose normalized AUC0-96h parameter at the baseline PK assessment will be calculated using NCA for VWF:RCo, VWF:Ag, and VWF:CB.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Maximum Plasma Concentration (Cmax) for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Cmax parameter at the baseline PK assessment will be calculated using NCA for VWF:RCo, VWF:Ag, and VWF:CB.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Dose Normalized Cmax for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Dose normalized Cmax parameter at the baseline PK assessment will be calculated using NCA for VWF:RCo, VWF:Ag, and VWF:CB.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Time to Reach Maximum Plasma Concentration (Tmax) for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Tmax parameter at the baseline PK assessment will be calculated using NCA for VWF:RCo, VWF:Ag, and VWF:CB.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Mean Residence Time for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Mean residence time parameter at the baseline PK assessment will be calculated using NCA for VWF:RCo, VWF:Ag, and VWF:CB.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Clearance for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Clearance parameter at the baseline PK assessment will be calculated using NCA for VWF:RCo, VWF:Ag, and VWF:CB.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Terminal Half-life (T1/2) for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
T1/2 parameter at the baseline PK assessment will be calculated using NCA for VWF:RCo, VWF:Ag, and VWF:CB.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Volume of Distribution at Steady State (Vss) for VWF:RCo, VWF:Ag and VWF:CB
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Vss parameter at the baseline PK assessment will be calculated using NCA for VWF:RCo, VWF:Ag, and VWF:CB.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Incremental Recovery (IR) at Cmax for VWF:RCo and VWF:Ag
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
IR parameter at the baseline PK assessment will be calculated using NCA for VWF:RCo and VWF:Ag.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
AUC From Time 0 to Last time of Measurable Activity (AUC0-tlast) for Factor VIII:Coagulation Activity (FVIII:C)
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
AUC0-tlast parameter at the baseline PK assessment will be calculated using NCA for FVIII: C.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Dose Normalized AUC0-tlast for FVIII:C
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Dose Normalized AUC0-tlast parameter at the baseline PK assessment will be calculated using NCA for FVIII: C.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
AUC0-96h for FVIII:C
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
AUC0-96h parameter at the baseline PK assessment will be calculated using NCA for FVIII: C.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Dose Normalized AUC0-96h for FVIII:C
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Dose normalized AUC0-96h parameter at the baseline PK assessment will be calculated using NCA for FVIII: C.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Cmax for FVIII:C
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Cmax parameter at the baseline PK assessment will be calculated using NCA for FVIII: C.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Dose Normalized Cmax for FVIII:C
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Dose Normalized Cmax parameter at the baseline PK assessment will be calculated using NCA for FVIII: C.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Tmax for FVIII:C
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Tmax parameter at the baseline PK assessment will be calculated using NCA for FVIII: C.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Plasma Level of VONVENDI based on Von Willebrand Factor: Ristocetin Cofactor (VWF:Rco)
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Plasma level of VONVENDI based on VWF:Rco will be reported.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Plasma Level of VONVENDI based on Von Willebrand Factor Antigen (VWF:Ag)
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Plasma level of VONVENDI based on VWF:Ag will be reported.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Plasma Level of VONVENDI based on Von Willebrand Factor Collagen Binding (VWF:CB)
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Plasma level of VONVENDI based on VWF:CB will be reported.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Plasma Level of Factor VIII Clotting (FVIII:C)
Time Frame: At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Plasma level of FVIII:C will be reported.
At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion
Total Hemostatic Efficacy Assessment 24 hours After Last Perioperative IP Infusion or at Day 14 Postoperative Visit
Time Frame: At 24 hours or Day 14
The overall hemostatic efficacy of VONVENDI, with or without ADVATE, will be assessed by the investigator using the hemostatic efficacy assessment rating scale either 24 hours after the last perioperative infusion or at the Day 14 postoperative visit, whichever comes first.
At 24 hours or Day 14
Total Volume of Actual and Predicted Intraoperative Blood Loss After the Surgery as Assessed by the Operating Surgeon
Time Frame: From Intraoperative through completion of surgery (up to Day 14)
Intraoperative actual blood loss will be assessed by the operating surgeon at the completion of surgery using a predefined 4-point rating scale. 1- Excellent (intraoperative blood loss was less than or equal to the maximum expected for the type of procedure performed in a hemostatically normal individual [<=]100 percent [%]); 2- Good (intraoperative blood loss up to 50% more than the maximum expected blood loss for the type of procedure performed in a hemostatically normal individual [101%-150%]); 3- Moderate (intraoperative blood loss exceeding 50% of the expected for the type of procedure performed in a hemostatically normal individual [greater than (>)150%]); 4- None (uncontrolled hemorrhage due to an inadequate therapeutic response despite appropriate dosing, necessitating a change of clotting factor replacement regimen). Lower scores indicate better hemostatic efficacy. Scoring is calculated by comparing the actual blood loss versus predicted blood loss.
From Intraoperative through completion of surgery (up to Day 14)
Intraoperative Hemostatic Efficacy Score as Assessed by Operating Surgeon at Completion of Surgery
Time Frame: At completion of surgery (up to Day 14)
The hemostasis assessment rating scale ranges from 1 to 4, where 1 = Excellent, 2 = Good, 3 = Moderate, and 4 = None. Lower scores indicate better hemostatic efficacy.
At completion of surgery (up to Day 14)
Daily Intra- and Postoperative Weight-adjusted Dose of VONVENDI With or Without ADVATE Through Postoperative Day 14
Time Frame: From day of surgery through postoperative Day 14
Daily intra- and postoperative weight-adjusted dose of VONVENDI with or without ADVATE through postoperative day 14 will be reported.
From day of surgery through postoperative Day 14

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Takeda

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Study Director, Takeda

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 13, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 25, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 25, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 12, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 12, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 19, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 8, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 7, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • TAK-577-3002

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

IPD Sharing Access Criteria

IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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