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

NCT07129343 | Recruiting Phase 3 FDA Drug

• 1 other identifier: TAK-577-3002
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 7

Brief Summary

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.

Conditions

Von Willebrand Disease (VWD)

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (9)

• Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

  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

  Number of participants with severity of TEAE will be reported.

  Up to 14 months

• Number of Participants With TEAEs and SAEs by Causality

  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

  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

  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

  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

  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)

  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

  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 Outcomes (31)

• Number of Infusions of VONVENDI With or Without ADVATE per Bleeding Episode

  Up to 12 months

• Number of Infusions of ADVATE per Bleeding Episode

  Up to 12 months

• Weight-adjusted Consumption of VONVENDI and ADVATE per Bleeding Episode

  Up to 12 months

• Time to Resolution of Bleeding Episodes

  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)

  At baseline: Within 1 hour pre-infusion, 0.25, 0.50, 1, 3, 6, 12, 24, 30, 48, 72, and 96 hours post-infusion

Study Arms (1)

All Participants With VWD

EXPERIMENTAL

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; Biological: ADVATE

Interventions

VONVENDI BIOLOGICAL

VONVENDI is administered by intravenous injection.

Also known as: vonicog alfa, TAK-577, rVWF

All Participants With VWD

ADVATE BIOLOGICAL

ADVATE is administered by intravenous injection.

Also known as: rFVIII, Octocog alfa

All Participants With VWD

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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).
• Participant has a known hypersensitivity to any of the components of the study drugs, such as to mouse or hamster proteins.
• Participant has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies or animal allergies.
• Participant has a medical history of a thromboembolic event.
• Participant is human immunodeficiency virus (HIV) positive with an absolute cluster of differentiation 4 (CD4) count \<200/cubic millimeter (mm\^3).
• 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).
• Participant is pregnant or lactating at the time informed consent is obtained.
• 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.
• Participant is diagnosed with progressive fatal disease and/or has a life expectancy of less than 15 months.
• 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.
• Participant has an acute illness (eg, influenza, flu-like syndrome, allergic rhinitis/conjunctivitis, nonseasonal asthma) at screening.
• 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.

Sponsors & Collaborators

• Takeda: lead

Study Sites (7)

Peking Union Medical College Hospital

Beijing, 100006, China

RECRUITING

Nanfang Hospital Southern Medical University

Guangzhou, 510515, China

RECRUITING

Jinan Central Hospital

Jihan, 250013, China

RECRUITING

Ruijin Hospital Shanghai Jiaotong University School of Medicine

Shanghai, 201801, China

RECRUITING

The First Affiliated Hospital of Soochow University

Suzhou, 215005, China

RECRUITING

Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences

Tianjin, 300052, China

RECRUITING

Tongji Hospital Tongji Medical College Huazhong University of Science and Technology

Wuhan, 430032, China

RECRUITING

Related Links

• Click here for more information about this trial in easy-to-understand language, including a Plain Language Summary of the results if the trial has been completed.

MeSH Terms

Conditions

von Willebrand Diseases

Interventions

Factor VIII; F8 protein, human

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Central Study Contacts

Takeda Contact

CONTACT

+1-877-825-3327; medinfoUS@takeda.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 12, 2025
• First Posted: August 19, 2025
• Study Start: October 13, 2025
• Primary Completion (Estimated): November 25, 2027
• Study Completion (Estimated): November 25, 2027
• Last Updated: January 8, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• 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.

Locations

CN (7)