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New treatment option for Von Willebrand Disease (VWD)
Official title A Study of Recombinant Von Willebrand Factor (rVWF) in Chinese Participants With Von Willebrand Disease (vWD)
ClinicalTrials.gov ID: NCT07129343
What this study is testing
What is VONVENDI?
VONVENDI is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for von willebrand disease (vwd).
Also referred to as vonicog alfa, TAK-577.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
- Time commitment: about 14 months
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Participant must voluntarily sign an institutional review board (IRB)/independent ethics committee-approved written informed consent form after all...
- Participant has a documented diagnosis of severe VWD (baseline VWF:RCo less than [\<]20 international units [IU]deciliter [dL]) with a diagnosis of...
- 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...
- Type 3 (VWF:Ag \<=3 IU/dL). Diagnosis is confirmed, when applicable, by genetic testing and/or by multimer analysis.
You likely can't join if
- Participant has been diagnosed with pseudo VWD or another hereditary or acquired coagulation disorder other than VWD (example [eg], qualitative and...
- 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...
- 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...
See the full eligibility criteria
- 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...
- 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.
- 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...
- 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...
- 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...
- 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...
- 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...
- Participant has been diagnosed with renal disease, with a serum creatinine level \>=2.5 milligram per deciliter (mg/dL).
- 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...
- Participant has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia).
- 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.
- Participant is identified by the investigator as being unable or unwilling to cooperate with study procedures.
The study team makes the final eligibility decision.
Where it's taking place
- Beijing, China
- Guangzhou, China
- Jihan, China
- Shanghai, China
- Suzhou, China
- Tianjin, China
- Wuhan, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 14 months per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Beijing, China; Guangzhou, China; Jihan, China; Shanghai, China; Suzhou, China; Tianjin, China and 1 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.