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A Clinical Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Chinese Adults With Von Willebrand Disease
Led by Takeda · Updated on 2026-01-08
20
Participants Needed
7
Research Sites
N/A
Total Duration
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AI-Summary
What this Trial Is About
Researchers are evaluating the safety and how well the drug VONVENDI works in adult Chinese participants who have Von Willebrand Disease (VWD). The study focuses on participants with severe VWD and aims to see how VONVENDI controls bleeding, both with and without the use of another drug called ADVATE, especially during elective surgeries or dental procedures. This phase 3 study also examines how VONVENDI is processed in the body and how it affects the body's response to bleeding. Participants will receive a single intravenous dose of VONVENDI at the start for pharmacokinetic assessment. During a 12-month period, any bleeding episodes needing treatment with von Willebrand factor (VWF) replacement will be managed using VONVENDI, with or without ADVATE. The initial dose of VONVENDI ranges from 40 to 80 international units per kilogram of body weight. Additional infusions may be given as necessary for managing bleeding related to surgeries or dental procedures. Participants will be involved in the study for about 14 months, including the treatment and follow-up periods. They will have regular visits to clinics or telephone follow-ups to monitor their health, treatment effects, and any side effects. Researchers will track various safety measures such as adverse events, allergic reactions, antibody development, laboratory test results, and vital signs over this time. The study also measures how much of each drug is used per bleeding episode and how quickly bleeding resolves.
CONDITIONS
Brief Title
A Study of Recombinant Von Willebrand Factor (rVWF) in Chinese Participants With Von Willebrand Disease (vWD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant must voluntarily sign an approved informed consent form.
- Documented diagnosis of severe Von Willebrand Disease (VWD) with baseline VWF:RCo less than 20 IU/dL, confirmed by recommended criteria for types 1, 2A, 2B, 2N, 2M, or 3.
- At least 18 years old at screening.
- Ethnic Chinese living in China, Taiwan, Hong Kong, or Macao.
- If female of childbearing potential, negative pregnancy test and agreement to use birth control during the study.
- Willing and able to comply with study requirements.
- At least 3 documented bleeding episodes requiring VWF replacement therapy in the 12 months before enrollment.
You will not qualify if you...
- Diagnosis of pseudo VWD or other coagulation disorders besides VWD.
- History or presence of VWF inhibitor at screening.
- Documented VWF:RCo half-life less than 6 hours.
- Presence of FVIII inhibitor with titer >= 0.6 BU/mL.
- Known allergy to study drugs or components, including mouse or hamster proteins.
- History of immunological disorders except mild allergies or asthma.
- History of thromboembolic events.
- HIV positive with CD4 count less than 200/mm3.
- Recent treatment with immunomodulatory drugs or corticosteroids equivalent to hydrocortisone >10 mg/day.
- Pregnant or breastfeeding at consent.
- Participation in another investigational study within 30 days before enrollment.
- Progressive fatal disease or life expectancy under 15 months.
- Member of study team or dependent of team members.
- Acute illness at screening.
- Significant liver disease or liver cirrhosis class B or C.
- Renal disease with serum creatinine >= 2.5 mg/dL.
- Platelet count below 100,000/mL (except for type 2B VWD with special assessment).
- Cervical or uterine conditions causing heavy bleeding.
- Other clinically significant conditions posing risk or inability to comply with study procedures.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 12 months
Participants receive a single intravenous dose of VONVENDI at baseline for pharmacokinetic assessment. During the 12-month on-demand treatment period, participants receive VONVENDI with or without ADVATE to manage bleeding episodes and perioperative bleeding as needed.
Visits as needed for bleeding episodes and perioperative management
Duration - Up to 2 months after treatment period
Participants are monitored for safety and treatment outcomes up to 14 months, including assessments of adverse events and laboratory parameters.
Scheduled follow-up visits post-treatment
Trial Site Locations
Total: 7 locations
1
Peking Union Medical College Hospital
Beijing, China, 100006
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2
Nanfang Hospital Southern Medical University
Guangzhou, China, 510515
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3
Jinan Central Hospital
Jihan, China, 250013
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4
Ruijin Hospital Shanghai Jiaotong University School of Medicine
Shanghai, China, 201801
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5
The First Affiliated Hospital of Soochow University
Suzhou, China, 215005
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6
Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences
Tianjin, China, 300052
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7
Tongji Hospital Tongji Medical College Huazhong University of Science and Technology
Wuhan, China, 430032
Actively Recruiting
Research Team
T
Takeda Contact
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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