von Willebrand disease
Conditions
Interventions
Vonvendi Intravenous infusion group:Vonvendi Intravenous infusion
Sponsors
The First Affiliated Hospital of Soochow University
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Subject must voluntarily sign an IRB/independent EC-approved written ICF after all relevant aspects of the study have been explained and discussed with the subject; 2. Subject has a documented diagnosis of severe VWD (baseline VWF:RCo <20 IU/dL) with a diagnosis of VWD type verified per the following recommended criteria: • Type 1 (VWF:RCo <20 IU/dL and by VWF activity/VWF: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. Subject is at least 18 years of age at screening; 4. Subject is ethnic Chinese and lives in China, including those from Taiwan, Hong Kong, and Macao; 5. If female of childbearing potential, subject presents with a negative pregnancy test and agrees to employ adequate birth control measures for the duration of the study; 6. Subject is willing and able to comply with the requirements of the protocol; 7. Subject 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
Exclusion criteria: 1. Subject has been diagnosed with pseudo VWD or another hereditary or acquired coagulation disorder other than VWD (eg, qualitative and quantitative platelet disorders or elevated PT/INR >1.4); 2. Subject has a history or presence of a VWF inhibitor at screening; 3. Subject has a documented history of a VWF:RCo half-life of =0.6 BU/mL (by Bethesda assay or Bethesda method with Nijmegen modification); 5. Subject has a known hypersensitivity to any of the components of the study drugs, such as to mouse or hamster proteins; 6. Subject has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies or animal allergies; 7. Subject has a medical history of a thromboembolic event; 8. Subject is HIV positive with an absolute CD4 count =2.5 mg/dL; 17. Subject has a platelet count <100,000/mL at screening (except for subjects 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 subject’s condition); 18. Subject has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia);; 19. Subject has other clinically significant comorbid illness (e.g., uncontrolled hypertension) that may pose additional risk to the subject, in the judgment of the investigator; 20. Investigator determines that the subject is unable or unwilling to cooperate with study procedures.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence, severity, and causality of AEs and SAEs;Incidence of thromboembolic events, severe hypersensitivity reactions;Immuogenicity:Development of neutralizing (inhibitory) antibodies to VWF and FVIII;Development of binding antibodies to VWF and FVIII ;Development of antibodies to CHO proteins, murine IgG, and rFurin;Number of infusions of VONVENDI with or without ADVATE per bleeding episode;Clinically significant abnormal findings in clinical laboratory results, ECG, and vital signs; | — |
Secondary
| Measure | Time frame |
|---|---|
| Number of infusions of ADVATE per bleeding episode;Time to resolution of bleeding episodes;Weight adjusted consumption of VONVENDI and ADVATE per bleeding episode; | — |
Countries
China
Contacts
Public ContactZiqiang Yu
The First Affiliated Hospital of Soochow University
Outcome results
None listed