Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With VWD
Condition: Von Willebrand Diseases
MeSH terms: Von Willebrand Diseases
Intervention: Wilate
Intervention description: Produced from the plasma of human donors, Wilate is presented as a powder or solvent for intravenous injection containing normally 500 IU or 1000 IU human VWF and human FVIII per vial. The ratio between VWF ristocetin co-factor activity (VWF:RCo) and FVIII:C is 1:1. The product contains approximately 100 IU/ml human VWF when reconstituted with 5ml/10mL water for injection with 0.1% polysorbate 80.
The specific activity of Wilate is ≥67 IU VWF:RCo/mg protein. The injection or infusion rate should not exceed 2-3mL per minute.
Intervention MeSH terms: Factor VIII
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 43.0
Sex: All
Minimum age: 6 Years
Healthy volunteers: No

Summary:

      This is a prospective, non-controlled, international, multi-center phase 3 study
      investigating the efficacy and safety of Wilate in previously treated adult patients with
      VWD, to obtain additional data on the safety and efficacy of Wilate in previously treated
      patients with VWD undergoing regular prophylaxis.
    

Eligibility criteria:

        Inclusion Criteria:

        Patients who meet all of the following criteria are eligible for the study:

          -  Aged ≥6 years at the time of screening

          -  VWD type 1 (baseline von Willebrand factor activity [VWF:Ristocetin Co-factor (RCo)]
             <30 IU/dL, 2A, 2B, 2M, or 3 according to medical history requiring substitution
             therapy with a VWF-containing product to control bleeding

          -  Currently receiving on-demand treatment with a VWF-containing product with at least 1,
             and an average of ≥2, documented spontaneous BEs per month in the last 6 months, with
             at least 2 of these BEs requiring treatment with a VWF-containing product

          -  Availability of records to reliably evaluate type, frequency, and treatment of BEs for
             at least 6 months of on-demand treatment before screening

          -  Female patients of child-bearing potential must have a negative urine pregnancy test
             at screening and agree to use adequate birth control measures; in case hormonal
             contra-ception is used, the medication class should remain unchanged for the duration
             of the study

          -  All patients to provide voluntarily given, fully informed written and signed consent
             obtained before any study-related procedures are conducted

        Exclusion Criteria:

        Patients who meet any of the following criteria are not eligible for the study:

          -  Having received on-demand or prophylactic treatment with a VWF-containing product but
             having no records available to reliably evaluate the type, frequency, and treatment of
             BEs over a period of at least 6 months of on-demand treatment

          -  History, or current suspicion, of VWF or FVIII inhibitors

          -  Medical history of a thromboembolic event within 1 year before enrolment

          -  Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate
             trans-aminase [ASAT] levels >5 times of upper limit of normal, creatinine >120 µmol/L)

          -  Platelet count <100,000/µL at screening (except for VWD type 2B)

          -  Body weight <20 kg at screening

          -  Patients receiving, or scheduled to receive, immunosuppressant drugs (other than
             an-tiretroviral chemotherapy), such as prednisone (equivalent to >10 mg/day), or
             similar drugs

          -  Pregnant or breast-feeding at the time of enrolment

          -  Cervical or uterine conditions causing abnormal uterine bleeding (including infection,
             dysplasia)

          -  Treatment with any IMP in another interventional clinical study currently or within 4
             weeks before enrolment

          -  Other coagulation disorders or bleeding disorders due to anatomical reasons

          -  Known hypersensitivity to any of the components of the study drug