Clinical trial: Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With VWD (NCT04052698)

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
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 6 Years old.


Patient description (medical language): A 23-year-old female has prolonged oral bleeding immediately after a tooth extraction.  Despite several interventions, the bleeding persists for hours and stops only after desmopressin (DDAVP) administration.  The patient has heavy menstrual cycles each month.  She has no other medical problems and takes no medications.  Her mother and grandmother have also had excessive bleeding during menstrual period.  Review of systems is positive for mild bruising on his legs. Laboratory findings reveal a normal platelet count and an abnormal ristocetin cofactor assay, as well as CB <= 0.30 IU/mL and FVIII:C <= 0.40 IU/mL.

Patient description (patient's own words): I'm a 23 woman and I went to the ER because of non-stop bleeding after my tooth extraction. I tried to make the bleeding stop but it just kept going for hours and it finally stopped when I took some medication called DDAVP. I usually have heavy periods, just like my mom and grandma had. I don't have any other medical condition and I don't take any other medication. The doctor noticed the bruises on my legs. I had to do some lab tests. I had a normal platelet count but an abnormal ristocetin cofactor assay. I had low CB and FVIII:C levels.

Does the patient match the trial criteria? Entailment