Clinical trial: To Assess the Pharmacokinetics and Safety and Tolerability of Efanesoctocog Alfa (BIVV001)in Adults With Type 2N and 3 Von Willebrand Disease (VWD) (NCT04770935)

Eligibility criteria:
Inclusion criteria :

        -- Male and/or female participant, between 18 and 65 years of age, inclusive at the time of
        informed consent.

          -  The participant has been diagnosed with hereditary type 3 VWD or type 2N VWD as
             documented in historical medical records OR a documented genotype known to produce VWD
             type 3 or 2N VWD.

          -  Type 3 VWD participants are included if they have a medical history of at least 25
             exposure days to VWF-containing coagulation factor concentrates - - - -

        Exclusion criteria:

          -  Hereditary or acquired coagulation disorder other than VWD (including qualitative and
             quantitative platelet disorders, and thrombocytopenia < 100,000 cells/uL at Screening)

          -  The participant has a FVIII activity levels >20 IU/dL, at Screening

          -  History or presence of a VWF inhibitor or clinical suspicion of a VWF inhibitor

          -  History of a positive FVIII inhibitor test, defined as ≥0.6 BU/mL (by Nijmegen
             modified Bethesda assay) or a clinical suspicion of a FVIII inhibitor

          -  Positive FVIII inhibitor test, defined as ≥0.6 BU/mL, at Screening

          -  History of hypersensitivity or anaphylaxis associated with any FVIII- or VWF-
             containing product

          -  The participant has received or anticipates receiving systemic immunosuppressive or
             immunomodulatory treatment within 12 weeks prior to Baseline.

          -  The participant requires the use of acetylsalicylic acid, non-NSAID anti-platelets,
             and NSAIDs above the maximum dose product

        The above information is not intended to contain all considerations relevant to a patient's
        potential participation in a clinical trial.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 18 Years old.
Subject must be at most 65 Years

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