Clinical trial: A Study of the Pharmacokinetics, Pharmacodynamics, and Safety of ARC1779 Injection in Patients With Von Willebrand Disease Type 2B (NCT00694785)

Eligibility criteria:
Inclusion Criteria:

          -  Male or female patients;

          -  ≥ 16 to ≤ 75 years of age;

          -  Diagnosis of VWD-2B according to national expert guidelines for the USA [1] and Europe
             [2] based on medical history and findings from a matrix of laboratory assays which may
             include: platelet count, concentration of VWF antigen (VWF:Ag), VWF ristocetin
             cofactor activity (VWF:RCo), Factor VIII (FVIII) activity, ristocetin-induced platelet
             aggregation (RIPA), platelet function analyzer (PFA-100®) closure time, bleeding time
             (BT), VWF multimer test, VWF: platelet-binding (VWF:PB) activity, etc.)

          -  Thrombocytopenia (defined as a platelet count < 100 per nL on at least 2 occasions
             within the month preceding enrollment;

          -  Female patients of reproductive age must be enrolled within 1 to 7 days of the
             cessation of preceding menses;

          -  Female patients must be non-pregnant and willing to use effective, redundant methods
             of contraception (i.e., for both self and male partner) throughout the study and for
             at least 30 days after discontinuation of study drug treatment;

          -  Male patients must agree to use a medically acceptable contraceptive (abstinence or
             use of a condom with spermicide) throughout the study and for at least 30 days after
             discontinuation of study drug treatment;

          -  All patients must be capable of understanding and complying with the protocol and must
             have signed the informed consent document.

        Exclusion Criteria:

          -  Patients with a possible co-existing or alternative hematologic diagnosis which can
             account for the laboratory findings of thrombocytopenia, etc.;

          -  Any significant medical co-morbidity which would pose an increased risk of bleeding
             (e.g., recent trauma or surgery, a history of gastrointestinal ulcers, etc.) or
             thrombosis (e.g., history of recurrent deep vein thrombosis (DVT).
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 16 Years old.
Subject must be at most 75 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