Clinical trial: Pharmacokinetic, Safety and Tolerability Study of Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex in Type 3 Von Willebrand Disease (NCT00816660)

Eligibility criteria:
Inclusion Criteria:

          -  Subject has voluntarily given written informed consent (before conduct of any
             study-related procedures)

          -  The subject has hereditary type 3 VWD (<= 3 IU/dL VWF:Ag)or severe type 1 or type 2A
             VWD (VWF:RCo <= 10% and FVIII:C <20%)

          -  The subject has a medical history of at least 25 exposure days to VWF/FVIII
             coagulation factor concentrates

          -  The subject has a Karnofsky score >= 70%

          -  The subject is between 18 to 60 years of age (on the day of signing the informed
             consent)

          -  NOT APPLICABLE IN ITALY: Female subjects of child-bearing potential must have a
             negative pregnancy test and agree to practice contraception using a method of proven
             reliability from the day of screening until the study completion visit

          -  APPLICABLE ONLY IN ITALY: Female subjects of child-bearing potential must have a
             negative pregnancy test and agree to practice non-hormonal-based contraception using a
             method of proven reliability (IUD acceptable) from the day of screening until 96 hours
             after the last investigational drug infusion

          -  NOT APPLICABLE IN ITALY: The subject must agree not to be on any therapy
             (hormone-based contraception acceptable) interfering with coagulation factor
             pharmacokinetics until 96 hours after the last investigational drug infusion

          -  APPLICABLE ONLY IN ITALY: The subject must agree not to be on any therapy interfering
             with coagulation factor pharmacokinetics until 96 hours after the last investigational
             drug infusion

        Exclusion Criteria:

          -  The subject has been diagnosed with a hereditary or acquired coagulation disorder
             other than VWD (including qualitative and quantitative platelet disorders and/or an
             international normalized ratio (INR) > 1.4)

          -  The subject has been diagnosed with an ADAMTS13 deficiency with less than 10% ADAMTS13
             activity

          -  The subject has a history or presence of VWF inhibitor

          -  The subject has a history or presence of FVIII inhibitor with a titer >= 0.4 BU (by
             Nijmegen assay) or >= 0.6 BU (by Bethesda assay)

          -  The subject has a known hypersensitivity to mouse or hamster proteins

          -  The subject has a medical history of immunological disorders, excluding seasonal
             allergic rhinitis/conjunctivitis, food allergies or animal allergies

          -  The subject has a medical history of a thromboembolic event

          -  The subject is HIV positive with an absolute CD4 count < 200/mm3

          -  The subject has been diagnosed with cardiovascular disease (New York Heart Association
             (NYHA) classes 1-4)

          -  The subject has been diagnosed with insulin-dependent diabetes mellitus

          -  The subject has an acute illness (e.g. influenza, flu-like syndrome, allergic
             rhinitis/conjunctivitis)

          -  The subject has been diagnosed with liver disease, as evidenced by, but not limited
             to, any of the following: serum ALT three times the upper limit of normal,
             hypoalbuminemia, portal vein hypertension (e.g. presence of otherwise unexplained
             splenomegaly, history of esophageal varices)

          -  The subject has been diagnosed with renal disease, with a serum creatinine level >= 2
             mg/dL

          -  In the judgment of the investigator, the subject has another clinically significant
             concomitant disease (e.g. uncontrolled hypertension, diabetes type II) that may pose
             additional risks for the subject

          -  The subject has been treated with an immunomodulatory drug, excluding topical
             treatment (e.g. ointments, nasal sprays) within 30 days before enrollment

          -  The subject has been treated with drugs known to induce thrombotic thrombocytopenic
             purpura (TTP) (e.g. Adenosine diphosphate (ADP) receptor inhibitors (Clopidogrel,
             Ticlopidine)) within 60 days before enrollment

          -  The subject is receiving or anticipates receiving another investigational and/or
             interventional drug within 30 days before enrollment

          -  The subject is a lactating female

          -  The subject has a history of drug or alcohol abuse within the last 5 years

          -  The subject has a progressive fatal disease and/or life expectancy of less than 3
             months

          -  The subject is identified by the investigator as being unable or unwilling to
             cooperate with study procedures

          -  The subject suffers from a mental condition rendering him/her unable to understand the
             nature, scope and possible consequences of the study and/or evidence of an
             uncooperative attitude

          -  Subject is in prison or compulsory detention by regulatory and/or juridical order
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 60 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