Clinical trial: Pharmacokinetics, Safety and Efficacy of Recombinant Von Willebrand Factor (rVWF) in the Treatment of Bleeding Episodes in Von Willebrand Disease (VWD) (NCT01410227)

Eligibility criteria:
Inclusion Criteria:

          -  Participant has been diagnosed with:

               1. Type 1 (Von Willebrand factor: Ristocetin cofactor activity (VWF:RCo) < 20 IU/dL)
                  or,

               2. Type 2A (VWF:RCo < 20 IU/dL), Type 2B (as diagnosed by genotype), Type 2N (Factor
                  VIII activity (FVIII:C) <10% and historically documented genetics), Type 2M or,

               3. Type 3 ( Von Willebrand factor antigen (VWF:Ag) ≤ 3 IU/dL) or,

               4. Severe Von Willebrand disease (VWD) with a history of requiring substitution
                  therapy with von Willebrand factor concentrate to control bleeding

          -  Participant, who participates in the treatment for bleeding episodes, has had a
             minimum of 1 documented bleed (medical history) requiring VWF coagulation factor
             replacement therapy during the previous 12 months prior to enrollment.

          -  Participant has a Karnofsky score ≥ 60%

          -  Participant is at least 18 and not older than 65 years of age at enrollment

          -  If female of childbearing potential, participant presents with a negative pregnancy
             test

          -  Participant agrees to employ adequate birth control measures for the duration of the
             study

          -  Participant is willing and able to comply with the requirements of the protocol

        Exclusion Criteria:

          -  Participant has been diagnosed with pseudo VWD or another hereditary or acquired
             coagulation disorder other than VWD (eg qualitative and quantitative platelet
             disorders or elevated PT/international normalized ratio [INR] >1.4).

          -  Participant has a documented history of a VWF:RCo half-life of <6 hours.

          -  Participant has a history or presence of a VWF inhibitor at screening.

          -  Participant has a history or presence of a factor VIII (FVIII) inhibitor with a titer
             ≥0.4 BU (by Nijmegen assay) or ≥ 0.6 BU (by Bethesda assay).

          -  Participant has a known hypersensitivity to any of the components of the study drugs,
             such as mouse or hamster proteins.

          -  Participant has a medical history of immunological disorders, excluding seasonal
             allergic rhinitis/conjunctivitis, mild asthma, food allergies or animal allergies.

          -  Participant has a medical history of a thromboembolic event.

          -  Participant is HIV positive with an absolute CD4 count <200/mm3.

          -  Participant has been diagnosed with cardiovascular disease (New York Heart Association
             [NYHA] classes 1-4.

          -  Participant has an acute illness (eg, influenza, flu-like syndrome, allergic
             rhinitis/conjunctivitis, non-seasonal asthma) at screening.

          -  Participant has been diagnosed with significant liver disease as evidenced by any of
             the following: serum alanine aminotransferase (ALT) 5 times the upper limit of normal;
             hypoalbuminemia; portal vein hypertension (eg, presence of otherwise unexplained
             splenomegaly, history of esophageal varices).

          -  Participant has been diagnosed with renal disease, with a serum creatinine level ≥2
             mg/dL.

          -  In the judgment of the investigator, the participant has another clinically
             significant concomitant disease (eg, uncontrolled hypertension) that may pose
             additional risks for the participant.

          -  Participant has been treated with an immunomodulatory drug, excluding topical
             treatment (eg, ointments, nasal sprays), within 30 days prior to enrollment

          -  Participant is pregnant or lactating at the time of enrollment.

          -  Participant has participated in another clinical study involving an IP or
             investigational device within 30 days prior to enrollment or is scheduled to
             participate in another clinical study involving an investigational product or
             investigational device during the course of this study.

          -  Participant has a history of drug or alcohol abuse within the 2 years prior to
             enrollment.

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

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

          -  Participant 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.

          -  Participant 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 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