Clinical trial: Study of a pd VWF/FVIII Concentrate, Biostate®, in Subjects With Von Willebrand Disease (NCT00941616)

Eligibility criteria:
Inclusion Criteria:

          -  Diagnosed with VWD

          -  Desmopressin acetate (DDAVP) treatment is ineffective or contraindicated or not
             available

          -  Evidence of vaccination against hepatitis A and B (or presence of antibodies against
             hepatitis A and B) within 10 years prior to their first dose of Biostate®

          -  Written informed consent given

        Exclusion Criteria (for participation in the PK component):

          -  Actively bleeding immediately prior to initial PK period

          -  Have received DDAVP or a VWF product in the 5 days prior to their first dose of study
             product

          -  Have Type 2B, 2N or 2M VWD

        Exclusion Criteria (for all subjects):

          -  Requiring a VWF product for a planned surgical procedure at enrolment

          -  Have received aspirin or other non-steroidal anti-inflammatory drugs within 7 days
             prior to their first dose of study product

          -  Known history of, or are suspected to have, VWF or FVIII inhibitors

          -  Suffering an acute or chronic medical condition, other than VWD, which may affect the
             conduct of the study

          -  Known or suspected hypersensitivity or previous evidence of severe side effects to
             Biostate®, VWF/FVIII concentrates, or human albumin

          -  Impaired liver function at screening

          -  Evidence or a history (within the previous 12 months) of abuse of any drug substance,
             licit or illicit

          -  Participation in a clinical study or use of an investigational compound in the 3
             months preceding the first day of study drug administration, or plans to enter such a
             study during the study period.

          -  Females who are pregnant, breast-feeding or who have a positive pregnancy test at
             screening
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 12 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