Clinical trial: A Study With OPTIVATE® in People With Von Willebrand Disease (NCT00387192)

Eligibility criteria:
Inclusion Criteria:

          1. Have given written informed consent.

          2. Be aged 12 years or older.

          3. Have severe VWD (VWF:RCo <20%) of known type. Severity will be confirmed by a current
             VWF:RCo result of <20%.

          4. Be known or expected to require a concentrate for management of VWD.

          5. Must have had at least one bleed in the last 12 months which required treatment with a
             FVIII and VWF concentrate.

          6. Have a known lack of, or poor response to, DDAVP.

          7. Have a prothrombin time (PT) of not more than 3 seconds above the upper limit of the
             reference range.

          8. At the Baseline Visit (Visit 1), patients must have had at least 5 days since their
             last infusion of replacement factor concentrate or DDAVP.

          9. Female patients of child-bearing potential must have a negative result on a human
             chorionic gonadotropin-based pregnancy test. If a female patient is or becomes
             sexually active, she must practice contraception by using a method of proven
             reliability for the duration of the study. Female patients must not be lactating.

        Exclusion Criteria:

          1. Have a history of inhibitor development to VWF or FVIII or a positive result at
             screening.

          2. Actively bleeding (Note: the patient can enter the study once the bleed is
             controlled).

          3. Presence of major systemic illnesses: renal disease, liver disease, or neurological or
             psychiatric disease which would compromise the outcome of the study in the opinion of
             the investigator.

          4. Known or suspected hypersensitivity to investigational medicinal product (IMP) or its
             excipients.

          5. Have a recent history of alcohol or drug abuse.

          6. Administration of a new chemical entity within the 4 months preceding enrolment.

          7. Participation in any other clinical study in which investigational or marketed drugs
             were employed in the 30 days preceding enrolment into this study, with the exception
             of the BPL clinical study Protocol 8VWF03.

          8. In the opinion of the investigator, the patient is unlikely to comply with the study
             protocol.
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