Clinical trial: Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients (NCT00555555)

Eligibility criteria:
Inclusion Criteria:

          1. Male or female 7 years of age or older

          2. The subject has been diagnosed of inherited VWD of Type 3 as determined by subject's
             medical records.

          3. The subject needs a surgical procedure (at least 10 surgical procedures have to be
             considered as "Major" according to the criteria of the protocol).

          4. The subject is expected to respond to exogenously administered FVIII/VWF according to
             Investigator's judgment.

          5. The subject freely gives written informed consent. Patients who are not legally
             permitted to provide written consent must sign a form of assent for study
             participation, and written consent must be provided by a parent or legal guardian.

        Exclusion Criteria:

          1. The subject has been diagnosed of acquired VWD.

          2. The subject is known to have history of intolerance to any Alphanate® containing
             substance.

          3. The subject is known to have history of anaphylactic reaction(s) to blood or blood
             components.

          4. Liver function tests (AST, ALT, bilirubin) > 2.5 x upper limit of normal (ULN).

          5. Renal function test (creatinine, BUN) > 1.5 x ULN.

          6. The subject is known or suspected to have present or past inhibitor activity
             (antibodies) directed against FVIII or VWF.

          7. The subject is known to abuse alcohol or illicit drug use within the past 12 months.

          8. The subject is participating in another clinical study involving an investigational
             treatment, or participated within the past 4 weeks (except if the patient is
             participating in another Alphanate® study). Studies consisting of data and blood
             sampling collections on a regular or long-term basis are exempt from this exclusion.

          9. The subject is unlikely to adhere to the protocol requirements of the study.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 7 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