Clinical trial: Study of a pd vWF/FVIII, Biostate®, in Subjects With Haemophilia A (NCT00879541)

Eligibility criteria:
Inclusion Criteria:

          -  Diagnosed with Haemophilia A with ≤ 1% Factor VIII (FVIII) levels in the absence of
             factor replacement

          -  Evidence of vaccination against hepatitis A and B (or presence of antibodies against
             hepatitis A and B due to either a previous infection or prior immunisation) within 10
             years prior to Day 1 documented in the medical notes

          -  At least 150 days of prior exposure to a FVIII replacement product

          -  Written informed consent given

        Exclusion Criteria (for participation in the pharmacokinetic (PK) component):

          -  Active bleeding

          -  Body weight > 100 kg

        Exclusion Criteria (for all subjects):

          -  Receipt of an infusion of any FVIII product, cryoprecipitate, whole blood, plasma, or
             desmopressin acetate (DDAVP) in the 4 days prior to Day 1

          -  Known history of FVIII inhibitors, or FVIII inhibitor level > 0.6 Bethesda Units (BU)
             at screening

          -  Receipt of aspirin or other Non-Steroidal Anti-Inflammatory Drugs (NSAIDs) within 7
             days of administration of study product.

          -  CD4 lymphocytes < 200/µL. Subjects wo are HIV-1 positive may be considered for the
             study if viral load ≤ 200 particles/µL at screening and all other eligibility criteria
             are met.

          -  Impaired liver function ie. bilirubin >1.5 x upper limit of normal (ULN) and/or
             AST/ALT > 2.5 x ULN at screening.

          -  Acute or chronic medical condition, other than haemophilia A, which may, in the
             opinion of the Investigator, affect the conduct of the study

          -  von Willebrand Disease (VWD) with Von Willebrand Factor:Ristocetin Cofactor (vWF:RCo)
             level < 50 IU/dL at screening

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

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

          -  Participation in a clinical study or use of an investigational compound (e.g. a new
             chemical entity not approved for clinical use) in the 3 months preceding the first day
             of study drug administration, or plans to enter such a study during the study period

          -  Not willing and/or not able to comply with study requirements
Male
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? Contradiction