Clinical trial: Study of Biostate® in Children With Hemophilia A (NCT01229007)

Eligibility criteria:
Inclusion Criteria:

          -  Male subjects between 0 and <12 years of age.

          -  Diagnosed with severe haemophilia A (FVIII:C <1%), and pre-treated for a minimum of 20
             to 50 exposure days.

          -  Have 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),
             as documented in the medical notes at enrolment.

          -  The subject and/or legal guardian understand(s) the nature of the study and has/have
             given written informed consent to participate in the study and is/are willing to
             comply with the protocol.

        Exclusion Criteria:

          -  For all subjects at Day 1: Are actively bleeding.

          -  Have received an infusion of any FVIII product, cryoprecipitate, whole blood, plasma
             or desmopressin acetate in the 4 days prior to their dosing within the PK component.

          -  Have a known history of, or who are suspected of having FVIII inhibitors.

          -  Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7
             days of administration of the IMP.

          -  Have an impaired liver function ie, bilirubin >1.5 x upper limit of normal (ULN)
             and/or aspartate/alanine aminotransferase (AST/ALT) >2.5 x ULN (referring to limits of
             the laboratory that performs the determination) at Screening.

          -  Are human immunodeficiency virus [HIV]-1/-2 antibody positive with a viral load of
             >200/µL.

          -  Suffer from an acute or chronic medical condition, other than haemophilia A, which
             may, in the opinion of the Investigator, affect the conduct of the study.

          -  Suffering from von Willebrand disease (VWD) with von Willebrand factor: ristocetin
             cofactor (VWF:RCo) level <50 IU/dL at Screening.

          -  Have a known or suspected hypersensitivity or previous evidence of severe side effects
             to a plasma-derived FVIII product or to human albumin.

          -  Have participated in a clinical study or used an investigational compound in another
             study (eg, a new chemical entity not registered for clinical use) in the 3 months
             preceding the first day of IMP administration, or are planning to enter such a study
             during the study period.

          -  Unwillingness and/or inability to comply with the study requirements.
Male
No healthy subjects accepted to join the trial.

Subject must be at most 12 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? Contradiction