Clinical trial: Efficacy and Safety of Fanhdi®, a High-purity Von Willebrand Containing FVIII Concentrate, in Pediatric Patients With Von Willebrand Disease (NCT02472665)

Eligibility criteria:
Inclusion Criteria:

          1. Subjects diagnosed with severe hereditary VWD (VWF:RCo<15-20 IU/dL) of any type

          2. Subjects less than 6 years of age

          3. Subjects that do not adequately respond to treatment with desmopressin (DDAVP) or had
             a previous treatment failure with DDAVP. Note: Inadequate response to DDAVP treatment
             or a previous treatment failure with DDAVP is not required for subjects with VWD type
             2b and 3. A confirmed diagnosis in medical history is sufficient for this criterion,
             since treatment with DDAVP is not indicated in or contraindicated for these VWD
             patients.

          4. Signed informed consent form provided by an authorized representative on behalf of the
             subject in accordance with local law and institutional policy.

        Exclusion Criteria:

          1. Subject has been diagnosed of acquired VWD

          2. Subjects bleeding at the time of the first infusion or the 10 days prior to the
             infusion

          3. Subjects treated with DDAVP or another FVIII containing VWF concentrate during the 5
             days prior to the infusion of the Fanhdi. This may be reduced to 3 days for subjects
             with type 3 VWD.

          4. Subject is known or suspected to have present or past inhibitor activity (antibodies)
             directed against FVIII or VWF

          5. Subject is known to have history of intolerance to any Fanhdi® containing substance

          6. Subject is known to have history of anaphylactic reaction(s) to blood or blood
             components

          7. Subjects presenting with severe platelet dysfunctions due to drugs (aspirin, other
             NSAIDs, etc.) or other pathologies (uremic thrombopathy, hematological diseases)

          8. Subjects with known previous infection with Hepatitis A Virus (HAV), Hepatitis B Virus
             (HBV), Hepatitic C Virus (HCV), or HIV or clinical signs and symptoms consistent with
             current HAV, HBV, HCV or HIV infection.

          9. Subjects presenting with anemia (hemoglobulin <11 g/dL)

         10. Subjects diagnosed with metabolic diseases that are not clinically controlled, such as
             diabetes mellitus, that could potentially interfere with the interpretations of the
             study

         11. Subjects participating in another clinical study within 30 days prior to screening or
             has received any investigational treatment within 3 months prior to screening

         12. If it is anticipated that the subject will be treated with other products containing
             FVIII or VWF different from Fanhdi® during a period of one year

         13. 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 2 Months old.
Subject must be at most 6 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