Clinical trial: ATHN 2: Factor Switching Study (NCT02546622)

Eligibility criteria:
Inclusion Criteria

          -  Moderate or Severe Congenital Hemophilia A or B (FVIII or FIX clotting activity less
             than or equal to 5% of normal).

          -  Able to give informed consent (by patient or parent/authorized guardian).

          -  Previously treated with plasma-derived or recombinant clotting factor replacement
             products with at least 50 exposure days (as assessed either from direct clinical
             records in children under age 5, or by clinical history of dosing in older patients).
             For Arm B being enrolled retrospectively, this previous treatment must be prior to
             product switch under study.

          -  Planning to switch, or recently switched within the previous 50 weeks, to a new brand
             or type of replacement factor VIII or IX, FDA approved after January 1, 2013.

          -  Arm B only: Negative inhibitor screen within the last 6 months prior to switching.

        Note: History of prior transient inhibitor or inhibitor eradicated by immune tolerance
        induction (ITI) are eligible.

        Exclusion Criteria

          -  Presence of any known bleeding disorder other than hemophilia A or B (i.e., patients
             with concurrent hemophilia and a second hemostatic defect are NOT eligible). Low Von
             Willebrand Factor (VWF) without VWF diagnosis are not excluded.

          -  Presence of an active inhibitor >0.6 BU for factor VIII, > 0.4 BU for factor IX at the
             time of eligibility assessment. Detection of such an inhibitor at the baseline visit
             prior to dosing with the new product (Arm A), or after dosing with new factor dosing
             (Arm B), would result in early termination without other study assessments.

          -  Currently undergoing ITI.

          -  Immunosuppressive therapy (cyclophosphamide, mycophenolate, IVIG) within 90 days and
             Rituximab within 6 months; topical steroid treatments and short course steroids for
             asthma or allergy allowed.

          -  Previous participation in Phase I, II or III interventional trials of the factor
             product being switched to.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.



Patient description (medical language): A 20-year-old man comes to the emergency due to bleeding after a tooth extraction.  The bleeding has persisted for approximately 30 minutes despite constant direct pressure.  He is a known case of Hemophilia type A treated with FVIII. Blood pressure is 95/60 mm Hg and pulse is 105/min. His weight is 70 Kg. Family history is positive for Hemophilia type A in his maternal uncle. He also has a lipoma on his left arm which he plans to remove surgically. His FVIII activity is 40%.

Patient description (patient's own words): I got my tooth extracted and I could not stop bleeding for 30 min. I'm a 20 yo dude but I have Hemophilia type A treated with FVIII. My blood pressure was 95/60 and my pulse 105/min. I'm 70 kg. My uncle on my mom's side also has type A hemophilia. I also have a fatty swelling on my left arm and I was supposed to remove it. My FVIII activity was 40%.

Does the patient match the trial criteria? Contradiction