Clinical trial: A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors (NCT03191799)

Eligibility criteria:
Inclusion Criteria:

          -  As per investigator's judgement, a willingness and ability to comply with scheduled
             visits, treatment plans, laboratory tests, and other study procedures, including the
             patient-reported outcome (PRO) questionnaires and bleed diaries through the use of an
             electronic device or paper

          -  Aged 12 years or older at the time of informed consent

          -  Diagnosis of congenital hemophilia A with persistent inhibitors against FVIII

          -  Documented treatment with bypassing agents or FVIII concentrates in the last 6 months
             (on-demand or prophylaxis). Prophylaxis needs to be discontinued the latest by a day
             before starting emicizumab

          -  Adequate hematologic, hepatic, and renal function

          -  For women of childbearing potential: agreement to remain abstinent (refrain from
             heterosexual intercourse) or use a highly effective contraceptive method with a
             failure rate of <1% per year during the treatment period and for at least five
             elimination half-lives (24 weeks) after the last dose of emicizumab

        Exclusion Criteria:

          -  Inherited or acquired bleeding disorder other than hemophilia A

          -  Ongoing (or plan to receive during the study) immune tolerance induction (ITI) therapy
             (prophylaxis regimens with FVIII and/or bypassing agents must be discontinued prior to
             enrollment). Patients receiving ITI therapy will be eligible following the completion
             of a 72-hour washout period prior to the first emicizumab administration

          -  History of illicit drug or alcohol abuse within 12 months prior to screening, as per
             the investigator's judgment

          -  High risk for thrombotic microangiopathy (TMA) (e.g., have a previous medical or
             family history of TMA), as per the investigator's judgment

          -  Previous (in the past 12 months) or current treatment for thromboembolic disease (with
             the exception of previous catheter-associated thrombosis for which antithrombotic
             treatment is not currently ongoing) or current signs of thromboembolic disease

          -  Other conditions (e.g., certain autoimmune diseases) that may increase the risk of
             bleeding or thrombosis

          -  History of clinically significant hypersensitivity reaction associated with monoclonal
             antibody therapies or components of the emicizumab injection

          -  Known human immunodeficiency virus (HIV) infection with CD4 count <200 cells/μL within
             6 months prior to screening

          -  Use of systemic immunomodulators (e.g., interferon or rituximab) at enrollment or
             planned use during the study, with the exception of antiretroviral therapy

          -  Concurrent disease, treatment, or abnormality in clinical laboratory tests that could
             interfere with the conduct of the study or that would, in the opinion of the
             investigator or Sponsor, preclude the patient's safe participation in and completion
             of the study or interpretation of the study results

          -  Receipt of: Emicizumab in a prior investigational study; An investigational drug to
             treat or reduce the risk of hemophilic bleeds within five half-lives of last drug
             administration; A non-hemophilia-related investigational drug within last 30 days or
             five half-lives, whichever is shorter; or, Any concurrent investigational drug.

          -  Pregnancy or lactation, or intent to become pregnant during the study

          -  Positive serum pregnancy test result within 7 days prior to initiation of emicizumab
             (females only)
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 12 Years old.


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