Clinical trial: An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors (NCT03154437)

Eligibility criteria:
Inclusion Criteria:

          -  Diagnosis of congenital hemophilia A of any severity and documented history of
             high-titer inhibitor (that is [i.e.], greater than or equal to [>/=] 5 Bethesda Units)

          -  History of treatment with episodic or prophylactic bypassing agents for at least the
             last 24 weeks

          -  >/=6 (if on an episodic bypassing agent regimen) or >/=2 (if on a prophylactic
             bypassing agent regimen) bleeds within 24 weeks prior to screening

          -  Currently using recombinant activated factor VII (rFVIIa) or are willing to switch to
             rFVIIa as primary bypassing agent for the treatment of breakthrough bleeds

          -  Adequate hematologic function, defined as platelet count >/= 100,000 per microliters
             (mcL) and hemoglobin >/=8 grams per deciliter (g/dL) at screening

          -  Adequate hepatic and renal function

        Exclusion Criteria:

          -  Inherited or acquired bleeding disorder other than hemophilia A

          -  Ongoing (or plan to receive during the study) immune tolerance induction (ITI) therapy
             or prophylaxis with FVIII with the exception of participants who have received a
             treatment regimen of FVIII prophylaxis with concurrent bypassing agent prophylaxis

          -  Treatment for thromboembolic disease within 12 months before Day 1 (with the exception
             of previous catheter-associated thrombosis for which antithrombotic treatment is not
             currently ongoing) or current signs of thromboembolic disease

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

          -  High risk for thrombotic microangiopathy (TMA), in the investigator's judgment

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

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

          -  Treatment with any of the following: An investigational drug to treat or reduce the
             risk of hemophilic bleeds within 5 half-lives of last drug administration before Day
             1; A non-hemophilia-related investigational drug within the last 30 days or 5
             half-lives before Day 1, whichever is longer; An investigational drug concurrently

          -  Any serious medical condition, treatment, or abnormality in clinical laboratory tests
             that, in the investigator's judgment, precludes the participant's safe participation
             in the study
No condition on gender to be admitted to 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? 0