Clinical trial: SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis (NCT04563520)

Eligibility criteria:
Inclusion Criteria:

          -  Moderately severe hemophilia A, defined as FVIII level <0.02 IU/mL in the central
             laboratory prior to development of an inhibitor

          -  Age ≥6 years of age at time of informed consent

          -  Documented on 2 occasions a high titer inhibitor (>5 BU/mL) with a 72-hour washout
             within 2 years of enrollment

          -  Parent/guardian (caregiver henceforth) or patient has provided written informed
             consent

          -  Adequate hematologic function (Hgb >8 g/dL and platelet count >100,000 µL)

          -  Adequate hepatic function (total bilirubin ≤1.5 x ULN and both AST/ALT ≤3x ULN at
             screening (excluding known Gilbert's)

          -  Adequate renal function (≤2.5 x ULN and CrCl ≥30 mL/min)

        Exclusion Criteria:

          -  Inherited or acquired bleeding disorder other than hemophilia A excluding low VWF
             (>30% VWF:RCo or VWF:GP1bm)

          -  Previous or current treatment for thromboembolic disease or signs of thromboembolic
             disease (excluding previous resolved line associated thrombosis)

          -  Conditions that may increase risk of bleeding or thrombosis

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

          -  Known HIV infection with CD4 count <200 cells/µL within 24 weeks prior to screening.
             Testing not required if <35 years of age.

          -  Use of systemic immunomodulators at enrollment or planned use during the study

          -  Participants who are at high risk for TMA (for example, have a previous medical/family
             history of TMA), in the investigator's judgment

          -  Concurrent disease, treatment, or abnormality in clinical laboratory tests that could
             interfere with the conduct of the study, may pose additional risk, or would, in the
             opinion of the investigator, preclude the participant's safe participation in and
             completion of the study

          -  Every effort will be made to include participants that are considering minor and major
             procedures over the next 2 years to capture this important data with the goal of 10
             procedures.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 6 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? 1