Clinical trial: Hemlibra in Mild Hemophilia A (NCT04567511)

Eligibility criteria:
Inclusion Criteria:

          -  Signed informed consent form from the subject, parent or guardian

          -  Male sex

          -  Diagnosis of mild congenital hemophilia A (baseline FVIII level of >5% to 30%) without
             a current FVIII inhibitor or a history of FVIII inhibitor

          -  Any number of FVIII exposure days, including PUPs

          -  BMI <30

          -  Age ≥5 years to ≤45 years

          -  Medical documentation of bleeding events, outcomes and hemostatic product usage for 12
             months prior to study enrollment

          -  Willingness and ability to comply with scheduled visits, treatment plans, laboratory
             tests, and other study procedures, including the health-related questionnaires,
             activity tracking, and bleed diaries, using systems provided during the study

          -  Willingness to undergo a Stimate/DDAVP challenge (only if the subject reports no
             adverse event associated with prior Stimate [DDAVP/desmopressin acetate] use);
             Stimate/DDAVP challenge will not be performed if the patient has a documented history
             of lack of response as defined by an increase of FVIII < 2 times baseline level

          -  Adequate hepatic function, defined as total bilirubin ≤1.5 × age-adapted upper limit
             of normal (ULN) (excluding Gilbert's syndrome) and both AST and ALT ≤3 × age-adapted
             ULN at the time of screening, and no clinical signs or known laboratory/radiographic
             evidence consistent with cirrhosis

          -  Adequate hematologic function, defined as a platelet count ≥100,000/μL and a PT≤1.5
             times the ULN at the time of screening

          -  Adequate renal function, defined as serum creatinine ≤2.5 × age-adapted ULN and
             creatinine clearance ≥30 mL/min by Cockcroft-Gault formula

        Exclusion Criteria:

          -  Inherited or acquired bleeding disorder other than mild congenital hemophilia A
             (baseline FVIII level of >5% to 30%)

          -  Any bleeding disorder other than or in addition to mild hemophilia A

          -  Current or prior inhibitor to FVIII (any titer)

          -  Female sex

          -  History of CVD, risk of CVD by the ASCVD risk estimator (defined as a subject having
             >20% risk of a cardiovascular event within the next 10 years if the subject is ≥20
             years of age) and/or a history of ischemic heart disease [ICD codes 120-125]

          -  High risk for TMA (eg, have a previous medical or family history of TMA), in the Study
             Investigator's judgment

          -  History of illicit drug or alcohol abuse by report or in the Study Investigator's
             judgment

          -  Previous (within the last 12 months) or current treatment for thromboembolic disease
             (with the exception of previous catheter-associated thrombosis for which
             anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease

          -  Other conditions (eg, certain autoimmune diseases) that may currently increase the
             risk of bleeding or thrombosis

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

          -  Known HIV infection with CD4 counts <200 cells/μL. HIV infection with CD4 counts ≥200
             cells/μL permitted

          -  Use of systemic immunomodulators (eg, interferon) at enrollment or planned use during
             the study, with the exception of anti-retroviral therapy

          -  Concomitant disease, condition, significant abnormality on screening evaluations or
             laboratory tests, or treatment that could interfere with the conduct of the study, or
             that would, in the opinion of the Study Investigator, pose an additional unacceptable
             risk in administering study drug to the patient

          -  Receipt of any of the following:

               -  Hemlibra (emicizumab) in a prior investigational study

               -  An investigational drug to treat or reduce the risk of hemophilic bleeds within 5
                  half-lives of last drug administration

               -  A non-hemophilia-related investigational drug within last 30 days or 5
                  half-lives, whichever is shorter

               -  Any other investigational drug currently being administered or planned to be
                  administered

          -  Inability to comply with the study protocol in the opinion of the Study Investigator
Male
No healthy subjects accepted to join the trial.
Subject must be at least 5 Years old.
Subject must be at most 45 Years

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