Clinical trial: LR769 in Congenital Hemophilia Patients With Inhibitors Undergoing Elective Surgery or Invasive Procedures (NCT02548143)

Eligibility criteria:
Inclusion Criteria:

        Each patient must meet the following criteria to be enrolled in this study:

          1. be male with a diagnosis of congenital hemophilia A or B of any severity

          2. have one of the following:

               1. a positive inhibitor test Bethesda Unit (BU) ≥5 (as confirmed at screening by the
                  institutional lab), OR

               2. a BU <5 but expected to have a high anamnestic response to FVIII or FIX, as
                  demonstrated from the patient's medical history, precluding the use of FVIII or
                  FIX products to treat or prevent bleeding, OR

               3. a BU <5 but expected to be refractory to increased dosing of FVIII or FIX, as
                  demonstrated from the patient's medical history, precluding the use of FVIII or
                  FIX products to treat or prevent bleeding episodes

          3. be ≥6 months to ≤75 years of age; different age restrictions may apply per local
             regulation and ethical considerations (enrollment of children <12 years of age will
             not begin until after review of data from the PERSEPT 2 study by the DMC)

          4. be scheduled for an elective surgical or other invasive procedure

          5. be capable of understanding and willing to comply with the conditions of the protocol
             OR in the case of a patient <18 years of age, parent(s)/legal guardian(s) must be
             capable of understanding and willing to comply with the conditions of the protocol

          6. have read, understood, and provided written Informed Consent (patient and/or
             parent(s)/legal guardian(s) if the patient is <18 years of age) or Assent, if
             applicable

        Exclusion Criteria:

        Patients who meet any of the following criteria will be excluded from the study:

          1. have any coagulation disorder other than hemophilia A or B

          2. be immunosuppressed (ie, the patient should not be receiving systemic
             immunosuppressive medication; cluster of differentiation 4 (CD4) counts at screening
             should be >200/µL)

          3. known intolerance to LR769 or any of its excipients

          4. currently receiving immune tolerance induction (ITI) therapy

          5. have a known allergy or hypersensitivity to rabbits

          6. have a platelet count<100,000/µL

          7. have received an investigational drug within 30 days of the planned first LR769
             administration , or is expected to receive such drug during participation in this
             study (with the exception of patients who are or were participating in another LR769
             study, eg, a study assessing the treatment of bleeding episodes with LR769)

          8. have a clinically relevant hepatic (aspartate aminotransferase (AST) and/or alanine
             aminotransferase (ALT) >3 times the upper limit of normal (ULN)) and/or renal
             impairment (creatinine >2 times the ULN)

          9. have a history of arterial and/or venous thromboembolic events (such as myocardial
             infarction, ischemic strokes, transient ischemic attacks, deep venous thrombosis (DVT)
             or pulmonary embolism (PE)) within 2 years prior to the planned first dose of LR769,
             uncontrolled arrhythmia, or current New York Heart Association (NYHA) functional
             classification score of stages II - IV

         10. have an active malignancy (those with non-melanoma skin cancer are allowed)

         11. have any life-threatening disease or other disease or condition which, according to
             the investigator's judgment, could imply a potential hazard to the patient, or
             interfere with the trial participation or trial outcome (eg, a history of
             non-responsiveness to bypassing products)

         12. be using aspirin, NSAIDS, herbs, natural medications, or other drugs with platelet
             inhibitory properties within one week prior to surgery and for the duration of
             treatment with LR769

         13. have active gastric or duodenal ulcer disease
Male
No healthy subjects accepted to join the trial.
Subject must be at least 6 Months old.
Subject must be at most 75 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