Clinical trial: A Pharmacokinetic and Clotting Activity Study of FVIII-PEGLip (NCT04592692)

Eligibility criteria:
Inclusion Criteria:

          -  Male adult patients aged 18 to 60 years;

          -  Severe Haemophilia A (FVIII plasma level <1IU/dL) with documented history of bleeds
             (for at least 6 months prior to enrolment);

          -  For patients without inhibitors: inhibitor titre < 0,6 Bethesda units and no medi-cal
             history of inhibitors;

          -  For patients with inhibitors: inhibitor titre ≥0,6 Bethesda units or documented
             medical history of inhibitors titre ≥0,6 Bethesda units;

          -  Adequate hematologic function, defined as platelet count ≥ 100,000/μL and hemoglobin ≥
             8 g/dL (≥ 4.97 mmol/L) at the time of screening;

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

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

          -  Patient's written informed consent, confirming his willingness to comply with the
             requirements of this protocol.

        Exclusion Criteria:

          -  Low platelet counts (<100000 / μl);

          -  Congenital or acquired bleeding defects (including acquired hemophilia) other than
             Hemophilia A;

          -  Abnormal renal function (serum creatinine concentrations greater than 1.3 mg/dL);

          -  Active hepatic disease (persistent aspartate aminotransferase [AST] or alanine
             aminotransferase [ALT] increases to greater than five times the upper limit of
             normal);

          -  A history of severe adverse reactions to blood products and/or plasma derived FVIII
             concentrates or liposomes, or PEG, or Nuwiq;

          -  A history of allergic reactions to bypassing agents;

          -  Any concomitant immunological disease (e.g. autoimmune chronic active hepati-tis,
             autoimmune thrombocytopenic purpura or Immune Thrombocytopenic Pur-pura (ITP), lupus,
             Multiple Sclerosis (MS));

          -  Patients receiving immunosuppressive treatment (excluding glucocorticoids);

          -  Patients receiving therapy with interferon;

          -  Patients receiving any immune tolerance induction (ITI) therapy at the moment of the
             screening;

          -  Any individual with known dyslipidemia disease or actively taking cholesterol lowering
             drugs for the treatment of hypercholesterolemia or hyperlipidemia (e.g., statins,
             cholesterol absorption inhibitors, bile acid sequestrates, nicotinic acid or
             fibrates);

          -  Intake of NSAIDs (except COX-2 inhibitors), acetylsalicylic acid (Aspirin) or any
             other antiplatelet agents, opioids.;

          -  Patients who have participated in another Clinical Trial (including medical device
             studies) within the past 60 days;

          -  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, according to the Investigator.

          -  For patients without inhibitors - a history of demonstrating long half-lives for
             FVIII.
Male
No healthy subjects accepted to join the trial.
Subject must be at least 18 Years old.
Subject must be at most 60 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? 1