Efficacy and Safety of Turoctocog Alfa Pegol (N8-GP) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A (pathfinder10)
Condition: Haemophilia A
MeSH terms: Hemophilia A
Intervention: turoctocog alfa pegol (N8-GP)
Intervention description: N8-GP will be injected into a vein (intravenous injections) every 4 days in at least 28 weeks
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 36.0
Sex: Male
Minimum age: 12 Years
Healthy volunteers: No

Summary:

      The study investigates how well the medicine called turoctocog alfa pegol (N8-GP) works in
      previously treated Chinese patients with severe haemophilia A.

      Participants will be treated with N8-GP. This is a medicine that doctors can already
      prescribe in other countries.

      The medicine will be injected into a vein (intravenous injections) and blood samples will be
      collected.

      The study will last for about 7-8 months. Participants will have between 8 and 15 visits to
      the clinic and possibly a number of phone calls with the study doctor.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Informed consent obtained before any trial-related activities. Trial-related
             activities are any procedures that are carried out as part of the trial, including
             activities to determine suitability for the trial.

          -  Male Chinese patient with severe congenital haemophilia A with a FVIII activity below
             1% according to medical records.

          -  Aged greater than or equal to 12 years at the time of signing informed consent.

          -  History of at least 150 exposure days (EDs) to other FVIII products.

          -  The patient and/or caregiver is capable of assessing a bleeding episode, keeping a
             diary, performing home treatment of bleeding episodes and otherwise following the
             trial procedures at the discretion of the investigator.

        Exclusion Criteria:

          -  Known or suspected hypersensitivity to trial product or related products.

          -  Previous participation in this trial. Participation is defined as signed informed
             consent.

          -  Participation in any clinical trial of an approved or non-approved investigational
             medicinal product within 5 half-lives or 30 days from screening, whichever is longer.

          -  Known history of FVIII inhibitors based on existing medical records, laboratory report
             reviews and patient and/or caregiver interviews.

          -  Current FVIII inhibitors greater than or equal to 0.6 BU.

          -  Congenital or acquired coagulation disorder other than haemophilia According to
             medical records.

          -  HIV positive, defined by medical records, with CD4+ count less than or equal 200/L and
             a viral load greater than 200 particles/μl or greater than 400000 copies/mL within 6
             months of the trial entry. If the data are not available in medical records within
             last 6 months, then the test must be performed at screening visit.

          -  Previous significant thromboembolic events (e.g. myocardial infarction,
             cerebrovascular disease or deep venous thrombosis) as defined by available medical
             records.

          -  Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine
             aminotransferase (ALT) greater than 3 times limit of normal combined with total
             bilirubin greater than 1.5 times the upper limit of normal at screening, as defined by
             central laboratory

          -  Renal impairment defined as estimated glomerular filtration rate (eGFR) below or equal
             to 30 mL/min/1.73 m^2 for serum creatinine measured at screening, as defined by
             central laboratory.

          -  Platelet count below 50×109/L at screening based on central laboratory values at
             screening.

          -  Ongoing immune modulating or chemotherapeutic medication.

          -  Any disorder, except for conditions associated with haemophilia A, which in the
             investigator's opinion might jeopardise the patient's safety or compliance with the
             protocol.

          -  Mental incapacity, unwillingness or language barriers precluding adequate
             understanding or cooperation.