Clinical trial: Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle". (NCT03588299)

Eligibility criteria:
Inclusion Criteria:

          -  Males age 18 years or older.

          -  Confirmed diagnosis of hemophilia A as evidenced by their medical history with plasma
             FVIII activity levels < 1% of normal or at screening.

          -  Have >150 exposure days (EDs) to FVIII concentrates (recombinant or plasma-derived).

        If on prophylaxis, are required to be willing to stop prophylactic treatment at specified
        time points throughout the study or If on-demand: should have had > 4 bleeding events in
        the last 52 weeks

        - Agree to use reliable barrier contraception.

        Exclusion Criteria:

          -  History of allergic reaction to any FVIII product.

          -  Clinically relevant findings in the physical examination considered critical by the
             treating physician, including obesity with BMI > 35 kg/m*2

          -  Current evidence of measurable inhibitor against factor VIII, as assessed by the
             central laboratory and/or prior history of inhibitors to FVIII protein.

          -  Evidence of active hepatitis B or C.

          -  Currently on antiviral therapy for hepatitis B or C.

          -  Significant underlying liver disease.

          -  Serological evidence of HIV-1 or HIV-2 with CD4 counts ≤200/mm*3; HIV+ and stable
             participants with CD4 count >200/mm*3 and undetectable viral load are eligible to
             enroll.

          -  Detectable antibodies reactive with AAVhu37capsid.

          -  Participant with another bleeding disorder that is different from hemophilia A (e.g.,
             von Willebrand disease, hemophilia B).

          -  Participated in a gene transfer trial within the last 52 weeks or in a clinical trial
             with an investigational product within the last 12 weeks.

          -  Known or suspected hypersensitivity or allergic reaction to trial product(s) or
             related FVIII products or any component of BAY2599023 (DTX201), or a contraindication
             to prednisolone
Male
No healthy subjects accepted to join the trial.
Subject must be at least 18 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? Contradiction