Clinical trial: A Phase 3, Open-label Interventional Study of an Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A (NCT04161495)

Eligibility criteria:
Inclusion criteria :

          -  Participant, male or female, must be equal to or greater than 12 years of age
             inclusive, at the time of signing the informed consent.

          -  Severe hemophilia A, defined as <1 IU/dL (<1%) endogenous FVIII activity as documented
             either by central laboratory testing at Screening or in historical medical records
             from a clinical laboratory demonstrating <1% FVIII coagulant activity (FVIII:C) or a
             documented genotype known to produce severe hemophilia A

          -  Previous treatment for hemophilia A (prophylaxis or on demand) with any recombinant
             and/or plasma-derived FVIII, or cryoprecipitate for at least 150 EDs.

          -  Current regimen includes one of the following:

               -  Prophylactic treatment regimen with a FVIII product or prophylactic emicizumab
                  therapy for at least 6 months during the previous 12 months. Appropriate washout
                  time needs to be taken into account.

               -  On-demand regimen with a FVIII product with a history of at least 12 bleeding
                  episodes in the previous 12 months or at least 6 bleeding episodes in the
                  previous 6 months prior to study enrollment.

                    -  On-demand participant is accepting to move to a prophylaxis treatment
                       regimen after 26-week on-demand period.

          -  Willingness and ability of the participant or surrogate (a caregiver or a family
             member ≥18 years of age) to complete training in the use of the study electronic
             Patient Diary (ePD) and to use the ePD throughout the study.

          -  Ability of the participant or his or her legally authorized representative (eg.,
             parent or legal guardian) to understand the purpose and risks of the study, willing
             and able to comply with study requirements and provide signed and dated informed
             consent or assent (as applicable) and authorization to use protected health
             information in accordance with national and local participant privacy regulations.

        Exclusion criteria:

          -  Clinically significant liver disease.

          -  Serious active bacterial or viral infection (other than chronic hepatitis or HIV)
             present within 30 days of Screening.

          -  Other known coagulation disorder(s) in addition to hemophilia A.

          -  History of hypersensitivity or anaphylaxis associated with any FVIII product

          -  Positive inhibitor results, defined as ≥0.6 BU/mL at Screening. History of a positive
             inhibitor test defined as ≥0.6 BU/mL. Family history of inhibitors will not exclude
             the participant.

          -  Use of Emicizumab within the 20 weeks prior to Screening

          -  Major surgery within 8 weeks prior to Screening.

        The above information is not intended to contain all considerations relevant to a patient's
        potential participation in a clinical trial.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 12 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? 0