A Phase 3 Open-label Interventional Study of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A
Condition: Factor VIII Deficiency
MeSH terms: Hemophilia A
Intervention: efanesoctocog alfa (BIVV001)
Intervention description: Pharmaceutical form: solution for injection Route of administration: IV injection
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 159.0
Sex: All
Minimum age: 12 Years
Healthy volunteers: No

Summary:

      Primary Objective:

      - To evaluate the efficacy of BIVV001 as a prophylaxis treatment in prophylaxis treatment
      arm.

      Secondary Objectives:

        -  To evaluate the efficacy of BIVV001 as a prophylaxis treatment.

        -  To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes.

        -  To evaluate BIVV001 consumption for the prevention and treatment of bleeding episodes.

        -  To evaluate the effect of BIVV001 prophylaxis on joint health outcomes.

        -  To evaluate the effect of BIVV001 prophylaxis on Quality of Life outcomes.

        -  To evaluate the efficacy of BIVV001 for perioperative management.

        -  To evaluate the safety and tolerability of BIVV001 treatment.

        -  To assess the pharmacokinetics (PK) of BIVV001 based on the 1-stage activated partial
           thromboplastin time (aPTT) and 2-stage chromogenic coagulation factor VIII (FVIII)
           activity assays.
    

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 less than (<) 1 international units per deciliter
             (IU/dL) (<1 percent [%]) 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 exposure days.

          -  Current regimen included 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 was accepted 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 greater than or equal to [>=] 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 Bethesda unit per milliliter (BU/mL) at
             screening. History of a positive inhibitor test defined as >=0.6 BU/mL. Family history
             of inhibitors would 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 was not intended to contain all considerations relevant to a
        participant's potential participation in a clinical trial.