Clinical trial: Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A (NCT04644575)

Eligibility criteria:
Inclusion criteria :

        For participants rolling over into Arm A

          -  Participants who have completed the studies EFC16923, EFC16925, Arm B or Arm C of the
             current study, or any other potential BIVV001 study.

          -  Male or Female For participants new to BIVV001 (Arm B and C)

          -  Participants who have 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 or 50 EDs for
             participants aged <6 years.

          -  Platelet count ≥100 000 cells/μL at screening.

          -  A participant known to be human immunodeficiency virus (HIV) antibody positive, either
             previously documented or identified from screening assessments, must have the
             following results prior to enrollment: CD4 lymphocyte count >200 cells/mm³ and viral
             load of <400 000 copies/mL

          -  Male

          -  Only for Arm B: Chinese participants

          -  Only for Arm C: planned major surgery within 6 months after Day 1.

        Exclusion criteria:

        For participants rolling over into Arm A

          -  Positive inhibitor result, defined as ≥0.6 Bethesda units (BU)/mL.

          -  Participation in another study. For participants new to BIVV001 (Arm B and Arm C)

          -  Any concurrent clinically significant liver disease that, in the opinion of the
             Investigator, would make the participant unsuitable for enrollment. This may include,
             but is not limited to cirrhosis, portal hypertension, and acute hepatitis.

          -  Serious active bacterial, fungal, 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.

          -  History of a positive inhibitor (to FVIII) test defined as ≥0.6 BU/mL, or any value
             greater than or equal to the lower sensitivity cut-off for laboratories with cut-offs
             for inhibitor detection between 0.7 and 1.0 BU/mL, or clinical signs or symptoms of
             decreased response to FVIII administrations. Family history of inhibitors will not
             exclude the participant.

          -  Positive inhibitor test (FVIII) result, defined as ≥0.6 BU/mL at screening.

          -  Treatment with acetylsalicylic acid (ASA) or antiplatelet agents that are not
             nonsteroidal anti-inflammatory drugs (NSAIDs) within 2 weeks prior to screening.

          -  Treatment with NSAIDs greater than the maximum dose specified in the regional
             prescribing information within 2 weeks prior to screening.

          -  Systemic treatment within 12 weeks prior to Screening with chemotherapy and/or other
             immunosuppressive drugs (except for the treatment of hepatitis C virus [HCV] or HIV).

          -  Emicizumab use 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.



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