BAX 802 in CHA With Inhibitors

A Phase 3, Multicenter, Open-label Study of the Efficacy and Safety of B-Domain Deleted Recombinant Porcine Factor VIII (BAX 802) in Subjects With Congenital Hemophilia A With Factor VIII Inhibitors Undergoing Surgical or Other Invasive Procedures

The purpose of this study is to evaluate the efficacy and safety of BAX 802 in males with congenital hemophilia A (CHA) with inhibitors who are undergoing major or minor elective surgical, dental, or other invasive procedures.

Inclusion Criteria

1. Participant requires a major or minor elective surgical, dental or other invasive procedure
2. Participant is male and ≥ 12 to ≤ 75 years old at the time of screening
3. Participant has provided signed informed consent (and assent for adolescent participants, as applicable) in accordance with local regulatory requirements
4. Participant has severe (factor VIII (FVIII) level < 1%) or moderately severe (FVIII level ≤ 2%) congenital hemophilia A (CHA) with inhibitors to human factor VIII (hFVIII) of ≥ 0.6 Bethesda units (BU), as tested at screening at the central laboratory
5. Participant is not currently receiving or has recently received (< 30 days) immune tolerance induction (ITI) therapy
6. Participant has a Karnofsky performance score of ≥ 60 at screening
7. Participant is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm^3 at screening
8. Participant is hepatitis C virus negative (HCV-) by antibody or polymerase chain reaction (PCR) testing; or HCV+ with chronic stable hepatitis disease. Positive serologies will be confirmed by PCR testing.
9. Participant is willing and able to comply with the requirements of the protocol.

Exclusion Criteria

1. The participant requires emergency surgery
2. Severe chronic liver dysfunction or disease (e.g., ≥ 5 × upper limit of normal [ULN] alanine aminotransferase [ALT], as confirmed by central laboratory at screening or a documented prothrombin time/international normalized ratio [PT/INR] > 1.5)
3. Clinically symptomatic renal disease (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening
4. Anti-porcine factor VIII (pFVIII) inhibitor > 10 BU prior to surgery
5. Platelet count < 100,000/μL at screening
6. Participant has another active coagulation disorder, other than hemophilia A, as per the medical history
7. Planned use of α-interferon with or without ribavirin for HCV infected patients or planned use of a protease inhibitor for HIV infected patients. Patients currently taking any of these medications for ≥ 30 days are eligible
8. Known hypersensitivity to recombinant porcine factor VIII (rpFVIII), or hamster or murine proteins
9. Participant has an ongoing or recent (within 3 months of screening) thrombo-embolic disease, fibrinolysis or disseminated intravascular coagulation (DIC)
10. Participant has been exposed to an IP within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an investigational product (IP) or investigational device during the course of this study
11. Participant is unable to tolerate quantity of blood to be drawn for protocol procedures
12. Participant is a family member or employee of the Investigator.