BAX 802 in CHA With Inhibitors

Brief Title

BAX 802 in CHA With Inhibitors

Official Title

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

Brief Summary

      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.

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Global Hemostatic Efficacy Assessment score (GHEA)- composed of 3 individual ratings: Day 0

Secondary Outcome

 Intra- and post-operative blood loss compared to the estimated volume of expected average blood loss in a comparable healthy individual

Condition

Hemophilia A

Intervention

Antihemophilic Factor (Recombinant), Porcine Sequence (BAX802)

Study Arms / Comparison Groups

 BAX802 in Surgery

Description:  Participants who are undergoing major or minor elective surgical, dental, or other invasive procedures.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Biological

Estimated Enrollment

Start Date

May 10, 2017

Completion Date

April 16, 2021

Primary Completion Date

April 2, 2021

Eligibility Criteria

        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.

Gender

Male

Ages

18 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

Study Director, ,

Location Countries

Bulgaria

Location Countries

Bulgaria

Administrative Informations

NCT ID

NCT02895945

Organization ID

Secondary IDs

2015-005521-39

Responsible Party

Sponsor

Study Sponsor

Baxalta now part of Shire

Collaborators

 Baxalta Innovations GmbH, now part of Shire

Study Sponsor

Study Director, Study Director, Shire

Verification Date

January 2020