Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

BAX 802 in CHA With Inhibitors

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02895945
Recruitment Status : Recruiting
First Posted : September 12, 2016
Last Update Posted : April 8, 2019
Sponsor:
Collaborator:
Baxalta Innovations GmbH, now part of Shire
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

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.

Condition or disease Intervention/treatment Phase
Hemophilia A Biological: Antihemophilic Factor (Recombinant), Porcine Sequence (BAX802) Phase 3

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : May 10, 2017
Estimated Primary Completion Date : April 2, 2021
Estimated Study Completion Date : April 16, 2021


Arm Intervention/treatment
Experimental: BAX802 in Surgery
Participants who are undergoing major or minor elective surgical, dental, or other invasive procedures.
Biological: Antihemophilic Factor (Recombinant), Porcine Sequence (BAX802)
In case of major surgery, FVIII target level is ≥80% for major surgeries/ procedures and ≥50% for minor surgeries/ procedures.
Other Names:
  • BAX 802
  • BAX802
  • recombinant porcine factor VIII
  • Obizur
  • rpFVIII




Primary Outcome Measures :
  1. Global Hemostatic Efficacy Assessment score (GHEA)- composed of 3 individual ratings: Day 0 [ Time Frame: Day 0 ]
    GHEA=Sum of 1-3 ratings. Excellent: 7-9 (no category <2) Good: 5-7 (no category <1) Fair: 3-4 (no category <1) None 0-2 (≥ category 0). a. Intra operative hemostatic efficacy (Excellent=3: Good=2: Fair=1: None =0). b. Hemostatic efficacy postoperative Day 1 (Excellent=3: Good=2: Fair=1: None =0). c. Overall perioperative hemostatic efficacy at discharge or within 24 to 72 hours after last perioperative treatment dose of BAX 802 (whichever is earlier) (Excellent=3: Good=2: Fair=1: None =0)

  2. Global Hemostatic Efficacy Assessment score (GHEA)- composed of 3 individual ratings: Day 1 [ Time Frame: Day 1 ]
    GHEA=Sum of 1-3 ratings. Excellent: 7-9 (no category <2) Good: 5-7 (no category <1) Fair: 3-4 (no category <1) None 0-2 (≥ category 0). a. Intra operative hemostatic efficacy (Excellent=3: Good=2: Fair=1: None =0). b. Hemostatic efficacy postoperative Day 1 (Excellent=3: Good=2: Fair=1: None =0). c. Overall perioperative hemostatic efficacy at discharge or within 24 to 72 hours after last perioperative treatment dose of BAX 802 (whichever is earlier) (Excellent=3: Good=2: Fair=1: None =0)

  3. Global Hemostatic Efficacy Assessment score (GHEA)- composed of 3 individual ratings: At discharge or within 24 to 72 hours after last perioperative treatment dose of BAX 802 (whichever is earlier) [ Time Frame: At discharge or within 24 to 72 hours after last perioperative treatment dose of BAX 802 (whichever is earlier) ]
    GHEA=Sum of 1-3 ratings. Excellent: 7-9 (no category <2) Good: 5-7 (no category <1) Fair: 3-4 (no category <1) None 0-2 (≥ category 0). a. Intra operative hemostatic efficacy (Excellent=3: Good=2: Fair=1: None =0). b. Hemostatic efficacy postoperative Day 1 (Excellent=3: Good=2: Fair=1: None =0). c. Overall perioperative hemostatic efficacy at discharge or within 24 to 72 hours after last perioperative treatment dose of BAX 802 (whichever is earlier) (Excellent=3: Good=2: Fair=1: None =0)


Secondary Outcome Measures :
  1. Intra- and post-operative blood loss compared to the estimated volume of expected average blood loss in a comparable healthy individual [ Time Frame: End of surgery; approximately 24 hours after surgery; and at discharge or 24 to 72 hours after the last perioperative treatment dose of BAX 802 (whichever is earlier) ]
    Predicted preoperatively by the investigator/surgeon

  2. Intra- and post-operative blood loss compared to the expected maximum blood loss in a comparable healthy individual [ Time Frame: End of surgery; approximately 24 hours after surgery; and at discharge or 24 to 72 hours after the last perioperative treatment dose of BAX 802 (whichever is earlier) ]
    Predicted preoperatively by the investigator/surgeon

  3. Major surgeries with good or excellent hemostatic score [ Time Frame: Day 0 (day of surgery) thru Day 14 or discharge (whichever is earlier) ]
    Proportion of major surgeries with good or excellent hemostatic score

  4. Daily weight-adjusted administration of BAX 802 [ Time Frame: Day 0 (postoperative) thru Day 14 or discharge (whichever is earlier) ]
    Daily weight-adjusted administration of BAX 802 per participant

  5. Total weight-adjusted administration of BAX 802 [ Time Frame: Day 0 (postoperative) thru Day 14 or discharge (whichever is earlier) ]
    Total weight-adjusted administration of BAX 802 per participant

  6. Volume of blood products transfused [ Time Frame: From initiation of the surgery until Day 14 or discharge (whichever is earlier) ]
    Amount of blood products (e.g., whole blood, red blood cells, platelets, and plasma) transfused

  7. Development of, and changes to, the titer of inhibitory and binding antibodies to porcine factor VIII (pFVIII) [ Time Frame: >14 days prior to Day 0 (surgery day); 7-14 days after last perioperative treatment dose of BAX 802; and day 42 after last perioperative treatment dose of BAX 802 ]
    Development of, and changes to, the titer of inhibitory and binding antibodies (IgG and IgM) to porcine factor VIII (pFVIII)

  8. Development of, and changes to, the titer of inhibitory and binding antibodies to human factor VIII (hFVIII) [ Time Frame: >14 days prior to Day 0 (surgery day); 7-14 days after last perioperative treatment dose of BAX 802; and day 42 after last perioperative treatment dose of BAX 802 ]
    Development of, and changes to, the titer of inhibitory and binding antibodies (IgG and IgM) to human factor VIII (hFVIII)

  9. Binding antibodies to baby hamster kidney (BHK) proteins [ Time Frame: ≤45 days prior to Day 0 (surgery day); 7-14 days after last perioperative treatment dose of BAX 802; and day 42 after last perioperative treatment dose of BAX 802 ]
    Development of binding antibodies to baby hamster kidney (BHK) proteins

  10. Thrombo-embolic events [ Time Frame: Day 0 (surgery day) through end of study visit (day 42 after discharge) ]
    Occurrence of thrombo-embolic events

  11. Incidence of severe allergic reactions [ Time Frame: Day 0 (surgery day) through end of study visit (day 42 after discharge) ]
    Incidence of severe allergic reactions (eg, anaphylaxis)

  12. Other investigational product (IP)-related adverse events (AEs) [ Time Frame: From Day 0 (day of surgery) through Day 42 post-surgery (End of Study Visit) ]
    Incidence of other investigational product (IP)-related adverse events (AEs)

  13. Clinically significant changes in vital signs [ Time Frame: Within 30 minutes pre, and 30 minutes post-dose ]
    Incidence of clinically significant changes in vital signs

  14. Clinically significant changes in routine laboratory parameters - hematology labs [ Time Frame: Screening visit (up to 45 days prior to day of surgery (Day 0)) through End of Study Visit (Day 42 post-surgery) ]
    Incidence of clinically significant changes in routine laboratory parameters - hematology labs

  15. Clinically significant changes in routine laboratory parameters - clinical chemistry [ Time Frame: Screening visit (up to 45 days prior to day of surgery (Day 0)) through End of Study Visit (Day 42 post-surgery) ]
    Incidence of clinically significant changes in routine laboratory parameters - clinical chemistry



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02895945


Contacts
Layout table for location contacts
Contact: Michael Krammer, MSc +43 1 20100 247 1372 michael.krammer@shire.com
Contact: Shire Contact +1 866 842 5335 ClinicalTransparency@shire.com

Locations
Layout table for location information
United States, Illinois
Bleeding and Clotting Disorders Institute Recruiting
Peoria, Illinois, United States, 61615
Canada, Quebec
Hopital Maisonneuve-Rosemont d/b/a CIUSSS de l'Est-de-l'Île-de-Montréal Recruiting
Montréal, Quebec, Canada, H1T 2M4
Germany
Zentrum für Hämostaseologie, Universitätsklinikum Leipzig AöR Recruiting
Leipzig, Germany, 04103
Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico-1-AMF7BT Recruiting
Milano, Italy, 20122
Netherlands
UMC Utrecht-1-1YA-3214 Recruiting
Utrecht, Netherlands, 3584 CX
Poland
Instytut Hematologii i Transfuzjologii-1-1Y7-1347 Recruiting
Warszawa, Poland, 02-776
South Africa
Bleeding Disorders Unit and Clinical Haematology Service at Charlotte Maxeke JHB Academic Hospital Recruiting
Johannesburg, South Africa, 2194
Principal Investigator: Johnny Mahlanghu, MBBCh         
Spain
Hospital Universitari i Politecnic La Fe Recruiting
Valencia, Comunidad Valenciana, Spain, 46026
Complejo Hospitalario Universitario A Coruña-1-2WKDXY Recruiting
La Coruña, Spain, 15006
Hospital Universitario La Paz-1-IKVQAW Recruiting
Madrid, Spain, 28046
United Kingdom
Royal Free Hospital Recruiting
London, Greater London, United Kingdom, NW3 2QG
St Thomas' Hospital-1-M6DQZK Recruiting
London, United Kingdom, SE1 7EH
Manchester Royal Infirmary-1-1PA3SZ Recruiting
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Baxalta now part of Shire
Baxalta Innovations GmbH, now part of Shire
Investigators
Layout table for investigator information
Study Director: Study Director Shire

Layout table for additonal information
Responsible Party: Baxalta now part of Shire
ClinicalTrials.gov Identifier: NCT02895945     History of Changes
Other Study ID Numbers: 241502
2015-005521-39 ( EudraCT Number )
First Posted: September 12, 2016    Key Record Dates
Last Update Posted: April 8, 2019
Last Verified: April 2019

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Layout table for MeSH terms
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants