BAX 802 in CHA With Inhibitors

NCT02895945 | Terminated Phase 3 Results DMC FDA Drug

• 2 other identifiers: 2415022015-005521-39
• Study Type: interventional
• Target: 8
• Locations: 12 countries
• Sites: 24

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.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

• Percentage of Surgeries With a "Good" or "Excellent" Response as Measured by the Global Hemostatic Efficacy Assessment (GHEA) Score

  GHEA score consisted of 3 individual rating scales: (1) Intra-operative Efficacy Assessment Scale, (2) Post-operative Efficacy Assessment Scale, and (3) Overall Peri-operative Efficacy Assessment Scale. Scales 1 and 2 was performed by the operating surgeon on Day 1, and Scale 3 was performed by the investigator on Day 14. Each rating scale was based on 4 points scale ranging from: 3 (Excellent), 2 (Good), 1 (Fair), and 0 (None). Total score ranged from 0 to 9, where scores evaluated as: excellent (7 to 9), good (5 to 7), fair (3 to 4), and none (0 to 2). The scores of 3 individual ratings scales were added together to form a GHEA score. For a GHEA score of 7 to be rated "excellent" with no individual assessment scores less than (\<) 2 and at least 1 assessment score equal to (=) 3; otherwise a score of 7 was rated "good". Percentage of Surgeries With a "Good" or "Excellent" response as measured by the GHEA score were reported.

  Day 1 up to discharge or Day 14 (whichever was earlier)

Secondary Outcomes (17)

• Actual Blood Loss, Estimated Volume of Expected Average Blood Loss and Expected Maximum Blood Loss During Intra-operative, Post-operative and Peri-operative Period

  Intra-operative: up to completion of surgery (Day 1), Post-operative: at 24 hours post-surgery, and Peri-operative: at discharge or Day 14 (whichever was earlier)

• Ratio of Actual Blood Loss and Estimated Volume of Expected Average Blood Loss During Intra-operative, Post-operative and Peri-operative Period

  Intra-operative: up to completion of surgery (Day 1), Post-operative: at 24 hours post-surgery, and Peri-operative: at discharge or Day 14 (whichever was earlier)

• Ratio of Actual Blood Loss and Expected Maximum Blood Loss During Intra-operative, Post-operative and Peri-operative Period

  Intra-operative: up to completion of surgery (Day 1), Post-operative: at 24 hours post-surgery, and Peri-operative: at discharge or Day 14 (whichever was earlier)

• Percentage of Major Surgeries With Good or Excellent Hemostatic Score

  Day 1 up to discharge or Day 14 (whichever was earlier)

• Average Daily Weight-adjusted Dose of BAX 802 Per Participant During Pre-operative, Intra-operative and Post-operative Period

  Pre-operative: before surgery, Intra-operative: up to completion of surgery (Day 1), Post-operative: from completion of surgical procedure till discharge or 14 days post surgery (whichever was earlier)

Study Arms (1)

BAX 802 in Surgery

EXPERIMENTAL

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

Biological: Antihemophilic Factor (Recombinant), Porcine Sequence (BAX 802)

Interventions

Antihemophilic Factor (Recombinant), Porcine Sequence (BAX 802) BIOLOGICAL

In case of major surgery, FVIII target level is ≥80% for major surgeries/ procedures and ≥50% for minor surgeries/ procedures.

Also known as: BAX 802, recombinant porcine factor VIII, Obizur, rpFVIII

BAX 802 in Surgery

Eligibility Criteria

• Age: 12 Years - 75 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant requires a major or minor elective surgical, dental or other invasive procedure
• Participant is male and ≥ 12 to ≤ 75 years old at the time of screening
• Participant has provided signed informed consent (and assent for adolescent participants, as applicable) in accordance with local regulatory requirements
• 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
• Participant is not currently receiving or has recently received (\< 30 days) immune tolerance induction (ITI) therapy
• Participant has a Karnofsky performance score of ≥ 60 at screening
• Participant is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm\^3 at screening
• 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.
• Participant is willing and able to comply with the requirements of the protocol.

You may not qualify if:

• The participant requires emergency surgery
• 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)
• Clinically symptomatic renal disease (serum creatinine \> 2.0 mg/dL), as confirmed by central laboratory at screening
• Anti-porcine factor VIII (pFVIII) inhibitor \> 10 BU prior to surgery
• Platelet count \< 100,000/μL at screening
• Participant has another active coagulation disorder, other than hemophilia A, as per the medical history
• 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
• Known hypersensitivity to recombinant porcine factor VIII (rpFVIII), or hamster or murine proteins
• Participant has an ongoing or recent (within 3 months of screening) thrombo-embolic disease, fibrinolysis or disseminated intravascular coagulation (DIC)
• 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
• Participant is unable to tolerate quantity of blood to be drawn for protocol procedures
• Participant is a family member or employee of the Investigator.

Sponsors & Collaborators

• Baxalta now part of Shire: lead
• Baxalta Innovations GmbH, now part of Shire: collaborator

Study Sites (24)

Bleeding and Clotting Disorders Institute

Peoria, Illinois, 61615, United States

Location

Wake Forest University Baptist Medical Center

Winston-Salem, North Carolina, 27157, United States

Location

Case Western Reserve University

Cleveland, Ohio, 44106-7284, United States

Location

UMHAT 'Tsaritsa Yoanna - ISUL', EAD

Sofia, 1527, Bulgaria

Location

UMHATEM 'N.I. Pirogov', EAD

Sofia, 1606, Bulgaria

Location

Hopital Maisonneuve-Rosemont d/b/a CIUSSS de l'Est-de-l'Île-de-Montréal

Montreal, Quebec, H1T 2M4, Canada

Location

Universitaetsklinikum Bonn AoeR

Bonn, 53127, Germany

Location

Zentrum für Hämostaseologie, Universitätsklinikum Leipzig AöR

Leipzig, 04103, Germany

Location

Presidio Ospedaliero di Castelfranco Veneto

Castelfranco Veneto, Treviso, 31033, Italy

Location

Azienda Ospedaliera Universitaria Careggi

Florence, 50134, Italy

Location

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

Azienda Ospedaliera di Padova

Padua, 35128, Italy

Location

UMC Utrecht

Utrecht, 3584 CX, Netherlands

Location

Oslo Universitetssykehus - Rikshospitalet

Oslo, 0372, Norway

Location

Instytut Hematologii i Transfuzjologii-1-1Y7-1347

Warsaw, 02-776, Poland

Location

FSBI of Science "Kirov Scientific and Research Institute of Hematology and Blood Transfusion of FMBA.

Kirov, 610027, Russia

Location

Bleeding Disorders Unit and Clinical Haematology Service at Charlotte Maxeke JHB Academic Hospital

Johannesburg, 2193, South Africa

Location

Hospital Universitari i Politecnic La Fe

Valencia, Valencia, 46026, Spain

Location

Complejo Hospitalario Universitario A Coruña

A Coruña, 15006, Spain

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Ankara University Medical Faculty

Ankara, 6590, Turkey (Türkiye)

Location

Ege University Medical Faculty

Izmir, 35040, Turkey (Türkiye)

Location

Ege University Medical Faculty

Izmir, 35100, Turkey (Türkiye)

Location

Kocaeli University Medical Faculty

Kocaeli, 41300, Turkey (Türkiye)

Location

Related Links

• To obtain more information on the study, click here/on this link

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Limitations and Caveats

Study was discontinued as Takeda determined that the benefit/risk profile did not support continuation of the surgery study for this specific Congenital Hemophilia A with Inhibitors participant population.

Results Point of Contact

• Title: Study Director
• Organization: Takeda

TrialDisclosures@takeda.com

+1-877-825-3327

Study Officials

• Study Director

  Shire

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 15, 2016
• First Posted: September 12, 2016
• Study Start: December 22, 2016
• Primary Completion: January 22, 2021
• Study Completion: January 22, 2021
• Last Updated: October 20, 2021
• Results First Posted: October 20, 2021

Record last verified: 2021-10

Data Sharing

• IPD Sharing: Will not share

Locations

ES (3); NO (1); CA (1); DE (2); ZA (1); US (3); RU (1); BU (2); IT (4); NL (1); TU (4); PL (1)