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    Summary
    EudraCT Number:2015-005521-39
    Sponsor's Protocol Code Number:241502
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2017-01-17
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2015-005521-39
    A.3Full title of the trial
    A Phase 3, Multicenter, Single-arm, 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
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of the Efficacy and Safety of Drug BAX 802 in Subjects with Congenital Hemophilia A with Factor VIII Inhibitors Undergoing Surgical or Other Invasive Procedures
    A.3.2Name or abbreviated title of the trial where available
    BAX802 in Congenital Hemophilia A with Inhibitors
    A.4.1Sponsor's protocol code number241502
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation PlanP/381/2017
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBaxalta Innovations GmbH
    B.1.3.4CountryAustria
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportBaxalta Innovations GmbH
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBaxalta Innovation GmbH
    B.5.2Functional name of contact pointClinical Development
    B.5.3 Address:
    B.5.3.1Street AddressIndustriestrasse 67
    B.5.3.2Town/ cityVienna`
    B.5.3.3Post codeA-1221
    B.5.3.4CountryAustria
    B.5.4Telephone number+43120100 2472930
    B.5.5Fax number+43120100 2475733
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name OBIZUR
    D.2.1.1.2Name of the Marketing Authorisation holderBaxalta Innovations GmbH
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameObizur
    D.3.2Product code BAX802
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSusoctocog alfa
    D.3.9.2Current sponsor codeBAX802
    D.3.9.3Other descriptive nameSUSOCTOCOG ALFA
    D.3.9.4EV Substance CodeSUB166390
    D.3.10 Strength
    D.3.10.1Concentration unit U unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Congenital Haemophilia A with Factor VIII Inhibitors
    E.1.1.1Medical condition in easily understood language
    Congenital Haemophilia A with Factor VIII Inhibitors
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10053751
    E.1.2Term Hemophilia A with anti factor VIII
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to evaluate the perioperative hemostatic efficacy of BAX 802 in male subjects with CHA with inhibitors to hFVIII undergoing major or minor elective surgical, dental, or other invasive procedures as determined by the Global Hemostatic Efficacy Assessment (GHEA) score.
    E.2.2Secondary objectives of the trial
    To determine:
    1. the safety of BAX 802 in the perioperative setting by assessing
    2. The development of and change in titer of anti-porcine FVIII (pFVIII) and anti-hFVIII antibodies (binding and neutralizing), and development of binding antibodies to baby hamster kidney (BHK)
    3. The occurrence of thrombo-embolic events and/or allergic reactions to
    BAX 802.
    4. The occurrence of adverse events (AEs) related to BAX 802
    5. The occurrence of clinically significant changes in vital signs and routine
    laboratory parameters related to BAX 802.
    To determine the intraoperative, postoperative, and overall
    perioperative blood loss, compared to the estimated volume of expected
    average and maximum blood loss in a comparable healthy individual as
    predicted preoperatively by the Investigator/surgeon
    3. To determine the proportion of major surgeries with good or excellent
    hemostatic score
    4. To determine the daily and total weight-adjusted administration of
    BAX 802 per subject
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Subject requires elective , dental or other invasive procedures
    2. Subject is male and ≥ 12 to ≤ 75 years old at the time of screening
    2. Subject has provided signed informed consent (and assent for
    adolescent subjects, as applicable) in accordance with local regulatory
    requirements
    4. Subject has severe (FVIII level < 1%) or moderately severe (FVIII
    level ≤ 2%) CHA with inhibitors to hFVIII of ≥ 0.6 BU, as tested at
    screening at the central laboratory
    5. Subject is not currently receiving or has recently received (< 30 days)
    ITI therapy
    6. Subject has a Karnofsky performance score of ≥ 60 at screening
    7. Subject is human immunodeficiency virus negative (HIV-); or HIV+
    with stable disease and CD4+ count ≥ 200 cells/mm3 at screening
    8. Subject 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. Subject is willing and able to comply with the requirements of the
    protocol.
    .
    E.4Principal exclusion criteria
    1.1. The subject requires emergency surgery
    2. Severe chronic liver dysfunction or disease (e.g., ≥ 5 x 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-pFVIII inhibitor > 10 BU prior to surgery
    5. Platelet count < 100,000/μL at screening
    6. Subject has another active coagulation disorder other than
    haemophilia A, as per the medical history
    7. Planned use of α-interferon with or without ribavarin 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 rpFVIII, or hamster or murine proteins
    9. Subject has an ongoing or recent (within 3 months of screening)
    thrombo-embolic disease, fibrinolysis or disseminated intravascular
    coagulation (DIC)
    10. Subject has been exposed to an IP within 30 days prior to enrollment
    or is scheduled to participate in another clinical study involving an IP or
    investigational device during the course of this study
    11. Subject is unable to tolerate quantity of blood to be drawn for
    protocol procedures
    12. Subject is a family member or employee of the investigator.
    E.5 End points
    E.5.1Primary end point(s)
    The primary outcome measure is the proportion of all surgical, dental, or
    other invasive procedures with a "good" or "excellent" response as
    measured by GHEA score which is composed of 3 individual ratings:
    -GHEA1: Assessment of intraoperative (Day 0) hemostatic efficacy of
    BAX 802 performed by the operating surgeon at the end of surgery.
    -GHEA2: Assessment of postoperative hemostatic efficacy of BAX 802 at
    postoperative Day 1 (approximately 24 hours [+/- 6 hours] postsurgery)
    performed by the operating surgeon. Note: If a patient is
    discharged <24 hours following surgery, then the GHEA2 hemostatic
    efficacy assessment will require a return visit the following day.)
    -GHEA3: Assessment of overall perioperative hemostatic efficacy of BAX802 at the GHEA3 Visit (discharge or within 24 to 72 hours after the last
    perioperative treatment dose of BAX 802 [whichever is earlier]),
    performed by the investigator, and where possible, also by the operating
    surgeon. Assessment by both is strongly recommended. In cases where
    there are differing assessments, the Investigator's
    assessment will be used.
    The scores of each of the 3 individual ratings (GHEA1, GHEA2, and
    GHEA3) described above, will be added together to form a GHEA score.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Varying times as defined by the protocol.
    E.5.2Secondary end point(s)
    Efficacy
    1. 1. Intra- and post-operative blood loss compared to the estimated
    volume of expected average blood loss and expected maximum blood
    loss in a comparable healthy individual with similar demographic
    characteristics as predicted preoperatively by the investigator/surgeon
    at the following time points:
    • Intraoperative, from start until the end of surgery
    • Postoperative Day 1, from end of surgery to approximately 24 hours
    (+/- 6 hours) after surgery
    • Overall perioperative at discharge or 24 to 72 hours after the last
    perioperative treatment dose of BAX 802 (whichever is earlier)
    2. Proportion of major surgeries with good or excellent hemostatic score
    3. Daily and total weight-adjusted administration of BAX 802 per subject
    4. Amount of blood products (e.g. whole blood, red blood cells, platelets,
    and plasma transfused

    Safety
    Development of, and changes to, the titer of inhibitory and binding
    antibodies (IgG and IgM) to pFVIII
    2. Development of, and changes to, the titer of inhibitory and binding
    antibodies (IgG and IgM) to hFVIII
    3. Development of binding antibodies to BHK proteins
    4. Occurrence of thrombo-embolic events
    5. Incidence of severe allergic reactions (e.g., anaphylaxis)
    6. Incidence of other IP-related AEs
    7. Incidence of clinically significant changes in vital signs and routine
    laboratory parameters (hematology, clinical chemistry)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Varying times as defined by the protocol.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA22
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Bulgaria
    Canada
    France
    Germany
    Hungary
    Italy
    Netherlands
    Norway
    Russian Federation
    South Africa
    Spain
    Sweden
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months4
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 2
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 9
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    F.4.2.2In the whole clinical trial 12
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Normal treatment to be continued as discussed with the subject's doctor.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-03-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-01-18
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2021-01-11
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