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    Summary
    EudraCT Number:2013-001359-11
    Sponsor's Protocol Code Number:261204
    National Competent Authority:Bulgarian Drug Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-07-30
    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 ConcernedBulgarian Drug Agency
    A.2EudraCT number2013-001359-11
    A.3Full title of the trial
    A Phase 3, Multi-Center, Open Label Study of Efficacy and Safety of PEGylated rFVIII (BAX 855) in Previously Treated Patients With Severe Hemophilia A 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 for safety and effectiveness of blood clotting factor VIII (Bax 855) for patients with blood clotting disorder called Hemophilia A, undergoing surgeries.
    A.3.2Name or abbreviated title of the trial where available
    Ph 3 Efficacy and Safety Study of BAX 855 in Hemophilia A Patients Undergoing Surgical Procedures
    A.4.1Sponsor's protocol code number261204
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/72/2013
    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 Innovations GmbH
    B.5.2Functional name of contact pointEli Taube
    B.5.3 Address:
    B.5.3.1Street AddressWagramerstrasse 17-19
    B.5.3.2Town/ cityVienna
    B.5.3.3Post codeA-1221
    B.5.3.4CountryAustria
    B.5.4Telephone number43120100 247 1240
    B.5.5Fax number43120100 534
    B.5.6E-maileli_taube@baxalta.com
    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 No
    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 namePEGylated rFVIII
    D.3.2Product code BAX 855
    D.3.4Pharmaceutical form Lyophilisate and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous bolus use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNnot applicable
    D.3.9.2Current sponsor codeBAX 855
    D.3.9.3Other descriptive nameBAX 855 (PEGYLATED RFVIII)
    D.3.9.4EV Substance CodeSUB90763
    D.3.10 Strength
    D.3.10.1Concentration unit IU/kg international unit(s)/kilogram
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number250 to 2000
    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 Yes
    D.3.11.13.1Other medicinal product typeRecombinant product with covalently bound polyethyleneglycol.
    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
    Severe hemophilia A (FVIII<1%)
    E.1.1.1Medical condition in easily understood language
    Hemophilia A
    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 19.0
    E.1.2Level LLT
    E.1.2Classification code 10060612
    E.1.2Term Hemophilia A
    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 is to evaluate the peri-operative hemostatic
    efficacy of BAX 855 in male PTPs aged 2- 75 years with severe
    hemophilia A (FVIII <1%) undergoing major or minor, elective or minor
    emergency surgical, dental or other invasive procedures.
    E.2.2Secondary objectives of the trial
    To determine the safety of BAX855 used in the perioperative setting
    o The occurrence of all AEs
    o The occurrence of thrombotic events and/or allergic reactions to BAX855
    o To determine the development of FVIII inhibitors and binding antibodies to FVIII, BAX855 and PEG, as well as antibodies to Chinese Hamster Ovary (CHO) proteins and murine IgG.
    • To determine the intra-and postoperative blood loss at the end of surgery and until drain removal, if applicable, compared to the estimated volume of expected average and maximum blood loss in a comparable healthy individual as predicted preoperatively by the investigator/surgeon
    To determine the volume of blood, red blood cells, platelets, and other blood products transfused
    • To determine the daily and total weight-adjusted consumption of BAX855 per subject
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subject requires an elective major or minor surgical, dental or other invasive procedure (e.g. biopsy, endoscopy).
    2. Subject and/or legal representative has/have provided signed informed consent.
    3. Subject is 2 to 75 years of age at the time of enrollment.
    4. Subject is male with severe hemophilia A (FVIII level <1%) as confirmed by the central lab at screening or a documented FVIII activity level <1%.
    5. Subject was previously treated with FVIII concentrates with ≥150 documented exposure days (EDs).
    6. Subject is currently receiving prophylaxis or on-demand therapy with FVIII concentrate.
    7. Subject has a Karnofsky performance score of ≥60 at screening.
    8. Subject is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count ≥200 cells/mm3, as confirmed by central laboratory at screening.
    9. Subject is Hepatitis C virus negative (HCV-) by antibody or PCR testing, as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis as assessed by the investigator. If positive, antibody titer will be confirmed by PCR.
    10. Subject is willing and able to comply with the requirements of the study protocol.
    11. For subjects transitioning from parent Bax855 studies, the subjects
    contunies to meet the entry criteria.
    E.4Principal exclusion criteria
    1. Subject has detectable FVIII inhibitory antibodies (≥0.4 BU using the Nijmegen modification of the Bethesda assay) at screening as determined by the central laboratory or at any timepoint prior to screening (≥0.4 BU using the Nijmegen modification of the Bethesda assay or ≥0.6 BU using the Bethesda assay).
    2. History of ongoing or recent thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC).
    3. Subject has a platelet count <100 x 109/L, as confirmed by central laboratory at screening.
    4. Subject has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening.
    5. Subject has severe chronic hepatic dysfunction (eg ≥5 X upper limit of normal alanine aminotransferase (ALT), as confirmed by central laboratory at screening, or a documented INR > 1.5).
    6. Subject has a known hypersensitivity towards mouse or hamster proteins, polysorbate 80 or to PEG.
    7. Subject is currently using or has recently (< 30 days) used pegylated drugs (other than BAX855) prior to study participation or is scheduled to use such drugs during trial participation.
    8. Subject is currently participating in another clinical drug (other than BAX855) or device study or use of another investigational product or device within 30 days prior to study entry.
    9. Subject has a diagnosis of an inherited or acquired hemostatic defect other than hemophilia A.
    10. Subject is currently receiving, or scheduled to receive during the course of the study, an immunomodulating drug (e.g., systemic corticosteroid agent at a dose equivalent to hydrocortisone greater than 10 mg/day, or alpha interferon) other than anti-retroviral chemotherapy.
    11. Subject has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject safety or compliance.
    12. The subject has an incremental recovery <1.5 IU/dL:IU/kg as determined in the parent study, if applicable.
    13. Subjects undergoing minor or major emergency surgery.
    E.5 End points
    E.5.1Primary end point(s)
    Global Hemostatic Efficacy score, which is composed of 3 individual ratings:
    1. Assessment of intra-operative hemostatic efficacy of BAX855 performed by the operating surgeon
    2. Assessment of postoperative hemostatic efficacy of BAX855 at postoperative day 1 performed by the investigator
    3. Assessment of postoperative hemostatic efficacy of BAX855 at EOS visit performed by the investigator (day 14 or discharge, whichever is
    first).
    E.5.1.1Timepoint(s) of evaluation of this end point
    6 months (±2 weeks) for first and secondary endpoints
    E.5.2Secondary end point(s)
    SAFETY
    • Development of neutralizing FVIII inhibitors
    • Development of binding antibodies to FVIII, BAX855, and PEG
    • Development of anti-CHO antibodies.
    • Occurrence of thrombotic events
    • Other IP-related adverse events (AEs)
    • Clinically significant changes in vital signs and routine laboratory parameters (hematology, clinical chemistry and virology)
    Pharmacokinetics of BAX855
    Primary PK:IR and T1/2. Secondary PK: AUC0-∞/dose (area under the plasma concentration /time curve from time 0 to infinity), MRT, CL, Vss, AUC0-96h/dose (area under the plasma concentration/time curve from time 0 to 96 hours post-infusion.
    Blood loss
    The observed versus predicted operative blood loss will be described for the period from initiation of the intervention to 24 hours after completion of the intervention or drain removal, as applicable,
    The intraoperative blood loss will be measured by determining the volume of blood and fluid removal through suction into the collection container (waste box and/or cell saver) and the estimated blood loss into swabs and towels during the procedure, per the anesthesiologist’s record. Post-operatively, blood loss will be determined by the drainage volume collected, which will likely consist mainly of drainage fluid via vacuum or gravity drain, as applicable.
    Transfusion requirements
    The type and number of units and amount (in mL) of blood products will be recorded. Furthermore, also salvage of blood obtained from autologous transfusion systems, e.g. cell savers, will be recorded. In addition, the type and amount of fluid replacement and volume expanders will be recorded as concomitant medication (e.g. amount and number of units of salvaged blood, red blood cells, platelets and other blood products transfused).
    Bleeding Episodes
    Any clinically relevant bleeding episodes, as well as the need for any further surgical interventions, are to be recorded. Study subjects will be closely monitored for the occurrence of bleeding episodes during the entire intra- and post-operative period, until the time of discharge from the investigative site or treatment facility.
    Drug consumption
    Daily and total weight-adjusted dose of BAX855 per subject.
    E.5.2.1Timepoint(s) of evaluation of this end point
    6 months (±2 weeks) for first and secondary endpoints
    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 Yes
    E.8.1.7.1Other trial design description
    single group
    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.8.2.4Number of treatment arms in the trial1
    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 EEA24
    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
    Belgium
    Bulgaria
    Lithuania
    Netherlands
    Poland
    Russian Federation
    Spain
    Ukraine
    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
    Last subject last visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months3
    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: 40
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 24
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 40
    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)
    Continue with the expected normal treatment for Hemophilia A
    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 Decision2013-08-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-07-24
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-09-25
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