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    Summary
    EudraCT Number:2011-002437-19
    Sponsor's Protocol Code Number:251101
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2011-09-30
    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 ConcernedUK - MHRA
    A.2EudraCT number2011-002437-19
    A.3Full title of the trial
    BAX326 (RECOMBINANT FACTOR IX): A PHASE 2/3 PROSPECTIVE, UNCONTROLLED, MULTICENTER STUDY EVALUATING PHARMACOKINETICS, EFFICACY, SAFETY, AND IMMUNOGENICITY IN PREVIOUSLY TREATED PEDIATRIC PATIENTS WITH SEVERE (FIX LEVEL < 1%) OR MODERATELY SEVERE (FIX LEVEL ≤ 2%) HEMOPHILIA B
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    BAX 326 (Recombinant factor nine): A clinical study evaluating pharmacokinetics, efficacy, safety, and immunogenicity in previously treated pediatric patients with severe or moderately severe hemophilia B.
    A.3.2Name or abbreviated title of the trial where available
    BAX326 Pediatric
    A.4.1Sponsor's protocol code number251101
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBaxter 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 supportBaxter Innovations GmbH
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBaxter Innovations GmbH
    B.5.2Functional name of contact pointGuido Wuerth, Clinical Operations
    B.5.3 Address:
    B.5.3.1Street AddressWagramer Strasse 17-19
    B.5.3.2Town/ cityVienna
    B.5.3.3Post code1220
    B.5.3.4CountryAustria
    B.5.4Telephone number+431201003489
    B.5.5Fax number+43120100717
    B.5.6E-mailguido_wuerth@baxter.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 nameRecombinant Coagulation Factor IX
    D.3.2Product code BAX326
    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 INNNONACOG ALFA
    D.3.9.1CAS number 181054-95-5
    D.3.9.2Current sponsor codeBAX326
    D.3.9.3Other descriptive nameRecombinant coagulation factor IX
    D.3.10 Strength
    D.3.10.1Concentration unit IU/kg international unit(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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 typeHuman Coagulation Factor IX (rDNA) produced by recombinant DNA technology in mammalian cell culture (CHO)
    D.IMP: 2
    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 nameRecombinant Coagulation Factor IX
    D.3.2Product code BAX326
    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 INNNONACOG ALFA
    D.3.9.1CAS number 181054-95-5
    D.3.9.2Current sponsor codeBAX326
    D.3.9.3Other descriptive nameRecombinant coagulation factor IX
    D.3.10 Strength
    D.3.10.1Concentration unit IU/kg international unit(s)/kilogram
    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 Yes
    D.3.11.13.1Other medicinal product typeHuman Coagulation Factor IX (rDNA) produced by recombinant DNA technology in mammalian cell culture (CHO)
    D.IMP: 3
    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 nameRecombinant Coagulation Factor IX
    D.3.2Product code BAX326
    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 INNNONACOG ALFA
    D.3.9.1CAS number 181054-95-5
    D.3.9.2Current sponsor codeBAX326
    D.3.9.3Other descriptive nameRecombinant coagulation factor IX
    D.3.10 Strength
    D.3.10.1Concentration unit IU/kg international unit(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000
    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 typeHuman Coagulation Factor IX (rDNA) produced by recombinant DNA technology in mammalian cell culture (CHO)
    D.IMP: 4
    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 nameRecombinant Coagulation Factor IX
    D.3.2Product code BAX326
    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 INNNONACOG ALFA
    D.3.9.1CAS number 181054-95-5
    D.3.9.2Current sponsor codeBAX326
    D.3.9.3Other descriptive nameRecombinant coagulation factor IX
    D.3.10 Strength
    D.3.10.1Concentration unit IU/kg international unit(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2000
    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 typeHuman Coagulation Factor IX (rDNA) produced by recombinant DNA technology in mammalian cell culture (CHO)
    D.IMP: 5
    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 nameRecombinant Coagulation Factor IX
    D.3.2Product code BAX326
    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 INNNONACOG ALFA
    D.3.9.1CAS number 181054-95-5
    D.3.9.2Current sponsor codeBAX326
    D.3.9.3Other descriptive nameRecombinant coagulation factor IX
    D.3.10 Strength
    D.3.10.1Concentration unit IU/kg international unit(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3000
    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 typeHuman Coagulation Factor IX (rDNA) produced by recombinant DNA technology in mammalian cell culture (CHO)
    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
    Pediatric previously treated patients (PTPs) with severe (FIX level < 1%) or moderately severe (FIX level ≤ 2%) hemophilia B.
    E.1.1.1Medical condition in easily understood language
    Previously treated children with severe or moderately severe hemophilia B.
    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 14.1
    E.1.2Level LLT
    E.1.2Classification code 10018939
    E.1.2Term Haemophilia B (Factor IX)
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess BAX326 pharmacokinetic (PK) parameters, to evaluate its hemostatic efficacy, safety, immunogenicity, and changes in health-related quality of life (HR QoL) in pediatric patients.
    E.2.2Secondary objectives of the trial
    • To evaluate the PK parameters of BAX326 in pediatric PTPs < 12 years of age
    • To monitor incremental recovery (IR) of BAX326 over time
    • To evaluate the hemostatic efficacy of BAX326 in the management and prevention of acute bleeding episodes for a period of 6 months
    • To evaluate safety in terms of immunogenicity for a minimum of 50 exposure days (EDs), the occurrence of thrombotic events, as well as clinically significant changes in routine laboratory parameters (hematology/clinical chemistry) and vital signs.
    • To evaluate changes in HR QoL and health resource use
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Subject and/or legal representative has/have voluntarily provided signed informed consent
    • Subject has severe (FIX level < 1%) or moderately severe (FIX level ≤ 2%) hemophilia B based on the one-stage activated partial thromboplastin time (aPTT) assay, as determined by the central laboratory
    • Subject is < 12 years old at the time of screening
    • Subject 6 to <12 years of age is previously treated with plasma-derived and/or recombinant FIX concentrate(s) for a minimum of 150 EDs (based on the subject’s medical records).
    If a subject does not have a verifiable, documented history of 150 EDs, s/he can be enrolled if the following criteria are met:
    1) there are an estimated 100 - 150 EDs to any FIX product (plasma-derived or recombinant FIX concentrate(s), prothrombin complex concentrate PCC or fresh frozen plasma FFP that are not fully documented (assumption based on the severity of disease and treatment history), and
    2) s/he has participated in Immunine Study 050901 and accumulated either at least 50 EDs to Immunine or a total of at least 150 EDs to a plasma-derived and/or recombinant FIX concentrate prior to enrollment
    • Subject < 6 years of age is previously treated with plasma-derived and/or recombinant FIX concentrate(s) for > 50 EDs (based on the subject’s medical records).
    If a subject does not have a verifiable, documented history of >50 EDs, s/he can be enrolled if the following criteria are met:
    1) there are approximately 20 - 50 EDs to any FIX product (plasma-derived or recombinant FIX concentrate(s), PCC or FFP) that are not fully documented, and
    2) s/he has participated in Immunine Study 050901 and accumulated a minimum of 30 EDs to Immunine or a total of > 50 EDs to a plasma-derived and/or recombinant FIX concentrate prior to enrollment
    • Subject has no evidence of a history of FIX inhibitors (based on the subject’s medical records). If a verifiable, documented history is unavailable, the subject can be enrolled if s/he has participated in Study 050901 for at least 30 EDs (< 6 years of age) or at least 50 EDs (6 to < 12 years of age) to Immunine prior to enrollment
    • Subject and/or legal representative accepts prophylactic treatment over a period of 6 months
    • Subject is immunocompetent as evidenced by a CD4 count ≥ 200 cells/mm3
    • Subject is human immunodeficiency (HIV) negative or is HIV+ with a viral load < 200 particles/μL ~ < 400,000 copies/mL
    • Subject and/or the legal representative is willing and able to comply with the requirements of the Protocol
    E.4Principal exclusion criteria
    • Subject has a history of FIX inhibitors with a titer ≥ 0.6 BU (as determined by the Nijmegen modification of the Bethesda assay or the assay employed in the respective local laboratory with the corresponding detection limit) at any time prior to screening
    • Subject has a detectable FIX inhibitor at screening, with a titer ≥ 0.6 BU as determined by the Nijmegen modification of the Bethesda assay in the central laboratory
    • Subject has a history of allergic reaction, e.g. anaphylaxis, following exposure to FIX concentrate(s)
    • Subject has a known hypersensitivity to hamster proteins or rFurin
    • Subject has evidence of an ongoing or recent thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC)
    • Subject has an abnormal renal function (serum creatinine >1.5 times the upper limit of normal)
    • Subject has an International Normalized Ratio (INR) >1.4
    • Subject has active hepatic disease with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels > 5 times the upper limit of normal
    • Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia B
    • Subject’s platelet count is < 100,000/mL
    • Subject has a clinically significant medical, psychiatric, or cognitive illness, that, in the opinion of the Investigator, would affect subject’s safety or compliance
    • Subject is currently receiving, or is scheduled to receive during the course of the study, an immunomodulating drug (e.g. corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or α-interferon) other than anti-retroviral chemotherapy
    • Subject has participated in another investigational study within 30 days of enrollment. However, participation in Study 050901 with Immunine is allowed.
    E.5 End points
    E.5.1Primary end point(s)
    • All AEs possibly or probably related to BAX326
    E.5.1.1Timepoint(s) of evaluation of this end point
    Ongoing (see protocol)
    E.5.2Secondary end point(s)
    • PK:
    o Area under the plasma concentration versus time curve from 0 to 72 hours post-infusion (AUC0-72 h/dose), total AUC/dose, mean residence time (MRT), clearance (CL), incremental recovery (IR), elimination phase half-life (T1/2), volume of distribution at steady state (Vss)
    o IR over time
    • Hemostatic efficacy:
    o Treatment of bleeding episodes: number of infusions per bleeding episode, overall hemostatic efficacy rating at resolution of bleed
    o Prophylaxis: annualized bleeding rate
    o Prophylaxis: number of bleeding episodes beginning within 24 and 48 hours of an infusion as exploratory outcome measures
    o Consumption of BAX326: number of infusions and weight-adjusted consumption per month and per year; weight-adjusted consumption per event
    • Safety and immunogenicity:
    o Development of inhibitory and total binding antibodies to FIX
    o Occurrence of severe allergic reactions, e.g. anaphylaxis
    o Occurrence of thrombotic events
    o Clinically significant changes in routine laboratory parameters (hematology and clinical chemistry) and vital signs
    o Development of antibodies to Chinese hamster ovary (CHO) proteins and recombinant furin (rFurin)
    • Changes in the following HR QoL parameters and health resource use
    For subjects who are between 2 to 7 years of age:
    o Generic: PedsQL™ (Parent-proxy versions: age group 2-4 years and age group 5-7 years)
    o Health resource use (hospitalizations, emergency room visits, doctor office visits, etc.)
    For subjects who are between 8 to 11 years of age:
    o Disease-specific: Haemo-QoL, short version
    o Generic: PedsQL™ Child version
    o Health resource use (hospitalizations, emergency room visits, doctor office visits, etc.)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Ongoing (see protocol)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity
    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) Yes
    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 No
    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 EEA7
    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
    Czech Republic
    Germany
    India
    Poland
    Romania
    Russian Federation
    Ukraine
    United Kingdom
    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
    • If two or more subjects develop a high titer inhibitory antibody > 5 Bethesda Unit (BU) confirmed by the central laboratory, or if two or more subjects develop anaphylaxis following exposure to BAX326, the recruitment will be halted.
    • The study may be stopped following any of the planned interim safety reviews or at any time by the Sponsor based on DSMC recommendations in case of unacceptable risks to subjects, such as increased inhibitor development, thrombotic events, or anaphylaxis.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    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: 24
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 4
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 20
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children < 12 years of age
    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 13
    F.4.2.2In the whole clinical trial 24
    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)
    Every subject may be entered into the Continuation Study (Protocol 251001) until s/he has accumulated a total of 100 EDs to BAX326, depending on local or national regulations.
    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 Decision2011-10-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-10-26
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2013-05-14
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