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    Summary
    EudraCT Number:2010-022726-33
    Sponsor's Protocol Code Number:251001
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-06-24
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2010-022726-33
    A.3Full title of the trial
    BAX 326 (recombinant Factor IX): Evaluation of Safety, Immunogenicity, and Hemostatic Efficacy in Previously Treated Patients with Severe (FIX level < 1%) or Moderately Severe (FIX level 1-2%) Hemophilia B - A Continuation Study
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    BAX 326 (recombinant cogulation factor IX): Continuation of a study of safety and effectiveness of the product in previously treated patients with severe or moderately severe hemophilia B.
    A.3.2Name or abbreviated title of the trial where available
    BAX 326 Continuation
    A.4.1Sponsor's protocol code number251001
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/293/2011
    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
    B.5.3 Address:
    B.5.3.1Street AddressDonau City Strasse 7
    B.5.3.2Town/ cityVienna
    B.5.3.3Post code1220
    B.5.3.4CountryAustria
    B.5.4Telephone number+431201002473489
    B.5.5Fax number+431201002475727
    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 gamma
    D.3.9.1CAS number 181054-95-5
    D.3.9.2Current sponsor codeBAX 326
    D.3.9.3Other descriptive nameRecombinant coagulation factor IX
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    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 gamma
    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 international 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 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 gamma
    D.3.9.1CAS number 181054-95-5
    D.3.9.2Current sponsor codeBAX 326
    D.3.9.3Other descriptive nameRecombinant coagulation factor IX
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    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 gamma
    D.3.9.1CAS number 181054-95-5
    D.3.9.2Current sponsor codeBAX 326
    D.3.9.3Other descriptive nameRecombinant coagulation factor IX
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    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 gamma
    D.3.9.1CAS number 181054-95-5
    D.3.9.2Current sponsor codeBAX 326
    D.3.9.3Other descriptive nameRecombinant coagulation factor IX
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    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
    Previously treated patients (PTPs) with severe (FIX level < 1%) or moderately severe (FIX level 1-2%) hemophilia B.
    E.1.1.1Medical condition in easily understood language
    Previously treated patients (PTPs) 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 17.0
    E.1.2Level LLT
    E.1.2Classification code 10018939
    E.1.2Term Haemophilia B (Factor IX)
    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
    -To further evaluate the safety, hemostatic efficacy, and immunogenicity of BAX326, and changes in health-related quality of life (HR QoL) in subjects who completed Pivotal Study 250901 or Pediatric Study 251101 or newly recruited, BAX326 naïve subjects, until BAX326 is licensed in the subject’s country or until the accumulation of up to approximately 100 exposure days (EDs) of treatment with BAX326, whichever occurs last
    E.2.2Secondary objectives of the trial
    -To further evaluate safety of BAX 326 in terms of IP-related AEs as well as clinically significant changes in routine laboratory parameters (hematology/clinical chemistry) and vital signs
    -To further evaluate the hemostatic efficacy of BAX326 in the prevention and routine prophylaxis of acute bleeding episodes using various prophylactic treatment regimens
    -To further evaluate the hemostatic efficacy of BAX326 in the management of acute bleeding episodes
    -To further evaluate immunogenicity for up to approximately 100 EDs to BAX326. To monitor incremental recovery (IR) of BAX326 over time
    -To evaluate changes in health-related quality of life (HR QoL), Patient Activity Level and health resource use
    -Exploratory: To correlate pre-infusion thrombin generation assay (TGA) parameters with preinfusion FIX levels and spontaneous breakthrough bleeds in a subset of subjects receiving twice weekly standard or modified prophylactic treatment, including PK-tailored prophylaxis
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    FOR SUBJECTS TRANSITIONING FROM BAX STUDIES 250901 and 251101
    • The subject and/or legal representative has/have voluntartily provided signed informed consent.
    • The subject has completed Baxter Pivotal Study 250901 or Pediatric Study 251101. The subject was 12 to 65 years old at the time of screening for 250901 study or <12 years old at the time of screening for Pediatric Study 251101.
    • Subject has not developed an inhibitory FIX antibody during Baxter clinical study 250901 or Pediatric Study 251101.
    • Subject is human immunodeficiency (HIV) negative or is HIV+ with a viral load < 200 particles/μL ~ <400,000 copies/mL
    • Subject is immunocompetent as evidenced by a CD4 count ≥ 200 cells/mm3.
    • If female of childbearing potential, subject presents with a negative pregnancy test and agrees to continue employing adequate birth control measures for the duration of the study.
    • Subject is willing and able to comply with the requirements of the protocol.

    FOR NEW SUBJECTS
    - Subject and/or legal representative has/have provided signed informed consent.
    - Subject is 2 to 70 years old at the time of screening.
    - Subject is naïve to BAX326
    - Subject has severe (FIX level < 1%) or moderately severe (FIX level 1-2%) hemophilia B (based on the one stage activated partial thromboplastin time (aPTT) assay), as tested at screening at the central laboratory.
    - Subject aged ≥ 6 years is previously treated with plasma-derived and/or recombinant FIX concentrate(s) for a minimum of 150 EDs.
    - Subject aged < 6 years year is previously treated with plasma-derived and/or recombinant FIX concentrate(s) for a minimum of 50 EDs.
    - Subject has no evidence of a history of FIX inhibitors.
    - Subject is immunocompetent as evidenced by a CD4 count ≥ 200 cells/mm3 at screening.
    - Subject is human immunodeficiency (HIV) negative or is HIV+ with a viral load
    < 200 particles/μL ~ < 400,000 copies/mL
    - If female of childbearing potential, subject presents with a negative pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
    - Subject is willing and able to comply with the requirements of the protocol.
    E.4Principal exclusion criteria
    FOR SUBJECTS TRANSITIONING FROM BAX STUDIES 250901 and 251101
    • The subject received factor IX product(s) other than BAX 326 upon completion of Baxter pivotal study 250901 or Pediatric Study 251101.
    • The subject has been diagnosed with an acquired hemostatic defect other than hemophilia B.
    • For subjects transferring from Pivotal Study 250901: The subject’s weight is < 35 kg or > 120 kg.
    • The subject’s platelet count is < 100,000/mL.
    • The subject has an abnormal renal function (serum creatinine > 1.5 times the upper limit of normal).
    • The subject has active hepatic disease with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels ≥ 5 times the upper limit of normal.
    • The subject 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.
    • The 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’s safety or compliance.
    • The subject is planned to take part in any other clinical study, with the exception of BAX 326 Surgery study as described in this protocol, during the course of the Continuation Study.
    • The subject is a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (i.e., children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.

    FOR NEW SUBJECTS
    - Subject has a history of FIX inhibitors with a titer ≥ 0.6 Bethesda Units (BU) (as determined by the Nijmegen modification of the Bethesda assay or the assay employed in the respective local laboratory) 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.
    - Subjects ≥ 12 years of age: Subject’s weight is < 35 kg or > 120 kg
    - Subject has a history of allergic reaction, eg, 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 severe chronic liver disease as evidenced by, but not limited to, any of the following: International Normalized Ratio (INR) > 1.4 hypoalbuminemia, portal vein hypertension including presence of otherwise unexplained splenomegaly and history of esophageal varices.
    - 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, or recreational drug/alcohol use 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 (eg, 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.
    - Subject is a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.
    E.5 End points
    E.5.1Primary end point(s)
    • Adverse events possibly or probably related to IP
    E.5.1.1Timepoint(s) of evaluation of this end point
    Continuous evaluation
    E.5.2Secondary end point(s)
    Safety and Immunogenicity
    • Development of inhibitory and total binding antibodies to FIX.
    • Development of antibodies to CHO proteins and rFurin.
    • Occurrence of severe allergic reactions, e.g. anaphylaxis.
    • Occurrence of thrombotic events
    • Clinically significant changes in routine laboratory parameters (hematology and clinical chemistry), and vital signs.

    PK
    • Incremental recovery (IR) over time.

    Hemostatic efficacy
    • Treatment of bleeding episodes: number of infusions per bleeding episode, overall hemostatic efficacy rating at resolution of bleed.
    • Prophylaxis: annualized bleeding rate.
    • Consumption of BAX326:
    - Number of infusions and weight-adjusted consumption per month and per year;
    - Weight-adjusted consumption per event (prophylaxis and on-demand).

    Health Related Quality of Life and Pharmacoeconomic Parameters
    Changes in the following HR QoL parameters and health resource use:
    • For subjects who are between 2 to 7 years of age (at the time of screening for Study 251101):
    - Generic: PedsQL™ (Parent-proxy versions: age group 2-4 years and age group 5-7 years)
    - Health resource use (hospitalizations, emergency room visits, doctor office visits, etc.)
    • For subjects who are between 8 to 11 years of age (at the time of screening for Study 251101):
    - Disease-specific: Haemo-QoL, short version
    - Generic: PedsQL™ Child version
    - Health resource use (hospitalizations, emergency room visits, doctor office visits, etc.)
    • For subjects who are between 12 and 16 years of age (at the time of screening for Study 250901) the following questioninaires will be used:
    - Disease-specific: Haemo-QoL short version
    - Generic: PedsQL™
    - Health utility: EQ-5D
    - General pain assessment through a visual analog scale (VAS)
    - Health resource use
    • For subjects aged 17 years and older (at the time of screening for Study 250901) the following questionnaires will be used:
    - Disease-specific: Haem-A-QoL
    - Generic: SF-36
    - Health Utility: EQ-5D
    - General pain assessment through a visual analog scale (VAS)
    - Health resource use
    E.5.2.1Timepoint(s) of evaluation of this end point
    Safety and immunogenicity: continuous
    Other parameters: End of the trial
    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 No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity, hemostatic efficacy
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA31
    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
    Argentina
    Brazil
    Chile
    Colombia
    Germany
    Ireland
    Japan
    Russian Federation
    Taiwan
    Ukraine
    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 years4
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months7
    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: 30
    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: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 15
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 70
    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 Yes
    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
    Minors > 2 years might be included
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 100
    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)
    Not applicable.
    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 Decision2014-08-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-09-17
    P. End of Trial
    P.End of Trial StatusCompleted
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