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    The EU Clinical Trials Register currently displays   38204   clinical trials with a EudraCT protocol, of which   6275   are clinical trials conducted with subjects less than 18 years old.
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    EudraCT Number:2009-016720-31
    Sponsor's Protocol Code Number:250901
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2010-03-19
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedGermany - PEI
    A.2EudraCT number2009-016720-31
    A.3Full title of the trial
    BAX 326 (recombinant Factor IX): A Phase 1/3 Prospective, Controlled, Multicenter Study Evaluating Pharmacokinetics, Efficacy, Safety, Immunogenicity in Previously Treated Patients with Severe (FIX level < 1%) or Moderately Severe (FIX level ≤ 2%) Hemophilia B
    A.3.2Name or abbreviated title of the trial where available
    BAX 326 pivotal
    A.4.1Sponsor's protocol code number250901
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBaxter Innovations GmbH
    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 support
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    B.Sponsor: 2
    B.1.1Name of SponsorBaxter Healthcare Corporation
    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 support
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 nameBAX326
    D.3.2Product code BAX326
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 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 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) Information not present in EudraCT
    D. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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. medicinal product typeHuman Coagulation Factor IX (rDNA) produced by recombinant DNA technology in mammalian cell culture (CHO) INN: Nonacog alfa
    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 with severe (FIX level < 1%) or moderately severe (FIX level ≤ 2%) hemophilia B.
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 12.1
    E.1.2Level LLT
    E.1.2Classification code 10018939
    E.1.2Term Haemophilia B (Factor IX)
    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 BAX 326 pharmacokinetic (PK) parameters and to determine PK equivalence with BeneFIX, to evaluate its hemostatic efficacy, safety, immunogenicity, and changes in health-related quality of life (HR QoL)
    E.2.2Secondary objectives of the trial
    To compare the PK parameters of BAX 326 with those of BeneFIX
    To monitor incremental recovery (IR) of BAX 326 over time
    To evaluate the hemostatic efficacy of BAX 326 in the management and prevention of acute bleeding episodes for a period of 6 months
    To evaluate the safety in terms of BAX 326 related adverse events, immunogenicity for a minimum of 50 exposure days (EDs), thrombogenicity during the PK parts, 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 is 12 to 65 years old at the time of screening.
    • Subject and/or legal representative has/have 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 tested at screening at the central laboratory.
    • Subject 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 1) there are 100 - 150 EDs to any FIX product (plasma-derived or recombinant FIX concentrate(s), cryoprecipitate, or fresh frozen plasma) that are not fully documented, and
    2) s/he has participated in the Immunine protocol 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 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 50 EDs to Immunine prior to enrollment.
    • Subject is willing to receive 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
    • 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
    • The 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.
    • The 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.
    • The subject's weight is < 35kg or > 120 kg.
    • The subject has a history of allergic reaction, eg, anaphylaxis, following exposure to FIX concentrate(s).
    • The subject has a known hypersensitivity to hamster proteins or rFurin.
    • The subject has evidence of an ongoing or recent thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC).
    • The subject has an abnormal renal function (serum creatinine > 1.5 times the upper limit of normal).
    • The subject has severe chronic liver disease as evidenced by, but not limited to, any of the following: International Normalized Ratio (INR) exceeding the upper limit of normal hypoalbuminemia, portal vein hypertension including presence of otherwise unexplained splenomegaly and history of esophageal varices.
    • The subject has active hepatic disease with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels > 5 times the upper limit of normal.
    • The subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia B.
    • The subject’s platelet count is < 100,000/mL.
    • 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 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.
    • The subject has participated in another investigational study within 30 days of enrollment. Participation in study 050901 with Immunine is allowed.
    • 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.
    E.5 End points
    E.5.1Primary end point(s)
    • PK:
    o Primary PK: AUC0-72 h/dose (area under the plasma concentration versus time curve from 0 to 72 hours post-infusion)
    o Secondary PK: AUC0-∞ /dose (area under the plasma concentration/time curve from time 0 to infinity) MRT (mean residence time), CL (clearance), IR, T1/2 (elimination phase half-life), Vss (Volume of distribution at steady state).
    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 endpoints.
    o Consumption of BAX 326: number of infusions and weight-adjusted consumption per month and per year; weight-adjusted consumption per event (prophylaxis and on-demand).
    • Safety:
    o Development of inhibitory and total binding antibodies to factor IX
    o Development of antibodies to Chinese hamster ovary (CHO) proteins and recombinant furin (rFurin)
    o Occurrence of severe allergic reactions, eg, anaphylaxis
    o Occurrence of thrombotic events and clinically significant changes in thrombogenic markers during the PK parts of the study: prothrombin fragment 1.2 (F 1.2), thrombin-antithrombin III (TAT), D-dimer.
    o IP-related AEs
    o Clinically significant changes in routine laboratory parameters (hematology and clinical chemistry), and vital signs
    • HR QoL and pharmacoeconomic parameters
    o Changes in the following HR QoL parameters and health resource use
    • For subjects who are between 12 to 16 years of age:
    o Disease-specific: Haemo-QoL short version
    o Generic: PedsQL™
    o Health utility: EQ-5D
    o General pain Assessment through a visual analog scale (VAS)
    o Health resource use
    • For subjects aged 17 years and older:
    o Disease-specific: Haemo-A-QoL
    o Generic: SF-36
    o Health utility: EQ-5D
    o General pain assessment through VAS
    o Health resource use
    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 Yes
    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, thrombogenicity
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E. trial type description
    Phase 1/3 study
    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 Yes
    E.8.1.1Randomised Yes
    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 Yes
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E. description
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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
    Not applicable.
    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 months8
    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 months8
    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.1.1.1In 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.1.2Adults (18-64 years) Yes
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2010-03-19. Yes
    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. of subjects incapable of giving consent
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 60
    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)
    After study completion, subjects may enter a Continuation Study covered by a separate protocol until BAX 326 is licensed in his/her respective country.
    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 Decision2010-09-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2010-06-29
    P. End of Trial
    P.End of Trial StatusCompleted
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