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    Summary
    EudraCT Number:2009-016719-39
    Sponsor's Protocol Code Number:050901
    National Competent Authority:Czech Republic - SUKL
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2010-02-18
    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 ConcernedCzech Republic - SUKL
    A.2EudraCT number2009-016719-39
    A.3Full title of the trial
    IMMUNINE – Purified Factor IX Concentrate Virus-Inactivated: A Phase IV, Prospective, Open-label Multicenter Study to Prospectively Document the Exposure of IMMUNINE and to Monitor FIX Inhibitors in Previously Treated Patients with Severe (FIX level < 1%) or Moderately Severe (FIX level ≤2%) Hemophilia B Who are Planned to Enter BAX 326 Study 250901 to investigate a New Recombinant FIX Concentrate
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    IMMUNINE - A Clinical Study with IMMUNINE (A Purified Factor IX Concentrate Virus-Inactivated) in Previously Treated Patients with Severe or Moderately Severe Hemophilia B Who are Planned to Enter the BAX 326 Study (250901, Investigating a new Factor IX Product)
    A.3.2Name or abbreviated title of the trial where available
    IMMUNINE Pre-Treatment Study
    A.4.1Sponsor's protocol code number050901
    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 pointClinical Study Team Lead
    B.5.3 Address:
    B.5.3.1Street AddressIndustriestrasse 67
    B.5.3.2Town/ cityVienna
    B.5.3.3Post code1220
    B.5.3.4CountryAustria
    B.5.4Telephone number+49 (0) 89 357 187 08
    B.5.5Fax number+49(0) 89 358 282 19
    B.5.6E-mailbarbara_gastl@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 Yes
    D.2.1.1.1Trade name IMMUNINE Baxter 600 IU
    D.2.1.1.2Name of the Marketing Authorisation holderBaxter Czech spol. S.r.o
    D.2.1.2Country which granted the Marketing AuthorisationCzech Republic
    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 nameIMMUNINE Baxter 600 IU
    D.3.2Product code n.a.
    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 INNHuman Coagulation Factor IX
    D.3.10 Strength
    D.3.10.1Concentration unit IU/l international unit(s)/litre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number600
    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 Yes
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.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 Yes
    D.2.1.1.1Trade name IMMUNINE BAXTER 1200 IU
    D.2.1.1.2Name of the Marketing Authorisation holderBaxter Czech spol S.r.o
    D.2.1.2Country which granted the Marketing AuthorisationCzech Republic
    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 nameIMMUNINE BAXTER 1200 IU
    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 INNHuman Coagulation Factor IX
    D.3.10 Strength
    D.3.10.1Concentration unit IU/l international unit(s)/litre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1200
    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 Yes
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    To expose up to 50 patients aged 12-64 years, and approximately 20 pediatric patients up to 11 years of age with severe or moderately severe hemophilia B to IMMUNINE for a period of approximately 20 to 50 exposure days (EDs), prospectively document the exposure days (EDs) and factor IX (FIX) inhibitors while receiving prophylactic treatment so that these
    patients can transition into the BAX 326 pivotal or pediatric study evaluating a new rFIX, provided all eligibility criteria are met.
    E.1.1.1Medical condition in easily understood language
    Severe to moderately severe Hemophilia B, i.e. lack of (active) blood clotting factor IX
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 15.0
    E.1.2Level LLT
    E.1.2Classification code 10060614
    E.1.2Term Hemophilia 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
    E.2.1 Main objective: To prospectively document the exposure to IMMUNINE and to monitor FIX inhibitors while
    receiving prophylactic treatment over a period of approximately 20 to 50 EDs in up to
    50 PTPs aged 12-64 years and approximately 20 pediatric subjects up to 11 years of age with
    severe (FIX level < 1%) or moderately severe (FIX level ≤ 2%) hemophilia B who are
    planned to enter the BAX 326 pivotal or pediatric study evaluating a new rFIX, provided all
    eligibility criteria are met.
    E.2.2Secondary objectives of the trial
    E.2.2 S econdary objectives:
    •To evaluate safety in terms of investigational product related adverse events, immunogenicity
    and thrombogenicity
    • To monitor hemostatic efficacy of IMMUNINE in the management and prevention of acute
    bleeding episodes, and surgical prophylaxis if necessary
    • To evaluate the subject’s compliance with the protocol specific treatment regimen
    • To monitor incremental recovery of IMMUNINE over time
    • To evaluate HR QoL at baseline
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • - Subject and/or legal representative has/have provided signed informed consent.
    • Subject is up to 64 years old at the time of screening.
    • 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 or recombinant FIX concentrate(s), cryoprecipitate or fresh frozen plasma (FFP) for approximately 100 - 150 exposure days (EDs) if > = 6 years old or 20-50 ED if < 6 years old and is planned to enter either the study BAX326 pivotal or BAX 326 pediatric study 250901. The number of EDs is derived from the subject’s treatment regimen and his/her bleeding pattern.
    • Subject/legal representative is willing/accepts to to receive prophylactic treatment for the duration of the study.
    • Subject is immunocompetent as evidenced by a CD4 count ≥ 200 cells/mm.
    • Subject is human immunodeficiency virus (HIV) negative or is HIV+ with a viral load < 200 particles/μL, approximately <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/legal representative is willing and able to comply with the requirements of the protocol.
    E.4Principal exclusion criteria
    • 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 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) at any time prior to screening.
    • 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.
    • The subject has evidence of a thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC).
    • Subject is scheduled for elective surgery, unless the surgery is medically required within the anticipated study period

    • The subject has an abnormal renal function (serum creatinine > 1.5 times the upper limit of normal [ULN]).
    • 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. During the study, subjects with chronic hepatitis B or C may have fluctuations of up to 5 times the upper limit of normal (ULN) but will not require discontinuation.
    • 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 other than anti-retroviral chemotherapy (eg, α-interferon, corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day).
    • The subject is pregnant or lactating at the time of study enrollment.
    • The subject is unwilling to consider further participation in the BAX 326 pivotal or pediatric study
    • Subject has participated in another clinical study involving an investigational product (IP) or device within 30 days prior to study enrollment.
    • 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 (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)
    The primary objective of the study is to prospectively document exposure to IMMUNINE and to monitor FIX inhibitors for approximately 20 to 50 EDs in up to 50 PTPs with severe or moderately severe hemophilia B so that these patients can transition into BAX326 study 250901 provided all eligibility criteria are met.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Study End
    E.5.2Secondary end point(s)
    Secondary study objectives are the evaluation of efficacy, safety, immunogenicity, thrombogenicity, HR QoL, and the subject’s compliance with the study specific treatment regimen.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Study End
    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 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 Yes
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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 EEA10
    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
    Bulgaria
    Chile
    Colombia
    Czech Republic
    Poland
    Romania
    Russian Federation
    Ukraine
    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 Visit of the Last Subject
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months4
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 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) Yes
    F.1.1.4.1Number of subjects for this age range: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 18
    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: 35
    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
    In case of minor patients consent of legal representative(s) is required.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 70
    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)
    Patients may transition into BAX 326 pivotal or pediatric study investigating a new recombinant FIX concentrate.
    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 Decision2010-03-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2010-04-07
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2012-09-28
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