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    Summary
    EudraCT Number:2013-002686-19
    Sponsor's Protocol Code Number:HIPS-2013
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2014-09-19
    Trial 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 ConcernedSweden - MPA
    A.2EudraCT number2013-002686-19
    A.3Full title of the trial
    Hemophilia Inhibitor PUP Study
    Anti-faktor VIII hämmande antikroppar hos tidigare obehandlade patienter med ärftlig blödarsjuka
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Inhibitor study in patients with hemophilia never treated in the past
    Anti-faktor VIII hämmande antikroppar hos tidigare obehandlade patienter med ärftlig blödarsjuka
    A.3.2Name or abbreviated title of the trial where available
    HIPS
    A.4.1Sponsor's protocol code numberHIPS-2013
    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 SponsorLund University, Clinical Coagulation Research Unit, Skane University Hospital
    B.1.3.4CountrySweden
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportBaxter
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationLund University, Clinical Coagulation Research Unit, Skane University Hospital
    B.5.2Functional name of contact pointKoagulationsmottagningen
    B.5.3 Address:
    B.5.3.1Street AddressJan Waldenströms gata 14
    B.5.3.2Town/ cityMalmö
    B.5.3.3Post code20502
    B.5.3.4CountrySweden
    B.5.6E-mailjenny.klintman@med.lu.se
    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 Advate
    D.2.1.1.2Name of the Marketing Authorisation holderBaxter AG - Wien - Austria
    D.2.1.2Country which granted the Marketing AuthorisationSweden
    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 nameAdvate
    D.3.2Product code no info available
    D.3.4Pharmaceutical form Lyophilisate for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    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 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
    Infants or children with severe hemophilia A previously untreated with factor concentrates.
    Spädbarn eller småbarn med svår hemofili A som tidigare inte erhållit behandling
    E.1.1.1Medical condition in easily understood language
    Infants or children with severe hemophilia A never treated in the past for this disease
    Spädbarn eller småbarn med svår form av ärftlig blödarsjuka typ A som inte fått någon behandling ännu
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to evaluate the changes in the immune system upon exposure to FVIII with severe hemophilia A
    Huvudsyftet med studien är att studera immunförsvarets reaktion vid exponering av faktor VIII hos patienter med svår form av hemofili A
    E.2.2Secondary objectives of the trial
    The secondary objective is to identify predictors of FVIII inhibitor development or tolerance to FVIII infusions
    I andra hand syftar studien till att identifiera markörer för inhibitorutveckling och toleransutveckling vid faktor VIII-behandling
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) An informed consent, approved by the appropriate institutional Review Board (IRB)/Independent Ethics Committee (IEC) has been administered, signed and dated by the parents or legal representative of the parents
    2) Subject diagnosed with severe hemophilia A defined by baseline FVIII:C <0.01 IU/mL. FVIII activity has to be confirmed by a central lab. If the confirmatory level is not <0.01IU/mL the child must exit the study.
    3) Subject weighs 3.5 kg or more at the time of his baseline study evaluation
    1) Informerat samtycke är inhämtat och signerat från föräldrar eller juridiskt ombud för dessa. Samtycke och patientinformation är dessförinnan prövad av oberoende etikprövningsnämnd.
    2) Patientens plasmanivå av FVIII är <0.01IU/mL och detta resultat är konfirmerat i centralt lab.
    3) Patienten väger minst 3,5 kg vid tiden för inklusion.
    E.4Principal exclusion criteria
    1) Subjects with prior exposure to clotting factor concentration or blood products.
    2) Subjects with a clinically significant chronic disease other than hemophilia A
    3) Subjects that are currently participating in another investigational clinical trial
    1) Patient som före inklusionen blivit exponerad för blodprodukter eller koagulationskoncentrat
    2) Patient med annan kronisk sjukdom utöver hemofili.
    3) Patient som för närvarande deltar i annan klinisk studie eller prövning
    E.5 End points
    E.5.1Primary end point(s)
    In relation to the primary endpoints, an inhibitor is defined by a Nijmegen test >0.6 BU in two consecutive tests conducted in the central lab. During the first 50 days of exposure to a single FVIII product, the following items will be evaluated:
    - FVIII inhibitor
    - FVIII antibody titer, subclasses and FVIII-binding affinity
    - FVIII-specific T-cell responses
    - Total number of FOXP-3-positive T reg
    - RNA expression for the whole transcriptome
    - F8 gene mutation and other known genomic predictors for inhibitor development
    En inhibitor definieras som ett Nijmegen test >0.6BIU/mL i två upprepade mätningar. Under de första 50 exponeringsdagarna av FVIII kommer följande immunologiska analyser/faktorer studeras:

    - FVIII inhibitor
    - FVIII antibody titer, subclasses and FVIII-binding affinity
    - FVIII-specific T-cell responses
    - Total number of FOXP-3-positive T reg
    - RNA expression for the whole transcriptome
    - F8 gene mutation and other known genomic predictors for inhibitor development
    E.5.1.1Timepoint(s) of evaluation of this end point
    The inhibitor to FVIII will be evaluated during the following visits: day 1, 5, 10, 20, 30, 40 and 50.
    Blodprover kommer insamlas i relation till exponeringsdag 1, 5, 10, 20, 30, 40 och 50
    E.5.2Secondary end point(s)
    The secondary endpoints will be evaluated during the first 50 exposure days. The items recorded in this period are: FVIII usage, bleeding episodes, occurrence of infection and/or immunization
    Under de första 50 exponeringsdagarna kommer följande klinisk information insamlas: FVIII-användning, blödningsepisoder, förekomst av infektioner och immuniseringar.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At study completion (after 50 exposure days or three years, what ever comes first)
    Vid studiens avslutande vilket är efter 50 expo-dagar alt efter 3 års inklusionstid.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    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
    This is a multinational, multicenter study in which a single source of FVIII will be used (Advate) and treatment will be determined by the patient'e physician, including dosing, frequency and regimen (prophylaxis or on demand).
    Detta är en multicenterstudie där Advate används. Vilken behandlingsregim (vid behov eller prophy), behandlingsstart och dosering bestäms av den behandlande läkaren.
    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 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    Last visit last subject (LVLS)
    Last visit last subject (LVLS)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    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: 50
    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: 40
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    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 No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 9
    F.4.2.2In the whole clinical trial 50
    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 will continue to be treated with Advate
    Patienter kommer efter studien att fortsätta behandling med Advate.
    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 Decision2014-11-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-08-22
    P. End of Trial
    P.End of Trial StatusOngoing
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