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    Summary
    EudraCT Number:2009-011186-88
    Sponsor's Protocol Code Number:ABB–09–001
    National Competent Authority:Slovakia - SIDC (Slovak)
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2013-12-05
    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 ConcernedSlovakia - SIDC (Slovak)
    A.2EudraCT number2009-011186-88
    A.3Full title of the trial
    Inhibitor Development in Previously Untreated Patients (PUPs) or Minimally Blood Component-Treated Patients (MBCTPs) when Exposed to plasma-derived von Willebrand Factor-Containing Factor VIII (VWF/FVIII) Concentrates and to Recombinant Factor VIII (rFVIII) Concentrates: An Independent, International, Multicentre, Prospective, Controlled, Randomised, Open Label, Clinical Trial
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Development of antibody against FVIII (inhibitors) in Untreated Patients or Minimally Blood Component-Treated Patients when Exposed Factor concentrates which can be made from human blood (plasma-derived) or manufactured in laboratory using genetically engineered cells that carry a human factor gene (recombinant): A non-commercial, international study with lots of sites and it is known which of the two by random assigned drugs will be taken
    A.3.2Name or abbreviated title of the trial where available
    SIPPET
    A.4.1Sponsor's protocol code numberABB–09–001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01064284
    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 SponsorFONDAZIONE ANGELO BIANCHI BONOMI
    B.1.3.4CountryItaly
    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 supportFondazione Angelo Bianchi Bonomi
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSintesi Research s.r.l.
    B.5.2Functional name of contact pointClinical Operation
    B.5.3 Address:
    B.5.3.1Street AddressVia G. Ripamonti 89
    B.5.3.2Town/ cityMilan
    B.5.3.3Post code20141
    B.5.3.4CountryItaly
    B.5.4Telephone number+3902873512
    B.5.5Fax number+390297374301
    B.5.6E-mailsippet-info@sintesiresearch.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 Fanhdi 500 I.E.
    D.2.1.1.2Name of the Marketing Authorisation holderGrifols Deutschland GmbH
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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.3.4Pharmaceutical 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 INNOctocog alfa.
    D.3.9.1CAS number 0
    D.3.9.3Other descriptive nameHUMAN COAGULATION FACTOR VIII
    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) Yes
    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 KOGENATE Bayer 500 IU
    D.2.1.1.2Name of the Marketing Authorisation holderBayer HealthCare AG
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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.3.4Pharmaceutical 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.3.9.1CAS number 0
    D.3.9.3Other descriptive nameHUMAN COAGULATION FACTOR VIII
    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 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 Yes
    D.3.11.13.1Other medicinal product typerecombinat medicinal product produced by genetically modified baby hamster kidney cells.
    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
    Inhibitor Development in Previously Untreated Patients (PUPs) or Minimally Blood Component-Treated Patients (MBCTPs) when Exposed to plasma-derived von Willebrand Factor-Containing Factor VIII (VWF/FVIII) Concentrates and to Recombinant Factor VIII (rFVIII) Concentrates: An Independent, International, Multicentre, Prospective, Controlled, Randomised, Open Label, Clinical Trial
    E.1.1.1Medical condition in easily understood language
    Develop. of antibody against FVIII in Patients when Exposed Factor concentrates made from human blood or manufactured in lab using genetically engineered cells that carry a human factor gene
    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 16.1
    E.1.2Level LLT
    E.1.2Classification code 10018937
    E.1.2Term Haemophilia A
    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 assess the immunogenicity of VWF/FVIII and of rFVIII concentrates by determining the frequency of inhibitor development in PUPs and MBCTs in the first 50 EDs or in the first 3 years from enrolment, whichever comes first
    E.2.2Secondary objectives of the trial
    To evaluate
    •the anamnestic response of inhibitor patients
    •the frequency of transient inhibitors
    •the modality of occurrence of inhibitors (number of EDs, titre at onset, etc)
    •clinical factors potentially associated to inhibitor development (age at first treatment, severity of bleeding episodes, surgery, intensity of treatment, modality of treatment, time of treatment in relation to vaccinations, concurrent diseases (in particular viral infections), complications of venous access and/or medications, type of delivery, breast feeding, type of milk formula)
    •laboratory factors potentially associated to inhibitor development (severity of FVIII defect, FVIII gene defect, FVIII Ag, HLA phenotype, IL-10.G, TNF alpha, CTLA-4 polymorphisms), analyses of FVIII mRNA expression levels, epitope mapping, FII and •FV gain-of-function thrombophilic mutation
    the incidence of all other adverse events related and not related to the products used
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    •Male subjects
    •Any ethnicity
    •Age <6 years
    •Severe haemophilia A (FVIII:C <1%), as confirmed by the central laboratory
    o Patients with FVIII levels >1% and < 2% will be separately recorded in the screening list
    •Previously untreated (0 EDs to any FVIII concentrate or blood products) or minimally treated (<5 EDs) with blood components, namely whole blood, fresh frozen plasma, packed red blood cells, platelets or cryoprecipitate
    o Patients not meeting these criteria will be separately recorded in the screening list
    •Negative inhibitor measurement at both local and central laboratory at screening
    •Ability to comply with study requirements
    •Signed informed consent of legal tutors
    o Patients who will not accept to enter into the study or to be randomized will be separately recorded

    E.4Principal exclusion criteria
    •Plasma FVIII level ≥1%, as assayed at the central laboratory
    o Those patients originally diagnosed locally as severe but subsequently found to have FVIII levels ranging from 1% to 2% on testing at the central laboratory will be separately recorded in the screening list.
    •Previous history of FVIII inhibitor
    •Other congenital or acquired bleeding defects
    •Concomitant congenital or acquired immunodeficiency
    •Concomitant treatment with systemic immunosuppressive drugs
    •Concomitant treatment with any investigational drug
    E.5 End points
    E.5.1Primary end point(s)
    •To assess the immunogenicity of plasma derived VWF/FVIII and rFVIII concentrates by determining the frequency of inhibitor development in the first 50 EDs or in the first 3 years from enrolment, whichever comes first in PUPs and MBCTs
    E.5.1.1Timepoint(s) of evaluation of this end point
    after development of the inhibitor, first 50 EDs or 3 years from enrolment
    E.5.2Secondary end point(s)
    anamnestic response of inhibitor patients, frequency of transient inhibitors, modality of occurrence of inhibitors, clinical factors potentially associated to inhibitor development, lab factors potentially associated to inhibitor development, analyses of FVIII mRNA expression levels, epitope mapping, FII & FV gain-of-function thrombophilic mutations, incidence of all other adverse events related and not related to the products used
    E.5.2.1Timepoint(s) of evaluation of this end point
    after development of the inhibitor, first 50 EDs or 3 years from enrolment
    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 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 No
    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 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 Yes
    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) Yes
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA29
    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
    Belgium
    France
    Italy
    Austria
    Portugal
    Romania
    Argentina
    Brazil
    Chile
    Colombia
    Egypt
    Germany
    India
    Iran, Islamic Republic of
    Saudi Arabia
    Spain
    Mexico
    Poland
    South Africa
    Turkey
    United Kingdom
    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
    The trial ends for each after 50 Exposure Days (treatment of patients with IMP) or 3 years after enrolment, whichever comes first or after inhibitor development. If an inhibitor development occurs (confirmation of the central laboratory), the patient must returs to the site every month for 6 months to verify the inhibitor plasmatic levels.
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years8
    E.8.9.2In all countries concerned by the trial months0
    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: 300
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 300
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 300
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 300
    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 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 Yes
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    MALE SUBJECT <6 YEARS
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 300
    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 the end of the study, the investigator will decide together with the parent of the patient, if the treatment schedule of the trial is changed or not. Because the IMPs are both licensed in Germany, they are freely available at the market and are in use at the sites for treatment of hemophilia A.
    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 Decision2014-02-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-01-18
    P. End of Trial
    P.End of Trial StatusOngoing
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