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    Summary
    EudraCT Number:2016-002828-85
    Sponsor's Protocol Code Number:RG_15-114
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2017-07-21
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2016-002828-85
    A.3Full title of the trial
    Paediatric Hepatic International Tumour Trial
    Ensayo internacional pediátrico en tumor hepático
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Paediatric Hepatic International Tumour Trial
    Ensayo internacional pediátrico en tumor hepático
    A.3.2Name or abbreviated title of the trial where available
    PHITT
    A.4.1Sponsor's protocol code numberRG_15-114
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03017326
    A.5.4Other Identifiers
    Name:ClinicalTrials.govNumber:NCT03017326
    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 SponsorUniversity of Birmingham
    B.1.3.4CountryUnited Kingdom
    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 supportEuropean Commission
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversity of Birmingham
    B.5.2Functional name of contact pointSean Jennings
    B.5.3 Address:
    B.5.3.1Street AddressResearch Governance, University of Birmingham, Edgbaston
    B.5.3.2Town/ cityBirmingham
    B.5.3.3Post codeB15 2TT
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number01214158011
    B.5.6E-mailresearchgovernance@contacts.bham.ac.uk
    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 Cisplatin
    D.2.1.1.2Name of the Marketing Authorisation holderPharmacia Nostrum, S.A.
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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 nameCisplatin
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    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 INNCisplatin
    D.3.9.1CAS number 15663-27-1
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 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 Carboplatin
    D.2.1.1.2Name of the Marketing Authorisation holderAccord Healthcare, S.L.U.
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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 nameCarboplatin
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    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 INNCarboplatin
    D.3.9.1CAS number 41575-94-4
    D.3.9.4EV Substance CodeAS2
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    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 Yes
    D.2.1.1.1Trade name Doxorubicin
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer, S.L.
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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 nameDoxorubicin
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    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 INNDoxorubicin hydrochloride
    D.3.9.1CAS number 25316-40-9
    D.3.9.4EV Substance CodeAS3
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    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 Yes
    D.2.1.1.1Trade name Etoposide
    D.2.1.1.2Name of the Marketing Authorisation holderTeva Pharma, S.L.U.
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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 nameEtoposide
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    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 INNEtoposide
    D.3.9.1CAS number 33419-42-0
    D.3.9.4EV Substance CodeAS4
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    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 Yes
    D.2.1.1.1Trade name Fluorouracil
    D.2.1.1.2Name of the Marketing Authorisation holderAccord Healthcare S.L.U.
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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 nameFluorouracil
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous bolus use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNFluorouracil
    D.3.9.1CAS number 51-21-8
    D.3.9.4EV Substance CodeAS5
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    D.IMP: 6
    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 Gemcitabine
    D.2.1.1.2Name of the Marketing Authorisation holderAurovitas Spain, S.A.U.
    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 nameGemcitabine
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    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 INNGemcitabine
    D.3.9.1CAS number 95058-81-4
    D.3.9.4EV Substance CodeAS6
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    D.IMP: 7
    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 Irinotecan
    D.2.1.1.2Name of the Marketing Authorisation holderHospira UK Limited
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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 nameIrinotecan
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    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 INNIrinotecan
    D.3.9.1CAS number 97682-44-5
    D.3.9.3Other descriptive nameCampto
    D.3.9.4EV Substance CodeAS7
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    D.IMP: 8
    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 Oxaliplatin
    D.2.1.1.2Name of the Marketing Authorisation holderHospira UK Limited
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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 nameOxaliplatin
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    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 INNOxaliplatin
    D.3.9.1CAS number 61825-94-3
    D.3.9.4EV Substance CodeAS8
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    D.IMP: 9
    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 Nexavar
    D.2.1.1.2Name of the Marketing Authorisation holderBayer Pharma AG
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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 nameSorafenib
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSorafenib
    D.3.9.1CAS number 284461-73-0
    D.3.9.4EV Substance CodeAS9
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    D.IMP: 10
    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 Vincristine
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer, S.L.
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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 nameVincristine
    D.3.4Pharmaceutical form Injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous bolus use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNVincristine Sulphate
    D.3.9.1CAS number 57-22-7
    D.3.9.4EV Substance CodeAS10
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 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
    Hepatoblastoma and Hepatocellular Carcinoma.
    Hepatoblastoma y carcinoma hepatocelular.
    E.1.1.1Medical condition in easily understood language
    Hepatoblastoma and Hepatocellular Carcinoma are types of liver cancer.
    El hepatoblastoma y el carcinoma hepatocelular son tipos de cáncer de hígado.
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10019828
    E.1.2Term Hepatocellular carcinoma non-resectable
    E.1.2System Organ Class 100000143675
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10062001
    E.1.2Term Hepatoblastoma
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10019830
    E.1.2Term Hepatocellular carcinoma resectable
    E.1.2System Organ Class 100000143675
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - To evaluate if the outcome with 4 cycles of treatment is not inferior to the outcome with 6 cycles of treatment, for patients with Low Risk HB, resectable tumours.
    - To compare outcome and toxicity in patients with Intermediate Risk HB, treated with one of three treatment regimens: C5VD (cisplatin/5-fluorouracil/vincristine/doxorubicin), SIOPEL-3 high risk (cisplatin, carboplatin and doxorubicin) and cisplatin monotherapy.
    - To compare the outcomes in patients with High Risk HB with metastatic disease, treated with one of two post induction treatments (carboplatin and doxorubicin alternating with carboplatin and etoposide, carboplatin and doxorubicin alternating with vincristine and irinotecan)
    - to determine whether the addition of gemcitabine and oxaliplatin (GEMOX) to cisplatin, doxorubicin and sorafenib improves outcome in unresectable HCC patients
    - to collect biology in all HB and HCC patients
    -Evaluar si la supervivencia con 4 ciclos de tratamiento no es inferior a la supervivencia con 6 ciclos de tratamiento en pacientes con Hepatoblastoma (HB) de bajo riesgo, tumores resecables
    -Comparar la supervivencia y la toxicidad en pacientes con HB de riesgo intermedio, tratados con 1 de los 3 regímenes: C5VD (cisplatino/5-fluorouracilo/vincristina/doxorrubicina), SIOPEL-3 de alto riesgo (cisplatino, carboplatino y doxorrubicina) y monoterapia con cisplatino
    -Comparar supervivencia en pacientes con HB de Alto Riesgo con enfermedad metastásica, tratados con 1 de los 2 tratamientos post inducción (carboplatino y doxorrubicina alternados con carboplatino y etopósido, carboplatino y doxorrubicina alternando con vincristina e irinotecán)
    -Determinar si adición de gemcitabina y oxaliplatino a cisplatino, doxorrubicina y sorafenib mejora la supervivencia en pacientes con Carcinoma Hepatocelular no resecable
    -Recopilar la biología de todos los pacientes con HB y Carcinoma Hepatocelular
    E.2.2Secondary objectives of the trial
    - to report event free survival in all patient groups
    - to evaluate prognostic factors, including the following:
    > to provide a comprehensive and highly validated panel of diagnostic and prognostic biomarkers
    > to determine if paediatric HCC is a biologically different entity to adult HCC
    > to validate prospectively a clinical risk stratification
    > to develop genomic and/or biomarker analysis to predict children who may have an increased risk of developing toxicity with chemotherapy
    - Determinar la supervivencia libre de eventos en todos los grupos de pacientes
    - Evaluar los factores pronósticos, incluyendo los siguientes:
        > Proporcionar un panel completo y altamente validado de biomarcadores diagnósticos y pronósticos
        > Determinar si el Carcinoma Hepatocelular pediátrico es una entidad biológicamente distinta del Carcinoma Hepatocelular adulto
        > Validar prospectivamente una estratificación de riesgo clínico
        > Desarrollar análisis genómicos y / o de biomarcadores para predecir los niños que pueden tener un mayor riesgo de desarrollar toxicidad con la quimioterapia
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    For Trial Entry:
    • Clinical diagnosis of HB or histologically defined diagnosis of HB or HCC.
    • Age ≤30 years
    • Written informed consent for trial entry

    For Allocation/Randomisation to Treatment Group:
    All Groups
    • Written Informed Consent for trial treatment participation
    • Patient assessed as fit to receive group specific treatment
    • For females of child-bearing potential, a negative pregnancy test prior to trial entry is required. Any patient who is of reproductive age must agree to use adequate contraception for the duration of the trial.

    Group A (no treatment arm)
    At diagnosis:
    • Resected Tumour.
    • Patient meets Very Low Risk definition according to CHIC guidelines.
    Group A1 – No treatment arm
    • Central pathology review confirming WDF histology.
    Group A2 - Treatment arm
    • Central pathology review confirming non-WDF histology.
    • Adequate renal function determined by:
    o Serum creatinine in the normal range based on age appropriate local reference values or glomerular filtration rate (GFR) ≥60mL/min/1.73m2
    • Adequate haematology:
    o Absolute neutrophil count (ANC) >0.75 x 109/L
    o Platelet count >75 x 109/L
    o Prothrombin time (PT) <1.2x ULN for age-based local reference values

    Group B
    • Patient meets Low Risk definition according to CHIC Guidelines
    • Adequate renal function determined by:
    o Serum creatinine in the normal range based on age appropriate local reference values or glomerular filtration rate (GFR) ≥60mL/min/1.73m2
    • Adequate haematology:
    o Absolute neutrophil count (ANC) >0.75 x 109/L
    o Platelet count >75 x 109/L
    o Prothrombin time (PT) <1.2x ULN for age-based local reference values

    Group C
    • Patient meets Intermediate Risk definition according to CHIC Guidelines
    • Adequate renal function determined by:
    o Serum creatinine in the normal range based on age appropriate local reference values or glomerular filtration rate (GFR) ≥60mL/min/1.73m2
    • Adequate cardiac function determined by:
    o Shortening fraction ≥28% by local assessment method
    o OR Ejection fraction ≥47% by local assessment method
    • Adequate haematology:
    o Absolute neutrophil count (ANC) >0.75 x 109/L
    o Platelet count >75 x 109/L
    o Prothrombin time (PT) <1.2x ULN for age-based local reference values

    Group D
    • Patient meets High Risk definition according to CHIC Guidelines
    • Adequate renal function determined by:
    o Serum creatinine in the normal range based on age appropriate local reference values or glomerular filtration rate (GFR) ≥60mL/min/1.73m2
    • Adequate cardiac function determined by:
    o Shortening fraction ≥28% by local assessment method
    o OR Ejection fraction ≥47% by local assessment method
    • Adequate haematology:
    o Absolute neutrophil count (ANC) >0.75 x 109/L
    o Platelet count >75 x 109/L
    o Prothrombin time (PT) <1.2x ULN for age-based local reference values

    Group E
    • Patient has been diagnosed with HCC
    • Tumour has been locally assessed as resectable
    • Adequate renal function determined by:
    o Serum creatinine in the normal range based on age appropriate local reference values or glomerular filtration rate (GFR) ≥60mL/min/1.73m2
    • Adequate cardiac function determined by:
    o Shortening fraction ≥28% by local assessment method
    o OR Ejection fraction ≥47% by local assessment method
    • Adequate haematology:
    o Absolute neutrophil count (ANC) >0.75 x 109/L
    o Platelet count >75 x 109/L
    o Prothrombin time (PT) <1.2x ULN for age-based local reference values

    Group F
    • Patient diagnosed with HCC
    • Tumour locally assessed as un-resectable, or metastatic HCC disease
    • Adequate renal function determined by:
    o Serum creatinine in the normal range based on age appropriate local reference values or glomerular filtration rate (GFR) ≥60mL/min/1.73m2
    • Adequate cardiac function determined by:
    o Shortening fraction ≥28% by local assessment method
    o OR Ejection fraction ≥47% by local assessment method
    • Adequate haematology:
    o Absolute neutrophil count (ANC) >0.75 x 109/L
    o Platelet count >75 x 109/L
    o Prothrombin time (PT) <1.2x ULN for age-based local reference values
    Para la entrada en el ensayo:
    • Diagnóstico clínico de Hepatoblastoma (HB) o diagnóstico histológico de Hepatoblastoma o Carcinoma Hepatocelular (CHC).
    • Edad ≤ 30 años
    • Consentimiento informado por escrito para la entrada en el ensayo

    Para asignación / aleatorización al grupo de tratamiento:
    Todos los Grupos
    • Consentimiento informado por escrito para la participación en el tratamiento en estudio
    • Paciente evaluado como adecuado para recibir tratamiento específico del grupo
    • Para mujeres potencialmente fértiles, se requiere una prueba de embarazo negativa antes de entrar en el estudio. Cualquier paciente que esté en edad reproductiva debe acordar el uso de anticoncepción adecuada durante la duración del ensayo.

    Grupo A (sin brazo de tratamiento)
    Al momento del diagnóstico:
    • Tumor Resecado.
    • El paciente cumple la definición de HB de riesgo muy bajo según las pautas de CHIC.
    Grupo A1 - Sin brazo de tratamiento
    • Revisión de patología central confirmando la histología de WDF.
    Grupo A2 - Brazo de tratamiento
    • Revisión de la patología central confirmando la histología no-WDF.
    • Función renal adecuada determinada por:
      - Creatinina sérica en el rango normal basado en valores de referencia locales apropiados para la edad o tasa de filtración glomerular (TFG) ≥60mL / min / 1.73m2
    • Hematología adecuada:
      - Número absoluto de neutrófilos (ANC)> 0,75 x 109 / L
      - Recuento de plaquetas> 75 x 109 / L
      - Tiempo de protrombina (PT) <1,2x LSN para valores de referencia locales basados ​​en la edad

    Grupo B
    • El paciente cumple la definición de HB de bajo riesgo según las directrices de CHIC
    • Función renal adecuada determinada por:
      - Creatinina sérica en el rango normal basado en valores de referencia locales apropiados para la edad o tasa de filtración glomerular (TFG) ≥60mL / min / 1.73m2
    • Hematología adecuada:
      - Número absoluto de neutrófilos (ANC)> 0,75 x 109 / L
      - Recuento de plaquetas> 75 x 109 / L
      - Tiempo de protrombina (PT) <1,2x LSN para valores de referencia locales basados ​​en la edad

    Grupo C
    • El paciente cumple la definición de HB de riesgo intermedio de acuerdo con las directrices de CHIC
    • Función renal adecuada determinada por:
       - Creatinina sérica en el rango normal basado en valores de referencia locales apropiados para la edad o tasa de filtración glomerular (TFG) ≥60mL / min / 1.73m2
    • Función cardíaca adecuada determinada por:
      - Fracción de acortamiento ≥28% según el método de evaluación local
      - O Fracción de eyección ≥47% según el método de evaluación local
    • Hematología adecuada:
      - Número absoluto de neutrófilos (ANC)> 0,75 x 109 / L
      - Recuento de plaquetas> 75 x 109 / L
      - Tiempo de protrombina (PT) <1,2x LSN para valores de referencia locales basados ​​en la edad

    Grupo D
    • El paciente cumple la definición de HB de Alto Riesgo según las directrices de CHIC
    • Función renal adecuada determinada por:
       - Creatinina sérica en el rango normal basado en valores de referencia locales apropiados para la edad o tasa de filtración glomerular (TFG) ≥60mL / min / 1.73m2
    • Función cardíaca adecuada determinada por:
      - Fracción de acortamiento ≥28% según el método de evaluación local
      - O Fracción de eyección ≥47% según el método de evaluación local
    • Hematología adecuada:
      - Número absoluto de neutrófilos (ANC)> 0,75 x 109 / L
      - Recuento de plaquetas> 75 x 109 / L
      - Tiempo de protrombina (PT) <1,2x LSN para valores de referencia locales basados ​​en la edad

    Grupo E
    • El paciente ha sido diagnosticado con CHC
    • El tumor se ha evaluado localmente como resecable
    • Función renal adecuada determinada por:
       - Creatinina sérica en el rango normal basado en valores de referencia locales apropiados para la edad o tasa de filtración glomerular (TFG) ≥60mL / min / 1.73m2
    • Función cardíaca adecuada determinada por:
      - Fracción de acortamiento ≥28% según el método de evaluación local
      - O Fracción de eyección ≥47% según el método de evaluación local
    • Hematología adecuada:
      - Número absoluto de neutrófilos (ANC)> 0,75 x 109 / L
      - Recuento de plaquetas> 75 x 109 / L
      - Tiempo de protrombina (PT) <1,2x LSN para valores de referencia locales basados ​​en la edad

    Grupo F
    • Paciente diagnosticado con CHC
    • Tumor evaluado localmente como enfermedad de CHC no resecable o metastásica
    • Función renal adecuada determinada por:
       - Creatinina sérica en el rango normal basado en valores de referencia locales apropiados para la edad o tasa de filtración glomerular (TFG) ≥60mL / min / 1.73m2
    • Función cardíaca adecuada determinada por:
      - Fracción de acortamiento ≥28% según el método de evaluación local
      - O Fracción de eyección ≥47% según el método de evaluación local
    • Hematología adecuada:
      - Número absoluto de neutrófilos (ANC)> 0,75 x 109 / L
      - Recuento de plaquetas> 75 x 109 / L
      - Tiempo de protrombina (PT) <1,2x LSN para valores de referencia locales basados ​​en la edad
    E.4Principal exclusion criteria
    • Any previous chemotherapy or currently receiving anti-cancer agents
    • Recurrent disease
    • Previously received a solid organ transplant
    • Uncontrolled infection
    • Unable to follow the protocol for any reason
    • Second malignancy
    • Pregnant or breastfeeding women
    • Cualquier quimioterapia previa o que reciba actualmente agentes anticancerígenos
    • Enfermedad recurrente
    • Recibió previamente un trasplante de órgano sólido
    • Infección no controlada
    • Incapaz de seguir el protocolo por cualquier motivo
    • Segunda malignidad
    • Mujeres embarazadas o lactantes
    E.5 End points
    E.5.1Primary end point(s)
    Event Free Survival (EFS) as defined as the time from randomisation (or registration into the trial for non-randomised patients) to the first failure event. Patients who have not had an event will be censored at their last follow-up date.
    Failure events are:
    • progression of existing disease or occurrence of disease at new sites,
    • death from any cause prior to disease progression,
    • diagnosis of a second malignant neoplasm.

    Response in HCC is defined as complete (CR) or partial (PR) response according to RECIST version 1.1 criteria. The assessment will be performed after 3 cycles of PLADO, or 4 cycles of PLADO+S/GEMOx+S in Group F patients. Patients who are not assessable for response, e.g. because of early stopping of treatment or death, will be assumed to be non-responders.
    Supervivencia Libre de Eventos, tal como se define como el tiempo desde la asignación al azar (o el registro en el ensayo para pacientes no aleatorios) hasta el primer evento. Los pacientes que no hayan tenido un evento serán censurados en su última fecha de seguimiento.
    Los eventos se definen como:
    • la progresión de la enfermedad existente o la aparición de la enfermedad en nuevas localizaciones,
    • muerte por cualquier causa antes de la progresión de la enfermedad,
    • diagnóstico de una segunda neoplasia maligna.

    La respuesta en CHC se define como una respuesta completa (CR) o parcial (PR) de acuerdo con los criterios de RECIST versión 1.1. La evaluación se realizará después de 3 ciclos de PLADO, o 4 ciclos de PLADO + S / GEMOx + S en pacientes del Grupo F. Los pacientes que no son evaluables para la respuesta, por ejemplo, debido a la detención temprana del tratamiento o la muerte, se supondrá que no responden.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Evaluation of EFS will be until first failure event.
    Evaluation of Response in HCC will be until end of treatment.
    La evaluación de la Supervivencia Libre de Eventos será hasta que ocurra el primer evento.
    La evaluación de la respuesta en CHC será hasta el final del tratamiento.
    E.5.2Secondary end point(s)
    Failure-Free Survival (FFS) is defined as per EFS (primary endpoint above) with the addition of failure to go to resection.
    Overall Survival (OS) is defined as the time from randomisation (or registration for non-randomised patients) to death from any cause.
    Toxicity will be recorded in relation to each cycle of randomised treatment and will be categorised and graded using Common Terminology Criteria for Adverse Events (CTCAE)
    Chemotherapy-related cardiac, nephro- and oto- toxicity will be recorded in relation to each cycle of non-randomised treatment and will be categorised by CTCAE.
    Hearing loss is defined as Grade 4 Boston grade oto-toxicity compared to baseline.
    Best Response is defined as Complete Response (CR) or Partial Response (PR) and is defined in the protocol based on radiological (RECIST) response and AFP decline.Patients who are not assessable for response (e.g. because of early stopping of treatment or death) will be assumed to be non-responders.
    Surgical resectability id defined as complete resection, partial resection or transplant following randomisation (or enrolement for non-randomised patients).
    Adherence to surgical guidelines is defined as the local clinician's surgical decision to resect or not compared to the current SIOPEL surgical guidelines.
    La Supervivencia Libre de Fracaso está definida por la Supervivencia Libre de Eventos (criterio de valoración primario anterior) con la adición del fallo a la hora de resecar el tumor.
    Supervivencia global se define como el tiempo desde la asignación al azar (o registro para pacientes no aleatorizados) hasta la muerte por cualquier causa.
    La toxicidad se registrará en relación con cada ciclo de tratamiento aleatorizado y será categorizada y graduada utilizando Criterios Terminológicos Comunes para Eventos Adversos (CTCAE, por sus siglas en inglés)
    Se registrará la toxicidad cardíaca, nefro- y oto-tóxica relacionada con la quimioterapia en relación con cada ciclo de tratamiento no aleatorio y se clasificará por CTCAE.
    La pérdida auditiva se define como toxicidad de grado de grado 4 de Boston comparada con el estado basal.
    La mejor respuesta se define como Respuesta Completa (CR) o Respuesta Parcial (PR) y se define en el protocolo basado en la respuesta radiológica (RECIST) y la disminución de la AFP. Los pacientes que no son evaluables por respuesta (por ejemplo, debido a la detención temprana del tratamiento o la muerte ) se supondrán como no respondedores.
    La resecabilidad quirúrgica se define como resección completa, resección parcial o trasplante después de la asignación al azar (o enrolamiento para pacientes no aleatorios).
    La adhesión a las guías quirúrgicas se define como la decisión quirúrgica del clínico local de resecar o no en comparación con las directrices quirúrgicas actuales de SIOPEL.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Evaluation of FFS will be until first failure event, including failure to resect.
    Evaluation of OS and Best Response will be until the last follow up.
    Evaluation of Toxicity and Chemotherapy-related toxicity will be until 30 days after End of Treatment.
    Evaluation of Hearing Loss, Surgical resectability and Adherence to surgical guidelines will be until End of Treatment.
    La evaluación de Supervivencia Libre de Fracaso será hasta el primer evento de fallo, incluyendo la falta de resección.
    Evaluación de la Supervivencia Global y la mejor respuesta será hasta el último seguimiento.
    La evaluación de la toxicidad y de la toxicidad relacionada con la quimioterapia será hasta 30 días después del fin del tratamiento.
    Evaluación de la Pérdida Auditiva, resecabilidad quirúrgica y Adherencia a las directrices quirúrgicas será hasta el final del tratamiento.
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic Yes
    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) 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 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 trial6
    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 concerned15
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA140
    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
    Czech Republic
    France
    Germany
    Ireland
    Italy
    Netherlands
    Norway
    Poland
    Spain
    Sweden
    Switzerland
    United Kingdom
    E.8.7Trial has a data monitoring committee No
    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 will remain open until the date of the the last patient's last visit.
    Última visita del último paciente.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days31
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months5
    E.8.9.2In all countries concerned by the trial days31
    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: 270
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Yes
    F.1.1.2.1Number of subjects for this age range: 10
    F.1.1.3Newborns (0-27 days) Yes
    F.1.1.3.1Number of subjects for this age range: 20
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 150
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 50
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 40
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 30
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 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 state60
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 300
    F.4.2.2In the whole clinical trial 1140
    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)
    On completing protocol defined treatment, their treating physician will discuss further treatment options with them and their family as per standard care.
    Una vez completado el tratamiento indicado en el protocolo, su médico discutirá con los sujetos y sus familias las opciones terapéuticas existentes dentro de la práctica clínica habitual.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation CINECA
    G.4.3.4Network Country Italy
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-08-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-08-21
    P. End of Trial
    P.End of Trial StatusOngoing
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