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    Summary
    EudraCT Number:2011-002184-17
    Sponsor's Protocol Code Number:TRA115450
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-03-08
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2011-002184-17
    A.3Full title of the trial
    A two part, double-blind, randomized, placebo-controlled and open-label study to investigate the efficacy, safety and tolerability of eltrombopag, a thrombopoietin receptor agonist, in pediatric patients with previously treated chronic immune (idiopathic) thrombocytopenic purpura (ITP).PETIT2: Eltrombopag in PEdiatric patients with Thrombocytopenia from ITP
    Studio suddiviso in due parti, una in doppio cieco, randomizzata e controllata verso placebo e una in aperto, per studiare l'efficacia, la sicurezza e la tollerabilita' di eltrombopag, un agonista del recettore della trombopoietina, in pazienti pediatrici con porpora trombocitopenica immune (idiopatica) (PTI) cronica precedentemente trattati. PETIT2: Eltrombopag in pazienti PEdiatrici con Trombocitopenia da PTI
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of a new medication for childhood chronic immune thrombocytopenic purpura, ITP, a blood disorder of low platelet counts that can lead to bruising easily, bleeding gums, and/or bleeding inside the body.
    Studio su un nuovo medicinale per la porpora trombocitopenica immune (PTI) cronica infantile, un disturbo del sangue caratterizzato da un livello basso di piastrine, che puo' facilitare la formazione di ematomi e provocare sanguinamento gengivale e/o emorragia interna.
    A.3.2Name or abbreviated title of the trial where available
    PETIT2
    PETIT2
    A.4.1Sponsor's protocol code numberTRA115450
    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 SponsorGLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD.
    B.1.3.4CountryUnited Kingdom
    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 supportGlaxoSmithKline R&D
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGlaxoSmithKline Research & Development
    B.5.2Functional name of contact pointClinical Trial Helpdesk
    B.5.3 Address:
    B.5.3.1Street Address1-3 Iron Bridge Road
    B.5.3.2Town/ cityUxbridge, Middlesex
    B.5.3.3Post codeUB11 1BU
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+44 02089904466
    B.5.5Fax number+44 02089901234
    B.5.6E-mailGSKClinicalSupportHD@gsk.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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/07/467
    D.3 Description of the IMP
    D.3.1Product nameELTROMBOPAG
    D.3.2Product code SB-497115
    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 INNELTROMBOPAG
    D.3.9.1CAS number 496775-61-2
    D.3.9.2Current sponsor codeSB-497115
    D.3.9.3Other descriptive nameREVOLADE, PROMACTA
    D.3.9.4EV Substance CodeSUB30140
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number12.5
    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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/07/467
    D.3 Description of the IMP
    D.3.1Product nameELTROMBOPAG
    D.3.2Product code SB-497115
    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 INNELTROMBOPAG
    D.3.9.1CAS number 496775-61-2
    D.3.9.2Current sponsor codeSB-497115
    D.3.9.3Other descriptive nameREVOLADE, PROMACTA
    D.3.9.4EV Substance CodeSUB30140
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/07/467
    D.3 Description of the IMP
    D.3.1Product nameELTROMBOPAG
    D.3.2Product code SB-497115
    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 INNELTROMBOPAG
    D.3.9.1CAS number 496775-61-2
    D.3.9.2Current sponsor codeSB-497115
    D.3.9.3Other descriptive nameREVOLADE, PROMACTA
    D.3.9.4EV Substance CodeSUB30140
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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: 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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/07/467
    D.3 Description of the IMP
    D.3.1Product nameELTROMBOPAG
    D.3.2Product code SB-497115
    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 INNELTROMBOPAG
    D.3.9.1CAS number 496775-61-2
    D.3.9.2Current sponsor codeSB-497115
    D.3.9.3Other descriptive nameREVOLADE, PROMACTA
    D.3.9.4EV Substance CodeSUB30140
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number75
    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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/07/467
    D.3 Description of the IMP
    D.3.1Product nameELTROMBOPAG
    D.3.2Product code SB-497115
    D.3.4Pharmaceutical form Powder for oral suspension
    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 INNELTROMBOPAG
    D.3.9.1CAS number 496775-61-2
    D.3.9.2Current sponsor codeSB-497115
    D.3.9.3Other descriptive nameREVOLADE, PROMACTA
    D.3.9.4EV Substance CodeSUB30140
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 3
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral suspension
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    To assess the efficacy of eltrombopag, relative to placebo, in achieving platelet counts of ≥ 50 Gi/L, when administered to pediatric subjects with previously treated chronic ITP during the first 12 weeks of Part 1, the randomized treatment period.
    Valutare l'efficacia di eltrombopag rispetto al placebo nell'ottenimento di conte piastriniche ≥ 50 Gi/l, quando somministrato a soggetti pediatrici con ITP cronica, precedentemente trattata, durante le prime 12 settimane della Parte 1 dello studio, il periodo di trattamento randomizzato.
    E.1.1.1Medical condition in easily understood language
    childhood chronic immune thrombocytopenic purpura, ITP, a blood disorder of low platelet counts that can lead to bruising easily, bleeding gums, and/or bleeding inside the body
    PTI cronica infantile,disturbo del sangue caratterizzato da livello basso di piastrine,che può facilitare la formazione di ematomi e provocare sanguinamento gengivale e/o emorragia interna
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10021245
    E.1.2Term Idiopathic thrombocytopenic purpura
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    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 efficacy of eltrombopag, relative to placebo, in achieving platelet counts of ≥ 50 Gi/L, when administered to pediatric subjects with previously treated chronic ITP during the first 12 weeks of Part 1, the randomized treatment period.
    Valutare l'efficacia di eltrombopag rispetto al placebo nell'ottenimento di conte piastriniche ≥ 50 Gi/l, quando somministrato a soggetti pediatrici con ITP cronica, precedentemente trattata, durante le prime 12 settimane della Parte 1 dello studio, il periodo di trattamento randomizzato.
    E.2.2Secondary objectives of the trial
    • efficacy of eltrombopag in achieving platelet counts ≥ 50 Gi/L when administered to pediatric subjects with previously treated chronic ITP.• efficacy of eltrombopag in achieving sustained platelet counts ≥ 50 Gi/L when administered to pediatric subjects with previously treated chronic ITP.• effect of eltrombopag on reduction and/or interruption of concomitant ITP therapies, when administered for 24 weeks to pediatric subjects with previously treated chronic ITP.• effect of eltrombopag on the need for rescue ITP medication when administered to pediatric subjects with previously treated chronic ITP.• efficacy of eltrombopag in decreasing the incidence and severity of bleeding symptoms when administered in pediatric subjects with previously treated chronic.Please refer to the protocol for The remaining objectives P12
    ·efficacia di eltrombopag nell'ottenimento di conte piastriniche ≥ 50 Gi/l quando somministrato a soggetti pediatrici con ITP cronica precedentemente trattata.·efficacia di eltrombopag nell'ottenimento duraturo di conte piastriniche ≥ 50 Gi/l quando somministrato a soggetti pediatrici con ITP cronica precedentemente trattata.·effetti di eltrombopag sulla riduzione e/o l'interruzione delle terapie concomitanti per l’ITP, quando somministrato per 24 settimane a soggetti pediatrici con ITP cronica precedentemente trattata.·effetti di eltrombopag relativamente alla necessità di utilizzare farmaci di salvataggio per l'ITP in soggetti pediatrici con ITP cronica precedentemente trattata.·efficacia di eltrombopag nella riduzione dell'incidenza e della gravità dei sintomi emorragici quando somministrato a soggetti pediatrici con ITP cronica precedentemente trattata.Vedi protocollo obiettivi P12
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Written informed consent must be obtained from the subject's guardian and accompanying informed assent from the subject (for children over 6 years old). 2. Subjects must be between 1 year and <18 years of age at Day 1. 3. Subjects will have a confirmed diagnosis of chronic ITP for at least 1 year, at screening, according to the guidelines published in the International Working Group Report [Rodeghiero, 2009]. 4. A peripheral blood smear or bone marrow examination will support the diagnosis of ITP with no evidence of other causes of thrombocytopenia. 5. Subjects must be refractory or have relapsed after at least one prior ITP therapy, or subjects must be unable, for a medical reason, to continue other ITP treatments. 6. Subjects must have a Day 1 (or within 48 hours prior) platelet count <30 Gi/L. 7. Previous therapy for ITP with immunoglobulins (IVIg and anti-D) must have been completed at least 2 weeks prior to Day 1, or these therapies must have been completed at least 1 week prior to Day 1 and have been clearly ineffective. 8. Previous treatment for ITP with splenectomy, rituximab and cyclophosphamide must have been completed at least 4 weeks prior to Day 1. 9. Subjects treated with concomitant ITP medication (e.g. corticosteroids or azathioprine) must be receiving a dose that has been stable for at least 4 weeks prior to Day 1. 10. Subjects must have a complete blood count (CBC) not suggestive of another hematological disorder. 11. Subjects must have the following laboratory results: • prothrombin time international normalized ratio (INR) and activated partial thromboplastin time (aPTT) within 80 to 120% of the normal range. • clinical chemistries that do NOT exceed the upper limit of normal (ULN) reference range by more than 20% for the following: creatinine, ALT, AST, total bilirubin, and alkaline phosphatase. • total albumin that is not below the lower limit of normal (LLN) by more than 10%. 12. Female subjects of child-bearing potential (after menarche) must: • have a negative pregnancy test within 24 hours of first dose of study treatment, • agree and be able to provide a blood or urine specimen for pregnancy testing during the study, • agree to use effective contraception, as defined in Section 7.3.3, during the study and for 28 days following the last dose of study treatment, and • not be lactating. 13. Male subjects with a female partner of childbearing potential must agree to use effective contraception as described in Section 7.3.3 from 2 weeks prior to administration of the first dose of study treatment until 3 months after the last dose of study treatment. 14. In France, a subject will be eligible for inclusion in this study only if either affiliated to or a beneficiary of a social security category.
    1.Deve essere stato ottenuto il consenso informato scritto dal tutore del soggetto e l'assenso informato che lo accompagna dal soggetto (per i bambini sopra ai 6 anni di età). 2.Il Giorno 1 i soggetti devono avere un'età compresa tra 1 e &lt; 18 anni. 3.I soggetti allo screening devono avere una diagnosi confermata di ITP cronica da almeno 1 anno, secondo le linee guida pubblicate nell'International Working Group Report [Rodeghiero, 2009]. 4.Uno striscio di sangue periferico o un esame del midollo osseo confermerà la diagnosi di ITP in assenza di evidenze di altre cause di trombocitopenia. 5.I soggetti devono essere refrattari o recidivanti dopo almeno una terapia precedente per l'ITP, o devono essere impossibilitati, per motivi medici, a proseguire altri trattamenti per l'ITP. 6.I soggetti devono avere una conta piastrinica al Giorno 1 (o entro le 48 ore precedenti) &lt;30 Gi/l. 7.La terapia precedente per l'ITP con immunoglobuline (IVIg e anti-D), deve essere stata completata almeno 2 settimane prima del Giorno 1, o queste terapie devono essere state completate almeno 1 settimana prima del Giorno 1 ed essere state chiaramente inefficaci. 8.Il trattamento precedente per l'ITP con splenectomia, rituximab e ciclofosfamide deve essere stato completato almeno 4 settimane prima del Giorno 1. 9.I soggetti trattati con farmaci concomitanti per l'ITP (ad es. corticosteroidi o azatioprina) devono ricevere una dose stabile da almeno 4 settimane prima del Giorno 1. 10.I soggetti devono avere un emocromo completo (CBC) non indicativo di un altro disturbo ematologico. 11.I soggetti devono ottenere i seguenti risultati di laboratorio: •rapporto internazionale normalizzato (INR) del tempo di protrombina e tempo di tromboplastina parziale attivata (aPTT) entro l'80 e il 120% dell'intervallo di normalità. • valori dei test di chimica clinica NON superi il limite superiore di normalità (ULN) di oltre il 20% per i seguenti analiti: creatinina, ALT, AST, bilirubina totale e fosfatasi alcalina.•albumina totale non al di sotto del limite inferiore di normalità (LLN) di oltre il 10%. 12.I soggetti di sesso femminile potenzialmente fertili (dopo il menarca) devono: •avere un risultato negativo al test di gravidanza negativo nelle 24 ore precedenti la prima dose di trattamento sperimentale,•accettare ed essere in grado di fornire un campione di sangue o di urine per i test di gravidanza durante lo studio,•accettare di utilizzare un metodo contraccettivo efficace, durante lo studio e per 28 giorni dopo l'ultima dose di trattamento sperimentale, e•non allattare. 13.I soggetti di sesso maschile con una partner femminile potenzialmente fertile devono accettare di utilizzare un metodo contraccettivo efficace, a partire da 2 settimane prima della prima dose di trattamento sperimentale e fino a 3 mesi dopo l'ultima dose di trattamento sperimentale.
    E.4Principal exclusion criteria
    1. Subjects with any clinically relevant abnormality, other than ITP, identified on the screening examination or any other medical condition or circumstance, which in the opinion of the investigator makes the subject unsuitable for participation in the study or suggests another primary diagnosis (e.g. Thrombocytopenia is secondary to another disease). 2. Subjects with concurrent or past malignant disease, including myeloproliferative disorder.3. Subjects expected not to be suitable for continuation of their current therapy for at least 13 additional weeks. 4. Subjects with a history of platelet agglutination abnormality that prevents reliable measurement of platelet counts. 5. Subjects with a diagnosis of secondary immune thrombocytopenia, including those with laboratory or clinical evidence of HIV infection, antiphospholipid antibody syndrome, chronic hepatitis B infection, hepatitis c virus infection, or any evidence of active hepatitis at the time of subject screening. 6. Subjects with Evans syndrome (autoimmune thrombocytopenia and autoimmune hemolysis). 7. Subjects with known inherited thrombocytopenia (e.g. MYH9 disorders). 8. Subjects treated with any medication that affects platelet function (including but not limited to aspirin, clopidogrel and/or NSAIDS) or anticoagulants for >3 consecutive days within 2 weeks of Day 1, or subjects treated with any prohibited medication as described in Section 6.2. 9. Subjects who have received treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) preceding Day 1. 10. Subjects who have previously received eltrombopag or any other thrombopoietin receptor agonist. 11. Any subject considered to be a child in care, defined as one who has been placed under the control or protection of an agency, organization, institution or entity by the courts, the government or a government body, acting in accordance with powers conferred on them by law or regulation. This can include a child cared for by foster parents or living in a care home or institution, provided that the arrangement falls within the definition above. The definition of a child in care does not include a child who is adopted or who has an appointed legal guardian. 12. Subjects who have a known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to eltrombopag or excipients that contraindicates their participation. 13. Any serious and/or unstable pre-existing medical, psychiatric disorder, or other conditions that could interfere with the subject's safety or compliance to the study procedures.
    1. Soggetti con un'anomalia clinicamente rilevante, diversa dall'ITP, individuata all'esame di screening, o un'altra condizione medica o circostanza che, a giudizio dello sperimentatore, rende il soggetto non idoneo a partecipare allo studio o suggerisca un'altra diagnosi primaria (ad es. trombocitopenia secondaria a un'altra malattia).
    2. Soggetti con malignità concomitante o pregressa, compreso il disordine mieloproliferativo.
    3. Soggetti che si prevede non saranno idonei a proseguire la terapia corrente per almeno altre 13 settimane.
    4. Soggetti con storia di anomalie dell'agglutinazione piastrinica che impedisce una misurazione affidabile del livello di piastrine.
    5. Soggetti con diagnosi di trombocitopenia immune secondaria, compresi quelli con evidenze di laboratorio o cliniche di infezione da HIV, sindrome da anticorpi antifosfolipidi, infezione cronica da epatite B, infezione da virus dell'epatite C o evidenze di epatite attiva al momento dello screening.
    6. Soggetti con sindrome di Evans (trombocitopenia autoimmune ed emolisi autoimmune).
    7. Soggetti con trombocitopenia ereditaria nota (ad es. disordini di MYH9).
    8. Soggetti trattati con farmaci che danneggiano la funzione piastrinica (come, a titolo di esempio, aspirina, clopidogrel e/o FANS) o anticoagulanti per &gt;3 giorni consecutivi nelle 2 settimane precedenti il Giorno 1, o soggetti trattati con i farmaci vietati elencati nella Sezione 6.2 del protocollo.
    9. Soggetti che hanno ricevuto un trattamento con un farmaco sperimentale nei 30 giorni o 5 emivite (a seconda del periodo più lungo) precedenti il Giorno 1.
    10. Soggetti che hanno ricevuto in precedenza eltrombopag o un altro agonista del recettore della trombopoietina.
    11. I soggetti considerati bambini sotto tutela, sono definiti come coloro che sono stati collocati sotto il controllo o la protezione di un ente, organizzazione, istituto o entità da tribunali, dal governo o da un organo governativo secondo i poteri a essi conferiti da leggi o regolamenti. Tale definizione può comprendere un bambino in affido presso una famiglia o che risiede in una casa di accoglienza o istituto, purché tale sistemazione rientri nella definizione di cui sopra. La definizione di bambino sotto tutela non comprende un bambino adottato o dotato di un tutore legale.
    12. Soggetti con reazione di ipersensibilità nota immediata o ritardata o idiosincrasia a farmaci chimicamente correlati a eltrombopag o a eccipienti, tale da controindicarne la partecipazione.
    13. Patologia medica, disturbo psichiatrico o altre condizioni gravi e/o instabili preesistenti che potrebbero interferire con la sicurezza del soggetto o il rispetto delle procedure dello studio.
    E.5 End points
    E.5.1Primary end point(s)
    The odds of achieving platelet counts ≥ 50 Gi/L during the first 12 weeks of Part 1, the randomized treatment period, for subjects receiving eltrombopag relative to placebo.
    Probabilità di ottenere conte piastriniche ≥ 50 Gi/l durante le prime 12 settimane della Parte 1 dello studio, il periodo di trattamento randomizzato, nei soggetti che ricevono eltrombopag rispetto al placebo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Primary endpoint will be based on platelet counts obtained through the first 12 weeks of Part 1.
    L'end point primario si baserà sulla conta piastrinica ottenuta durante le prime 12 settimane della parte 1 dello studio.
    E.5.2Secondary end point(s)
    The proportion of subjects receiving eltrombopag, compared to placebo, who achieve platelet counts ≥ 50 Gi/L for at least 6 out of 8 weeks, between weeks 5-12 of Part 1. Weighted mean platelet change (area under the platelet-time curve divided by duration), for subjects receiving eltrombopag relative to placebo, from baseline to Week 12 of Part 1. The proportion of subjects receiving eltrombopag, compared to placebo, who achieve platelet counts ≥ 50 Gi/L at any time during the first 6 weeks of Part 1. The proportion of subjects receiving eltrombopag, compared to placebo, who achieve platelet counts ≥ 50 Gi/L at any time during the first 12 weeks of Part 1.The proportion of subjects achieving platelet counts ≥ 50 Gi/L at any time during Part 2. Maximum period of time with platelet counts continuously ≥ 50 Gi/L for subjects receiving eltrombopag relative to placebo during the first 12 weeks of Part 1. The proportion of weeks in which subjects achieve platelet counts ≥ 50 Gi/L, between weeks 4-24 of Part 2. Maximum period of time with platelet counts continuously ≥ 50 Gi/L during Part 2. The proportion of subjects who reduced or discontinued baseline concomitant ITP medications during Part 2. The proportion of subjects receiving eltrombopag, relative to placebo, who required protocol-defined rescue treatment during Part 1. The proportion of subjects who required protocol-defined rescue treatment during Part 2. Incidence and severity of symptoms associated with ITP, including bleeding, bruising and petechiae, measured using the World Health Organization (WHO) Bleeding Scale for subjects receiving eltrombopag relative to placebo, during Part 1. Incidence and severity of symptoms associated with ITP, including bleeding, bruising and petechiae, measured using the WHO Bleeding Scale during Part 2. Safety and tolerability parameters including blood pressure and heart rate, ophthalmic examinations, clinical laboratory assessments and frequency of all adverse events, categorized using Common Terminology Criteria for Adverse Events (CTCAE) v4 toxicity grades. PK data collected in this study will be included in a population PK analysis in order to estimate primary model-based PK parameters such as CL/F, Q/F, Vc/F, Vp/F, and ka and the influence of potential covariates on these parameters.
    •La percentuale di soggetti trattati con eltrombopag, rispetto al placebo, che ottengono conte piastriniche ≥ 50 Gi/l per almeno 6 settimane su 8 nelle settimane 5-12 della Parte 1.
    •La variazione media ponderata delle piastrine (area sotto la curva piastrine/tempo divisa per la durata) dal basale alla Settimana 12 della Parte 1.
    •La percentuale di soggetti trattati con eltrombopag, rispetto al placebo, che ottengono conte piastriniche ≥ 50 Gi/l in qualsiasi momento nelle prime 6 settimane della Parte 1.
    •La percentuale di soggetti trattati con eltrombopag, rispetto al placebo, che ottengono conte piastriniche ≥ 50 Gi/l in qualsiasi momento nelle prime 12 settimane della Parte 1.
    •Il periodo di tempo massimo per cui le conte piastriniche rimangono costantemente ≥ 50 Gi/l durante le prime 12 settimane della Parte 1.
    •La percentuale di soggetti trattati con eltrombopag, rispetto al placebo, che richiedono un trattamento di salvataggio definito dal protocollo durante la Parte 1.
    •Parametri di sicurezza e tollerabilità quali pressione arteriosa, frequenza cardiaca, esami oculistici, analisi cliniche di laboratorio e frequenza di tutti gli eventi avversi, classificati in base ai gradi di tossicità di Common Terminology Criteria for Adverse Events (CTCAE) v4 nella Parte 1.
    •Incidenza e gravità dei sintomi associati all'ITP, come sanguinamento, lividi e petecchie, misurate secondo la Bleeding Scale dell'Organizzazione Mondiale della Sanità (OMS) durante la Parte 1.
    •La percentuale di soggetti che ottengono conte piastriniche ≥ 50 Gi/l in qualsiasi momento durante la Parte 2.•La percentuale di settimane per le quali i soggetti ottengono conte piastriniche ≥ 50 Gi/l nelle settimane 4-24 della Parte 2.
    •Il periodo di tempo massimo in cui le conte piastriniche rimangono costantemente ≥ 50 Gi/l durante la Parte 2.
    •La percentuale di soggetti che riducono o interrompono i farmaci concomitanti basali per l'ITP durante la Parte 2.
    •La percentuale di soggetti che richiedono un trattamento di salvataggio definito dal protocollo durante la Parte 2.
    •Parametri di sicurezza e tollerabilità quali pressione arteriosa, frequenza cardiaca, esami oculistici, analisi cliniche di laboratorio e frequenza di tutti gli eventi avversi, classificati in base ai gradi di tossicità di Common Terminology Criteria for Adverse Events (CTCAE) v4 nella Parte 2.
    •Incidenza e gravità dei sintomi associati all'ITP, come sanguinamento, lividi e petecchie, misurate secondo la Bleeding Scale dell'Organizzazione Mondiale della Sanità (OMS) durante la Parte 2.Parametri di sicurezza e tollerabilità quali pressione arteriosa e frequenza cardiaca, esami oculistici, analisi cliniche di laboratorio e frequenza di tutti gli eventi avversi, classificati in base ai gradi di tossicità di Common Terminology Criteria for Adverse Events (CTCAE) v4.I dati sulla PK raccolti in questo studio saranno inclusi in un'analisi PK della popolazione al fine di stimare parametri PK primari basati sul modello come CL/F, Q/F, Vc/F, Vp/F e ka e l'influenza di potenziali covariate su tali parametri
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary endpoints span up to 13 weeks in Part 1 and up to 24 weeks in Part 2.
    Gli end points secondari saranno valutati fino a 13 settimane nella Parte 1 e fino a 24 settimane nella Parte 2.
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    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 Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA27
    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
    Chile
    Hong Kong
    Israel
    Russian Federation
    Taiwan
    Thailand
    United States
    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
    LSLV
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months25
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months26
    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: 75
    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: 43
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 30
    F.1.2Adults (18-64 years) No
    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 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 state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 75
    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)
    If eltrombopag is not commercially available after completion of the study treatment, up to 37 weeks with eltrombopag in this study, provisions will be made to allow patients with a continued response to maintain therapy with eltrombopag through a post-trial supply where permitted by local regulations.
    Se Eltrombopag non sarà in commercio dopo che è stato completato il trattamento dello Studio (37 settimane) dove permesso dalle leggi locali il farmaco verrà fornito con un programma di uso nominale, dopo approvazione del Comitato Etico
    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 Decision2012-03-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-01-25
    P. End of Trial
    P.End of Trial StatusCompleted
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