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    Summary
    EudraCT Number:2012-000650-64
    Sponsor's Protocol Code Number:CICL670E2419
    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:2012-12-11
    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 number2012-000650-64
    A.3Full title of the trial
    An open label, multi-center, efficacy and safety study of deferasirox in iron overloaded patients with nontransfusion dependent thalassemia.(THETIS)
    Studio multicentrico, in aperto, per valutare l'™efficacia e la sicurezza di deferasirox in pazienti affetti da talassemia non trasfusione dipendente con sovraccarico di ferro.(THETIS)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An open label, multi-center, efficacy and safety study of deferasirox in iron overloaded patients with nontransfusion dependent thalassemia.(THETIS)
    Studio multicentrico, in aperto, per valutare l'efficacia e la sicurezza di deferasirox in pazienti affetti da talassemia non trasfusione dipendente con sovraccarico di ferro.(THETIS)
    A.3.2Name or abbreviated title of the trial where available
    THETIS
    THETIS
    A.4.1Sponsor's protocol code numberCICL670E2419
    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 SponsorNOVARTIS FARMA
    B.1.3.4CountryItaly
    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 supportNovartis Pharma Services AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNOVARTIS FARMA
    B.5.2Functional name of contact pointDrug Regulatory Affairs
    B.5.3 Address:
    B.5.3.1Street AddressLargo Umberto Boccioni, 1
    B.5.3.2Town/ cityORIGGIO
    B.5.3.3Post code21040
    B.5.3.4CountryItaly
    B.5.4Telephone number+39 02 96541
    B.5.5Fax number+39 02 9659066
    B.5.6E-mailinfo.studiclinici@novartis.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 EXJADE*28CPR DISP 125MG
    D.2.1.1.2Name of the Marketing Authorisation holderNOVARTIS FARMA SpA
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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/3/02/092
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Dispersible 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 INNDEFERASIROX
    D.3.9.1CAS number 201530-41-8
    D.3.9.4EV Substance CodeSUB21981
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number125
    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 EXJADE*28CPR DISP 250MG
    D.2.1.1.2Name of the Marketing Authorisation holderNOVARTIS FARMA SpA
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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/3/02/092
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Dispersible 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 INNDEFERASIROX
    D.3.9.1CAS number 201530-41-8
    D.3.9.4EV Substance CodeSUB21981
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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 EXJADE*28CPR DISP 500MG
    D.2.1.1.2Name of the Marketing Authorisation holderNOVARTIS FARMA SpA
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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/3/02/092
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Dispersible 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 INNDEFERASIROX
    D.3.9.1CAS number 201530-41-8
    D.3.9.4EV Substance CodeSUB21981
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(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 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
    Iron overload in patients with non-transfusion dependent thalassemia
    Sovraccarico di ferro in pazienti affetti da talassemia non trasfusione dipendente
    E.1.1.1Medical condition in easily understood language
    Iron overload
    Sovraccarico di ferro
    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 14.1
    E.1.2Level PT
    E.1.2Classification code 10065973
    E.1.2Term Iron overload
    E.1.2System Organ Class 10027433 - Metabolism and nutrition 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 deferasirox in patients with NTDT based on change in LIC from baseline after 52 weeks of treatment.
    Valutare l’efficacia di deferasirox in pazienti con talassemia non trasfusione dipendente in base alla variazione della concentrazione epatica di ferro (LIC) rispetto al basale dopo 52 settimane di trattamento.
    E.2.2Secondary objectives of the trial
    • To evaluate the impact of deferasirox on Quality of Life • To evaluate the efficacy of deferasirox based on change in LIC from baseline after 24 weeks of treatment • To assess the correlation of change in SF and LIC at baseline and end of study (EOS) • To assess the efficacy of deferasirox based on change in LIC from baseline after 52 weeks of treatment by genotype • To assess the efficacy of deferasirox based on change in SF from baseline over 52 weeks of treatment • To evaluate the safety of deferasirox doses up to 30 mg/kg/day • To assess the efficacy of deferasirox on endocrine function • To conduct pharmacokinetic (pK) analysis in a subset of patients
    -Valutare l’impatto di deferasirox sulla Qualità della Vita nell’adulto utilizzando il questionario SF-36;-Valutare l’impatto di deferasirox sulla QoL pediatrica utilizzando il questionario Pediatric Quality of Life;-Valutare l’efficacia di deferasirox in base alla variazione della LIC rispetto al basale dopo 24 settimane di trattamento;-Valutare la correlazione della variazione di SF e LIC al basale e alla fine dello studio;-Valutare l’efficacia di deferasirox in base alla variazione della LIC rispetto al basale dopo 52 settimane di trattamento per genotipo;-Valutare l’efficacia di deferasirox in base alla variazione di SF rispetto al basale nel corso di 52 settimane di trattamento;-Valutare la sicurezza d’impiego di dosaggi di deferasirox fino a 30 mg/kg/die. Fare riferimento al paragrafo 3 del protocollo.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Male or female aged ≥ 10 years with non-transfusion-dependent congenital or chronic anemias inclusive of beta-thalassemia intermedia, HbE beta-thalassemia or alpha-thalassemia intermedia (HbH disease) • LIC ≥ 5 mg Fe/g dw measured by R2 MRI at screening • Serum ferritin ≥ 300 ng/mL at screening (two consecutive values at least 14 days apart from each other) • Written informed consent obtained prior to any screening procedures
    -Pazienti di sesso maschile e femminile di età ≥ 10 affetti da anemia congenita o cronica non trasfusione dipendente, comprese β-talassemia intermedia, HbE β-talassemia o α-talassemia intermedia (malattia HbH); -LIC ≥ 5 mg Fe/g dw misurata tramite RMN R2 allo screening; -Ferritina sierica ≥ 300 ng/mL allo screening (due valori consecutive ad un intervallo di almeno 14 giorni uno dall’altro);- Consenso informato scritto ottenuto prima dell’effettuazione di qualsiasi procedura di screening.
    E.4Principal exclusion criteria
    • HbS-beta Thalassemia • Anticipated regular transfusion program during the study • Any blood transfusion 6 months prior to study start • Significant proteinuria; creatinine clearance ≤40 ml/min; serum creatinine >ULN; ALT >5xULN
    -Pazienti con diagnosi di HbS β-talassemia;-Programma di trasfusioni regolari previsto durante lo studio. I pazienti sottoposti a trasfusioni sporadiche nel corso dello studio non saranno esclusi;-Qualsiasi trasfusione di sangue nei 6 mesi precedenti l’inizio dello studio;-Pazienti non in grado di sottoporsi alle valutazioni dello studio compresa la RMN, ad esempio pazienti claustrofobici, portatori di pacemaker o di impianti metallici ferromagnetici diversi da quelli approvati per l’utilizzo sicuro per la RMN e pazienti obesi;-Proteinuria significativa come indicato da un rapporto proteine/creatinina urinaria &gt; 1.0 mg/mg in un campione di urine non della prima minzione del mattino alla visita 1 o alla visita 2;-Clearance della creatinina ≤ 40 ml/min in due misurazioni durante la visita 1 e la visita 2;-Creatinina sierica &gt; ULN in due misurazioni durante la visita 1 la visita 2;-ALT &gt; 5 x ULN alla visita 1 e alla visita 2;-Evidenza clinica di epatite B attiva o epatite C;-Diagnosi nota di cirrosi;-Storia di tossicità oculare e/o uditiva clinicamente rilevante correlata alla terapia ferro chelante;-Storia di sierologia positiva per HIV;-Presenza di una condizione chirurgica o medica che possa significativamente alterare l’assorbimento, la distribuzione o l’escrezione del farmaco in studio;-Pazienti con malattie infiammatorie attive che interferiscono con una misurazione accurata della ferritina sierica;-Pazienti in gravidanza o allattamento, o pazienti potenzialmente in grado di avere figli che non utilizzano un metodo contraccettivo efficace;-Pazienti partecipanti ad un altro studio clinico o che hanno ricevuto un farmaco sperimentale sistemico nelle 4 settimane precedenti o un farmaco sperimentale ad uso topico nei 7 giorni precedenti lo screening;-Storia di mancata aderenza a regimi medici o pazienti che sono considerati potenzialmente non affidabili e/o non cooperativi, non disponibili o non in grado di aderire al protocollo;-Storia di ipersensibilità al trattamento in studio o ai suoi eccipienti;-Condizione medica significativa che interferisce con la capacità di partecipare a questo studio;-Storia di abuso di droghe o alcol nei 12 mesi precedenti l’arruolamento.Criterio di esclusione per i pazienti pediatrici:Si richiede un peso del paziente di almeno 20 kg per consentire la somministrazione di 5 mg/kg con una compressa da 125 mg.
    E.5 End points
    E.5.1Primary end point(s)
    Change in LIC from baseline
    Variazione della concentrazione epatica del ferro (Liver Iron Concentration – LIC) dal basale
    E.5.1.1Timepoint(s) of evaluation of this end point
    52 weeks
    52 settimane
    E.5.2Secondary end point(s)
    Change in SF from baseline
    Variazione della ferritina sierica (Serum Ferritin – SF) dal basale
    E.5.2.1Timepoint(s) of evaluation of this end point
    52 weeks
    52 settimane
    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 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 No
    E.8.1.1Randomised Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial1
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    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
    China
    Lebanon
    Russian Federation
    Thailand
    Turkey
    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
    Completion of this study as a whole will occur upon the availability and accuracy verification of the last data point required for statistical analysis.
    Il completamento dello studio avverrà dopo disponibilità e verifica accurata degli ultimi dati necessari all'analisi statistica.
    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 months30
    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: 17
    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) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 3
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 14
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 102
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 16
    F.4.2.2In the whole clinical trial 120
    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)
    Open IPSP (individual patient supply program) program.
    Programma di uso compassionevole (IPSP, individual patient supply program).
    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 Decision2012-12-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-11-13
    P. End of Trial
    P.End of Trial StatusCompleted
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