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    The EU Clinical Trials Register currently displays   43862   clinical trials with a EudraCT protocol, of which   7285   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2006-003337-32
    Sponsor's Protocol Code Number:CICL670A2204
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2010-03-23
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2006-003337-32
    A.3Full title of the trial
    ?Estudio Fase II, multicéntrico, abierto y no comparativo para evaluar la eficacia y la seguridad de ICL670 administrado durante 1 año ajustando la dosis en función de los niveles de ferritina en suero, en pacientes con anemia crónica y hemosiderosis transfusional?
    A.4.1Sponsor's protocol code numberCICL670A2204
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorNovartis Farmacéutica, S.A.
    B.1.3.4CountrySpain
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationSwitzerland
    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.3.1Product nameEXJADE
    D.3.2Product code ICL670
    D.3.4Pharmaceutical form Dispersible tablet
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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.3.9.2Current sponsor codeICL670
    D.3.9.3Other descriptive nameEXJADE
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationSwitzerland
    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.3.1Product nameEXJADE
    D.3.2Product code ICL670
    D.3.4Pharmaceutical form Dispersible tablet
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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.3.9.2Current sponsor codeICL670
    D.3.9.3Other descriptive nameEXJADE
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationSwitzerland
    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.3.1Product nameEXJADE
    D.3.2Product code ICL670
    D.3.4Pharmaceutical form Dispersible tablet
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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.3.9.2Current sponsor codeICL670
    D.3.9.3Other descriptive nameEXJADE
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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
    Pacientes con diagnóstico de sobrecarga de hierro crónica con anemia dependiente de transfusiones distintas a ß-talasemia y drepanocitosis
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    ?Evaluar la eficacia del tratamiento con deferasirox según el cambio de la CHH, con dosis iniciales de deferasirox según la tasa de transfusión y el objetivo terapéutico, en pacientes con anemias crónicas y hemosiderosis transfusional distintas a la ?-talassemia2 y la drepanocitosis
    E.2.2Secondary objectives of the trial
    ? ? Evaluar la eficacia del tratamiento con deferasirox según el cambio en los niveles de ferritina
    ? Evaluar la seguridad y la tolerabilidad de deferasirox durante un año
    ? Evaluar la eficacia y la seguridad en pacientes japoneses
    ? Analizar el balance del hierro después de un año de tratamiento
    ? Confirmar el valor de la ferritina como marcador para la monitorización precisa del tratamiento quelante
    ? Realizar exploraciones oftalmológicas en pacientes tratados con deferasirox. Se monitorizarán al menos 60 pacientes durante 2 años.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Anexo nº 4 estudio de extensión fecha 05-08-2008
    El propósito de este anexo es ampliar la duración del estudio clínico un año más, para poder recoger datos de eficacia y de seguridad a largo plazo de ICL670.
    La evaluación continuará durante un año utilizando el esquema definido en el protocolo del estudio principal.
    ? Las determinaciones de los parámetros hematológicos, bioquímicos y de orina continuarán analizándose en una base mensual.
    ? La exploración física y las evaluaciones audiométricas se repetirán cada 6 meses.
    ? El contenido de hierro hepático, las audiometrías y la medición del tiempo de protrombina se realizará de nuevo después de un año de tratamiento.
    ? Además en los pacientes pediátricos se evaluará la velocidad de crecimiento y el desarrollo sexual.
    Los pacientes iniciarán la extensión con la dosis que recibían al final del estudio principal. Se permitirán ajustes de la dosis y se basarán en los algoritmos descritos en el protocolo del estudio principal
    Los valores basales definidos al inicio del estudio principal se utilizarán como una referencia para los ajustes de la dosis
    E.3Principal inclusion criteria
    ? ? Pacientes con anemia cr?nica que requieren transfusi?nes de sangre distinta a la ?-talasemia y a la drepanocitosis.
    ? Varones o mujeres ? 2 años de edad con sobrecarga de hierro transfusional debido a:
    Riesgo de SMD bajo o intermedio (INT-1) determinado mediante los criterios del IPSS.
    Otras anemias congénitas o adquiridas, excluyendo la ? -talasemia y la drepanocitosis
    ? Antecedentes de haber recibido ? 20 unidades (aproximadamente 100 ml/kg) de concentrado de eritrocitos a lo largo de toda la vida del paciente o signos de sobrecarga de hierro (ferritina en suero > 1000 µg/l) .
    ? Capaz de proporcionar el consentimiento informado escrito
    ? Esperanza de vida ? 12 meses
    ? Si el paciente ya había sido tratado con deferiprona se debe realizar un periodo de lavado de un mes antes de administrar la primera dosis de deferasirox.
    E.4Principal exclusion criteria
    ? ? Se excluyen los pacientes con ß-talasemia o drepanocitosis.
    ? Pacientes con síndrome mielodisplásico con una puntuación IPSS intermedia -2 o elevada.
    ? Pacientes con niveles de creatinina en suero > LSN1
    ? Pacientes con niveles de ALT (SGPT) > 5 x LSN
    ? Proteinuria significativa indicada por un índice en orina de proteína/creatinina > 0,5 mg/mg en una muestra de orina que no sea de primera hora en dos evaluaciones durante el periodo de selección.
    ? Historia de resultados positivos en la prueba del VIH(ELISA o transferencia Western) .
    ? Antecedentes de indicios clínicos o de laboratorio de hepatitis C o B activa (HBsAg en ausencia de HBsAb O BIEN positividad para Ac frente al VHC con positividad para ARN del VHC y niveles de ALT por encima del intervalo normal)
    ? Los pacientes que están recibiendo tratamientos experimentales para el SMD, incluidos lenalidomida, talidomida, azacitidina y trióxido arsénico, deben someterse a un periodo de lavado ? 4 semanas antes de la primera dosis del fármaco del estudio.
    ? Pacientes con hipertensión sistémica no controlada
    ? Pacientes con enfermedad cardíaca inestable no controlada mediante el tratamiento médico convencional
    ? Enfermedad sistémica (cardiovascular, renal, hepática, etc.) que impediría el tratamiento del estudio
    ? Embarazo (confirmado mediante la prueba de laboratorio requerida en el momento de la selección) o periodo de lactancia.
    ? Pacientes tratados con un fármaco experimental sistémico en las últimas 4 semanas o con un fármaco experimental tópico en los últimos 7 días
    ? Otro trastorno quirúrgico o médico que podría alterar de forma significativa la absorción, distribución, metabolismo o excreción de la medicación del estudio
    ? Pacientes que el investigador considere potencialmente poco fiables y/o no cooperadores en relación con el protocolo del ensayo
    ? Antecedentes de hipersensibilidad a la medicación del estudio o a los excipientes
    ? Mujeres sexualmente activas en periodo premenopáusico que no utilicen un método de anticoncepción adecuado. Las mujeres deben utilizar un método de anticoncepción eficaz o deben haber sido sometidas a una histerectomía total y/o ooforectomía, ligadura de trompas confirmadas clínicamente o estar en periodo posmenopáusico, definido como la presencia de amenorrea durante al menos 12 meses.
    E.5 End points
    E.5.1Primary end point(s)
    ? Como principales parámetros de la eficacia se emplearán las variaciones en la CHH en la población global después de un año de 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 No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans Information not present in EudraCT
    E.7.1.2Bioequivalence study Information not present in EudraCT
    E.7.1.3Other Information not present in EudraCT
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    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.8.3 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 EEA3
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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
    Extensión de un año del estudio utilizando el esquema definido en el protocolo del estudio principal.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months6
    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.1.1.1In 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.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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 64
    F.4.2.2In the whole clinical trial 114
    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)
    Después de la finalización del estudio central, los pacientes que cumplan con los criterios de inclusión y ninguno de exclusión del estudio de extensión y que requieran la continuación del tratamiento quelante (en opinión del investigador) se les ofrecerá un protocolo de extensión (Anexo nº 4) durante un año para recoger datos de seguridad y eficacia a largo plazo.
    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 Decision2007-03-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2007-01-22
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2012-02-02
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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