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    The EU Clinical Trials Register currently displays   43857   clinical trials with a EudraCT protocol, of which   7284   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:2005-001290-10
    Sponsor's Protocol Code Number:CICL670A2203
    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:2005-12-05
    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 number2005-001290-10
    A.3Full title of the trial
    Estudio de acceso expandido para proporcionar EXJADE® (deferasirox) a pacientes con trastornos congénitos de eritrocitos y sobrecarga crónica de hierro debido a transfusiones de sangre, que no pueden ser tratados adecuadamente con otros quelantes aprobados localmente
    A.4.1Sponsor's protocol code numberCICL670A2203
    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 Farmaceútica, 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 Information not present in EudraCT
    D.2.1.1.1Trade name EXJADE
    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 Information not present in EudraCT
    D.3.11.8Extractive medicinal product Information not present in EudraCT
    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 Information not present in EudraCT
    D.2.1.1.1Trade name EXJADE
    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 Information not present in EudraCT
    D.3.11.8Extractive medicinal product Information not present in EudraCT
    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 Information not present in EudraCT
    D.2.1.1.1Trade name EXJADE
    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 Information not present in EudraCT
    D.3.11.8Extractive medicinal product Information not present in EudraCT
    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
    Sobrecarga crónica de hierro debido a transfusiones de sangre en pacientes con trastornos congénitos de eritrocitos

    Congenital disorders of red blood cell and chronic iron overload from blood transfusions
    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
    • Proporcionar un acceso expandido a Exjade a pacientes con trastornos congénitos de eritrocitos, con una sobrecarga crónica de hierro causada por transfusiones de sangre, que no pueden recibir tratamiento con otros quelantes aprobados localmente debido a un incumplimiento terapéutico documentado, a contraindicaciones, a toxicidades inaceptables y/o a una respuesta deficiente documentada.

    • Continuar evaluando el perfil de seguridad de Exjade en función de la administración del fármaco y la notificación de los acontecimientos adversos graves.
    E.2.2Secondary objectives of the trial
    Evaluar el efecto del tratamiento con Exjade en los cambios en el nivel de ferritina sérica.
    E.2.3Trial contains a sub-study Information not present in EudraCT
    E.3Principal inclusion criteria
    • Pacientes de ambos sexos con una edad igual o superior a los 2 años.

    • Pacientes con un trastorno congénito de eritrocitos documentado (por ejemplo, Beta-talasemia mayor, anemia drepanocítica, anemia de Diamond-Blackfan) que necesiten transfusiones de sangre continuadas.

    • Pacientes que no puedan recibir un tratamiento adecuado con un quelante de hierro localmente aprobado debido a uno de los siguientes motivos:

    • Incumplimiento terapéutico documentado, definido como la toma de menos del 50% de las dosis de la terapia quelante prescrita en los 12 meses previos a la inclusión en el estudio.

    • Contraindicaciones, toxicidades inaceptables y/o respuesta deficiente documentada a los quelantes aprobados localmente, a pesar de un cumplimiento terapéutico adecuado.

    • Pacientes con un historial de, al menos, 20 transfusiones de sangre (equivalente a 100 mL/kg de concentrados de eritrocitos).

    • Pacientes con un valor de ferritina sérica superior o igual a 1000 microg/L en la visita de selección

    • Pacientes capaces de cumplir con todos los procedimientos, medicamentos y evaluaciones relacionados con el estudio.

    • Las mujeres pre-menopáusicas sexualmente activas deberán utilizar un método anticonceptivo de doble barrera, un anticonceptivo oral + un anticonceptivo de barrera o se deberán haber sometido a una histerectomía y/o ooforectomía total, a una ligadura de trompas clínicamente documentadas, o ser posmenopáusicas, definida como amenorrea durante, al menos, 12 meses.


    • El paciente otorgara un consentimiento informado por escrito y en el caso de los pacientes pediátricos será el tutor legal del paciente quien lo otorgue. La definición del término “pediátrico” para la inclusión y desarrollo del estudio será la que indique la legislación local. El investigador informará exhaustivamente a los padres o tutores legales sobre los requisitos del estudio. También se informará a los pacientes pediátricos según su capacidad, utilizando un lenguaje y unos términos que puedan comprender. Se obtendrá el consentimiento informado por escrito de su tutor legal que actuará en nombre del paciente según la legislación nacional. Si tuvieran capacidad, todos los pacientes pediátricos deberían firmar personalmente su consentimiento informado por escrito.
    E.4Principal exclusion criteria
    • Pacientes que reciban un tratamiento continuado con otro quelante de hierro (cualquier tratamiento con un quelante de hierro se deberá interrumpir, al menos 24 horas antes de la inclusión en el estudio).

    • Pacientes que cumplan los criterios de elegibilidad para cualquier otro protocolo de ensayo clínico en progreso promovido por Novartis con Exjade y que tengan acceso geográfico a estos centros.

    • Pacientes que no puedan tolerar (o que tengan toxicidades inaceptables al) un tratamiento previo con Exjade.

    • Pacientes con un nivel de creatinina sérica por encima del límite superior de la normalidad en la visita de selección.

    • Pacientes con ALT > ó = 500 U/L en la visita de selección.

    • Pacientes con evidencia de cataratas o pérdida auditiva relacionada con la quelación en las 4 semanas previas a la visita basal.

    • Pacientes embarazadas (demostrado por un test de embarazo Beta-HCG en suero en la visita de selección realizado a todas las pacientes susceptibles de quedarse embarazadas) y pacientes en periodo de lactancia.

    • Pacientes tratados con un fármaco experimental sistémico en las 4 semanas previas a la visita basal o tratados con un fármaco experimental tópico en los 7 días previos a la visita basal.
    E.5 End points
    E.5.1Primary end point(s)
    Las evaluaciones de seguridad consistirán en la monitorización de todos los acontecimientos adversos y en la notificación de todos los acontecimientos adversos graves, la monitorización regular de los niveles hematológicos y bioquímicos, y la monitorización regular del peso corporal, y las condiciones oftalmológicas y auditivas.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Information not present in EudraCT
    E.6.2Prophylaxis Information not present in EudraCT
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Information not present in EudraCT
    E.6.7Pharmacodynamic Information not present in EudraCT
    E.6.8Bioequivalence Information not present in EudraCT
    E.6.9Dose response Information not present in EudraCT
    E.6.10Pharmacogenetic Information not present in EudraCT
    E.6.11Pharmacogenomic Information not present in EudraCT
    E.6.12Pharmacoeconomic Information not present in EudraCT
    E.6.13Others Information not present in EudraCT
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Information not present in EudraCT
    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) Information not present in EudraCT
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) Information not present in EudraCT
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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.8.5The trial involves multiple Member States Yes
    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 Information not present in EudraCT
    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
    La última visita del último paciente que continue en el estudio.
    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 months0
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1.1In Utero Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    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 Information not present in EudraCT
    F.3.3.1Women of childbearing potential not using contraception For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2005-12-05. Yes
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Pacientes Pediátricos.Se obtendrá el CI por escrito de sus padres ó tutor legal que actuará en nombre del paciente según la legislación nacional. Si tuvieran capacidad, todos los pacientes pediátricos deberían firmar personalmente su CI por escrito
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 1727
    F.4.2.2In the whole clinical trial 3000
    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 Decision2006-01-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2006-01-13
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2008-04-11
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    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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