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    Summary
    EudraCT Number:2010-022583-13
    Sponsor's Protocol Code Number:CRAD001MIC02
    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:2011-01-25
    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 number2010-022583-13
    A.3Full title of the trial
    Estudio multicéntrico, abierto, de acceso expandido de RAD001, en pacientes con astrocitoma subependimario de células gigantes (SEGA) asociado con el complejo esclerosis tuberosa (TSC). ESTUDIO EFFECTS: Everolimus para acceso expandido acelerado en SEGA con TSC.
    A.3.2Name or abbreviated title of the trial where available
    EFFECTS STUDY
    A.4.1Sponsor's protocol code numberCRAD001MIC02
    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 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/3/10/764
    D.3 Description of the IMP
    D.3.1Product nameeverolimus
    D.3.2Product code RAD001
    D.3.4Pharmaceutical form 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 INNeverolimus
    D.3.9.1CAS number 159351-69-6
    D.3.9.3Other descriptive nameEVEROLIMUS
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2.5
    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) 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 astrocitoma subependimario de células gigantes (SEGA) asociado con el complejo esclerosis tuberosa (TSC)
    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 seguridad de RAD001 en pacientes con SEGA asociado con TSC.
    E.2.2Secondary objectives of the trial
    Evaluar la mejor tasa de respuesta global a criterio del investigador de RAD001 en pacientes con SEGA asociado con TSC.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Hombres o mujeres iguales o mayores a 3 años de edad
    Diagnóstico clínicamente definitivo de esclerosis tuberosa según los criterios de Gomez modificados (Roach 1998, Hyman 2000, Tabla 5-1).
    El diagnóstico clínicamente definitivo se define por la presencia de alguno de los siguientes criterios:
    Dos criterios mayores de la Tabla 5-1
    Un criterio mayor más dos criterios menores de la Tabla 5-1
    Presencia de por lo menos una lesión de SEGA identificada con RM o TC (según los requisitos locales)
    Nota: las lesiones de SEGA sólo se diagnostican en pacientes con TSC. Surgen en la capa subependimaria del ventrículo lateral y normalmente están localizadas cerca del agujero de Monro y aumentan homogéneamente con contraste con RM sin evidencia de edema circundante.
    Las mujeres físicamente fértiles deberán presentar documentación de prueba de embarazo negativa antes de la inclusión. Las mujeres premenopáusicas sexualmente activas (y las parejas de los pacientes varones) deberán utilizar métodos anticonceptivos eficaces mientras participen en el estudio y durante hasta 8 semanas después de finalizar el tratamiento.
    Consentimiento informado por escrito según las pautas locales
    E.4Principal exclusion criteria
    Pacientes con cirugía requerida y planeada relacionada con el SEGA
    Antecedentes de infarto de miocardio, angina o accidente cerebrovascular relacionado con aterosclerosis
    Deterioro conocido de la función pulmonar (por ejemplo, FEV1 o DLCO 70% del valor pronóstico)
    Anomalías hepáticas o hematológicas significativas (es decir, niveles de transaminasas > 2.5 x LSN o bilirrubina sérica > 1.5 x LSN, hemoglobina < 9 g/dL, plaquetas < 80,000/mm3, recuento de neutrófilos absoluto < 1,000/mm3).
    Infección activa o en curso en el momento de la inclusión
    Pacientes con hepatitis B ó C
    Antecedentes previos de trasplante de órganos
    Cirugía reciente (que implique la entrada en una cavidad corporal o precise suturas) dentro del mes previo a la inclusión
    Uso de un fármaco en investigación dentro de los 30 días antes de la inclusión
    Hiperlipidemia incontrolada: Colesterol sérico en ayunas > 300 mg/dL O >7.75 mmol/L Y triglicéridos en ayunas > 2.5 x LSN
    Diabetes mellitus incontrolada definida por glucosa sérica en ayunas > 1.5 x LSN
    Pacientes con diátesis hemorrágica o que reciban medicación oral antivitamina K
    Creatinina sérica > 1.5 x LSN.
    Cualquier condición médica severa y/o incontrolada que pudiese causar riesgos de seguridad inaceptables:
    Hipercolesterolemia/hipertrigliceridemia incontrolada
    Deterioro de la función gastrointestinal o enfermedad gastrointestinal que pueda alterar significativamente la absorción de la medicación del estudio (por ejemplo, enfermedad ulcerosa, náuseas incontroladas, vómitos, diarrea, síndrome de mala absorción).
    Pacientes con hipersensibilidad conocida a RAD001 o a otros análogos de la rapamicina (sirolimus, temsirolimus) o a sus excipientes.
    Para los fines de las evaluaciones de RM (si aplicable):
    Otros implantes metálicos ferromagnéticos que no sean los aprobados como seguros para uso en el escáner de RM (por ejemplo, aparatos ortopédicos, algunos tipos de clips de aneurisma, metralla)
    Pacientes que sufran claustrofobia incontrolable o que físicamente no puedan colocarse en la máquina (por ejemplo, obesidad).
    Pacientes con antecedentes conocidos de seropositividad frente al VIH.
    Que no puedan acudir a las visitas programadas a la clínica e incumplimiento con las visitas programadas a la clínica necesarias para monitorización, ajuste de la dosis y manejo de la toxicidad.
    Mujeres embarazadas o en periodo de lactancia o adultos con potencial reproductor que no utilicen métodos anticonceptivos eficaces. Si se utilizan métodos anticonceptivos de barrera, ambos sexos deberán continuar utilizándolos durante todo el estudio.
    E.5 End points
    E.5.1Primary end point(s)
    Acontecimientos adversos de grado 3 y 4, acontecimientos adversos graves
    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 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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA59
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months1
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    niños, adolescentes
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 250
    F.4.2.2In the whole clinical trial 250
    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)
    treatment upon the discretion of the investigator
    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 Decision2011-03-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-03-11
    P. End of Trial
    P.End of Trial StatusCompleted
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