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    The EU Clinical Trials Register currently displays   42567   clinical trials with a EudraCT protocol, of which   7008   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2013-004291-35
    Sponsor's Protocol Code Number:CACZ885N2301
    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:2014-04-04
    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 number2013-004291-35
    A.3Full title of the trial
    A randomized, double-blind, placebo controlled study of canakinumab in patients with Hereditary Periodic Fevers (TRAPS, HIDS, or crFMF), with subsequent randomized withdrawal/ dosing frequency reduction and open-label long term treatment epochs
    Estudio aleatorizado, doble ciego, controlado con placebo de canakinumab en pacientes con fiebres periódicas hereditarias (TRAPS, HIDS o FMFrc) con retirada aleatorizada/reducción de la frecuencia de la dosis y posterior fase de tratamiento abierto a largo plazo
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of efficacy and safety of canakinumab in patients with Hereditary Periodic Fevers
    Estudio de la eficacia y seguridad de canakinumab en pacientes con fiebres periódicas hereditarias
    A.4.1Sponsor's protocol code numberCACZ885N2301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02059291
    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 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 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 Farmacéutica, S.A.
    B.5.2Functional name of contact pointDepartamento Medico (ICRO)
    B.5.3 Address:
    B.5.3.1Street AddressGran Via Corts Catalanes, 764
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08013
    B.5.3.4CountrySpain
    B.5.4Telephone number+34933064342
    B.5.5Fax number+34933064290
    B.5.6E-maileecc.novartis@novartis.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleComparator
    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 Ilaris
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Ltd.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namecanakinumab
    D.3.2Product code ACZ885
    D.3.4Pharmaceutical form Powder for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCANAKINUMAB
    D.3.9.1CAS number 914613-48-2
    D.3.9.2Current sponsor codeACZ885
    D.3.9.4EV Substance CodeSUB30137
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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 placeboSolution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Hereditary Periodic Fevers (TRAPS, HIDS, or crFMF)
    Fiebres periódicas hereditarias (TRAPS, HIDS o FMFrc)
    E.1.1.1Medical condition in easily understood language
    Hereditary Periodic Fevers
    Fiebres periódicas hereditarias
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.1
    E.1.2Level LLT
    E.1.2Classification code 10067783
    E.1.2Term Tumor necrosis factor receptor-associated periodic syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.1
    E.1.2Level PT
    E.1.2Classification code 10016207
    E.1.2Term Familial mediterranean fever
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.1
    E.1.2Level PT
    E.1.2Classification code 10072010
    E.1.2Term Hyper IgD syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the randomized treatment epoch and for the overall
    study is to demonstrate that subcutaneous canakinumab administered every 4 weeks is superior to placebo in achieving a clinically meaningful reduction of disease activity defined as resolution of the index flare at Day 15 and no new disease flares over 16 weeks of treatment.
    El objetivo principal de la fase de tratamiento aleatorizado y del estudio global es demostrar que canakinumab subcutáneo administrado cada 4 semanas es superior a placebo en la consecución de una reducción clínicamente significativa de la actividad de la enfermedad definida como la remisión del número de brotes el día 15 y ningún nuevo brote de la enfermedad a lo largo de 16 semanas de tratamiento.
    E.2.2Secondary objectives of the trial
    ? To evaluate the percentage of patients who achieve a Physician Global Assessment of Disease Activity (PGA) <2 (?minimal? or ?none?) at Week 16
    ? To evaluate the percentage of patients with the serologic remission at Week 16 (defined as C-reactive protein [CRP] <10 mg/L)
    ? To evaluate the percentage of patients with normalized Serum Amyloid A (SAA) level at Week 16 (defined as SAA <10 mg/L)
    ? To evaluate the percentage of canakinumab responders in Epoch 2 who maintain a clinically meaningful response (absence of new flares) when switched to canakinumab every 8 weeks compared to placebo (Epoch 3)
    ? To evaluate the long-term safety and tolerability and immunogenicity of canakinumab
    ? To evaluate the pharmacokinetics/ pharmacodynamics of canakinumab
    ? Evaluar el porcentaje de pacientes que alcancen una evaluación global de la actividad de la enfermedad por parte del médico (PGA) <2 (?mínima? o ?ninguna?) en la semana 16.
    ? Evaluar el porcentaje de pacientes con remisión serológica en la semana 16 (definida como proteína C reactiva [PCR] <10 mg/l).
    ? Evaluar el porcentaje de pacientes con nivel normalizado de amiloide A sérico (AAS) en la semana 16 (definida como AAS <10 mg/l).
    ? Evaluar el porcentaje de respondedores a canakinumab en la fase 2 que mantengan una respuesta clínicamente significativa (ausencia de nuevos brotes) cuando cambien a canakinumab cada 8 semanas en comparación con placebo (fase 3)
    ? Evaluar la seguridad, tolerabilidad e inmunogenicidad a largo plazo de canakinumab
    ? Evaluar la farmacocinética/farmacodinámica de canakinumab.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patient's written informed consent (or parent's written informed consent in case of pediatric patient) at screening
    - Male and female patients at least 2 years of age at the time of the screening visit.
    - Confirmed diagnosis and active flare at randomization
    - CRP >10mg/L at randomization
    ? Consentimiento informado escrito del paciente (o consentimiento informado escrito de los padres en caso de pacientes pediátricos) en la selección.
    ? Pacientes de ambos sexos de al menos 2 años de edad en el momento de la visita de selección.
    ? Diagnóstico confirmado de TRAPS, HIDS o FMFrc en la selección.
    ? Brote activo de TRAPS, HIDS o FMFrc en la randomizacion.
    ? PCR >10mg/l (rango normal de PCR ?10 mg/l) en la aleatorización.
    E.4Principal exclusion criteria
    Use of the following therapies (within varying protocol defined timeframes): Corticosteroids, anakinra, canakinumab, rilonacept, tocilizumab, TNF inhibitors, abatacept, tofacitinib, rituximab, leflunomide, thalidomide, cyclosporine, intravenous immunoglobulin, 6-Merceptopurine, azathioprine, cyclophosphamide, or chlorambucil, any other investigational biologics
    - History of malignancy of any organ system (other than localized basal cell carcinoma of the skin or in - situ cervical cancer), treated or untreated
    - Significant medical diseases, including but not limited to the following:
    a. History of organ transplantation
    b. Elevated liver enzymes ?2x ULN
    d. Increase in total bilirubin
    e. Serious hepatic disorder (Child-Pugh scores B or C)
    f. Chronic Kidney Disease
    g. Thyroid disease
    h. Diagnosis of active peptic ulcer disease
    i. Coagulopathy j. Significant CNS effects including vertigo and dizziness
    - Any conditions or significant medical problems which immunecompromise the patient and/or places the patient at unacceptable risk for immunomodulatory therapy
    - Live vaccinations within 3 months prior to the start of the trial, during the trial, and up to 3 months following the last dose
    ? Uso de los siguientes tratamientos (dentro de los distintos plazos definidos en el protocolo):
    Corticosteroides (prednisona oral) > 0,2 mg/kg/día (o superior al máximo de 15 mg/ día en niños con un peso superior a 60 kg), anakinra, canakinumab, rilonacept, tocilizumab, inhibidores de TNF, abatacept, tofacitinib, rituximab, leflunomida, talidomida, ciclosporina, inmunoglobulina intravenosa, 6-merceptopurina, azatioprina, ciclofosfamida o clorambucilo, inyecciones intraarticulares, periarticulares o intramusculares de corticosteroides, cualquier otro producto biológico en investigación.
    ? Antecedentes de tumores malignos de cualquier sistema orgánico (salvo carcinoma cutáneo de células basales localizado o cáncer de cuello uterino in situ) tratados o no tratados.
    ? Enfermedades médicas significativas, incluyendo entre otras:
    a. Antecedentes de trasplante de órganos.
    b. Nivel elevado de alanina aminotransferasa (ALT) ? 2x LSN.
    c. Nivel elevado de aspartato aminotransferasa (AST) ?2x LSN.
    d. Aumento de la bilirrubina total definido según los criterios comunes de terminología (CTC) de grado ?2.
    e. Trastorno hepático grave (puntuaciones B o C de Child-Pugh).
    f. Enfermedad renal crónica según los estadios de NKF ?4.
    g. Enfermedad tiroidea.
    h. Diagnóstico de enfermedad de úlcera péptica activa.
    i. Coagulopatía.
    j. Efectos significativos en el SNC incluidos vértigo y mareos.
    ? Cualquier condición o problema médico significativo que inmunocomprometa al paciente y/o le ponga en una situación de riesgo inaceptable para el tratamiento inmunomodulador, p. ej.,
    a. Disminución del recuento absoluto de neutrófilos según grado ?1 de los CTC.
    b. Trombocitopenia de grado 1 de los CTC.
    c. Cualquier infección bacteriana, fúngica (salvo onicomicosis) o vírica activa o recurrente.
    d. Infección del VIH, infecciones de hepatitis B o hepatitis C.
    e. Presencia de tuberculosis
    f. Requisito de administración de antibióticos contra la TB latente.
    g. Signos clínicos o antecedentes de esclerosis múltiple u otras enfermedades desmielinizantes o síndrome de Felty.
    ? Administración de vacunas vivas durante los 3 meses anteriores al inicio del ensayo, durante el ensayo y hasta los 3 meses posteriores a la última dosis.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of participants with resolution of initial flare at time of the randomization and absence of new flares.
    To demonstrate significant reduction of disease activity with canakinumab versus placebo
    Proporción de pacientes con resolucion del brote incial en el momento de la randomizacion y ausencia de nuevos brotes. Demostrar la reduccion significativa de la actividad de la enfermedad con canakinumab comparado con placebo
    E.5.1.1Timepoint(s) of evaluation of this end point
    16 weeks
    16 semanas
    E.5.2Secondary end point(s)
    Percentage of participants who achieve Physician's global assessment < 2
    Percentage of participants with the serologic remission
    Porcentaje de pacientes que consigan una Evaluación global de la actividad de la enfermedad por parte del médico < 2 Porcentaje de pacientes con remision serologica
    E.5.2.1Timepoint(s) of evaluation of this end point
    16 weeks
    16 semanas
    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 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 No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 EEA47
    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
    Belgium
    Canada
    Denmark
    Finland
    France
    Germany
    Greece
    Hungary
    Ireland
    Israel
    Italy
    Japan
    Netherlands
    Russian Federation
    Spain
    Switzerland
    Turkey
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    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
    LVLS
    ultima visita del ultimo paciente
    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 months7
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months7
    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: 100
    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: 40
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 60
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 80
    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 Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Parent or legal guardian?s written informed consent and child?s assent, if appropriate, are required before any assessment is performed for patients < 18 years of age.
    Se deberá obtener el consentimiento informado escrito de los padres o tutor legal y el asentimiento del niño, si aplica, antes de realizar cualquier evaluación en el caso de pacientes < 18 años de edad
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state13
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 140
    F.4.2.2In the whole clinical trial 180
    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)
    None
    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 Decision2014-05-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-05-05
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-07-04
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