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    Summary
    EudraCT Number:2014-002388-13
    Sponsor's Protocol Code Number:AZA-JMML-001
    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:2014-11-04
    Trial 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 number2014-002388-13
    A.3Full title of the trial
    A Phase 2, multicenter, open-label study to evaluate the pharmacokinetics, pharmacodynamics, safety and activity of azacitidine and to compare azacitidine to historical controls in pediatric subjects with newly diagnosed advanced myelodysplastic syndrome or juvenile myelomonocytic leukemia before hematopoietic stem cell transplantation.
    Estudio de fase 2, multicéntrico, abierto para evaluar la farmacocinética, farmacodinámica, seguridad y actividad de azacitidina y para comparar azacitidina con los controles históricos en sujetos pediátricos con nuevo diagnóstico de síndrome mielodisplásico avanzado o leucemia mielomonocítica juvenil antes de trasplante de células madre hematopoyéticas
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    To determine the safety, clinical efficacy, and effect of azacitidine on the body,within the body and interactions within the body in children and young adults with newly diagnosed advanced Myelodysplastic Syndromes or Juvenile myelomonocytic leukemia before transplantation of the bone marrow and blood. At the end of the study, the data will be compared to data from an older study of patients with the same diseases.
    Para determinar la seguridad, eficacia clínica, y el efecto de azacitidina en el cuerpo, dentro del cuerpo y las interacciones dentro del cuerpo en niños y jóvenes adultos con nuevo diagnóstico de síndromes mielodisplásicos avanzados o leucemia mielomonocítica juvenil antes del trasplante de médula ósea y sangre. Al final del estudio, los datos serán comparados con los datos de un estudio anterior de pacientes con las mismas enfermedades.
    A.4.1Sponsor's protocol code numberAZA-JMML-001
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/031/2014
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCelgene Corporation
    B.1.3.4CountryUnited States
    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 supportCelgene Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCelgene Corporation
    B.5.2Functional name of contact pointClinicalTrialDisclosure
    B.5.3 Address:
    B.5.3.1Street Address9225 Indian Creek Parkway, Suite 900
    B.5.3.2Town/ cityOverland Park, Kansas
    B.5.3.3Post code66210
    B.5.3.4CountryUnited States
    B.5.4Telephone number+34 91 422 90 00
    B.5.5Fax number+1 913-266-0394
    B.5.6E-mailClinicalTrialDisclosure@celgene.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 Vidaza 25 mg/ml polvo para suspensión inyectable
    D.2.1.1.2Name of the Marketing Authorisation holderCelgene Europe 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 Yes
    D.2.5.1Orphan drug designation numberEU/3/01/084
    D.3 Description of the IMP
    D.3.1Product nameAzacitidina
    D.3.4Pharmaceutical form Powder for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAZACITIDINA
    D.3.9.1CAS number 320-67-2
    D.3.9.4EV Substance CodeSUB05624MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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 Vidaza 25 mg/ml polvo para suspensión inyectable
    D.2.1.1.2Name of the Marketing Authorisation holderCelgene Europe 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 Yes
    D.2.5.1Orphan drug designation numberEU/3/01/084
    D.3 Description of the IMP
    D.3.1Product nameAzacitidina
    D.3.4Pharmaceutical form Powder for suspension 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 INNAZACITIDINA
    D.3.9.1CAS number 320-67-2
    D.3.9.4EV Substance CodeSUB05624MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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
    newly diagnosed advanced myelodysplastic syndrome (MDS) or juvenile myelomonocytic leukemia (JMML) prior to hematopoietic stem cell transplantation (HSCT)
    Tratamiento de sujetos pediátricos con nuevo diagnóstico de síndrome mielodisplásico (SMD) avanzado o leucemia mielomonocítica juvenil (LMMJ) antes del trasplante de células madre hematopoyéticas (TCMH)
    E.1.1.1Medical condition in easily understood language
    JMML is a chronic cancer of blood affecting children whereby liver and spleen enlarges due to abnormal growth of bone marrow.MDS is a disorder whereby bone marrow produces fewer/immature blood cells
    LMMJ es cáncer crónico de sangre que afecta a niños por el que hígado y bazo se agrandan por crecimiento anormal de médula ósea.SMD es trastorno por el que médula ósea produce menos/inmaduros glóbulos
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level LLT
    E.1.2Classification code 10054439
    E.1.2Term Juvenile chronic myelomonocytic leukemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level HLT
    E.1.2Classification code 10028536
    E.1.2Term Myelodysplastic syndromes
    E.1.2System Organ Class 100000004851
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level PT
    E.1.2Classification code 10023249
    E.1.2Term Juvenile chronic myelomonocytic leukaemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to assess the treatment effect on response rate (MDS: CR, PR, or marrow CR; JMML: either cCR or cPR) at Cycle 3 Day 28 and to compare against standard therapy using a matched-pairs analysis with historical data.
    El objetivo principal consiste en evaluar el efecto del tratamiento sobre la tasa de respuesta (SMD: remisión completa [RC], remisión parcial [RP] o RC en médula ósea; LMMJ: remisión completa clínica [RCc] o remisión parcial clínica [RPc]) el día 28 del ciclo 3 y en compararlo con el tratamiento convencional mediante un análisis por parejas de datos históricos.
    E.2.2Secondary objectives of the trial
    The secondary objectives are to further evaluate safety, efficacy, pharmacodynamics (PD), and pharmacokinetics (PK) of azacitidine in this subject population.
    El objetivo secundario consiste en evaluar mejor la seguridad, eficacia, farmacocinética (FC) y farmacodinámica (FD) de azacitidina en esta población de sujetos.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    MDS:

    1. Patient has newly diagnosed advanced primary or secondary MDS with an amount of immature cells in blood or bone marrow or chromosomal abnormality linked to secondary MDS. Blood or bone marrow samples confirming diagnosis within 14 days prior to ICF as for the MDS.

    JMML:

    1. Patient has newly diagnosed JMML, with samples from blood and bone marrow confirming diagnosis within the 14 days prior to informed consent/informed assent signature, with specific alteration in genes (which carry the information that determines a person characteristics) in the body

    Both MDS and JMML:

    2.Patient has a Lansky play score/ Karnofsky performance status at least equal to 60
    3.Patient has a normal renal function and a normal liver function.
    4.Subjects should be between 1 month to less than 18 years at time of signing ICF/ IAF
    SMD:

    1.Nuevo diagnóstico de SMD primario o secundario avanzado con una cantidad de células inmaduras en sangre o médula ósea o anomalía cromosómica ligada a SMD secundario, con análisis de SP y MO que confirmen el diagnóstico en los 14 días previos a la firma del consentimiento informado

    LMMJ:

    1.Nuevo diagnóstico de LMMJ, con análisis de SP y MO que confirmen el diagnóstico en los 14 días previos a la firma del consentimiento informado, con alteración específica en los genes (que llevan la información que determina las características de una persona) en el cuerpo

    Para SMD y LMMJ:

    2. Puntuación de juego de Lansky o estado funcional de Karnofsky mayor o igual a 60
    3. Pacientes con función hepática y renal normal
    4. Pacientes de edad comprendida entre 1 mes y menos de 18 años en el momento de otorgar el consentimiento o asentimiento informado
    E.4Principal exclusion criteria
    MDS exclusions:

    1.Patient has an illness caused by 'genetic defects' (which cause abnormalities in the information that determine a person's characteristics).
    2.Patient has inherited disease that cause bone marrow (the soft tissue inside of the bone) failures.

    JMML Exclusion:
    1.Patient has a specific deviation in so called Germline.

    Both:
    1.Patient has any other organ dysfunction that will interfere with the administration of the therapy according to this protocol.
    2.Hypersensitivity to azacitidine
    SMD:

    1.Paciente con una enfermedad causada por "defectos genéticos" (que causan anomalías en la información que determina las características de una persona).
    2. Paciente que ha heredado la enfermedad que causa los fracasos de la médula ósea (el tejido blando del interior de los huesos).

    LMMJ:

    1. Pacientes con una desviación específica en la llamada línea germinal

    Ambos:
    1. Cualquier otra disfunción orgánica que interfiera en la administración del tratamiento conforme a este protocolo
    2. Alergia a azacitidina
    E.5 End points
    E.5.1Primary end point(s)
    MDS:
    Proportion of subjects with CR, PR or marrow CR according to modified criteria based on Table 3 in Cheson 2006, adapted to pediatric reference values at 3 months (Cycle 3, Day 28). Response must be sustained for at least 4 weeks either in the 4-week period preceding or succeeding Cycle 3 Day 28 (ie, sustained over the period Cycle 2 Day 28 to Cycle 3 Day 28, or Cycle 3 Day 28 to Cycle 4 Day 28).

    JMML:
    Proportion of subjects with sustained cCR or cPR according to the International JMML response criteria in Niemeyer 2014 at 3 months (Cycle 3, Day 28). Response must be sustained for at least 4 weeks either in the 4-week period preceding or succeeding Cycle 3 Day 28 (ie, sustained over the period Cycle 2 Day 28 to Cycle 3 Day 28, or Cycle 3 Day 28 to Cycle 4 Day 28).
    Tasa de respuesta el día 28 del ciclo 3
    -SMD: proporción de sujetos con RC, RP o RC en médula ósea con arreglo a los criterios modificados recogidos en la tabla 3 del artículo de Cheson 2006, adaptados a valores de referencia pediátricos, al cabo de 3 meses (día 28 del ciclo 2). La respuesta deberá mantenerse durante al menos 4 semanas en el período de 4 semanas que preceda o suceda al día 28 del ciclo 3 (es decir, mantenida durante el período comprendido entre el día 28 del ciclo 2 y el día 28 del ciclo 3 o entre el día 28 del ciclo 3 y el día 28 del ciclo 4).
    -LMMJ: proporción de sujetos con RCc o RPc mantenida con arreglo a los criterios internacionales de respuesta de la LMMJ (Niemeyer 2014) al cabo de 3 meses (día 28 del ciclo 2). La respuesta deberá mantenerse durante al menos 4 semanas en el período de 4 semanas que preceda o suceda al día 28 del ciclo 3 (es decir, mantenida durante el período comprendido entre el día 28 del ciclo 2 y el día 28 del ciclo 3 o entre el día 28 del ciclo 3 y el día 28 del ciclo 4).
    E.5.1.1Timepoint(s) of evaluation of this end point
    MDS:
    Response will be evaluated on Day 1 of Cycles 2 and 3, Day 28 of Cycle 3, if there is a suspected disease progression after Cycle 3 Day 28, Day 28 of Cycle 6 (if applicable), and pre-HSCT (Hematopoietic Stem Cell Transplantation , transplant of cells)

    JMML:
    Response will be evaluated on Day 1 of Cycles 2 and 3, Day 28 of Cycle 3, if there is a suspected disease progression after Cycle 3 Day 28, Day 28 of Cycle 6 (if applicable), and pre-HSCT (Hematopoietic Stem Cell Transplantation , transplant of cells)
    MDS:
    La respuesta será evaluada en el día 1 de los ciclos 2 y 3, el día 28 del ciclo 3, si hay una progresión de la enfermedad sospechada después de Ciclo 3 Día 28, Día 28 del Ciclo 6 (si procede), y pre-TCMH (trasplante de células madre hematopoyéticas, trasplante de células)

    LMMJ:
    La respuesta será evaluada en el día 1 de los ciclos 2 y 3, el día 28 del ciclo 3, si hay una progresión de la enfermedad sospechada después de Ciclo 3 Día 28, Día 28 del Ciclo 6 (si procede), y pre-TCMH (trasplante de células madre hematopoyéticas, trasplante de células)
    E.5.2Secondary end point(s)
    1.Cytogenetic Response for MDS subjects
    2.Cytogenetic and Molecular Response for JMML subjects
    3.Duration of response (DoR)
    4. Time to response (TTR)
    5.Time to progression (TTP)
    6.Leukemia free survival (LFS)
    7.Overall survival (OS)
    8.Deoxyribonucleic acid methylation status in BM on Days 1 and 15 of Cycle 1, Day 28 of Cycle 3, pre-HSCT, and at the time of relapse/progression
    9.Percentage of subjects undergoing HSCT
    10.Time to first HSCT
    11.Safety defined by frequency and severity of treatment related AEs
    12.Pharmacokinetics (PK)
    1 Respuesta citogenética en los sujetos con SMD;
    2 Respuesta citogenética y molecular en los sujetos con LMMJ.
    3 Duración de la respuesta (DR).
    4 Tiempo hasta la respuesta (THR).
    5 Tiempo hasta la progresión (THP).
    6 Supervivencia sin leucemia (SSL).
    7 Supervivencia global (SG).
    8 Estado de metilación del ADN en MO los días 1 y 15 del ciclo 1, el día 28 del ciclo 3, antes del TCMH y en el momento de la recidiva o progresión.
    9 Porcentaje de sujetos que se sometan a un TCMH.
    10 Tiempo hasta el primer TCMH.
    11 Seguridad definida por la frecuencia e intensidad de los AA relacionados con el tratamiento.
    12 Farmacocinética (FC).
    E.5.2.1Timepoint(s) of evaluation of this end point
    1,2.C1D1,C2D1,C3D1,D28 if there is suspected disease progression after C3D28,C6D28 (if applicable)&pre-HSCT(only if >21 days since the last BM aspirate)in order to evaluate response to treatment
    3.1st observed response until either disease progression/any cause of death
    4.1st study dose day until a response.
    5.1st study dose day until either disease progression/death due to progression
    6,7,9,10.during follow-up period
    8.C1D1,C1D15,C3D28, at time of relapse/disease progression&pre-HSCT for extraction of DNA
    11.during the course of study
    12.C1D5,D6 Prior to dosing,C1D7 prior to dosing,postdose 5mins(IV use azacitidine application) or 15mins postdose(SC use),postdose 30mins, 1hr, 2hrs, 4hrs, 6hrs IV and SC use.
    1,2.C1D1, C2D1, C3D1, D28 si hay sospecha de progresión de enfermedad después de C3D28,C6D28(si procede) y pre-TCMH(sólo si> 21 días desde último aspirado de MO)con el fin de evaluar la respuesta al tratamiento
    3.Primera respuesta observada hasta progresión de enfermedad/cualquier causa de muerte
    4.Primer día de dosis estudio hasta respuesta.
    5.Primer día de dosis estudio hasta progresión de enfermedad/muerte por progresión
    6,7,9,10.durante periodo de seguimiento
    8.C1D1, C1D15, C3D28, en momento de recaída/progresión de enfermedad y pre-TCMH para extracción de ADN
    11.durante curso de estudio
    12.C1D5, D6 Antes de dosis, C1D7 antes de dosis, 5 min después de dosis (azacitidina IV) o 15 min después de dosis (SC), después de dosis 30 minutos, 1h, 2hrs,4hrs, 6hrs IV y uso SC.
    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 Yes
    E.6.7Pharmacodynamic Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    comparación con grupo control histórico, utilizando datos recabados retrospectivos de registro EWOG
    comparison against a historical control arm using data collected retrospectively from EWOG registry
    E.8.2.4Number of treatment arms in the trial2
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA44
    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 No
    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
    End of trial is the date of the last visit of the last subject to complete the study, or the date of receipt of the last data point from the last subject that is required for primary, secondary and/or exploratory analysis, as pre-specified in the protocol and/or the Statistical Analysis Plan (SAP), whichever is the later date.
    El final del estudio se define como la fecha de la última visita del último sujeto que finalice el estudio o la fecha de recepción de los últimos datos correspondientes al último sujeto que sean necesarios para los análisis principales, secundarios o exploratorios, tal como se establece en el protocolo o el Plan de Análisis Estadístico (PAE), la que sea posterior
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    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: 55
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 33
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 17
    F.1.2Adults (18-64 years) No
    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 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
    Trial will involve children from one months old
    El ensayo involucrará a niños de un mes de edad
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 55
    F.4.2.2In the whole clinical trial 55
    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)
    Subjects should be treated as per standard of care and according to their institition?s procedures after they discontinue from the study. Some patients will go on to transplantation (which is also the standard of care).
    Los sujetos deben ser tratados según el estándar de tratamiento y de acuerdo con los procedimientos de las instituciones después de haber dejado el estudio. Algunos pacientes pasarán a trasplante (que también es el estándar de tratamiento).
    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 Decision2014-12-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-12-15
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-05-24
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