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    Summary
    EudraCT Number:2016-002814-29
    Sponsor's Protocol Code Number:CETB115E2403
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Restarted
    Date on which this record was first entered in the EudraCT database:2016-12-16
    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 number2016-002814-29
    A.3Full title of the trial
    SOAR Trial, A two-part study: Interventional phase II single-arm trial to assess efficacy and safety of Eltrombopag combined with cyclosporine as first line therapy in patients with severe acquired aplastic anemia, and an extension with up to 60-months follow-up.
    Ensayo SOAR, estudio con dos partes: fase II intervencionista de brazo único para evaluar la eficacia y seguridad de eltrombopag combinado con ciclosporina como tratamiento de primera línea en pacientes con aplasia medular adquirida grave, y un periodo de extensión de hasta 60 meses de seguimiento
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    SOAR Trial: Eltrombopag combined with cyclosporine as first line therapy in patients with severe acquired aplastic anemia with follow-up up to 60-months.
    Ensayo SOAR: Eltrombopag combinado con ciclosporina como tratamiento de primera línea en pacientes con aplasia medular adquirida grave con un seguimiento de hasta 60 meses
    A.3.2Name or abbreviated title of the trial where available
    SOAR
    A.4.1Sponsor's protocol code numberCETB115E2403
    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 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 Médico (GMO)
    B.5.3 Address:
    B.5.3.1Street AddressGran Vía de les Corts Catalanes, 764
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08013
    B.5.3.4CountrySpain
    B.5.4Telephone number+34 900 353036
    B.5.5Fax number+34 93 2479903
    B.5.6E-maileecc.novartis@novartis.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 REVOLADE
    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 nameEltrombopag
    D.3.2Product code ETB115
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    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 INNELTROMBOPAG
    D.3.9.1CAS number 496775-61-2
    D.3.9.2Current sponsor codeETB115
    D.3.9.4EV Substance CodeSUB30140
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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.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 REVOLADE
    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 nameEltrombopag
    D.3.2Product code ETB115
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    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 INNELTROMBOPAG
    D.3.9.1CAS number 496775-61-2
    D.3.9.2Current sponsor codeETB115
    D.3.9.4EV Substance CodeSUB30140
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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
    First-line severe aplastic anaemia
    Primera línea en pacientes con aplasia medular adquirida grave
    E.1.1.1Medical condition in easily understood language
    First-line severe aplastic anaemia
    Primera línea en pacientes con aplasia medular adquirida grave
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level PT
    E.1.2Classification code 10002967
    E.1.2Term Aplastic anaemia
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of eltrombopag + cyclosporine as first-line therapy on overall hematologic response (neutrophil, platelet, hemoglobin) by 6 months
    Evaluar la eficacia de eltrombopag + ciclosporina como tratamiento de primera línea en la respuesta hematológica global (neutrófilos, plaquetas, hemoglobina) a los 6 meses
    E.2.2Secondary objectives of the trial
    Obj. 1: Evaluate the effect of ETB + CsA on overall hematologic response (neutrophil, platelet, hemoglobin) by 3 and 12 months
    All of the following secondary objectives will be assessed by 6 months, 30 months and 60 months as appropriate and will be reported in a cumulative basis
    Obj. 2: Evaluate the duration of hematologic response
    Obj. 3: Evaluate disease relapse rate
    Obj. 4: Evaluate the clonal evolution to myelodysplasia, paroxysmal nocturnal hemoglobinuria (PNH), and leukemia
    Obj. 5: Evaluate the need for blood transfusion
    Obj. 6: Evaluate the need for platelet transfusion
    Obj. 7: Evaluate the duration of platelet and blood transfusion independence
    Obj. 8: Evaluate overall survival (OS)
    Obj. 9: Evaluate the effect of ETB and CsA on patient symptoms and health related quality of life
    Obj. 10: Evaluate the safety and tolerability of ETB + CsA
    Obj. 11: Characterize the PK of ETB when combined to CsA
    -1: evaluar el efecto de eltrombopag + ciclosporina en la respuesta hematológica global (neutrófilos, plaquetas, hemoglobina) a los 3 y 12 meses.
    Todos estos objetivos secundarios se evaluarán a los 6, 30 y 60 meses según proceda y se notificarán de manera acumulativa.
    -2: evaluar la duración de la respuesta hematológica.
    -3: evaluar la tasa de recidiva de la enfermedad.
    -4: evaluar la evolución clonal hacia mielodisplasia, HPN leucemia.
    - 5: evaluar la necesidad de una transfusión de hematíes.
    - 6: evaluar la necesidad de una transfusión de plaquetas.
    -7: evaluar la duración de la independencia de la transfusión de plaquetas y hematíes
    - 8: evaluar la supervivencia global (OS)
    - 9: evaluar el efecto de eltrombopag y ciclosporina en los síntomas y la calidad de vida relacionada con la salud del paciente.
    - 10: evaluar la seguridad y tolerancia de eltrombopag + ciclosporina.
    - 11: caracterizar la PK de eltrombopag combinado con ciclosporina.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Patient has signed the Informed Consent (ICF) prior to any screening procedures being performed.
    2.Patient is male/female ≥6 years old at the time of informed consent and able to swallow a tablet.
    3.Patient has SAA characterized by:
    a.Bone marrow cellularity <30% (excluding lymphocytes) and
    b.At least two of the following (peripheral blood):
    •Absolute neutrophil count <500/ µL
    •Platelet count <20,000/ µL
    •Absolute reticulocyte count <60,000/ µL
    4.Normal ECG defined as the following as determined via the mean of a triplicate ECG
    • Resting heart rate
    6-<12 years: 60-130 bpm
    12-<18 years: 60-120 bpm
    ≥18 years: 50-90 bpm
    •QTcF at screening <450 msec (male patients), 460 msec (female patients)
    1. Pacientes que hayan firmado el consentimiento informado (FCI) antes de realizar cualquier procedimiento de selección.
    2. Pacientes de ambos sexos ≥ 6 años de edad en el momento de la obtención del consentimiento informado que sean capaces de tragar un comprimido.
    3. Pacientes con AMG caracterizada por:
    a. Celularidad de la médula ósea < 30 % (salvo linfocitos) y
    b. al menos dos de los siguientes (sangre periférica):
    -Recuento absoluto de neutrófilos < 500/µl.
    -Recuento de plaquetas < 20.000/ µl.
    -Recuento absoluto de reticulocitos < 60.000/µl.
    4. ECG normal definido del siguiente modo y determinado mediante la media de un ECG triplicado.
    E.4Principal exclusion criteria
    1.Diagnosis of Fanconi anemia.
    2.Evidence of a clonal hematologic bone marrow disorder on cytogenetics. Patients with very severe neutropenia (ANC < 200 /μL) will not be excluded initially if cytogenetics are not available or pending. If a clonal disorder is identified, the patient will be excluded.
    3.Prior immunosuppressive therapy with cyclosporine, alemtuzumab, rabbit or horse ATG and thrombopoietin receptor (TPO-R) agonists.
    4.Hypersensitivity to eltrombopag or its components.
    5.AST or ALT >3 x ULN.
    6.Creatinine, total bilirubin, and alkaline phosphatase >3 x ULN .
    7.Patient with liver cirrhosis.
    8.Infection not adequately controlled with appropriate therapy.
    9.Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient’s ability to consent, be compliant with study procedures, tolerate protocol therapy, or that death within 30 days is likely.
    10.Patients with cancer who are not considered cure, are on active chemotherapeutic treatment or who take drugs with hematological effects.
    11.Administration of an investigational drug within 30 days or 5 half-lives, whichever is longer, preceding the first dose of study treatment.
    12.Pregnancy statements and contraception requirements:
    Pregnancy or nursing (lactating) women
    Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant (or female partners of male patients), unless they are using highly effective methods of contraception during dosing and for 3 months after stopping medication.
    In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment.
    13.Not able to understand the investigation nature of the study or to give informed consent.
    14.Clinically significant ECG abnormality including cardiac arrhythmias (e. g. ventricular tachycardia) complete left bundle branch block, high grade atrioventricular block, or inability to determine the QTcF interval on the ECG.
    15.Presence of cardiac disease, or family history of idiopathic sudden death or congenital long QT syndrome.
    16.Risk factors for Torsades de Pointe including uncorrected hypokalemia or hypomagnesemia, or use of concomitant medication(s) with a known risk to prolong the QT interval that cannot be discontinued.
    1. Diagnóstico de anemia de Fanconi.
    2. Evidencia de un trastorno hematológico clonal de la médula ósea en la citogenética. Los pacientes con neutropenia muy grave (RAN < 200 /μl) no quedarán excluidos inicialmente si la citogenética no está disponible o está pendiente. Si se identifica un trastorno clonal, el paciente quedará excluido
    3. Tratamiento inmunosupresor previo con ciclosporina, alemtuzumab, GAT de conejos o caballos y agonistas del receptor de trombopoyetina (TPO-R).
    4. Hipersensibilidad a eltrombopag o sus componentes.
    5. AST o ALT >3 x LSN.
    6. Creatinina, bilirrubina total y fosfatasa alcalina >3 x LSN.
    7. Paciente con cirrosis hepática.
    8. Infección no controlada adecuadamente con el tratamiento apropiado.
    9. Estado paliativo o enfermedad hepática, renal, cardíaca, neurológica,
    pulmonar, infecciosa o metabólica concurrente de tal gravedad que impidiera la capacidad del paciente para otorgar su consentimiento, cumplir los procedimientos del estudio, tolerar el tratamiento del protocolo, o que pudiera ocasionar la muerte en los 30 días posteriores.
    10. Pacientes con cáncer que no se consideren curados, que estén recibiendo tratamiento quimioterapéutico activo o que tomen fármacos con efectos hematológicos.
    11. Administración de un fármaco en investigación en los 30 días o 5 vidas medias antes a la primera dosis del tratamiento del estudio, aquel periodo que sea más largo.
    12. Declaraciones sobre embarazo y requisitos relativos a los métodos anticonceptivos:
    Mujeres embarazadas o en periodo de lactancia.
    Mujeres en edad fértil, definidas como toda mujer fisiológicamente capaz de quedarse embarazada (o parejas mujeres de pacientes varones), salvo que estén utilizando métodos anticonceptivos altamente eficaces durante la administración de la dosis y durante los 3 meses posteriores a la suspension de la medicación. Los métodos anticonceptivos altamente eficaces incluyen:
    Abstinencia total (cuando esté en consonancia con el estilo de vida habitual y preferido del sujeto). Abstinencia periódica (p. ej., calendario, ovulación, métodos sintotérmicos o postovulación) y el coitus interruptus no son métodos anticonceptivos aceptables.
    Esterilización femenina (cuando se le haya realizado una ooforectomía bilateral con o sin histerectomía), histerectomía total o ligadura de trompas al menos 6 semanas antes de recibir el tratamiento del estudio.
    Si sólo se ha realizado ooforectomía, únicamente cuando se haya confirmado el estado reproductor de la mujer mediante la evaluación de seguimiento del nivel hormonal.
    Esterilización masculina (al menos 6 meses antes de la selección). Los varones a quienes se les haya practicado una vasectomía deberán ser la única pareja del sujeto en cuestión.
    Uso de métodos anticonceptivos hormonales orales, inyectados o implantados, o implantación de un dispositivo intrauterino (DIU) o sistema intrauterino (SIU) u otros métodos anticonceptivos hormonales que tengan una eficacia comparable (tasa de fallo < 1%), por ejemplo, anillo vaginal hormonal o anticonceptivo hormonal transdérmico.
    Varones sexualmente activos, salvo que utilicen un preservativo durante el coito mientras estén tomando el fármaco durante el tratamiento y durante los 3 meses posteriores a la suspensión del tratamiento; no deberán engendrar un bebé durante este periodo. El uso de preservativos también es necesario en hombres sometidos a vasectomía, así como durante la relación sexual con una pareja varón a fin de evitar la emisión del fármaco a través del semen. Las mujeres que tomen anticonceptivos orales deberán tomar la misma píldora durante un mínimo de 3 meses antes de recibir el tratamiento del estudio.
    13. Pacientes que no puedan entender la naturaleza de investigación del estudio o que no puedan otorgar su consentimiento informado.
    14. Anomalía clínicamente significativa en el ECG que incluye arritmias cardíacas (p. ej., taquicardia ventricular), bloqueo completo de rama izquierda, bloqueo atrioventricular de grado alto o incapacidad de determinar el intervalo QTcF en el ECG.
    15. Presencia de enfermedad cardíaca o antecedentes familiares de muerte súbita idiopática o síndrome del QT prolongado congénito.
    16. Factores de riesgo de Torsades de Pointe que incluyen hipopotasemia o hipomagnesemia no corregidas, o uso de medicación concomitante que conlleva un riesgo conocido de prolongar el intervalo QT que no se pueda retirar.
    E.5 End points
    E.5.1Primary end point(s)
    Primary analysis will be done after all the patients enrolled have completed 6 months of treatment with eltrombopag+cyclosporine or discontinued treatment with eltrombopag prior to 6 months for evaluating the primary endpoint.
    El análisis principal se realizará después de que todos los pacientes incluidos hayan completado 6 meses de tratamiento con eltrombopag + ciclosporina o hayan retirado el tratamiento con eltrombopag antes de 6 meses para evaluar la variable principal.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The primary objective of the study is to evaluate the eltrombopag + cyclosporine as first-line therapy as assessed by the overall hematologic response rate by 6 months. Bayesian approach will be used for analysis of the primary endpoint. The primary analysis will be based on the calculation of observed overall hematologic response rate by 6 months and its posterior distribution using a beta-binomial model. Combination therapy of eltrombopag + cyclosporine will be declared efficacious if the following criteria are met:
    a. Observed hematologic ORR ≥ “clinically meaningful” threshold (30%)
    b. Probability of true ORR “not being clinically meaningful (response ≤ 20%) is less than 10%.
    Para el análisis de la variable principal se utilizará un método bayesiano. El análisis principal se basará en el cálculo de la tasa de respuesta hematológica global observada a los 6 meses y su distribución posterior mediante un modelo beta-binomial. El tratamiento de combinación de eltrombopag + ciclosporina se declarará eficaz si se cumplen los siguientes criterios:
    a. ORR hematológica observada ≥ umbral «clínicamente significativo»(30 %).
    b. La probabilidad de la ORR verdadera «no clínicamente significativa (respuesta ≤ 20 %)» es inferior al 10 %.
    E.5.2Secondary end point(s)
    Second analysis will be done after all the patients enrolled have completed 30 months of treatment with cyclosporine or discontinued treatment with cyclosporine prior to 30 months.
    El análisis secundario se realizará después de que todos los pacientes incluidos hayan completado 30 meses de tratamiento con ciclosporina o hayan retirado el tratamiento con ciclosporina antes 30 meses.
    E.5.2.1Timepoint(s) of evaluation of this end point
    The primary analysis will be conducted and the primary CSR will be written 6 M (months) after all the patients enrolled have completed 6 M of treatment with eltrombopag+cyclosporine or discontinued treatment with eltrombopag prior to 6 M.
    The second CSR will be written based on the cumulative data obtained after all the patients enrolled have completed 30 M tapering of cyclosporine or discontinued treatment with cyclosporine prior to 30 M.
    The final analysis will be done after all the patients enrolled completed 60 M in the study or discontinued the study prior to 60 M, based on which the final CSR will be written.
    Overall survival will be reported by 6 months (EOT with Eltrombopag), by 30 M (EOT with Cyclosporine) and by the completion of 60 M (end of study).
    El análisis principal y el Informe clinico (CSR) del estudio primario serán realizados 6M después de que todos los pacientes reclutados hayan completado 6M de tto. con Eltrombopag+ Ciclosporina o hayan retirado el tto. con eltrombopag antes de 6M.
    El segundo CSR será escrito en base a los datos obtenidos después de que todos los pacientes incluidos hayan completado 30M de tto. con ciclosporina o hayan retirado el tto. con ciclosporina antes 30M.
    El análisis final se realizará después de que todos los pacientes incluidos hayan permanecido 60M en el estudio o se hayan retirado del estudio antes de 60M, en base a lo cuál será escrito el CSR final.
    La supervivencia global será reportada a los 6M(EoT con eltrombopag), a los 30 meses (EoT con Ciclosporina) y a los 60M (final del studio)
    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 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 Yes
    E.6.13.1Other scope of the trial description
    Biomarkers
    Biomarcadores
    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 No
    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 No
    E.8.2.4Number of treatment arms in the trial1
    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 EEA10
    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
    Brazil
    Canada
    France
    Hong Kong
    Hungary
    India
    Italy
    Korea, Republic of
    Mexico
    Netherlands
    Qatar
    Spain
    Turkey
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months3
    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: 10
    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: 3
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 35
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 21
    F.4.2.2In the whole clinical trial 50
    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)
    Second treatment line for SAA
    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 Decision2017-03-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-01-18
    P. End of Trial
    P.End of Trial StatusRestarted
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