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    EudraCT Number:2018-003411-21
    Sponsor's Protocol Code Number:FEDR-MF-002
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-04-12
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2018-003411-21
    A.3Full title of the trial
    A Phase 3, multicenter, open-label, randomized study to evaluate the efficacy and safety of fedratinib compared to best available therapy in subjects with DIPSS - intermediate or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis and previously treated with ruxolitinib
    Estudio en fase III, multicéntrico, abierto y aleatorizado para evaluar la eficacia y la seguridad del fedratinib comparado con la mejor terapia disponible en pacientes con mielofibrosis primaria de riesgo intermedio o alto riesgo según el Sistema dinámico internacional de puntuación pronóstica (DIPSS), mielofibrosis post-policitemia vera o mielofibrosis post-trombocitemia esencial tratados previamente con ruxolitinib
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study comparing current standard therapies with fedratinib for the treatment of myelofibrosis (either diagnosed alone or after polycythemia vera or essential thrombocytopenia) in subjects who have previously received ruxolitinib
    Estudio en el que se comparan las actuales terapias de referencia con fedratinib para el tratamiento de la mielofibrosis (diagnosticada sola o después de una policitemia vera o una trombocitemia esencial) en pacientes que han recibido previamente ruxolitinib
    A.3.2Name or abbreviated title of the trial where available
    The Freedom 2 trial
    A.4.1Sponsor's protocol code numberFEDR-MF-002
    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 SponsorImpact Biomedicines, Inc., a Wholly Owned Subsidiary of Celgene 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+1888260 1599
    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/3/10/794, EU/3/10/810, EU/3/10/811
    D.3 Description of the IMP
    D.3.1Product nameFedratinib
    D.3.4Pharmaceutical form Capsule
    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 INNfedratinib
    D.3.9.2Current sponsor codefedratinib
    D.3.9.3Other descriptive nameFEDRATINIB
    D.3.9.4EV Substance CodeSUB126288
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    Primary myelofibrosis, post-polycythemia vera myelofibrosis , or post-essential thrombocythemia myelofibrosis
    Mielofibrosis primaria, mielofibrosis post-policitemia vera o mielofibrosis post-trombocitemia esencial
    E.1.1.1Medical condition in easily understood language
    Cancer of the bone marrow where normal blood cell growth is affected and collagen fibers begin to develop in the marrow
    Cáncer de médula ósea en el que el crecimiento normal de las células sanguíneas se ve afectado y empiezan a formarse fibras de colágeno en la médula
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10028537
    E.1.2Term Myelofibrosis
    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
    To evaluate the percentage of subjects with at least 35% spleen volume reduction in the fedratinib and the BAT arms.
    Evaluar el porcentaje de pacientes con una reducción del volumen esplénico de al menos el 35 % en el grupo de fedratinib y en el de la mejor terapia disponible (MTD).
    E.2.2Secondary objectives of the trial
    To evaluate myelofibrosis (MF)-associated symptoms as measured by the Myelofibrosis Symptom Assessment Form (MFSAF) (Appendix B)
    To evaluate the percentage of subjects with at least 25% spleen volume reduction (SVR)
    To evaluate the safety of fedratinib
    To evaluate the reduction of spleen size by palpation
    To evaluate durability of spleen response by MRI/CT and by palpation
    To evaluate the durability of symptoms response
    To evaluate spleen and disease progression free survival
    To assess the effectiveness of the risk mitigation strategy for gastrointestinal events and Wernicke encephalopathy (WE)
    To evaluate Health-Related Quality of Life (HRQoL) as measured by the European Organization for Research and Treatment of Cancer Quality of Life C30 (EORTC QLQ-C30) (Appendix C)
    To evaluate Patient Reported Outcomes (PRO) as measured by the EQ-5D-5L questionnaire (Appendix D)
    To evaluate Overall Survival (OS)
    -Evaluar síntomas asociados a mielofibrosis según Formulario evaluación síntomas mielofibrosis (MFSAF) (anexo B)
    -Evaluar % pacientes que presenten una reducción de al menos el 25 % de su volumen esplénico (RVE)
    -Evaluar seguridad fedratinib
    -Evaluar, mediante palpación, la reducción tamaño bazo
    -Evaluar durabilidad de la respuesta esplénica mediante resonancia magnética nuclear (RMN)/tomografía computarizada (TC) y palpación
    -Evaluar durabilidad respuesta sintomática
    -Evaluar bazo y supervivencia sin progresión de la enfermedad
    -Valorar eficacia de la estrategia de atenuación de riesgos para acontecimientos gastrointestinales y la encefalopatía de Wernicke (EW)
    -Evaluar calidad de vida relacionada con la salud mediante Cuestionario calidad vida C30 de Organización europea para la investigación y tratamiento cáncer (EORTC QLQ-C30) (anexo C)
    -Evaluar resultados percibidos por pacientes (RPP) según resultados cuestionario EQ-5D-5L (anexo D)
    -Evaluar supervivencia global (SG)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject is at least 18 years of age at the time of signing the informed consent form (ICF)
    2. Subject has an Eastern Cooperative Oncology Group (ECOG) Performance Score (PS) of 0, 1 or 2
    3. Subject has diagnosis of primary myelofibrosis (PMF) according to the 2016 World Health Organization (WHO) criteria, or diagnosis of post-ET or post-PV myelofibrosis according to the IWG-MRT 2007 criteria,
    onfirmed by the most recent local pathology report
    4. Subject has a DIPSS Risk score of Intermediate-2 or High
    5. Subject has a measurable splenomegaly during the screening period as demonstrated by spleen volume of ≥ 450 cm3 by MRI or CT-scan and by palpable spleen measuring ≥ 5 cm below the left costal margin
    6. Subject has a measurable total symptoms score (≥ 1) as measured by the Myelofibrosis Symptom Assessment Form (MFSAF)
    7. Subject has been previously exposed to ruxolitinib, and must meet at least one of the following criteria (a and/or b)
    a. Treatment with ruxolitinib for ≥ 3 months with inadequate efficacy response (refractory) defined as < 10% spleen volume reduction by MRI or < 30% decrease from baseline in spleen size by palpation or regrowth (relapse) to these parameters following an initial response
    b. Treatment with ruxolitinib for ≥ 28 days complicated by any of the following (intolerant):
     Development of a red blood cell transfusion requirement (at least 2 units/month for 2 months) or
     Grade ≥ 3 AEs of thrombocytopenia, anemia, hematoma, and/or hemorrhage while on treatment with ruxolitinib
    8. Subject must have treatment-related toxicities from prior therapy resolved to Grade 1 or pretreatment baseline before start of last therapy prior to randomization
    9. Subject must understand and voluntarily sign an ICF prior to any study-related assessments/procedures being conducted
    10. Subject is willing and able to adhere to the study visit schedule and other protocol requirements
    11. A female of childbearing potential (FCBP) must:
    a. Have 2 negative pregnancy tests as verified by the Investigator during screening prior to starting study treatment. She must agree to ongoing pregnancy testing during the course of the study, and after end of study treatment. This applies even if the subject practices true abstinence from heterosexual contact.
    b. Either commit to true abstinence from heterosexual contact (which must be reviewed on a monthly basis and source documented) or agree to use and be able to comply with highly effective contraception without interruption, –14 days prior to starting investigational product, during the study treatment (including dose
    interruptions), and for 30 days after discontinuation of study treatment.
    12. A male subject must:
    Practice true abstinence (which must be reviewed on a monthly basis) or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions and for at least 30 days following investigational product discontinuation, or longer if required for each compound and/or by local regulations, even if he has undergone a successful vasectomy.
    1. El sujeto debe tener como mínimo 18 años de edad en el momento de la firma del formulario de consentimiento informado (FCI)
    2. El sujeto debe tener una puntuación de 0, 1 o 2 en la escala de estado funcional (EF) del Grupo Oncológico Cooperativo del Este (ECOG, Eastern Cooperative Oncology Group)
    3. Al sujeto le deben haber diagnosticado mielofibrosis primaria (MFP) según los criterios de la Organización Mundial de la Salud (OMS) de 2016 o mielofibrosis post-policitemia vera (post-PV) o post-trombocitemia esencial (post-TE) según los criterios del Grupo internacional de trabajo para el estudio y tratamiento de la mielofibrosis (IWG-MRT, International Working Group for Myelofibrosis Research and Treatment) 2007, confirmado mediante el informe anatomopatológico más reciente
    4. El sujeto debe tener una puntuación de riesgo moderado-2 o alto según el DIPSS
    5. El sujeto debe tener esplenomegalia mensurable durante el periodo de selección, manifestada por un volumen esplénico >= 450 cm3 en una RMN o TC y por un tamaño del bazo a la palpación >= 5 cm bajo el margen costal izquierdo
    6. El paciente tiene una puntuación de síntomas totales mensurables >= 1 según el formulario de evaluación de los síntomas de mielofibrosis (MFSAF)
    7. El sujeto debe haber estado expuesto previamente al ruxolitinib y cumplir al menos uno de los siguientes criterios (a y/o b)

    a. Tratamiento con ruxolitinib durante un período >= 3 meses con una respuesta de eficacia insuficiente (resistente), definida como una reducción < 10 % del volumen esplénico determinada mediante RMN o una disminución< 30 % con respecto al momento basal del tamaño del bazo a la palpación o recaída (recidiva) a dichos parámetros tras una respuesta inicial
    b. Tratamiento con ruxolitinib durante un período >= 28 días, complicado por alguna de las siguientes circunstancias (intolerancia):

    - Necesidad de una transfusión de eritrocitos (al menos 2 unidades/mes durante 2 meses) o
    - AA de trombocitopenia, anemia, hematoma y/o hemorragia de grado >= 3 durante el tratamiento con ruxolitinib

    8. Las toxicidades relacionadas con la terapia previa deben haber remitido hasta el grado 1 o hasta el valor basal previo al tratamiento antes del inicio de la última terapia anterior a la aleatorización
    9. El sujeto debe entender y firmar voluntariamente un FCI antes de someterse a ninguna evaluación o procedimiento asociado al estudio
    10. El sujeto debe querer y poder cumplir el calendario de visitas del estudio y otros requisitos del protocolo
    11. Las mujeres en edad fértil (MEF) deben:
    a. Obtener 2 resultados negativos en sendas pruebas de embarazo, verificados por el investigador durante la selección antes de comenzar el tratamiento del estudio. Acceder a realizarse pruebas de embarazo continuadas a lo largo del estudio y tras finalizar el tratamiento del estudio. Esto es válido incluso si la paciente mantiene una abstinencia real en lo que respecta a las relaciones sexuales heterosexuales.
    b. Comprometerse a mantener una abstinencia real en lo que respecta a las relaciones sexuales heterosexuales (que deberá revisarse mensualmente y registrarse en el documento fuente) o estar de acuerdo y ser capaz de usar métodos anticonceptivos muy eficaces de manera ininterrumpida desde los 14 días anteriores al inicio del producto en investigación, durante el tratamiento del estudio (incluidas las interrupciones de su administración) y durante los 30 días posteriores a la interrupción de dicho tratamiento.
    12 Los pacientes varones deben:
    Mantener una abstinencia real (que deberá revisarse mensualmente) o aceptar usar un preservativo durante las relaciones sexuales con mujeres embarazadas o en edad fértil mientras participan en el estudio, durante las interrupciones de la administración y al menos durante los 30 días siguientes a la interrupción del producto en investigación o durante un plazo mayor si es necesario para cada compuesto o si así lo exige la normativa local, incluso si se han sometido a una vasectomía con resultado satisfactorio.
    E.4Principal exclusion criteria
    1. Any of the following laboratory abnormalities:
    a. Platelets < 50 x 10^9/L
    b. Absolute neutrophil count (ANC) < 1.0 x 10^9/L
    c. White blood count (WBC) > 100 x 10^9/L
    d. Myeloblasts ≥ 5 % in peripheral blood
    e. Estimated glomerular filtration rate < 30 mL/min/1.73 m2 (as per the Modification of Diet in Renal Disease [MDRD] formula)
    f. Serum amylase or lipase > 1.5 x upper limit of normal (ULN)
    g. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 3 x upper limit of normal (ULN)
    h. Total bilirubin > 1.5 x ULN, subject’s total bilirubin between 1.5 – 3.0 x ULN are eligible if the direct bilirubin fraction is < 25% of the total bilirubin
    2. Subject is pregnant or lactating female
    3. Subject with previous splenectomy
    4. Subject with previous or planned hematopoietic cell transplant
    5. Subject with prior history of Wernicke encephalopathy (WE)
    6. Subject with signs or symptoms of WE (eg, severe ataxia, ocular paralysis or cerebellar signs) without documented exclusion of WE by thiamine level and brain MRI
    7. Subject with thiamine deficiency, defined as thiamine levels in whole blood below normal range according to institutional standard and not demonstrated to be corrected prior to randomization
    8. Subject with concomitant treatment with or use of pharmaceutical, herbal agents or food known to be strong inducers of Cytochrome P450 3A4 (CYP3A4), sensitive CYP3A4 substrates with narrow therapeutic range, sensitive Cytochrome P450 2C19 (CYP2C19)
    substrates with narrow therapeutic range, or sensitive Cytochrome P450 2D6 (CYP2D6) substrates with narrow therapeutic range
    9. Subject on any chemotherapy, immunomodulatory drug therapy (eg, thalidomide, interferon-alpha), anagrelide, immunosuppressive therapy, systemic corticosteroids > 10 mg/day prednisone or equivalent. Subjects who have had prior exposure to hydroxyurea (eg, Hydrea) in the past may be enrolled into the study as long as it has not been administered within 14 days prior to randomization
    10. Subject has received ruxolitinib within 14 days prior to randomization
    11. Subject with previous exposure to Janus kinase (JAK) inhibitor(s) other than ruxolitinib treatment
    12. Subject on treatment with aspirin with doses > 150 mg daily
    13. Subject with major surgery within 28 days prior to randomization
    14. Subject with diagnosis of chronic liver disease (eg, chronic alcoholic liver disease, autoimmune hepatitis, sclerosing cholangitis, primary biliary cirrhosis, hemochromatosis, non-alcoholic steatohepatitis)
    15. Subject with prior malignancy other than the disease under study unless the subject has not required treatment for the malignancy for at least 3 years prior to randomization. However, subject with the following history/concurrent conditions provided successfully treated may enroll: non-invasive skin cancer, in situ cervical cancer, carcinoma in situ of the breast, incidental histologic finding of prostate cancer (T1a or T1b using the tumor, nodes, metastasis [TNM] clinical staging system), or is free of disease and on hormonal treatment only
    16. Subject with uncontrolled congestive heart failure (New York Heart Association Classification 3 or 4)
    17. Subject with known human immunodeficiency virus (HIV), known active infectious Hepatitis B (HepB), and/or known active infectious Hepatitis C (HepC)
    18. Subject with serious active infection
    19. Subject with presence of any significant gastric or other disorder that would inhibit absorption of oral medication
    20. Subject is unable to swallow capsule
    21. Subject with any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study
    22. Subject has any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study
    23. Subject has any condition that confounds the ability to interpret data from the study
    24. Subject with participation in any study of an investigational agent (drug, biologic, device) within 30 days prior to randomization
    25. Subject with a life expectancy of less than 6 months
    1. Cualquiera de las siguientes anomalías analíticas:
    a. Plaquetas < 50 × 109/l
    b. Recuento absoluto de neutrófilos (RAN) < 1,0 x 109/l
    c. Recuento de leucocitos (RL) >100 × 109/l
    d. Mieloblastos >= 5 % en sangre periférica
    e. Índice de filtración glomerular estimado < 30 ml/min/1.73 m2 (de acuerdo con la fórmula de la modificación de la dieta en la enfermedad renal [MDRD, Modification of Diet in Renal Disease])
    f. Amilasa o lipasa sérica > 1,5 veces el límite superior de la normalidad (LSN)
    g Aspartato-aminotransferasa (AST) o alanina-aminotransferasa (ALT) > 3 veces el límite superior de la normalidad (LSN)
    h. Bilirrubina total > 1,5 veces el LSN; los sujetos con una bilirrubina total entre 1,5 y 3,0 veces el LSN podrán participar si el porcentaje de bilirrubina directa es < 25 % de la bilirrubina total

    2. Mujeres embarazadas o en período de lactancia
    3. Sujetos que se hayan sometido a una esplenectomía
    4. Sujetos con un trasplante de células madre hematopoyéticas previo o programado
    5. Sujetos con antecedentes de encefalopatía de Wernicke (EW)
    6. Sujetos con signos o síntomas de EW (por ejemplo, ataxia grave, parálisis ocular o signos cerebelosos) sin descarte de EW confirmado mediante el nivel de tiamina y una RMN cerebral
    7. Sujetos con carencia de tiamina, definida como un nivel de tiamina en sangre entera por debajo del intervalo normal de referencia del centro y sin corrección demostrada antes de la aleatorización
    8. Sujetos que reciban tratamiento simultáneo o utilicen fármacos, plantas medicinales o alimentos que sean inductores potentes conocidos del citocromo P450 3A4 (CYP3A4), sustratos sensibles del CYP3A4 con estrecho margen terapéutico, sustratos sensibles del citocromo P450 2C19 (CYP2C19) con estrecho margen terapéutico o sustratos sensibles del citocromo P450 2D6 (CYP2D6) con estrecho margen terapéutico
    9. Sujetos que reciban quimioterapia o tratamiento con inmunomoduladores (por ejemplo, talidomida, interferón α), anagrelida, inmunodepresores, corticoesteroides sistémicos > 10 mg/día de prednisona o un equivalente.
    Los sujetos expuestos a la hidroxiurea (por ejemplo, Hydrea) en el pasado pueden incluirse en el estudio siempre que no se les haya administrado en los 14 días previos a la aleatorización
    10. Sujetos que hayan recibido ruxolitinib en los 14 días previos a la aleatorización
    11. Sujetos con exposición previa a inhibidores de la cinasa de Jano (JAK) aparte del tratamiento con ruxolitinib
    12. Sujetos en tratamiento con aspirina en dosis > 150 mg diarios
    13. Sujetos sometidos a cirugía mayor en los 28 días previos a la aleatorización
    14. Sujetos con diagnóstico de hepatopatía crónica (por ejemplo, hepatopatía alcohólica crónica, hepatitis autoinmune, colangitis esclerosante, cirrosis biliar primaria, hemocromatosis, esteatohepatitis no alcohólica)
    15. Sujetos con neoplasia maligna previa distinta de la enfermedad en estudio, a menos que no hayan necesitado tratamiento para ella durante al menos los 3 años previos a la aleatorización. No obstante, podrán participar los sujetos con las siguientes enfermedades pasadas/simultáneas tratadas satisfactoriamente: cáncer de piel no invasivo, cáncer cervicouterino localizado, carcinoma de mama localizado, hallazgo histológico casual de cáncer de próstata (T1a o T1b en el sistema de estadificación por tumor, ganglios y metástasis [TNM]) o que hayan superado la enfermedad y estén recibiendo solamente tratamiento hormonal
    16. Sujetos con insuficiencia cardiaca congestiva (clase III o IV de la Asociación de Cardiología de Nueva York [New York Heart Association])
    17. Sujetos con infección conocida por el virus de la inmunodeficiencia humana (VIH), hepatitis B infecciosa activa conocida y/o hepatitis C infecciosa activa conocida
    18. Sujetos con infección grave activa
    19. Sujetos con afecciones importantes gástricas o de otro tipo que inhibirían la absorción de medicamentos orales
    20. Sujetos que no sean capaces de tragar cápsulas
    21. Sujetos con alguna enfermedad, anomalía analítica o trastorno psiquiátrico importantes que les impedirían participar en el estudio

    22. Sujetos con alguna afección, incluida la presencia de anomalías analíticas, que, de participar en el estudio, los situaría en una situación de riesgo inaceptable
    23. Sujetos con alguna afección que pueda interferir con la capacidad de interpretar los datos del estudio
    24. Sujetos que participen en cualquier estudio de un producto en investigación (fármaco, sustancia biológica o dispositivo) en los 30 días previos a la aleatorización
    25. Paciente con una expectativa de vida inferior a 6 meses
    E.5 End points
    E.5.1Primary end point(s)
    Spleen volume response rate (RR)
    Tasa de respuesta del volumen esplénico (TR)
    E.5.1.1Timepoint(s) of evaluation of this end point
    From Screening to the end of Cycle 6
    Desde la selección hasta el final del ciclo 6
    E.5.2Secondary end point(s)
    1. Symptom response rate (SRR)
    2. Spleen volume response rate (RR25)
    1. Tasa de respuesta sintomática (TRS)
    2. Tasa de respuesta del volumen esplénico (TR25)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. From C1D1 to the end of Cycle 6
    2. From Screening to the end of Cycle 6
    1. Desde el D1C1 hasta el final del ciclo 6
    2. Desde la selección hasta el final del ciclo 6
    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 Yes
    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. 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 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. description
    Mejor terapia disponible (MTD)
    Best available therapy (BAT)
    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 concerned10
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA75
    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
    Czech Republic
    Korea, Republic of
    Russian Federation
    United Kingdom
    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 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 prespecified in the protocol, whichever is the later date.
    UVUS o la fecha en la que se obtenga el último dato del último sujeto necesario para el análisis principal, secundario y/o exploratorio, lo que ocurra más tarde, tal y como se especifica en el protocolo.
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months0
    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 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 38
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 154
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 170
    F.4.2.2In the whole clinical trial 192
    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)
    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 Decision2019-05-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-05-10
    P. End of Trial
    P.End of Trial StatusOngoing
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