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    Summary
    EudraCT Number:2020-003809-60
    Sponsor's Protocol Code Number:XPORT-MF-035
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Temporarily Halted
    Date on which this record was first entered in the EudraCT database:2022-02-18
    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 number2020-003809-60
    A.3Full title of the trial
    A phase 2, randomized, open-label, multicenter study to evaluate safety and efficacy of single agent selinexor versus treatment of physician’s choice in patients with previously treated myelofibrosis
    Estudio en fase II, aleatorizado, abierto y multicéntrico para evaluar la seguridad y la eficacia de selinexor en monoterapia frente al tratamiento de elección del médico en pacientes con mielofibrosis tratados previamente
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate Safety and Efficacy of Selinexor Versus Treatment of Physician's Choice in Participants With Previously Treated Myelofibrosis
    Un estudio para evaluar la seguridad y la eficacia de selinexor frente al tratamiento de elección del médico en participantes que han sido tratados de mielofibrosis previamente
    A.4.1Sponsor's protocol code numberXPORT-MF-035
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04562870
    A.5.4Other Identifiers
    Name:IND NumberNumber:151631
    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 SponsorKaryopharm Therapeutics Inc.
    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 supportKaryopharm Therapeutics Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationKaryopharm Therapeutics Inc.
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street Address85 Wells Avenue
    B.5.3.2Town/ cityNewton
    B.5.3.3Post codeMA 02459
    B.5.3.4CountryUnited States
    B.5.6E-mailclinicaltrials@karyopharm.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 No
    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 nameSelinexor
    D.3.2Product code KPT-330
    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 INNSelinexor
    D.3.9.1CAS number 1393477-72-9
    D.3.9.2Current sponsor codeKPT-330
    D.3.9.3Other descriptive nameSELINEXOR
    D.3.9.4EV Substance CodeSUB177942
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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
    Myelofibrosis
    Mielofibrosis
    E.1.1.1Medical condition in easily understood language
    Myelofibrosis
    Mielofibrosis
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    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.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10074689
    E.1.2Term Post polycythemia vera myelofibrosis
    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 21.0
    E.1.2Level LLT
    E.1.2Classification code 10074690
    E.1.2Term Post essential thrombocythemia myelofibrosis
    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 21.1
    E.1.2Level LLT
    E.1.2Classification code 10074691
    E.1.2Term Post polycythaemia vera myelofibrosis
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10077161
    E.1.2Term Primary 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 determine the clinical activity of selinexor monotherapy compared with physician’s choice (PC) in patients with previously treated myelofibrosis (MF).
    Determinar la actividad clínica de selinexor en monoterapia en comparación con la EM en pacientes con MF tratada previamente.
    E.2.2Secondary objectives of the trial
    Key Secondary
    • To evaluate additional measures of clinical activity of selinexor compared to physician’s choice (PC) in patients with myelofibrosis (MF).

    Other Secondary
    • To assess the survival outcome of patients with myelofibrosis (MF) receiving selinexor compared with physician’s choice (PC).
    • To evaluate additional measures of clinical activity of selinexor compared to physician’s choice (PC) in patients with myelofibrosis (MF).
    • To determine the safety profile of selinexor and compare to physician’s choice (PC) in patients with myelofibrosis (MF).
    • To characterize pharmacokinetics (PK) of selinexor in patients with myelofibrosis (MF).

    Exploratory:
    • To evaluate patient reported outcomes in
    patients with MF receiving selinexor compared to PC.
    • To identify predictive biomarkers of
    response to treatment and explore treatment
    mechanism of action in patients with MF.
    • Assess changes in transfusion requirements
    • Assess changes in serum LDH levels
    Secundario clave:
    • Evaluar las medidas adicionales de la actividad clínica de selinexor en comparación con la EM en pacientes con MF.

    Otros secundarios:
    • Evaluar el resultado de supervivencia de los pacientes con MF que reciben selinexor en comparación con los de EM.
    • Evaluar las medidas adicionales de la actividad clínica de selinexor en comparación con la EM en pacientes con MF.
    • Determinar el perfil de seguridad de selinexor y compararlo con el EM en pacientes con MF.
    • Caracterizar la farmacocinética (FC) de selinexor en pacientes con MF.

    Exploratorios:
    • Evaluar los resultados notificados por el paciente en pacientes con MF que reciben selinexor en comparación con el EM.
    • Identificar biomarcadores predictivos de la respuesta al tratamiento y analizar el mecanismo de acción del tratamiento en pacientes con MF.
    • Evaluar los cambios en las necesidades de transfusión
    • Evaluar los cambios en los niveles de LDH en suero
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients are eligible to be included in the study only if they meet all of the following criteria:
    Main Inclusion Criteria
    1. A diagnosis of primary MF or post-ET or post-PV MF according to the 2016 WHO classification of MPN, confirmed by the most recent local pathology report.
    2. Previous treatment with JAK inhibitors for at least 6 months.
    3. Measurable splenomegaly during the screening period as demonstrated by spleen volume of > or = 450 cm3 by MRI or CT scan
    4. Relapsed, refractory or intolerant to JAK inhibitors as defined as meeting one of the criteria below:
    a. <35% spleen volume reduction by MRI or CT-scan (from baseline) or
    b. <50% decrease in spleen size by palpation (from baseline) or an increase of at least 3 cm with the spleen at least 5 cm below the left costal margin or
    c. Spleen volume increase >25% from nadir or a return to within 10% of baseline after any initial response or
    d. Treatment with JAK inhibitor was complicated by development of RBC transfusion requirement (2 units per month for 2 month); or grade 3 thrombocytopenia, anemia, hematoma/hemorrhage; or Grade 2 non- hematologic toxicity while on JAK inhibitors
    5. Patients > or = 18 years of age
    6. ECOG < or = 2
    7. Platelet count > or = 75 × 10^9/L
    8. Absolute neutrophil count (ANC) > or = 1.5 × 10^9/L
    9. Serum direct bilirubin < or =1.5 × ULN; AST and ALT < or = 2.5 × ULN
    10. Calculated creatinine clearance (CrCl) >15 mL/min based on the Cockcroft and Gault formula.
    11. Patients with active hepatitis B virus (HBV) are eligible if antiviral therapy for hepatitis B has been given for >8 weeks and viral load is <100 IU/mL.
    12. Patients with untreated hepatitis C virus (HCV) are eligible if there is a documentation of negative viral load per institutional standard.
    13. Patients with history of human immunodeficiency virus (HIV) are eligible if they have CD4+ T-cell counts > or = 350 cells/microL, negative viral load per institutional standard, and no history of acquired immunodeficiency syndrome (AIDS)-defining opportunistic infections in the last year.
    14. Female patients of childbearing potential must have a negative serum pregnancy test at screening and agree to use highly effective methods of contraception throughout the study and for at least 90 days after the last dose of selinexor, or for the duration as stated on the label (SmPC/USPI) for those on the comparator drug (physician’s choice arm). Childbearing potential excludes: Age >50 years and naturally amenorrhoeic for >1 year, or previous bilateral salpingo-oophorectomy, or hysterectomy.
    15. Male patients who are sexually active must use highly effective methods of contraception throughout the study and for at least 90 days after the last dose of selinexor, or for the duration as stated on the label (SmPC/USPI) for those on the comparator drug (physician’s choice arm). Male patients must agree not to donate sperm during the study treatment period.
    16. Patients must sign written informed consent in accordance with federal, local and institutional guidelines.

    Crossover from physician’s choice (PC) arm to selinexor (S) arm will be allowed for patients who develop progressive disease (>25% increase from nadir) or for those with <35% reduction at the Week 36 MRI assessment.
    Patients should meet all inclusion and exclusion criteria prior to crossover.
    • Patients who crossover due to PD (>25% increase from nadir) confirmed by IRC or due to <35% reduction in spleen size at Week 36 MRI assessment confirmed by IRC, should have a wash-out of 10 days from last dose in Arm PC.
    Informed consent for participation in crossover part should be obtained prior to any key assessments are performed for the crossover part of the study. The consent can be obtained anytime between EoT in Arm PC to CID1 of the crossover part.
    1. Diagnóstico de MF primaria o MF después de trombocitemia esencial o policitemia vera según la clasificación de la OMS de 2016 de NMP, confirmado por el informe de patología local más reciente.
    2. Tratamiento previo con inhibidores de JAK durante al menos 6 meses.
    3. Esplenomegalia mensurable durante el periodo de selección, demostrada por un volumen del bazo > or = 450 cm3 mediante RM o TAC.
    4. Recidivante, refractaria o intolerante a los inhibidores de JAK según se define como el cumplimiento de uno de los criterios siguientes:
    a. <35 % de reducción del volumen del bazo mediante RM o TAC (con respecto al inicio) o
    b. <50 % de disminución en el tamaño del bazo mediante palpación (con respecto al inicio) o un aumento de al menos 3 cm con el bazo, al menos 5 cm por debajo del reborde costal izquierdo o
    c. aumento del volumen del bazo >25 % desde el nadir o un retroceso al 10 % con respecto al inicio) después de cualquier respuesta inicial o
    d. el tratamiento con inhibidor de JAK se complicó por el desarrollo de necesidad de transfusión de eritrocitos (2 unidades por mes durante 2 meses); o trombocitopenia de
    grado 3, anemia, hematoma/hemorragia; o toxicidad no hematológica de grado 2 durante el tratamiento con inhibidores de JAK.
    5. Pacientes > o = 18 años de edad
    6. ECOG < o = 2
    7. Recuento de plaquetas > o = 75 × 10 9 /l
    8. Recuento de neutrófilos absoluto > o = 1,5 × 10 9 /l
    9. Bilirrubina directa en serum < o = 1,5 × LSN; AST y ALT < o =2,5 × LSN
    10. Aclaramiento de creatinina (ACr) calculado >15 ml/min según la fórmula de Cockcroft y Gault.
    11. Los pacientes con virus de la hepatitis B (VHB) activo son aptos si se ha administrado tratamiento antivírico para la hepatitis B durante >8 semanas y la carga viral es <100 UI/ml.
    12. Los pacientes con el virus de la hepatitis C (VHC) no tratado son aptos si existe documentación de concentración vírica negativa según la norma institucional.
    13. Los pacientes con antecedentes de virus de inmunodeficiencia humana (VIH) son aptos si tienen recuentos de linfocitos T CD4+ > o = 350 células/microl, concentración vírica negativa según la norma institucional y no tienen antecedentes de infecciones oportunistas por síndrome de inmunodeficiencia adquirida (SIDA) en el último año.
    14. Las mujeres que participen y que tengan capacidad de concebir deben tener una prueba de embarazo en suero negativa en el momento de la selección y aceptar utilizar métodos anticonceptivos muy eficaces durante todo el estudio y, por lo menos, hasta 90 días después de la última dosis de Selinexor, o la duración indicada en la ficha técnica del medicamento comparador (grupo EM – Physician Choice (PC)). La capacidad de concebir excluye: Edad >50 años y amenorrea natural durante >1 año, salpingoooforectomía bilateral previa o histerectomía.
    15. Los hombres sexualmente activos deben utilizar métodos anticonceptivos muy eficaces durante todo el estudio y, por lo menos, hasta 90 días después de la última dosis de Selinexor, o la duración indicada en la ficha técnica del medicamento comparador (grupo EM – Physician Choice (PC)). Los pacientes deben aceptar no donar esperma durante el periodo de tratamiento del estudio.
    16. Los pacientes deben firmar un consentimiento informado por escrito de acuerdo con las directrices federales, locales e institucionales.

    Se permitirá el paso del brazo de elección del médico (PC) al brazo de selinexor (S) para los pacientes que desarrollen una enfermedad progresiva (>25% de aumento desde el nadir) o para aquellos con una reducción de <35% en la evaluación de la RM de la semana 36.
    Los pacientes deben cumplir todos los criterios de inclusión y exclusión antes del cruce.
    - Los pacientes que se cambien de grupo debido a la EP (>25% de aumento desde el nadir) confirmada por el IRC o debido a una reducción de <35% en el tamaño del bazo en la evaluación de la IRM de la semana 36 confirmada por el IRC, deben tener un lavado de 10 días desde la última dosis en el brazo PC.
    El consentimiento informado para la participación en la parte cruzada debe obtenerse antes de que se realice cualquier evaluación clave para la parte cruzada del estudio. El consentimiento puede obtenerse en cualquier momento entre la FdT en el brazo PC y la CID1 de la parte cruzada.
    E.4Principal exclusion criteria
    Patients are excluded from the study if any of the following criteria apply:
    1. >5% blasts in peripheral blood or >10% blasts in bone marrow (i.e., accelerated phase).
    2. Previous treatment with selinexor or other XPO1 inhibitors.
    3. Use of any standard or experimental anti-MF therapy <21 days prior to Cycle 1 Day 1 (hydroxyurea or growth factors are allowed).
    4. Impairment of gastrointestinal (GI) function or GI disease that could significantly alter the absorption of selinexor (Example: vomiting, or diarrhea that is CTCAE grade >1).
    5. Received strong cytochrome P450 3A (CYP3A) inhibitors < or = 7 days prior to selinexor dosing OR strong CYP3A inducers < or = 14 days prior to selinexor dosing.
    6. Major surgery <28 days prior to C1D1.
    7. Uncontrolled (i.e., clinically unstable) infection requiring parenteral antibiotics, antivirals, or antifungals within 7 days prior to first dose of study treatment; however, prophylactic use of these agents is acceptable (including parenteral).
    8. Any life-threatening illness, medical condition, or organ system dysfunction which, in the Investigator’s opinion, could compromise the patient’s safety, prevent the patient from giving informed consent, or being compliant with the study procedures.
    9. Female patients who are pregnant or lactating.
    10. Patients with contraindications to use of selinexor or all the drugs intended to be used in the comparative treatment arm.
    1. >5 % blastos en sangre periférica o >10 % blastos en médula ósea (p.ej. fase acelerada).
    2. Tratamiento previo con selinexor u otros inhibidores de XPO1.
    3. Uso de cualquier tratamiento anti-FM estándar o experimental <21 días antes del día 1 del ciclo 1 (se permiten hidroxiurea o factores de crecimiento).
    4. Deterioro de la función gastrointestinal (GI) o enfermedad GI que pudiera alterar significativamente la absorción de selinexor (ejemplo: vómitos o diarrea de grado >1 según los CTCAE).
    5. Recibieron inhibidores potentes del citocromo P450 3A (CYP3A) < o = 7 días antes de la administración de selinexor O inductores potentes del CYP3A < o =14 días antes de la
    administración de selinexor.
    6. Cirugía mayor <28 días antes del D1C1.
    7. Infección no controlada (es decir, clínicamente inestable) que requiera antibióticos, antivíricos o antifúngicos parenterales en los 7 días previos a la primera dosis del tratamiento del estudio; sin embargo, es aceptable el uso profiláctico de estos fármacos (incluidos los parenterales).
    8. Cualquier enfermedad, afección o disfunción de aparato o sistema potencialmente mortal que, en opinión del investigador, pueda poner en peligro la seguridad del paciente, impedir que el paciente dé su consentimiento informado o cumplir con los procedimientos del estudio.
    9. Mujeres embarazadas o en periodo de lactancia.
    10. Pacientes para los que esté contraindicado el consumo de Selinexor o cualquier otro medicamento que se vaya a utilizar en el brazo de tratamiento comparativo.
    E.5 End points
    E.5.1Primary end point(s)
    Rate of Spleen Volume Reduction of > or = 35% (SVR35) based on the response assessment by the Independent Radiology Committee (IRC)
    Tasa de reducción del volumen del bazo > o = 35 % (RVE35) según la evaluación de la respuesta por parte del Comité de Radiología Independiente (CRI)
    E.5.1.1Timepoint(s) of evaluation of this end point
    From Baseline up to Week 48
    Desde basal a la semana 48
    E.5.2Secondary end point(s)
    Key Secondary
    • Rate of Total Symptom Score reduction of > or = 50% (TSS50) in the myelofibrosis symptom assessment form (MFSAF) V4.0, based on local assessment
    • Rate of Spleen Volume Reduction of > or =25% (SVR25) based on the
    response assessment by the Independent Radiology Committee (IRC)

    Other Secondary
    • Overall survival (OS)
    • Anemia response as defined per the IWG-MRT (International Working Group – Myeloproliferative Neoplasms Research and Treatment) criteria, based on local
    assessment
    • Duration of SVR35 and SVR25, based on IRC assessment
    • Duration of TSS50, based on local assessment
    • ORR (Complete Response [CR] + Partial Response [PR] + Clinical Improvement [CI]) by IWG-MRT criteria, based on local assessment

    The occurrence, nature, and severity of AEs

    Population PK derived parameters:
    • AUC
    • Cmax
    Secundarios clave:

    • Tasa de reducción de la puntuación total de síntomas > o = 50 % (TSS50) en el formulario de evaluación de síntomas de mielofibrosis (MFSAF) V4.0, según la evaluación local
    • Tasa de reducción del volumen del bazo > o = 25 % (RVE25) según la evaluación de la respuesta por parte del CRI

    Otros secundarios:
    • Supervivencia global (SG)
    • Evaluar las medidas adicionales de la actividad clínica de selinexor en comparación con la EM en pacientes con MF.
    • Respuesta de anemia según la definición de los criterios del IWG-MRT, basado en la evaluación local
    • Duración de SVR35 y SVR25, según la evaluación del CRI
    • Duración de la TSS50 según la evaluación local
    • TRO (respuesta completa [RC] + respuesta parcial [RP] + mejoría clínica [MC]) según los criterios del IWG-MRT, basado en la evaluación local
    • La aparición, la naturaleza y la gravedad de los AA

    Parámetros derivados de la FC poblacional:
    • Área bajo la curva (AUC)
    • C máx
    E.5.2.1Timepoint(s) of evaluation of this end point
    Key Secondary Endpoints
    • From Baseline up to end of last cycle (approximately 48 months)
    • From Baseline up to Week 48

    Other Secondary Endpoints
    • From Baseline up to 12 months after end of treatment (approximately 60 months)
    • From Baseline up to 28 Days after last dose (approximately 48 months)
    • From Baseline up to 28 Days after last dose (approximately 48 months)
    • From Baseline up to 28 Days after last dose (approximately 48 months)
    • From Baseline up to 28 Days after last dose (approximately 48 months)

    From first dose of study treatment up to 30 days after end of treatment (approximately 48 months)

    PK parameter: Cycle 2 Day 1: 1, 2, 4, 6, and 24 hours post dose (each cycle is 28 days)
    Criterios de valoración secundarios clave
    - Desde basal hasta el final del último ciclo (unos 48 meses)
    - Desde basal hasta la semana 48

    Otros criterios de valoración secundarios
    - Desde basal hasta 12 meses después del final del tratamiento (aproximadamente 60 meses)
    - Desde basal hasta 28 días después de la última dosis (unos 48 meses)
    - Desde basal hasta 28 días después de la última dosis (unos 48 meses)
    - Desde basal hasta 28 días después de la última dosis (unos 48 meses)
    - Desde basal hasta 28 días después de la última dosis (unos 48 meses)

    Desde la primera dosis del tratamiento del estudio hasta 30 días después del final del tratamiento (unos 48 meses)

    Parámetros de FC: Ciclo 2 Día 1: 1, 2, 4, 6 y 24 horas después de la dosis (cada ciclo es de 28 días)
    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 Yes
    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 Yes
    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 Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over Yes
    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
    a elección del médico según práctica habitual
    physician’s choice as per clinical practice
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA24
    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
    France
    Hungary
    Italy
    Poland
    Spain
    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
    The end of study (Last Patient, Last Visit) (EoS) will occur upon completion of the Survival Follow-up period for the last patient treated who remains on treatment in the study. Completion of follow-up for the last patient will occur when this patient has been followed for up to 1 year after the End of Treatment (EoT) visit (discontinuation of treatment, patient has withdrawn consent, has been withdrawn from the study, has died, or has been lost to follow-up, whichever occurs first).
    El final del estudio (último paciente última visita) será al terminar el periodo de seguimiento de supervivencia del último paciente tratado. La finalización del seguimiento del último paciente se producirá cuando éste haya sido seguido hasta 1 año después de la visita de fin de tratamiento (interrupción del tratamiento, el paciente ha retirado su consentimiento, ha sido retirado del estudio, ha fallecido o se ha perdido el seguimiento, lo que ocurra primero).
    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 months2
    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 months4
    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: 23
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 89
    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 state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 92
    F.4.2.2In the whole clinical trial 112
    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)
    After the end of treatment visit each randomized patient will enter in a Survival Follow-up period. The Survival Follow-up period will last until the last patient will complete it. Completion of follow-up for the last patient will occur when this patient has been followed for up to 1 year after the End of Treatment (EoT) visit (discontinuation of treatment, patient has withdrawn consent, has been withdrawn from the study, has died, or has been lost to follow-up, whichever occurs first).
    Tras la visita de finalización del tratamiento, los pacientes aleatorizados entrarán en un periodo de seguimiento de supervivencia: hasta que el último paciente lo complete. La finalización del seguimiento del último paciente se producirá cuando éste haya sido seguido hasta 1 año después de la visita de Fin de Tratamiento (interrupción del tratamiento, el paciente ha retirado su consentimiento, ha sido retirado del estudio, ha fallecido o se ha perdido el seguimiento, lo que ocurra primero).
    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 Decision2022-06-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-06-14
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
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