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    Summary
    EudraCT Number:2021-001498-21
    Sponsor's Protocol Code Number:A011-14
    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:2021-08-31
    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 number2021-001498-21
    A.3Full title of the trial
    A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sotatercept When Added to Maximum Tolerated Background Therapy in Participants With Pulmonary Arterial Hypertension (PAH) World Health Organization (WHO) Functional Class (FC) III or FC IV at High Risk of Mortality
    Estudio fase 3, aleatorizado, doble ciego y controlado con placebo para evaluar sotatercept añadido al tratamiento de base máximo tolerado en pacientes con hipertensión arterial pulmonar (HAP) de clase funcional (CF) III o IV de la Organización Mundial de la Salud (OMS) con alto riesgo de mortalidad
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate Sotatercept in Participants with PAH WHO FC III or FC IV at High Risk of Mortality
    Estudio para evaluar sotatercept en pacientes con HAP con una CF de la OMS III o IV y riesgo elevado de mortalidad
    A.3.2Name or abbreviated title of the trial where available
    A Phase3 Study of Sotatercept in Participants with PAH WHO FC III or FC IV at High Risk of Mortality
    Estudio fase 3 de sotatercept en pacientes con HAP con CF III o IV (OMS) y riesgo alto de mortalidad
    A.4.1Sponsor's protocol code numberA011-14
    A.5.4Other Identifiers
    Name:INDNumber:136150
    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 SponsorAcceleron Pharma 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 supportAcceleron Pharma Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAcceleron Pharma Inc.
    B.5.2Functional name of contact pointSaraBeth Hahn
    B.5.3 Address:
    B.5.3.1Street Address128 Sidney Street
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA 02139
    B.5.3.4CountryUnited States
    B.5.4Telephone number34900 834 223
    B.5.6E-mailRegistroEspanolDeEstudiosClinicos@druginfo.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/20/2369
    D.3 Description of the IMP
    D.3.1Product nameSotatercept
    D.3.2Product code ACE-011
    D.3.4Pharmaceutical form Lyophilisate for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSOTATERCEPT
    D.3.9.1CAS number 1001080-50-7
    D.3.9.2Current sponsor codeACE-011
    D.3.9.3Other descriptive nameActRIIA-IgG1Fc
    D.3.9.4EV Substance CodeSUB179718
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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 No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboLyophilisate and solvent for solution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Pulmonary Arterial Hypertension (PAH)
    Hipertensión Arterial Pulmonar (HAP)
    E.1.1.1Medical condition in easily understood language
    Cardiovascular Disease
    Enfermedad Cardiovascular
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10064911
    E.1.2Term Pulmonary arterial hypertension
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The objective of this study is to evaluate the effects of sotatercept
    treatment (plus maximum tolerated background PAH therapy) versus
    placebo (plus maximum tolerated background PAH therapy) on time to
    first event of all-cause death, lung transplantation, or PAH worsening related hospitalization of ≥ 24 hours, in participants with WHO FC III
    or FC IV PAH at high risk of mortality.
    El objetivo de este estudio es evaluar los efectos del tratamiento con sotatercept (más el tratamiento de base máximo tolerado para la HAP) en comparación con placebo (más el tratamiento de base máximo tolerado para la HAP) en el tiempo transcurrido hasta el primer acontecimiento de muerte por cualquier causa, trasplante de pulmón u hospitalización relacionada con empeoramiento de la HAP de ≥24 horas, en participantes con HAP de CF III o CF IV de la OMS y riesgo elevado de mortalidad
    E.2.2Secondary objectives of the trial
    Not Applicable
    No Aplicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Eligible participants must meet all of the following inclusion criteria to be enrolled in the study:
    1. Age 18 to 75 years, inclusive
    2. Documented diagnostic right heart catheterization prior to screening confirming the diagnosis of WHO PAH Group 1 in any of the following subtypes:
    • Idiopathic PAH
    • Heritable PAH
    • Drug/toxin-induced PAH
    • PAH associated with CTD
    • PAH associated with simple, congenital systemic-to-pulmonary shunts at least 1 year following repair
    3. Symptomatic PAH classified as WHO FC III or IV
    4. REVEAL Lite 2 risk score of ≥ 10
    5. Right heart catheterization performed during screening (or within 2 weeks prior to screening, if done at the clinical study site) documenting a minimum PVR of ≥ 5 Wood units and a pulmonary capillary wedge pressure (PCWP) or left ventricular end-diastolic pressure (LVEDP) of ≤ 15 mmHg
    6. Clinically stable and on stable doses of maximum tolerated (per
    investigator’s judgment) double or triple background PAH therapies for at least 30 days prior to screening
    7. Females of childbearing potential must:
    • Have 2 negative urine or serum pregnancy tests as verified by the investigator prior to starting study therapy; must agree to ongoing urine or serum pregnancy testing during the course of the study and until 8 weeks after the last dose of the study drug
    • If sexually active, have used, and agree to use highly
    effective contraception without interruption; for at least28 days prior to starting the investigational product, during the study (including dose interruptions), and for 16 weeks (112 days) after discontinuation of study treatment
    • Refrain from breastfeeding a child or donating blood, eggs, or ovum for the duration of the study and for at least 16 weeks (112 days) after the last dose of study treatment
    8. Male participants must:
    • Agree to use a condom, defined as a male latex condom or nonlatex condom NOT made out of natural (animal) membrane (e.g., polyurethane), 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 16 weeks (112 days) following investigational product discontinuation, even if he has undergone a successful vasectomy
    • Refrain from donating blood or sperm for the duration of the study and for 16 weeks (112 days) after the last dose of study treatment
    9. Ability to adhere to study visit schedule and understand and
    comply with all protocol requirements
    10. Ability to understand and provide written informed consent
    Para poder participar en el estudio, los pacientes deberán cumplir todos los criterios de incorporación descritos a continuación:
    1. Entre 18 y 75 años de edad (ambos inclusive).
    2. Cateterismo cardíaco derecho documentado antes de la selección que confirme el diagnóstico de HAP del grupo 1 de la OMS en cualquiera de los siguientes subtipos:
    •HAP idiopática
    • HAP hereditaria
    • HAP inducida por medicamentos o toxinas
    • HAP asociada a enfermedad de tejido conectivo
    • HAP asociada a derivaciones sistémico pulmonares congénitas simples al menos 1 año después de la reparación
    3. HAP sintomática de clase funcional III o IV de la OMS.
    4. Puntuación de riesgo ≥10 según el registro REVEAL Lite 2
    5. Cateterismo cardíaco derecho realizado durante la selección (o en las 2 semanas previas a la selección, si se realizó en el centro del estudio clínico) con documentación de una RVP mínima de ≥5 unidades Wood y una presión de enclavamiento capilar pulmonar (PECP) o una presión telediastólica del ventrículo izquierdo (PTDVI) de ≤15 mm Hg.
    6. Clínicamente estables y con dosis estables de los tratamientos de base máximos tolerados (a criterio del investigador) dobles o triples para la HAP durante al menos 30 días antes de la selección
    7. Las mujeres con capacidad de concebir deben:
    • Tener 2 pruebas de embarazo en orina o suero negativas, verificadas por el investigador antes de iniciar el tratamiento del estudio; y acceder a hacerse pruebas de embarazo en orina o suero durante el estudio y hasta 8 semanas después de la última dosis del fármaco del estudio.
    • En caso de ser sexualmente activas, haber utilizado y comprometerse a utilizar un método anticonceptivo muy eficaz sin interrupción durante al menos 28 días antes de empezar a recibir el producto en investigación, durante el estudio (incluidas las interrupciones de la administración de dosis) y durante 16 semanas (112 días) después de suspender el tratamiento del estudio.
    •Abstenerse de amamantar o de donar sangre u óvulos durante el estudio y durante al menos 16 semanas (112 días) después de la última dosis del tratamiento del estudio.
    8. Los participantes varones tendrán que:
    • Comprometerse a utilizar preservativo, es decir, preservativo masculino de látex o de otro material diferente al látex que NO esté fabricado con membranas naturales (de origen animal), por ejemplo, de poliuretano, cuando mantengan relaciones sexuales con mujeres embarazadas o con capacidad de concebir mientras participen en el estudio, durante las interrupciones del tratamiento y durante al menos 16 semanas (112 días) después de la suspensión del producto en investigación, incluso si se han sometido a una vasectomía con éxito.
    • Abstenerse de donar sangre o semen durante el estudio y durante 16 semanas (112 días) después de la última dosis del tratamiento del estudio.
    9. Ser capaces de cumplir el calendario de visitas del estudio y comprender y cumplir todos los requisitos del protocolo.
    10. Ser capaces de comprender y otorgar su consentimiento informado por escrito.
    E.4Principal exclusion criteria
    1. Diagnosis of PAH WHO Groups 2, 3, 4, or 5
    2. Diagnosis of the following PAH Group 1 subtypes: human immunodeficiency virus-associated PAH and PAH associated
    with portal hypertension
    3. Diagnosis of pulmonary veno-occlusive diseases or pulmonary capillary hemangiomatosis or overt signs of capillary and/or venous involvement
    4. Hemoglobin at screening above gender-specific upper limit of
    normal (ULN), per local laboratory test
    5. Baseline platelet count < 50,000/mm3 (< 50.0 x 109/L) at
    screening
    6. Baseline systolic blood pressure < 85 mmHg at screening
    7. Pregnant or breastfeeding women
    8. Any of the following clinical laboratory values at the Screening
    Visit:
    • Estimated glomerular filtration rate < 30 mL/min/m2 (as
    defined by the Modification of Diet in Renal Disease Study
    equation)
    • Serum alanine aminotransferase or aspartate aminotransferase levels > 3 × ULN or total bilirubin > 2.0 × ULN
    9. Currently enrolled in or have completed any other investigational product study within 30 days for small-molecule drugs or within 5 half-lives for biologics prior to the date of signed informed consent
    10. Prior exposure to or known allergic reaction to sotatercept
    (ACE-011) or luspatercept (ACE-536)
    11. History of pneumonectomy
    12. Pulmonary function test values of forced vital capacity < 60%
    predicted within 1 year prior to the Screening Visit
    13. Untreated obstructive sleep apnea
    14. History of known pericardial constriction
    15. History of restrictive or congestive cardiomyopathy
    16. Electrocardiogram (ECG) with Fridericia’s corrected QT interval (QTcF) > 450 ms (or > 500 ms if right bundle branch block is present) during the Screening Period
    17. Personal or family history of long QT syndrome or sudden cardiac death
    18. Left ventricular ejection fraction < 50% on historical echocardiogram within 1 year prior to the Screening Visit
    19. Any current or prior history of symptomatic coronary disease (prior myocardial infarction, percutaneous coronary
    intervention, coronary artery bypass graft surgery, or cardiac anginal chest pain) in the past 6 months prior to the Screening Visit
    20. Cerebrovascular accident within 3 months prior to the Screening Visit
    21. Significant (≥ 2+ regurgitation) mitral regurgitation or aortic
    regurgitation valvular disease
    1. Diagnóstico de HAP de los grupos 2, 3, 4 o 5 de la OMS
    2. Diagnóstico de los siguientes subtipos del grupo 1 de HAP: HAP asociada al virus de la inmunodeficiencia humana (VIH) y HAP asociada a hipertensión portal.
    3. Diagnóstico de enfermedades venooclusivas pulmonares o hemangiomatosis capilar pulmonar o signos manifiestos de afectación capilar o venosa
    4. Hemoglobina en la selección por encima del límite superior de la normalidad (LSN) específico del sexo, según el análisis del laboratorio local.
    5. Recuento basal de plaquetas <50 000/mm3 (< 50,0 x 109/l) en la selección
    6. Presión arterial sistólica basal <85 mm Hg en la selección
    7. Mujeres embarazadas o en periodo de lactancia
    8. Cualquiera de los siguientes valores analíticos en la visita de selección:
    • Filtración glomerular estimada <30 ml/min/m2 (calculada mediante la fórmula de la modificación de la dieta en enfermedad renal).
    • Niveles séricos de alanina aminotransferasa o aspartato aminotransferasa > 3 veces el LSN o bilirrubina total >2.0 veces el LSN.
    9. Participación actual o finalización de cualquier otro estudio con un producto en investigación en los 30 días (fármacos de molécula pequeña) o el equivalente a 5 semividas (fármacos biológicos) previos a la fecha de la firma del consentimiento informado
    10. Exposición previa o reacción alérgica conocida a sotatercept (ACE 011) o luspatercept (ACE-536).
    11. Antecedentes de neumonectomía
    12. Valores de la prueba de función pulmonar de capacidad vital forzada < 60 % del valor teórico en el año previo a la visita de selección
    13. Apnea obstructiva del sueño no tratada
    14. Antecedentes de constricción pericárdica conocida
    15. Antecedentes de miocardiopatía restrictiva o congestiva
    16. Electrocardiograma con un intervalo QT corregido con la fórmula de Fridericia >450 ms (o >500 ms en presencia de bloqueo de rama derecha) durante el período de selección.
    17. Antecedentes personales o familiares de síndrome del QT largo o muerte súbita de origen cardíaco.
    18. Fracción de eyección del ventrículo izquierdo < 50 % en un ecocardiograma realizado en el año previo a la visita de selección.
    19.Presencia o antecedentes de cualquier enfermedad coronaria sintomática (infarto de miocardio previo, intervención coronaria percutánea, injerto de revascularización coronaria o dolor cardiaco anginoso) en los 6 meses previos a la visita de selección.
    20. Accidente cerebrovascular en los 3 meses anteriores a la visita de selección
    21. Enfermedad valvular por insuficiencia mitral o insuficiencia aórtica significativa (reflujo ≥2+).
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is the time to first event of all-cause
    death, lung transplantation, or PAH worsening-related hospitalization
    of ≥ 24 hours.
    El criterio de valoración principal de la eficacia es el tiempo transcurrido hasta el primer acontecimiento de muerte por cualquier causa, trasplante de pulmón u hospitalización de ≥24 horas relacionada a un empeoramiento de la HAP.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The primary endpoint, time to first event of all-cause death, lung transplantation, or PAH worsening-related hospitalization of ≥ 24 hours, will be analyzed using the log-rank test with randomization factors as strata. The Cui-Hung-Wang (CHW) method will be used to control the overall Type I error rate.47 If there is no increase in the number of events and sample size following the planned interim analysis, then the CHW method will be the same as a conventional analysis of this data. The Chen-DeMets-Lan method will be used as a supportive analysis to the CHW method.48 The point estimate of the hazard ratio with 95% CI will be estimated by a Cox regression model stratified by the randomization factors.
    El criterio de valoración principal,tiempo hasta el primer evento de muerte por cualquier causa,trasplante de pulmón u hospitalización ≥ 24 horas relacionada al empeoramiento de la HAP, serán analizadas mediante la prueba de rango logarítmico con factores de aleatorización como estratos. El método CuiHungWang(CHW)se usará para controlar la tasa general de error Tipo I. Si no hay aumento en el número de eventos y el tamaño de la muestra después del análisis intermedio planificado, entonces el método CHW será el mismo al del análisis convencional de los datos. El método ChenDeMetsLan se utilizará como análisis de apoyo al método CHW. El puntaje estimado de la razón de riesgo con un IC de 95% se calculará mediante un modelo de regresión de Cox estratificado por los factores de aleatorización
    E.5.2Secondary end point(s)
    The secondary endpoints are ranked as follows:
    1. Overall survival
    2. Transplant-free survival
    3. Proportion of participants who experienced a mortality event at
    EOS
    4. Change from baseline in REVEAL Lite 2 risk score at Week 24
    5. Proportion of participants achieving a low or intermediate (≤ 7)
    REVEAL Lite 2 risk score at Week 24
    6. Change from baseline in NT-proBNP levels at Week 24
    7. Change from baseline in mean pulmonary artery pressure (mPAP) at Week 24
    8. Change from baseline in PVR at Week 24
    9. Proportion of participants who improve in WHO FC at the end
    of the DBPC Treatment Period
    10. Change from baseline in 6MWD at Week 24
    11. Change from baseline in cardiac output (CO) at Week 24
    12. Change from baseline in EuroQoL-5 dimensions scale 5 levels
    (EQ-5D-5L) index score at Week 24
    Los criterios de valoración secundarios se clasifican de la siguiente manera:
    1. Supervivencia global
    2. Supervivencia sin trasplante
    3. Proporción de participantes que presenten un acontecimiento de mortalidad al FDE.
    4. Variación de la puntuación total del REVEAL Lite 2 en la semana 24 con respecto al momento basal.
    5. Proporción de participantes que logren una puntuación de riesgo baja o intermedia (≤7) en la escala REVEAL Lite 2 en la semana 24.
    6. Variación de las concentraciones de NT-proBNP entre el momento basal y la semana 24
    7. Variación de la presión arterial pulmonar media (PAPm) entre el momento basal y la semana 24
    8. Variación de la RVP entre el momento basal y la semana 24
    9.Proporción de participantes que mejoran en la clase funcional de la OMS al final del período de tratamiento doble ciego y controlado con placebo
    10. Variación de la DR6M entre el momento basal y la semana 24
    11. Variación del gasto cardiaco (GC) entre el momento basal y la semana 24
    12.Variación de la puntuación del índice EuroQol de 5 dimensiones y 5 niveles (EQ-5D-5L) entre el momento basal y la semana 24
    E.5.2.1Timepoint(s) of evaluation of this end point
    The first-ranked secondary endpoint of overall survival will be measured at the end of the double blind placebo-controlled period (up to approximately 46 months) at the primary analysis period which will occur when either 92 events occur if promising zone is not triggered or when 128 events occur if promising zone is triggered. Transplant-free survival and proportion of participants who experienced a mortality event will be measured at the end of the study (up to approximately 49 months). All other secondary endpoints will be measured at week 24.
    El criterio de valoración secundario clasificado en primer lugar de la supervivencia general se medirá al final del período doble ciego controlado con placebo (hasta aproximadamente 46 meses) en el período de análisis primario que ocurrirá cuando se produzcan 92 eventos si no se activa la zona prometedora o cuando ocurran 128 eventos si se activa la zona prometedora. La supervivencia sin trasplante y la proporción de participantes que experimentaron un evento de mortalidad se medirán al final del estudio (hasta aproximadamente 49 meses). Todos los demás criterios de valoración secundarios se medirán en la semana 24.
    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 No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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
    Australia
    Belgium
    Canada
    France
    Germany
    Israel
    Italy
    Mexico
    Netherlands
    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
    A participant is considered to have completed the study if he/she has completed all phases of the study, including the EOT and/or EOS Visits. Participants who discontinue the study early or decline enrollment into the LTFU Study A011-12 (SOTERIA) will be asked to return to the clinic for the EOS Visit.
    The end of the study is defined as when the last participant completes the last visit.
    Se considera que un paciente ha completado el estudio si el/ella ha completado todas las fases del estudio, incluidas las visitas FDT y / o FDE. A los pacientes que interrumpan el estudio antes de tiempo o rechacen la participación en el Estudio LTFU A011-12 (SOTERIA) se les pedirá que regresen al centro para la Visita FDE.
    El final del estudio se define cuando el último participante completa la última visita.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months1
    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: 83
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 83
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 68
    F.4.2.2In the whole clinical trial 166
    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)
    At the time of study unblinding, after the required number of events are met, the participants will complete an EOT Visit and may continue into the LTFU Study A011-12 (SOTERIA).
    En el momento del desenmascaramiento del estudio, una vez alcanzado el número exigido de acontecimientos, los pacientes acudirán a una visita de FDT y podrán continuar en el estudio de SLP A011-12 (SOTERIA).
    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 Decision2021-11-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-10-06
    P. End of Trial
    P.End of Trial StatusOngoing
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