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    EudraCT Number:2021-000199-12
    Sponsor's Protocol Code Number:A011-13
    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:2021-08-23
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2021-000199-12
    A.3Full title of the trial
    A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate Sotatercept When Added to Background Pulmonary Arterial Hypertension
    (PAH) Therapy in Newly Diagnosed Intermediate- and High-risk PAH Patients.
    Estudio de fase III, aleatorizado, doble ciego y controlado con placebo para evaluar sotatercept añadido al tratamiento de base de la hipertensión arterial pulmonar (HAP) en pacientes con HAP de riesgo intermedio y alto recién diagnosticada
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 Study of Sotatercept in Newly Diagnosed Intermediate- and High-risk Pulmonary arterial hypertension (PAH) Patients.
    Estudio de fase III de Sotatercept en pacientes con HAP de riesgo intermedio y alto recién diagnosticada.
    A.3.2Name or abbreviated title of the trial where available
    A Phase 3 Study of Sotatercept in Newly Diagnosed Intermediate- and High-risk PAH Patients
    Estudio de fase III de Sotatercept en pacientes con HAP de riesgo intermedio y alto recién diagnosti
    A.4.1Sponsor's protocol code numberA011-13
    A.5.4Other Identifiers
    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 number001484767-5150
    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. 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboLyophilisate 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 background PAH therapy) versus placebo (plus background PAH therapy) on TTCW in participants who are newly diagnosed with PAH and are at intermediate or high risk of PAH disease progression.
    El objetivo de este estudio es evaluar los efectos del tratamiento con sotatercept (más el tratamiento de base para la HAP) en comparación con un placebo (más el tratamiento de base para la HAP) sobre el tiempo transcurrido hasta el empeoramiento clínico (THEC) en participantes con diagnóstico reciente de HAP y riesgo intermedio o alto de progresión de la enfermedad.
    E.2.2Secondary objectives of the trial
    not applicable
    No aplicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age ≥ 18 years
    2. Documented diagnostic right heart catheterization (RHC) within 6 months of screening documenting a minimum PVR of ≥ 4 Wood units and pulmonary capillary wedge pressure (PCWP) or left ventricular end-diastolic pressure (LVEDP) of ≤ 15 mmHg, with the diagnosis of WHO PAH Group 1 in any of the following subtypes:
    - Idiopathic PAH
    - Heritable PAH
    - Drug/toxin-induced PAH
    - PAH associated with connective tissue disease
    - PAH associated with simple, congenital systemic-to-pulmonary shunts at least 1 year following repair
    3. Symptomatic PAH classified as WHO FC II or III
    4. REVEAL Lite 2 risk score ≥ 6
    5. Diagnosis of PAH within 6 months of screening and on stable doses of a double combination of background PAH therapies for at least 90 days prior to screening. A triple combination of therapies, with stable doses for 90 days, may be allowed per local standard-of-care guidelines, but is restricted to 10% of the study population.
    6. Six-minute walk distance ≥ 150 m repeated twice at screening at least 4 hours apart, but no longer than 1 week apart, and both values are within 15% of each other (calculated from the highest value)
    7. Females of childbearing potential must meet the following criteria:
    -Have 2 negative urine or serum pregnancy tests as verified by the investigator prior to starting study drug administration; she 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 least 28 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 meet the following criteria:
    -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
    1. Edad ≥ 18 años.
    2. Cateterismo cardíaco derecho (CCD) documentado, realizado con fines diagnósticos en los 6 meses previos a la selección, que documente una RVP mínima ≥ 4 unidades Wood y presión de enclavamiento capilar pulmonar (PECP) o presión telediastólica ventricular izquierda (PTDVI) ≤ 15 mm Hg, con el diagnóstico de HAP del grupo 1 de la OMS de cualquiera de los siguientes subtipos:
    • HAP idiopática
    • HAP hereditaria
    • HAP inducida por fármacos o toxinas
    • HAP asociada a enfermedad del tejido conjuntivo
    • HAP asociada a cortocircuitos sistémico-pulmonares simples congénitos al menos un año después de la reparación del cortocircuito.
    3. HAP sintomática de clase funcional II o III de la OMS
    4. Puntuación de riesgo ≥ 6 según el REVEAL Lite 2
    5. Diagnóstico de HAP en los 6 meses previos a la selección y en tratamiento con dosis estables de una combinación doble de tratamientos de base para la HAP durante al menos 90 días antes de la selección. Podrá permitirse una combinación triple de tratamientos, con dosis estables durante 90 días, conforme a las normas asistenciales locales, pero se limita al 10 % de la población del estudio.
    6. Distancia recorrida en seis minutos ≥ 150 m repetida dos veces en la selección con una diferencia mínima de 4 horas y no superior a 1 semana, y ambos valores están dentro del 15 % entre sí (calculado a partir del valor más alto).
    7. Las mujeres con capacidad de concebir deberán cumplir los criterios siguientes:
    • Tener 2 pruebas de embarazo en orina o suero negativas, verificadas por el investigador antes de iniciar la administración del fármaco del estudio; deberá 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) y durante 16 semanas (112 días) después de suspender el tratamiento del estudio.
    • Abstenerse de dar el pecho 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 varones deberán cumplir los criterios siguientes:
    • Comprometerse a utilizar un 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. Capacidad de seguir el calendario de visitas del estudio y de comprender y cumplir todos los requisitos del protocolo.
    10. Capacidad de comprender y otorgar el 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 (HIV)-associated PAH and PAH associated with portal hypertension
    3. Hemoglobin at screening above gender-specific upper limit of normal (ULN), per local laboratory test
    4. Uncontrolled systemic hypertension as evidenced by sitting systolic blood pressure (BP) > 160 mmHg or sitting diastolic BP > 100 mmHg during the Screening Visit after a period of rest
    5. Baseline systolic BP < 90 mmHg at screening
    6. Pregnant or breastfeeding women
    7. Any of the following clinical laboratory values at the Screening
    -Estimated glomerular filtration rate < 30 mL/min/m2 (as defined by MDRD equation)
    -Serum alanine aminotransferase or aspartate aminotransferase levels > 3 × ULN or total bilirubin > 1.5 × ULN -Platelet count < 50,000/mm3 (< 50.0 × 109/L)
    8. 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
    9. Known allergic reaction to sotatercept (ACE-011)
    10. History of pneumonectomy
    11. Pulmonary function test values of forced vital capacity < 60% predicted within 1 year prior to the Screening Visit
    12. Stopped receiving any pulmonary hypertension chronic general supportive therapy (e.g., diuretics, oxygen, anticoagulants, and
    digoxin) within 60 days prior to the Screening Visit
    13. Initiation of an exercise program for cardiopulmonary rehabilitation within 90 days prior to the Screening Visit or
    planned initiation during the study (participants who are stable in the maintenance phase of a program and who will continue for the duration of the study are eligible)
    14. Untreated obstructive sleep apnea
    15. History of known pericardial constriction
    16. History of restrictive or congestive cardiomyopathy
    17. History of atrial septostomy within 180 days prior to the Screening Visit
    18. Electrocardiogram with Fridericia’s corrected QT interval > 450 ms (or > 500 ms if right bundle branch abnormality is present) during the Screening Period
    19. Personal or family history of long QT syndrome or sudden cardiac death
    20. Left ventricular ejection fraction < 50% on historical echocardiogram within 1 year prior to the Screening Visit or pulmonary capillary wedge pressure > 15 mmHg as determined by historical RHC within 6 months prior to the Screening Visit
    21. 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
    22. Cerebrovascular accident within 3 months prior to the Screening Visit
    23. Acutely decompensated heart failure within 30 days prior to the Screening Visit, as per investigator assessment
    24. Significant (≥ 2+ regurgitation) mitral regurgitation or aortic regurgitation valvular disease
    25. Received intravenous inotropes (e.g., dobutamine, dopamine, norepinephrine, and vasopressin) within 30 days prior to the Screening Visit
    1. Diagnóstico de HAP de los grupos 2, 3, 4 o 5 de la OMS
    2. Diagnóstico de los siguientes subtipos de HAP del grupo 1: HAP asociada al virus de la inmunodeficiencia humana (VIH) y HAP asociada a hipertensión portal.
    3. Hemoglobina en la selección por encima del límite superior de la normalidad (LSN) específico del sexo, según el análisis del laboratorio local.
    4. Hipertensión sistémica no controlada, demostrada por una presión arterial (PA) sistólica en sedestación > 160 mm Hg o una PA diastólica en sedestación > 100 mm Hg durante la selección después de un período de reposo.
    5. PA sistólica basal < 90 mm Hg en la selección
    6. Mujeres embarazadas o en período de lactancia
    7. Cualquiera de los siguientes valores analíticos en la visita de selección:
    • Filtración glomerular estimada < 30 ml/min/m2 (definida mediante la ecuación MDRD).
    • Concentración sérica de alanina aminotransferasa o aspartato aminotransferasa > 3 veces el LSN o bilirrubina total > 1,5 veces el LSN.
    • Recuento de plaquetas < 50 000/mm3 (< 50,0 × 109/l).
    8. 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
    9. Reacción alérgica conocida a sotatercept (ACE-011).
    10. Antecedentes de neumonectomía
    11. Valores de la prueba de función pulmonar de capacidad vital forzada < 60 % del valor teórico en el año previo a la visita de selección
    12. Finalización de cualquier tratamiento complementario general crónico para la hipertensión pulmonar (p. ej., diuréticos, oxígeno, anticoagulantes y digoxina) en los 60 días previos a la visita de selección
    13. Inicio de un programa de ejercicio para rehabilitación cardiopulmonar en los 90 días previos a la visita de selección o inicio previsto durante el estudio (podrán incorporarse al estudio participantes que se encuentren estables en la fase de mantenimiento de un programa de este tipo y que lo mantengan durante todo el estudio).
    14. Apnea obstructiva del sueño no tratada
    15. Antecedentes de constricción pericárdica conocida
    16. Antecedentes de miocardiopatía restrictiva o congestiva
    17. Antecedentes de septostomía auricular en los 180 días previos a la visita de selección
    18. Electrocardiograma con un intervalo QT corregido con la fórmula de Fridericia > 450 ms (o > 500 ms en presencia de alteración de rama derecha) durante el período de selección.
    19. Antecedentes personales o familiares de síndrome del QT largo o muerte súbita de origen cardíaco.
    20. Fracción de eyección del ventrículo izquierdo < 50 % en un ecocardiograma histórico en el año previo a la visita de selección o presión de enclavamiento pulmonar > 15 mm Hg determinada en un CCD histórico en los 6 meses previos a la visita de selección.
    21. 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.
    22. Accidente cerebrovascular en los 3 meses anteriores a la visita de selección
    23. Insuficiencia cardíaca descompensada aguda en los 30 días previos a la visita selección, según la evaluación del investigador.
    24. Enfermedad valvular por regurgitación mitral o regurgitación aórtica significativa (reflujo ≥ 2+).
    25. Administración de inótropos intravenosos (p. ej., dobutamina, dopamina, norepinefrina y vasopresina) en los 30 días previos a la visita de selección
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is TTCW, defined as the first confirmed morbidity event or death. The events that will comprise this endpoint
    include the following:
    • All-cause death
    • Non-planned PAH-related hospitalization of ≥ 24 hours in duration
    • Atrial septostomy
    • Lung transplant
    • Deterioration in performance in exercise testing due to PAH, defined as a decrease in 6MWD from baseline on
    2 consecutive tests (which must be at least 4 hours apart) and at least 1 of the following:
    − Worsening of WHO FC from baseline
    − Signs/symptoms of increased right heart failure
    − Addition of a background PAH therapy or change in the composition of PAH background therapy, including an increase in parenteral prostacyclin of ≥ 10%
    All events will be adjudicated by a blinded, independent committee of clinical experts.
    El criterio de valoración principal de la eficacia es el tiempo transcurrido hasta el empeoramiento clínico, definido como el primer episodio de morbilidad confirmado, o la muerte. Los episodios que constituirán este criterio de valoración son los siguientes:
    • Muerte por cualquier causa
    • Hospitalización relacionada con la HAP no programada de ≥ 24 horas de duración
    • Septostomía auricular
    • Trasplante de pulmón
    • Deterioro del rendimiento en la prueba de esfuerzo debido a la HAP, definido como una disminución de la DR6M con respecto al valor basal en 2 pruebas consecutivas (obtenidas con una diferencia mínima de 4 horas) y al menos uno de los siguientes criterios:
    - Empeoramiento de la clase funcional de la OMS con respecto al valor basal
    - Signos/síntomas de aumento de la insuficiencia cardíaca derecha
    - Adición de un tratamiento de base para la HAP o cambio en la composición del tratamiento de base para la HAP, incluido un aumento de la prostaciclina parenteral ≥ 10 %.
    Todos los episodios serán validados por un comité de expertos clínicos independiente y desconocedor de las asignaciones del tratamiento.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The primary endpoint will be analyzed using a weighted stratified log-rank test with randomization stratification factors as strata. The Cui-Hung-Wang (CHW) method will be used to control the overall Type I error rate. The point estimate of the hazard ratio with 95% CI will be estimated by a Cox regression model stratified by the randomization factors. 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.
    El criterio de valoración principal se analizará mediante una prueba del orden logarítmico estratificada y ponderada con los factores de estratificación de la aleatorización como estratos. Se utilizará el método de Cui Hung-Wang (CHW) para controlar la tasa global de error de tipo I. La estimación puntual de la razón de riesgos instantáneos, con su IC del 95 %, se calculará mediante un modelo de regresión de Cox estratificado en función de los factores de aleatorización. Si no se produce un aumento del número de episodios y del tamaño de la muestra después del análisis intermedio previsto, el método de CHW será el mismo que el de un análisis convencional de estos datos.
    Se utilizará el método de Chen-DeMets-Lan como análisis complementario del método de CHW.
    E.5.2Secondary end point(s)
    1. Multicomponent improvement endpoint measured by the proportion of participants achieving all of the following at Week 24 relative to baseline:
    -Improvement in 6MWD (increase ≥ 30 m)
    -Improvement in NT-proBNP (decrease in NT-proBNP ≥ 30%) or maintenance/achievement of NT-proBNP level < 300 ng/L
    -Improvement in WHO FC or maintenance of WHO FC II
    2. Proportion of participants who achieve a low REVEAL Lite 2 risk score at Week 24 versus baseline
    3. Proportion of participants who maintain or achieve a low risk score at Week 24 versus baseline using the simplified French
    Risk score calculator
    4. Change from baseline in NT-proBNP levels at Week 24
    5. Proportion of participants who improve in WHO FC or maintain WHO FC II at 24 weeks from baseline
    6. Change from baseline in 6MWD at Week 24
    7. Change from baseline in EuroQol - 5 dimensions scale 5 levels (EQ-5D-5L) index score at Week 24
    8. Change from baseline in Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT)® at Week 24
    1. Criterio de valoración de mejoría multicomponente medido mediante la proporción de participantes que logren todo lo siguiente en la semana 24 con respecto al valor basal:
    • Mejoría de la DR6M (aumento ≥ 30 m)
    • Mejoría del NT-proBNP (disminución del NT-proBNP ≥ 30 %) o mantenimiento/consecución de una concentración de NT-proBNP < 300 ng/l
    • Mejoría de la clase funcional de la OMS o mantenimiento de la CF II de la OMS
    2. Proporción de participantes que obtienen una puntuación de riesgo bajo en REVEAL Lite 2 en la semana 24 en comparación con el valor basal
    3. Proporción de participantes que mantienen o alcanzan una puntuación de riesgo bajo en la semana 24 en comparación con el valor basal, utilizando la calculadora simplificada de la puntuación de riesgo francesa
    4. Variación de las concentraciones de NT-proBNP entre el momento basal y la semana 24
    5. Proporción de participantes que mejoran en la CF de la OMS o mantienen la CF II de la OMS entre el momento basal y la semana 24
    6. Variación de la DR6M entre el momento basal y la semana 24
    7. Variación de la puntuación del índice EuroQol de 5 dimensiones y 5 niveles (EQ 5D 5L) entre el momento basal y la semana 24
    8. Variación del cuestionario de síntomas y repercusión en la hipertensión arterial pulmonar (PAH-SYMPACT) ® entre el momento basal y la semana 24
    E.5.2.1Timepoint(s) of evaluation of this end point
    week 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. 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 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 concerned8
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA72
    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
    Korea, Republic of
    New Zealand
    South Africa
    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 the study is defined as when the last participant completes the last visit.
    El fin de ensayo está definido cuando el último paciente completa la última visita.
    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 months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years7
    E.8.9.2In all countries concerned by the trial months6
    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: 497
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 165
    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 state22
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 303
    F.4.2.2In the whole clinical trial 662
    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 Decision2021-10-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-10-07
    P. End of Trial
    P.End of Trial StatusOngoing
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