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    Summary
    EudraCT Number:2020-004142-11
    Sponsor's Protocol Code Number:A011-11
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-08
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2020-004142-11
    A.3Full title of the trial
    A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Compare the Efficacy and Safety of Sotatercept Versus Placebo When Added to Background Pulmonary Arterial Hypertension (PAH) Therapy for the Treatment of PAH
    Studio di fase 3, randomizzato, in doppio cieco, controllato con placebo volto a confrontare l’efficacia e la sicurezza di sotatercept rispetto al placebo per il trattamento dell’ipertensione arteriosa polmonare (IAP) quando aggiunto alla terapia di base per la IAP
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 Study of Sotatercept for the Treatment of PAH
    Studio di fase 3 su sotatercept per il trattamento della IAP
    A.3.2Name or abbreviated title of the trial where available
    -
    -
    A.4.1Sponsor's protocol code numberA011-11
    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 number0014847675150
    B.5.5Fax number00000000
    B.5.6E-mailshahn@acceleronpharma.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.3.9.1CAS number 1001080-50-7
    D.3.9.2Current sponsor codeACE-011
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 placeboSolution for injection/infusion
    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)
    Ipertensione arteriosa polmonare (IAP)
    E.1.1.1Medical condition in easily understood language
    Cardiovascular Disease
    Malattia cardiovascolare
    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 objectives of this study are to evaluate the efficacy and safety of sotatercept treatment (plus background PAH therapy) versus placebo (plus background PAH therapy) at 24 weeks in adults with PAH.
    Gli obiettivi di questo studio sono valutare l'efficacia e la sicurezza del trattamento con sotatercept (più terapia di base per PAH) rispetto al placebo (più terapia di base per PAH) a 24 settimane in adulti affetti da PAH
    E.2.2Secondary objectives of the trial
    -
    -
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age = 18 years
    2. Documented diagnostic right heart catheterization (RHC) at any time prior to screening confirming the diagnosis of WHO pulmonary arterial hypertension (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 pulmonary hypertension classified as WHO Functional Class II or III
    4. Baseline RHC performed during the Screening Period documenting a minimum pulmonary vascular resistance (PVR) of = 5 WU
    5. At stable doses of background PAH therapy and diuretics (i.e.,patientspecific dose goal for each therapy already achieved) for at least 90 days prior to screening; for infusion prostacyclins, dose adjustment within 10% of optimal dose is allowed per medical practice.
    6. 6MWD = 150 and = 500 m repeated twice at screening (measured at least 4 hours apart, but no longer than 1 week), and both values are within 15% of each other (calculated from the highest value)
    7. Females of childbearing potential must: • Have 2 negative urine or serum pregnancy tests as verified by the investigator prior to starting study drug administration; she must agree to ongoing 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:
    • Agree to use a condom, defined as a male latex condom or non-latex 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 112 days (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. Età = 18 anni
    2. Cateterismo cardiaco destro (CCD) documentato in qualsiasi momento prima dello screening che conferma la diagnosi di ipertensione arteriosa polmonare (PAH) Gruppo 1 secondo l'OMS in uno dei seguenti sottotipi:
    • PAH idiopatica
    • PAH ereditaria
    • PAH indotta da farmaco/tossina
    • PAH associata a malattia del tessuto connettivo
    • PAH associata a shunt semplice, da sistemico a polmonare congenito riparato, almeno 1 anno dalla riparazione
    3. Ipertensione polmonare sintomatica classificata come di classe funzionale II o III dell'OMS
    4. CCD alla baseline eseguito durante il periodo di screening che documenta una resistenza vascolare polmonare minima (PVR) di = 5 WU
    5. A dosi stabili di terapia di base per PAH e diuretici (ovvero,
    obiettivo di dose specifica per il paziente per ogni terapia già raggiunto) per almeno 90 giorni prima dello screening; per le prostacicline per infusione, è consentito un aggiustamento della dose entro il 10% della dose ottimale secondo la prassi medica.
    6. Test della distanza percorsa in 6 minuti (6MWD) = 150 e = 500 m ripetuti due volte allo screening (misurati ad almeno 4 ore ma non più di 1 settimana di distanza l'uno dall'altro), ed entrambi i valori sono entro il 15% l'uno dall'altro (calcolati a partire dal valore più alto)
    7. Le donne in età fertile devono:
    -• Avere 2 test di gravidanza sulle urine o sul siero negativi, verificati dallo sperimentatore prima di iniziare la somministrazione del farmaco in studio; devono acconsentire ad eseguire test di gravidanza durante il corso dello studio e fino a 8 settimane dopo l'ultima dose di farmaco in studio
    • Se sessualmente attive, hanno utilizzato e accettano di utilizzare metodi di contraccezione altamente efficaci senza interruzioni, per almeno 28 giorni prima dell'inizio della somministrazione del prodotto sperimentale, durante lo studio (comprese le interruzioni della dose) e per 16 settimane (112 giorni) dopo l'interruzione del trattamento in studio
    -• Astenersi dall'allattare un bambino o dal donare sangue e ovuli per la durata dello studio e per almeno 16 settimane (112 giorni) dopo l'ultima dose di trattamento in studio
    8. I partecipanti di sesso maschile devono:
    • Accettare di usare un preservativo, definito come profilattico maschile in lattice o altro profilattico non in lattice che NON sia costituito da membrana naturale (animale) (ad es., in poliuretano) durante il contatto sessuale con una donna in gravidanza o in età fertile durante la partecipazione allo studio, durante le interruzioni del dosaggio e per almeno 16 settimane (112) giorni dopo l'interruzione del trattamento con il prodotto sperimentale, anche in caso di vasectomia eseguita con successo
    -• Astenersi dal donare sangue o sperma per tutta la durata dello studio e per 112 giorni (112 giorni) dopo l'ultima dose di trattamento in studio
    9. Capacità di aderire al programma di visite dello studio e di comprendere e rispettare tutti i requisiti del protocollo
    10. Capacità di comprendere e fornire il consenso informato scritto
    E.4Principal exclusion criteria
    1. Diagnosis of pulmonary hypertension 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. Exclusions in PAH Group I should also include schistosomiasis APAH and pulmonary veno-occlusive disease
    3. Hemoglobin (Hgb) at screening above gender-specific upper limit of normal, per local laboratory test
    4. Uncontrolled systemic hypertension as evidenced by sitting systolic blood pressure (BP) > 160 mmHg or sitting diastolic blood pressure > 100 mmHg during 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 visit:
    • eGFR < 30 mL/min/m2 (as defined by MDRD equation)
    • Serum alanine aminotransferase or aspartate aminotransferase levels
    > 3 × upper limit of normal (ULN) or total bilirubin > 1.5 × ULN
    8. Currently enrolled in or have completed any other investigational product study within 30 days for small-molecule drugs or within 5 halflives for biologics prior to the date of signed informed consent
    9. Prior exposure to sotatercept (ACE-011) or luspatercept (ACE-536) or known allergic reaction to either one
    10. Have full or partial pneumonectomy
    11. Pulmonary function test (PFT) values of forced vital capacity (FVC) <60% predicted at the screening visit or within 6 months prior to the screening visit. If PFT is not available, a chest CT scan showing no more than mild interstitial lung disease (ILD) at the screening visit or 1 years prior to it
    12. 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)
    13. History of more than mild obstructive sleep apnea that is untreated
    14. Known history of portal hypertension or chronic liver disease, including hepatitis B and/or hepatitis C (with evidence of recent infection and/or active virus replication), defined as mild to severe hepatic impairment (Child-Pugh Class A-C).
    15. History of restrictive, constrictive or congestive cardiomyopathy
    16. History of atrial septostomy within 180 days prior to the screening visit
    17. Electrocardiogram (ECG) with Fridericia's corrected QT interval
    (QTcF) > 500 ms during screening visit
    18. Personal or family history of long QT syndrome (LQTS) or sudden cardiac death
    19. Left ventricular ejection fraction (LVEF) < 45% on historical ECHO within 6 months prior to the screening visit or pulmonary capillary wedge pressure (PCWP) > 15 mmHg as determined in the Screening Period RHC
    20. 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. Note: Anginal pain can be ignored as an exclusion criterion if coronary angiography shows no obstructions
    21. Cerebrovascular accident within 3 months prior to the screening visit
    22. Acutely decompensated heart failure within 30 days prior to the screening visit, as per investigator assessment
    23. Significant (= 2+ regurgitation) mitral regurgitation or aortic regurgitation valvular disease.
    1. Diagnosi di ipertensione polmonare Gruppi OMS 2, 3, 4 o 5
    2. Diagnosi dei seguenti sottotipi di PAH del Gruppo 1: PAH associata a infezione da virus dell'immunodeficienza umana (HIV) e PAH associata a ipertensione portale. Le esclusioni nel Gruppo I della PAH dovrebbero includere anche PAH associata a schistosomiasi e malattia veno-occlusiva polmonare
    3. Emoglobina (Hgb) allo screening al di sopra del limite superiore della norma specifico per sesso, secondo il test di laboratorio locale
    4. Ipertensione sistemica incontrollata come evidenziato dalla pressione (BP) sistolica da seduti > 160 mmHg o dalla pressione diastolica da seduti > 100 mmHg durante la visita di screening dopo un periodo di riposo
    5. BP sistolica alla baseline < 90 mmHg rispetto allo screening
    6. Donne in gravidanza o in allattamento
    7. Uno qualsiasi dei seguenti valori clinici di laboratorio alla visita di screening:
    • eGFR < 30 ml/min/m2 (come definito dall'equazione MDRD)
    • Livelli di alanina aminotransferasi (ALT) o aspartato aminotransferasi (AST) nel siero > 3 volte il limite superiore della norma (ULN) o bilirubina diretta > 1,5 x ULN
    8. Attualmente arruolato/a o che ha completato qualsiasi altro studio su prodotto sperimentale entro 30 giorni per i farmaci a piccole molecole o entro 5 emivite per i prodotti biologici prima della data di firma del consenso informato
    9. Precedente esposizione a sotatercept (ACE-011) o luspatercept (ACE-536) o nota reazione allergica ad uno dei due
    10. Aver eseguito una pneumonectomia completa o parziale
    11. Valori del test di funzionalità polmonare (PFT) o capacità vitale forzata (FVC) < 60% del valore previsto alla visita di screening o entro 6 mesi prima della visita di screening. Se il PFT non è disponibile, una TC toracica che non mostri più di una lieve malattia polmonare interstiziale (ILD) alla visita di screening o 1 anno prima di essa
    12. Inizio di un programma di esercizi per la riabilitazione cardiopolmonare nei 90 giorni precedenti la visita di screening o inizio programmato durante lo studio (sono ammessi i partecipanti che sono stabili nella fase di mantenimento di un programma e che continueranno per tutta la durata dello studio)
    13. Anamnesi di apnea ostruttiva del sonno più che lieve, non trattata
    14. Anamnesi nota di ipertensione portale o di malattia epatica cronica, compresa epatite B e/o epatite C (con evidenza di infezione recente e/o di replicazione attiva del virus), definita come compromissione epatica da lieve a grave (Child-Pugh Classe A-C).
    15. Anamnesi di cardiomiopatia restrittiva, costrittiva o congestizia
    16. Anamnesi di septostomia atriale nei 180 giorni precedenti la visita di screening
    17. Elettrocardiogramma (ECG) con intervallo QT corretto con formula di Fridericia (QTcF) > 500 ms durante la visita di screening
    18. Anamnesi personale o familiare di sindrome del QT lungo (LQTS) o morte cardiaca improvvisa
    19. Frazione di eiezione ventricolare sinistra (LVEF) < 45% su ecocardiogramma (ECHO) storico entro 6 mesi prima della visita di screening o pressione capillare polmonare di incuneamento (PCWP) > 15 mmHg come determinato nel CCD del periodo di screening
    20. Qualsiasi anamnesi attuale o precedente di malattia coronarica sintomatica (infarto miocardico pregresso, intervento coronarico percutaneo, intervento chirurgico di bypass dell'arteria coronarica o dolore toracico anginoso cardiaco) negli ultimi 6 mesi prima della visita di screening. Nota: Il dolore anginoso può essere ignorato come criterio di esclusione se l'angiografia coronarica non mostra ostruzioni
    21. Incidente cerebrovascolare entro 3 mesi prima della visita di screening
    22. Insufficienza cardiaca gravemente scompensata nei 30 giorni precedenti la visita di screening, come da valutazione dello Sperimentatore.
    23. Significativo (= 2+ rigurgito) rigurgito mitralico o malattia valvolare da rigurgito aortico.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is the change from baseline in 6MWD
    L’endpoint di efficacia primario è la variazione della 6MWD dalla visita baseline
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 24
    Settimana 24
    E.5.2Secondary end point(s)
    1. Multicomponent improvement endpoint measured by the proportion of participants achieving all of the following:
    • 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. Change from baseline in PVR
    3. Change from baseline in NT-proBNP levels
    4. Proportion of participants who improve in WHO FC
    5. Time to death or the first occurrence of any of the following clinical worsening events (TTCW)
    • Worsening-related listing for lung and/or heart transplant
    • Need to initiate rescue therapy with an approved background PAH therapy or the need to increase the dose of infusion prostacyclin by 10% or more
    • Need for atrial septostomy
    • Hospitalization for worsening of PAH (= 24 hours)
    • Deterioration of PAH, defined by both of the following events occurring
    at any time, even if they began at different times, as compared to their
    baseline values:
    - Worsened WHO FC
    - Decrease in 6MWD by = 15% confirmed by 2 tests at least 4 hours apart, but no more than 1 week
    6. Proportion of participants who maintain or achieve a low risk score using the simplified French Risk score calculator
    7. Change from baseline in EuroQoL - 5 dimensions (EQ-5D-5L) index score
    8. Change from baseline in Pulmonary Arterial Hypertension - Symptoms and Impact (PAH-SYMPACT®)
    1. Endpoint di miglioramento multicomponente misurato dalla percentuale di partecipanti che hanno raggiunto tutti i seguenti risultati:
    -• Miglioramento di NT-proBNP (diminuzione di NT-proBNP = 30%) o mantenimento/raggiungimento del livello di NT-proBNP < 300 ng/L
    -• Miglioramento della Classe funzionale (FC) OMS o mantenimento dell'FC II OMS
    2. Cambiamento rispetto alla baseline nella conta assoluta dei linfociti.
    3. Cambiamento rispetto alla baseline nei livelli di NT-proBNP
    4. Percentuale di partecipanti che migliorano nell’FC OMS
    5. Tempo al decesso o alla prima insorgenza di uno dei seguenti eventi di peggioramento clinico (TTCW)
    • Candidatura per il trapianto di polmoni e/o cuore correlata al peggioramento
    • Necessità di iniziare una terapia di salvataggio con una terapia di base approvata per PAH o necessità di aumentare la dose di prostaciclina per infusione del 10% o più
    • Necessità di una septostomia atriale
    • Ricovero in ospedale per peggioramento della PAH (= 24 ore)
    • Deterioramento della PAH, definito da entrambi i seguenti eventi che si verificano in qualsiasi momento, anche se hanno avuto inizio in tempi diversi, rispetto ai loro valori alla baseline:
    • Peggioramento dell'FC OMS
    • Diminuzione della 6MWD di = 15% confermata da 2 prove a distanza di almeno 4 ore ma non più di 1 settimana l'una dall'altra
    6. Percentuale dei partecipanti che mantengono o raggiungono un punteggio di basso rischio utilizzando il calcolatore di punteggio French Risk semplificato
    7. Cambiamento rispetto alla baseline nel punteggio dell'indice EuroQoL - 5 dimensioni (EQ-5D-5L)
    8. Cambiamento rispetto alla baseline nel questionario Sintomi e impatto - ipertensione arteriosa polmonare (PAH-SYMPACT®)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 24
    Settimana 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 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 EEA83
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Argentina
    Australia
    Austria
    Belgium
    Brazil
    Canada
    Czechia
    France
    Germany
    Ireland
    Israel
    Italy
    Korea, Democratic People's Republic of
    Korea, Republic of
    Mexico
    Netherlands
    New Zealand
    Poland
    Serbia
    Spain
    Sweden
    Switzerland
    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.
    End of Study visits are only required for participants who discontinue the study early or decline transition to a future sotatercept long-term follow-up study. The End of Study is defined as when the last participant completes the last visit
    Si considera che un partecipante abbia completato lo studio se ha completato tutte le fasi dello studio, comprese le visite EOT e/o EOS. Le visite di fine studio sono richieste solo per i partecipanti che interrompono anticipatamente lo studio o che rifiutano la transizione verso un futuro studio di follow-up a lungo termine. La fine dello studio è definita come la data in cui l'ultimo partecipante completa l’ultima visita
    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 months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    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: 142
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 142
    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 state16
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 130
    F.4.2.2In the whole clinical trial 284
    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)
    Patients who completed the study will be able to participate in a LTFU study
    I pazienti che hanno completato lo studio potranno partecipare a uno studio di follow-up a lungo termine (LTFU)
    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 Decision2021-04-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-06-23
    P. End of Trial
    P.End of Trial StatusOngoing
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