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    EudraCT Number:2017-000858-18
    Sponsor's Protocol Code Number:281102
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Temporarily Halted
    Date on which this record was first entered in the EudraCT database:2018-02-15
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2017-000858-18
    A.3Full title of the trial
    A phase 3, prospective, randomized, controlled, open-label, multicenter, 2-period crossover study with a single arm continuation evaluating the safety and efficacy of BAX 930 (rADAMTS13) in the prophylactic and on-demand treatment of subjects with severe congenital thrombotic thrombocytopenic purpura (cTTP, Upshaw-Schulman Syndrome [USS], hereditary thrombotic thrombocytopenic purpura [hTTP])
    Studio di crossover in 2 periodi di fase 3, prospettico, randomizzato, controllato, in aperto, multicentrico, con braccio singolo di continuazione volto a valutare la sicurezza e l’efficacia di BAX 930 (rADAMTS13) nel trattamento profilattico e su richiesta di soggetti con porpora trombotica trombocitopenica congenita (cTTP) (sindrome di Upshaw-Schulman) grave
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3, randomized, controlled study of severe congenital thrombotic thrombocytopenic purpura, with Bax 930.
    Studio di fase 3, randomizzato, controllato su soggetti con porpora trombotica trombocitopenica congenita grave, con BAX 930
    A.3.2Name or abbreviated title of the trial where available
    A phase 3, randomized, controlled study of prophylactic and on-demand treatment of cTTP with BAX 930
    Studio di fase 3, randomizzato, controllato su soggetti con porpora trombotica trombocitopenica con
    A.4.1Sponsor's protocol code number281102
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/48/2012
    B. Sponsor Information
    B.Sponsor: 1
    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 supportBaxalta Innovations GmbH
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBaxalta Innovation GmbH
    B.5.2Functional name of contact pointFrank Stout
    B.5.3 Address:
    B.5.3.1Street Address650 East Kenadall Street
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA 02191
    B.5.3.4CountryUnited States
    B.5.4Telephone number0016175888610
    B.5.5Fax number0
    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/3/08/588
    D.3 Description of the IMP
    D.3.1Product nameRecombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs 13
    D.3.2Product code BAX930 or SHP655
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNapadamtase alfa
    D.3.9.2Current sponsor codeSHP655 (BAX930)
    D.3.9.3Other descriptive nameRecombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs
    D.3.9.4EV Substance CodeSUB183727
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1500
    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 Information not present in EudraCT
    D. therapy medical product Information not present in EudraCT
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. 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 Yes
    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
    severe congenital thrombotic thrombocytopenic purpura (cTTP, Upshaw-Schulman Syndrome [USS], hereditary thrombotic
    thrombocytopenic purpura [hTTP])
    Porpora trombotica trombocitopenica congenita (cTTP) (sindrome di Upshaw-Schulman) grave
    E.1.1.1Medical condition in easily understood language
    severe congenital thrombotic thrombocytopenic purpura
    Porpora trombotica trombocitopenica congenita grave
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10043562
    E.1.2Term Thrombocytopenic purpura, thrombotic
    E.1.2System Organ Class 100000004851
    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 incidence of acute TTP episodes in subjects with severe cTTP receiving either standard of care (SoC) or BAX 930 as a prophylactic treatment
    Determinare l’incidenza di episodi TTP acuti in soggetti con cTTP grave che ricevono lo standard di cura (SoC) o BAX 930 come trattamento profilattico
    E.2.2Secondary objectives of the trial
    1. To evaluate the efficacy of BAX 930 in the treatment of acute TTP episodes as measured by the 1) number of acute TTP events responding to treatment and 2) time to resolution in both the prophylactic and the on-demand cohorts
    2. To evaluate the safety and tolerability of BAX 930 in terms of related adverse events (AEs) and serious adverse events (SAEs) in both the prophylactic and the on-demand cohorts
    3. To evaluate the pharmacokinetics (PK) of ADAMTS13 for up to 288 hours post-infusion in each treatment arm (BAX 930 and SoC) in the prophylactic cohort
    4. To evaluate the incidence of isolated TTP manifestations including thrombocytopenia,microangiopathic hemolytic anemia, renal dysfunction, neurologic signs and symptoms, and
    abdominal pain in the prophylactic cohort
    5. To assess the immunogenicity of BAX 930 as measured by the incidence of binding and inhibitory antibodies to ADAMTS13 in both the prophylactic and the on-demand cohorts
    1. Valutare l’efficacia di BAX 930 nel trattamento di episodi TTP acuti, misurata in base al 1) numero di eventi TTP acuti che rispondono al trattamento e 2) tempo alla risoluzione sia nella coorte profilattica che in quella su richiesta
    2. Valutare la sicurezza e la tollerabilità di BAX 930 in termini di eventi avversi (EA) correlati ed eventi avversi gravi (SAE) sia nella coorte profilattica che in quella su richiesta
    3. Valutare la farmacocinetica (PK) di ADAMTS13 per un massimo di 288 ore post-infusione in ciascun braccio di trattamento (BAX 930 e SoC) nella coorte profilattica
    4. Valutare l’incidenza di manifestazioni TTP isolate, comprese trombocitopenia, anemia emolitica microangiopatica, disfunzione renale, segni e sintomi neurologici e dolore addominale nella coorte profilattica
    5. Valutare l’immunogenicità di BAX 930 misurata in base all’incidenza di anticorpi leganti e inibitori diretti contro ADAMTS13 sia nella coorte profilattica che in quella su richiesta
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject or legally authorized representative has provided signed informed consent (≥18 years of age) and/or assent form (signed by legal representative if subject is <18 years of age).
    2. Subject is 0 to 70 years of age, inclusive, at the time of screening. (Subjects <18 years of age will be enrolled only after at least 10 exposures with BAX 930 have been completed in at least 5 subjects over 18 years of age and reviewed by the study medical director).
    3. Subject has a documented diagnosis of severe hereditary ADAMTS13 deficiency, defined as:
    · Confirmed by molecular genetic testing, documented in subject history or at screening, and
    · ADAMTS13 activity <10% as measured by the FRETS-VWF73 assay, documented in subject history or at screening (subjects currently receiving SoC prophylactic therapy may exceed 10% ADAMTS13 activity at screening).
    Note: Subjects currently receiving prophylactic therapy will be screened immediately prior to their usual prophylactic infusion
    4. Subject does not display any severe TTP symptoms signs (platelet count <100,000/µL and elevation of LDH >2×ULN) at screening. Subjects presenting with minor, but stable laboratory abnormalities at the time of screening may be enrolled (prophylactic cohort only).
    5. Subject is currently on a prophylactic dosing regimen or has a documented history of at least 1 TTP event and an ability to tolerate SoC prophylactic dosing (prophylactic cohort only).
    6. Subjects ≥16 years of age must have a Karnofsky score ≥70% and subjects <16 years of age must have a Lansky score ≥80%.
    7. Subject is hepatitis C virus (HCV)-negative as confirmed by antibody or polymerase chain reaction testing OR HCV-positive if their disease is chronic but stable.
    8. If female of childbearing potential, subject presents with a negative blood pregnancy test and agrees to employ adequate birth control measures for the duration of the study and to undergo quarterly pregnancy testing.
    9. Subject is willing and able to comply with the requirements of the protocol.
    1. Il soggetto o il rappresentante legalmente autorizzato deve aver fornito il modulo di consenso informato firmato (≥18 anni di età) e/o modulo di assenso (firmato dal rappresentante legale se il soggetto ha <18 anni di età).
    2. Il soggetto è di età compresa fra 0 e 70 anni al momento dello screening. (I soggetti di età <18 anni saranno arruolati soltanto dopo che almeno 5 soggetti di età superiore a 18 anni avranno completato almeno 10 esposizioni a BAX 930 che dovranno essere valutate dal DMC).
    3. Il soggetto presenta una diagnosi documentata di grave deficit ereditario di ADAMTS13, definito come:
    • confermato mediante analisi genetica molecolare, documentata nell’anamnesi del soggetto o allo screening;
    • attività di ADAMTS13 <10% misurata mediante il saggio FRETS-VWF73, documentata nell’anamnesi del soggetto o allo screening (i soggetti attualmente trattati con terapia profilattica del SoC possono eccedere il 10% dell’attività di ADAMTS13 allo screening).
    Nota: i soggetti che attualmente ricevono la terapia profilattica saranno sottoposti a screening immediatamente prima di ricevere la loro infusione profilattica di routine.
    4. Il soggetto non mostra alcun segno grave di TTP (conta piastrinica <100.000/µl e aumento della LDH >2 volte l’ULN) allo screening (solo nella coorte profilattica).
    5. Il soggetto sta attualmente ricevendo un regime posologico profilattico o presenta un’anamnesi documentata di almeno 1 evento TTP ed è in grado di tollerare la somministrazione profilattica del SoC (solo per la coorte profilattica).
    6. I soggetti di età ≥16 anni devono avere un punteggio Karnofsky ≥70% e i soggetti di età <16 anni devono avere un punteggio Lansky ≥80%.
    7. Il soggetto è negativo al virus dell’epatite C (HCV) con conferma mediante ricerca di anticorpi o analisi della reazione a catena della polimerasi OPPURE è positivo all’HCV se la sua malattia è cronica ma stabile.
    8. Se donna in età fertile, il soggetto deve risultare negativo al test di gravidanza sul sangue e acconsentire ad adottare misure contraccettive adeguate per tutta la durata dello studio e sottoporsi a test di gravidanza trimestrali.
    9. Il soggetto intende ed è in grado di attenersi ai requisiti previsti dal protocollo.
    E.4Principal exclusion criteria
    1. Subject has been diagnosed with any other TTP-like disorder (microangiopathic hemolytic anemia), including acquired TTP.
    2. Subject has known hypersensitivity to hamster proteins.
    3. Subject has experienced an acute TTP episode less than 30 days prior to screening (prophylactic cohort only).
    4. Subject has a medical history or presence of a functional ADAMTS13 inhibitor at screening.
    5. Subject has a medical history of a genetic or acquired immune deficiency that would interfere with the assessment of product immunogenicity, including subjects who are human immunodeficiency virus (HIV)-positive with an absolute cluster of differentiation 4 (CD4) count <200/mm3 or who are
    receiving chronic immunosuppressive drugs.
    6. Subject has been diagnosed with severe cardiovascular disease (New York Heart Association classes 3 to 4).
    7. Subject with end stage renal disease requiring chronic dialysis.
    8. Subject has been diagnosed with hepatic dysfunction, as evidenced by, but not limited to, any of the following:
    a. Serum alanine aminotransferase (ALT) ≥2xULN
    b. International normalized ratio >1.5
    c. Severe hypoalbuminemia <24 g/L
    d. Portal vein hypertension (e.g., presence of otherwise unexplained splenomegaly, history of esophageal varices)
    9. In the opinion of the investigator, the subject has another clinically significant concomitant disease that may pose additional risks for the subject.
    10. Subject has been treated with an immunomodulatory drug, excluding topical treatment (e.g., ointments, nasal sprays), within 30 days prior to enrollment. Use of corticosteroids in conjunction with administration of FFP to prevent allergic manifestations is permitted.
    11. Subject has an acute illness (e.g., influenza, flu-like syndrome, allergic rhinitis/conjunctivitis, bronchial asthma) at the time of screening (prophylaxis cohort only).
    12. Subject is receiving or anticipates receiving another investigational drug and/or interventional drug within 30 days before enrollment.
    13. Subject has a history of drug and/or alcohol abuse within the last 2 years.
    14. Subject has a progressive fatal disease and/or life expectancy of less than 3 months.
    15. Subject is identified by the investigator as being unable or unwilling to cooperate with study procedures.
    16. Subject suffers from a mental condition rendering him/her unable to understand the nature, scope, and possible consequences of the study and/or evidence of an uncooperative attitude.
    17. Subject is a family member or employee of the sponsor or investigator.
    18. If female, subject is pregnant or lactating at the time of enrolment.
    1. Al soggetto è stato diagnosticato qualsiasi altro disturbo simile alla TTP (anemia emolitica microangiopatica), compresa la TTP acquisita.
    2. Il soggetto presenta ipersensibilità nota alle proteine di criceto.
    3. Il soggetto ha manifestato un episodio TTP acuto meno di 30 giorni prima dello screening (solo per la coorte profilattica).
    4. Il soggetto ha un’anamnesi medica o presenta un inibitore funzionale di ADAMTS13 allo screening.
    5. Il soggetto presenta un’anamnesi medica di un deficit immunitario genetico o acquisito che interferirebbe con la valutazione dell’immunogenicità del prodotto, compresi quei soggetti positivi al virus dell’immunodeficienza umana (HIV) con una conta assoluta dei linfociti positivi per il recettore del cluster di differenziazione 4 (CD4) <200/mm3 o che stanno ricevendo un trattamento cronico con farmaci immunosoppressivi.
    6. Al soggetto è stata diagnosticata una grave malattia cardiovascolare (di classe da 3 a 4 secondo la New York Heart Association).
    7. Il soggetto è affetto da malattia renale allo stadio terminale che richiede dialisi cronica.
    8. Al soggetto è stata diagnosticata una disfunzione epatica, confermata, tra l’altro, da uno qualsiasi dei seguenti valori:
    a. Alanina aminotransferasi (ALT) sierica ≥2 volte l’ULN
    b. Rapporto normalizzato internazionale >1,5
    c. Grave ipoalbuminemia <24 g/l
    d. Ipertensione della vena porta (ad es., presenza di splenomegalia non giustificata altrimenti, anamnesi di varici esofagee)
    9. In base all’opinione dello sperimentatore, il soggetto è affetto da un’altra malattia concomitante clinicamente significativa che potrebbe comportare ulteriori rischi per il soggetto.
    10. Il soggetto è stato trattato con farmaci immunomodulatori, ad eccezione dei trattamenti topici (es. unguenti, spray nasali), nei 30 giorni precedenti l’arruolamento. È consentito l’uso di corticosteroidi in combinazione con la somministrazione di FFP per prevenire manifestazioni allergiche.
    11. Il soggetto presenta una malattia acuta (ad es., influenza, sindrome simil-influenzale, rinite/congiuntivite allergica, asma bronchiale) al momento dello screening (solo per la coorte profilattica).
    12. Il soggetto sta ricevendo o prevede di ricevere un altro farmaco sperimentale e/o farmaco interventistico nei 30 giorni precedenti l’arruolamento.
    13. Il soggetto presenta un’anamnesi di abuso di farmaci e/o alcol negli ultimi 2 anni.
    14. Il soggetto è affetto da una malattia progressiva con esito fatale e/o ha un’aspettativa di vita inferiore a 3 mesi.
    15. Il soggetto viene identificato dallo sperimentatore come incapace o riluttante a seguire le procedure dello studio.
    16. Il soggetto è affetto da una condizione mentale che lo rende incapace di comprendere la natura, l’ambito di applicazione e le possibili conseguenze dello studio e/o evidenzia un’attitudine di non collaborazione.
    17. Il soggetto è un familiare o un dipendente dello sponsor o dello sperimentatore.
    18. Se donna, il soggetto è in stato di gravidanza o in allattamento al momento dell’arruolamento.
    E.5 End points
    E.5.1Primary end point(s)
    1. Incidence of acute TTP episodes among subjects receiving either BAX
    930 or SoC prophylactically during the corresponding treatment periods
    1. Incidenza di manifestazioni subacute durante la terapia profilattica con BAX 930 o SoC nel corso dei periodi di trattamento corrispondente
    E.5.1.1Timepoint(s) of evaluation of this end point
    After first 24 subjects completed the 2x2 crossover phase of the study
    (interim analysis) and at the end of the study
    Dopo che i primi 24 soggetti hanno completato la fase di crossover 2x2 dello studio (analisi provvisoria) e alla fine dello studio
    E.5.2Secondary end point(s)
    1. Number and incidence of acute TTP episodes responding to BAX 930,
    defined as not requiring the use of another ADAMTS13 containing agent
    2. Time to resolution of clinical symptomatology, if present, and
    normalization of laboratory parameters (platelet count ≥150,000/μL;
    LDH ≤1.5×ULN) following initiation of treatment in acute TTP episodes
    with BAX 930 or SoC agent
    3. Incidence of thrombocytopenia defined as a drop in platelet count ≥
    25% of baseline or a platelet count <150,000/μL
    4. Incidence of microangiopathic hemolytic anemia defined as an
    elevation of LDH >1.5×ULN
    5. Incidence of neurological symptoms (e.g., confusion, dysphonia,
    dysarthria, focal or general motor symptoms including seizures)
    6. Incidence of renal dysfunction defined as an increase in serum
    creatinine >1.5×baseline
    7. Incidence of abdominal pain
    8. Incidence of supplemental doses prompted by subacute
    9. Incidence of dose modification not prompted by an acute event
    10. Incidence of acute TTP episodes while subjects are on their final
    dose and dosing regimen
    1. Incidence of product-related and unrelated AEs and SAEs during each
    treatment period
    2. Incidence of binding and inhibitory antibodies to ADAMTS13
    3. Clinically relevant changes in vital signs, clinical chemistry, and
    4. Estimated total quantity of ADAMTS13 administered during the
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    treatment of acute events
    1. Assessment of the PK parameters (incremental recovery [IR], area
    under the plasma curve [AUC], terminal half-life [t1/2], mean residence
    time [MRT], systemic clearance [CL], steady state volume of distribution
    [Vss], and maximum concentration following infusion [Cmax]) for both
    the SoC agent and BAX 930 at the beginning of the respective treatment
    periods with each agent and for BAX 930 at the conclusion of Treatment
    Period 2 in the prophylactic cohort
    2. Assessment of VWF:Ag, VWF:RCo, and VWF multimer pattern at
    baseline and following infusion of the SoC agent and BAX 930 treatment
    during the initial PK assessment
    3. Assessment of ADAMTS13 activity (trough levels) and VWF
    parameters prior to each infusion
    1. Numero e incidenza di episodi TTP acuti che rispondono a BAX 930, definiti come episodi che non richiedono l’uso di un altro agente contenente ADAMTS13
    2. Tempo alla risoluzione della sintomatologia clinica, se presente, e alla normalizzazione dei parametri di laboratorio (conta piastrinica ≥150.000/µl; LDH ≤1,5 volte l’ULN) dopo l’inizio del trattamento di episodi TTP acuti con BAX 930 o un agente del SoC
    3. Incidenza di trombocitopenia definita come un calo nella conta piastrinica ≥25% del basale o una conta piastrinica <150.000/µl
    4. Incidenza di anemia emolitica microangiopatica definita come un aumento della LDH >1,5 volte l’ULN
    5. Incidenza di sintomi neurologici (ad es., confusione, disfonia, disartria, sintomi motori focali o generali comprese le crisi convulsive)
    6. Incidenza di disfunzione renale definita come un aumento nella creatinina sierica >1,5 volte il basale
    7. Incidenza di dolore addominale
    8. Incidenza di dosi supplementari indotte da manifestazioni subacute
    9. Incidenza di modifiche della dose non indotte da un evento acuto
    10. Incidenza di episodi TTP acuti mentre i soggetti ricevono la loro dose finale e posologia
    1. Insorgenza di EA e SAE correlati e non correlati al prodotto durante ciascun periodo di trattamento
    2. Insorgenza di anticorpi leganti e inibitori contro ADAMTS13
    3. Variazioni clinicamente significative nei segni vitali, nella chimica clinica e nell’ematologia
    4. Quantità totale stimata di ADAMTS13 somministrato durante il trattamento di eventi acuti
    1. Valutazione di parametri PK (recupero incrementale [IR], area sotto la curva [AUC] della concentrazione plasmatica, emivita terminale [t1/2], tempo medio di permanenza [MRT], clearance sistemica [CL], volume di distribuzione allo stato stazionario [Vss] e concentrazione massima [Cmax] dopo l’infusione) sia per l’agente del SoC che per BAX 930 all’inizio dei rispettivi periodi di trattamento con ciascun agente e per BAX 930 al termine del Periodo di trattamento 2 nella coorte profilattica
    2. Valutazione di VWF:Ag, VWF:RCo e modello multimerico di VWF al basale e dopo infusione dell’agente del SoC e il trattamento con BAX 930 durante la valutazione PK iniziale
    3. Valutazione dell’attività di ADAMTS13 (livelli di ADAMTS13 pre-infusione) e dei parametri di VWF prima di ciascuna infusione di SoC o BAX 930 e al momento dell’insorgenza di un evento acuto
    E.5.2.1Timepoint(s) of evaluation of this end point
    Eifficacy analysis as above; final analysis at study end when all efficacy,
    safety and PK data are available
    Analisi dell'efficacia come sopra; analisi finale all'estremità di studio quando tutta l'efficacia,
    dati di sicurezza e PK sono disponibili
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    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) Yes
    E.8.2.2Placebo No
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    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
    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
    last subject last visit
    Ultima visita dell'ultimo paziente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 years3
    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 Yes
    F.1.1Number of subjects for this age range: 1
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F. of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) Yes
    F. of subjects for this age range: 1
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F. of subjects for this age range: 2
    F.1.1.5Children (2-11years) Yes
    F. of subjects for this age range: 3
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 50
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 Yes
    F.3.3.2Women of child-bearing potential using contraception No
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 36
    F.4.2.2In the whole clinical trial 60
    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)
    Standard treatment
    Trattamento standard
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-10-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-07-02
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
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