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    Summary
    EudraCT Number:2019-002921-31
    Sponsor's Protocol Code Number:C3391003
    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:2020-11-06
    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 number2019-002921-31
    A.3Full title of the trial
    A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
    UNO STUDIO DI FASE 3, MULTICENTRICO, RANDOMIZZATO, IN DOPPIO CIECO, CONTROLLATO VERSO PLACEBO, PER VALUTARE LA SICUREZZA E L'EFFICACIA DI PF-06939926 NEL TRATTAMENTO DELLA DISTROFIA MUSCOLARE DI DUCHENNE
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 3 study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne muscular dystrophy.
    UNO STUDIO DI FASE 3 PER VALUTARE LA SICUREZZA E L'EFFICACIA DI PF-06939926 NEL TRATTAMENTO DELLA DISTROFIA MUSCOLARE DI DUCHENNE
    A.3.2Name or abbreviated title of the trial where available
    -
    -
    A.4.1Sponsor's protocol code numberC3391003
    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 SponsorPFIZER 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 supportPfizer Inc., 235 East 42nd Street, New York, NY 10017
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPfizer Inc.
    B.5.2Functional name of contact pointClinical Trials.gov Call Centre
    B.5.3 Address:
    B.5.3.1Street Address235 East 42nd Street
    B.5.3.2Town/ cityNew York
    B.5.3.3Post codeNY10017
    B.5.3.4CountryUnited States
    B.5.4Telephone number+18007181021
    B.5.6E-mailClinicalTrials.gov_Inquiries@pfizer.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/3/16/1716
    D.3 Description of the IMP
    D.3.1Product namePF-06939926
    D.3.2Product code [PF-06939926]
    D.3.4Pharmaceutical form Solution for infusion
    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 INNPF-06939926
    D.3.9.2Current sponsor codePF-06939926
    D.3.9.4EV Substance CodeSUB193152
    D.3.10 Strength
    D.3.10.1Concentration unit ml millilitre(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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 placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    DUCHENNE MUSCULAR DYSTROPHY (DMD)
    DISTROFIA MUSCOLARE DI DUCHENNE (DMD)
    E.1.1.1Medical condition in easily understood language
    DMD is a severe, X-linked, progressive neuromuscular disease affecting approximately one in 3600 to 9300 live male births
    La DMD è una grave malattia neuromuscolare progressiva, legata a X, che colpisce circa uno tra 3600 e 9300 nati maschili vivi
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10013801
    E.1.2Term Duchenne muscular dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To demonstrate superior efficacy of treatment with PF-06939926 as compared to placebo based on change from Baseline in the North Star Ambulatory Assessment (NSAA).
    Dimostrare l’efficacia superiore del trattamento con PF-06939926 confrontato con placebo in base alla variazione rispetto al basale nella scala di valutazione della deambulazione North Star (NSAA).
    E.2.2Secondary objectives of the trial
    - To quantify the mini-dystrophin expression level in the muscle of participants treated with PF-06939926.
    - To characterize the distribution of mini-dystrophin expression in the muscle of participants treated with PF-06939926.
    - To characterize the change in serum creatine kinase (CK) concentration in participants treated with PF-06939926 as compared to placebo.
    - Quantificare il livello di espressione della minidistrofina a livello muscolare nei partecipanti trattati con PF-06939926.
    - Definire la distribuzione dell’espressione della mini-distrofina a livello muscolare nei partecipanti trattati con PF-06939926.
    - Definire la variazione della concentrazione di creatinchinasi sierica (CK) nei partecipanti trattati con PF-06939926 rispetto alla terapia con placebo.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male participants who are =4 and <8 years of age at Screening (Visit 1).
    - Confirmed diagnosis of DMD by prior genetic testing demonstrating the presence of a mutation in the dystrophin gene consistent with DMD at Screening (Visit 1). If the Investigator determines that the results are inconclusive, a repeat genetic testing will be allowed through the central laboratory at Screening (Visit 1).
    - Receipt of a stable daily dose of glucocorticoids (=0.5 mg/kg/day prednisone, prednisolone, or =0.75 mg/kg/day deflazacort) for at least 3 months prior to Screening (Visit 1) and during the period between
    Screening (Visit 1) and Day 1 (Visit 3). In order to comply with protocol procedures, there should also be a reasonable expectation that this daily dose of glucocorticoids will remain stable for the first 2 years of the study. A stable dose is defined as one in which any change is =0.2 mg/kg
    - A NSAA total score >16 and <30 at Screening (Visit 1).
    - Ambulatory, defined as being able to walk 10 meters unassisted, at Screening (Visit 1).
    - Participants/legally acceptable representatives who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations, and other study procedures including,
    potentially, open and/or needle muscle biopsies under general anesthesia.
    - Participants/legally acceptable representatives who are capable of giving assent/signed informed consent as described in Appendix 1, which includes compliance with the requirements and restrictions listed in the assent/informed consent document (ICD) and in this protocol.
    - Participants/legally acceptable representatives who are willing to protect the integrity of the study data by not actively seeking sensitive clinical data (eg, CK, ALT, AST, NAb to AAV9) through independent
    laboratory tests and by not sharing trial experiences with other participants or publicly (eg, through social media).
    I partecipanti possono essere inclusi nello studio solo se rispondono a tutti i seguenti criteri:
    1. Partecipanti di sesso maschile di età =4 e <8 anni allo screening (Visita 1).
    2. Diagnosi confermata di DMD previo test genetico comprovante la presenza di una mutazione nel gene della distrofina compatibile con la DMD allo screening (Visita 1). Se lo sperimentatore stabilirà che l’esito è inconcludente, i test genetici potranno essere nuovamente effettuati tramite il laboratorio centrale allo screening (Visita 1).
    3. Trattamento con una dose stabile giornaliera di glucocorticoidi (=0,5 mg/kg/giorno di prednisone, prednisolone, oppure =0,75 mg/kg/giorno di deflazacort) per almeno i 3 mesi precedenti lo screening (Visita 1) e nel periodo compreso tra lo screening (Visita 1) e il Giorno 1 (Visita 3). Al fine di rispettare le procedure previste dal protocollo, dovrebbe sussistere un’aspettativa ragionevole che tale dose giornaliera di glucocorticoidi rimanga stabile per i primi 2 anni dello studio. Per dose stabile si intende una dose soggetta a modifiche =0,2 mg/kg.
    4. Un punteggio totale NSAA >16 e <30 allo screening (Visita 1).
    5. Capacità di deambulazione, intesa come la capacità di camminare 10 metri senza assistenza, allo screening (Visita 1).
    6. Partecipanti/rappresentanti giuridicamente accettabili che desiderano e sono in grado di rispettare tutte le visite programmate, il piano terapeutico, gli esami di laboratorio, le considerazioni sullo stile di vita e altre procedure relative allo studio incluse, eventualmente, biopsie muscolari a cielo aperto e/o agobiopsie in
    anestesia generale.
    7. Partecipanti/rappresentanti giuridicamente accettabili in grado di fornire il proprio assenso/consenso informato opportunamente firmato, compresa la conformità ai requisiti e alle restrizioni elencate nel modulo dell’assenso/del consenso informato (ICD) e nel presente protocollo.
    8. Partecipanti/rappresentanti giuridicamente accettabili che desiderano proteggere l’integrità dei dati dello studio astenendosi dal cercare attivamente i dati clinici sensibili (ad es. CK, ALT, AST, NAb per AAV9) attraverso esami di laboratorio indipendenti nonché dal condividere esperienze cliniche pubblicamente (ad es. sui social media) o con altri partecipanti.
    E.4Principal exclusion criteria
    - Prior treatment with gene therapy, defined as any therapy introducing exogenous DNA or intended to permanently alter the endogenous DNA. Gene therapy (other than IP) will be prohibited for the duration of the study.
    - Exposure within 6 months prior to Screening (Visit 1) to any treatment designed to increase dystrophin expression (including, but not limited to exon-skipping and nonsense read-through). These treatments will also be prohibited during the period between Screening (Visit 1) and Day 1 (Visit 3) and for the first 2 years of the study.
    - Previous administration with an investigational drug within 30 days (or as determined by the local requirement) or 5 half-lives (whichever is longer) at Screening (Visit 1). These treatments will also be prohibited during the period between Screening (Visit 1) and Day 1 (Visit 3) and for
    the first 2 years of the study.
    - Known cognitive impairment or behavioral issues that would impede the ability to follow instructions, in the judgment of the Investigator, at Screening (Visit 1).
    - Any nonhealed injury at Screening (Visit 1) which, in the opinion of the Investigator, may impact functional testing; additionally, lower limb fractures must have been healed for at least 3 months prior to Screening (Visit 1).
    - Positive test for NAb to AAV9, based on the threshold determined by the Central Laboratory, from a sample taken at Screening (Visit 1).
    - Receipt of a live attenuated vaccination within 90 days prior to Screening (Visit 1). Receipt of a live attenuated vaccination will also be prohibited during the period between Screening (Visit 1) and Day 1
    (Visit 3), for 90 days prior to Year 2 IP administration, and for the first 2 months after each IP administration.
    -Receipt of an influenza vaccination, systemic antiviral and/or interferon therapy within 30 days prior to Screening (Visit 1). Receipt of an influenza vaccination, systemic antiviral and/or interferon therapy will
    also be prohibited during the period between Screening (Visit 1) and Day 1 (Visit 3), for 30 days prior to Year 2 IP administration, and for the first 2 months after each IP administration.
    -Receipt of any systemic immunosuppressant agents other than glucocorticoids within 30 days prior to Screening (Visit 1). Receipt of any systemic immunosuppressant agents other than glucocorticoids will also be prohibited during the period between Screening (Visit 1) and Day 1 (Visit 3) and during the first 2 years of the study, unless administered in response to immunologic reaction.
    -Abnormality in hematology or chemistry profiles at Screening (Visit 1).
    A single repeat for value(s) outside allowable limits is permitted to reassess eligibility:
    a. Absolute neutrophil count <1000 cells/mm3;
    b. Cystatin C >1.0 mg/L;
    c. Positive hepatitis A virus (anti-HAV) immunoglobulin M, hepatitis B surface antigen (HBsAg), and/or hepatitis C antibody (HCVAb);
    d. Markers of hepatic inflammation or overt or occult cirrhosis as evidenced by one or more of the following:
    1. Prothrombin time (PT) > upper limit of normal (ULN); prolonged international normalized ratio (INR) >ULN;
    2. GLDH >2 x ULN;
    3. Total bilirubin >1.5 x ULN(unless the participant has a history of Gilbert disease) and direct bilirubin >0.5 mg/dL;
    4. Gamma-glutamyl transferase (GGT) >1.5 x ULN.
    For full list of exclusion criteria please refer to study protocol.
    1. Precedente trattamento con terapia genica, intesa come qualsiasi terapia che prevede l’introduzione di DNA esogeno o l’alterazione permanente del DNA endogeno. Terapie geniche (diverse da quella a base del prodotto sperimentale) sono proibite per tutta la durata dello studio.
    2. Esposizione nei 6 mesi precedenti lo screening (Visita 1) a qualsiasi trattamento che miri ad aumentare l’espressione della distrofina (inclusi, a titolo esemplificativo, lo skipping dell’esone e la lettura delle mutazioni stop). Tali terapie saranno altresì proibite nel periodo compreso tra lo screening (Visita 1) e il Giorno 1 (Visita 3) e nei primi 2 anni dello studio.
    3. Precedente somministrazione di un farmaco sperimentale nei 30 giorni (o secondo quanto specificato dalle disposizioni locali) o entro 5 emivite (a seconda di quale dei due è maggiore) allo screening (Visita 1). Tali terapie saranno altresì proibite nel periodo compreso tra lo screening (Visita 1) e il Giorno 1 (Visita 3) e nei primi 2 anni dello studio.
    4. Deficit cognitivo o disturbi comportamentali noti che impedirebbero di seguire le istruzioni, a giudizio dello sperimentatore, allo screening (Visita 1).
    5. Eventuali lesioni non curate allo screening (Visita 1) che, secondo lo sperimentatore, potrebbero condizionare i test funzionali; inoltre, le eventuali fratture degli arti inferiori devono essere curate almeno 3 mesi prima dello screening (Visita 1).
    6. Esito positivo al test degli NAb per AAV9, in base alla soglia stabilita dal laboratorio centrale, di un campione prelevato allo screening (Visita 1).
    7. Ricezione di una vaccinazione viva attenuata entro i 90 giorni precedenti lo screening (Visita 1). La ricezione di una vaccinazione viva attenuata è altresì vietata nel periodo compreso tra lo screening (Visita 1) e il Giorno 1 (Visita 3), nei 90 giorni precedenti la somministrazione del prodotto sperimentale durante l’Anno 2, e nei primi 2 mesi dopo la somministrazione del prodotto sperimentale.
    8. Ricezione di una vaccinazione antinfluenzale, di una terapia sistemica antivirale e/o a base di interferone nei 30 giorni precedenti lo screening (Visita 1). La ricezione di una vaccinazione antinfluenzale, di una terapia sistemica antivirale e/o a base di interferone è altresì vietata nel periodo compreso tra lo screening (Visita 1) e il Giorno 1 (Visita 3), nei 30 giorni precedenti la somministrazione del prodotto sperimentale durante l’Anno 2, e nei primi 2 mesi dopo la somministrazione del prodotto sperimentale.
    9. Ricezione di agenti immunosoppressori sistemici diversi dai glucocorticoidi nei 30 giorni precedenti lo screening (Visita 1). La ricezione di agenti immunosoppressori sistemici diversi dai glucocorticoidi è altresì vietata nel periodo compreso tra lo screening (Visita 1) e il Giorno 1 (Visita 3) e durante i primi 2 anni dello studio, a meno che non vengano somministrati in risposta a una reazione immunologica.
    10. Alterazione del profilo ematologico ed ematochimico allo screening (Visita 1). È ammessa una singola ripetizione per valori al di fuori dei limiti consentiti per rivalutare l’idoneità:
    a. Conta dei neutrofili assoluti <1000 cellule/mm3;
    b. Cistatina C >1,0 mg/L;
    c. Positività a immunoglobulina M dell’epatite A (anti-HAV), antigene di superficie dell’epatite B (HBsAg) e/o anticorpo dell’epatite C (HCVAb);
    d. Marcatori di infiammazione epatica o cirrosi conclamata o occulta, come provato da uno o più dei seguenti valori:
    1. Tempo di protrombina (PT) > limite superiore della norma (ULN); rapporto internazionale normalizzato prolungato (INR) >ULN;
    2. GLDH >2 x ULN;
    3. Bilirubina totale >1,5 x ULN (a meno che il partecipante abbia un’anamnesi di sindrome di Gilbert) e bilirubina diretta >0,5 mg/dL;
    4. Gamma-glutamil transferasi (GGT)>1,5 x ULN.
    Per un elenco completo dei criteri di esclusione di prega di fare riferimento al Protocollo di studio.
    E.5 End points
    E.5.1Primary end point(s)
    Change from Baseline at Week 52 in the NSAA total score.
    Variazione dal basale alla Settimana 52 nel punteggio totale della NSAA.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 52
    Settimana 52
    E.5.2Secondary end point(s)
    - Change from Baseline in percent normal mini-dystrophin expression level in biceps brachii muscle biopsies at Day 60 (or Day 360) using an LC-MS assay.
    - Change from Baseline in percent of muscle fibers expressing minidystrophin in biceps brachii muscle biopsies at Day 60 (or Day 360) as assessed by immunofluorescence.
    - Change from Baseline at Week 52 in serum CK concentration.
    - Number of skills gained at Week 52 based on the individual items of the NSAA.
    - Number of skills either improved or maintained at Week 52 based on the individual items of the NSAA.
    -Change from Baseline at Week 52 in the 10 meter run/walk velocity.
    -Change from Baseline at Week 52 in the rise from floor velocity.
    -Change from Baseline at Week 52 in the Modified Pediatric Outcomes Data Collection Instrument (PODCI): Transfer and Basic Mobility Core Scale (Pediatric Parent).
    - Change from Baseline at Week 52 in the Modified PODCI: Sports and Physical Functioning Core Scale (Pediatric Parent).
    - Variazione dal basale nel livello di espressione, in percentuale, della minidistrofina nei campioni del muscolo bicipite branchiale al Giorno 60 (o al Giorno 360) analizzati mediante cromatografia liquida con spettrometro di massa (LCMS).
    - Variazione dal basale in percentuale dell’espressione della mini-distrofina all’interno delle fibre
    muscolari nei campioni di tessuto prelevato dal bicipite branchiale il Giorno 60 (o Giorno 360) analizzati mediante immunofluorescenza.
    - Variazione dal basale alla Settimana 52 nella concentrazione di CK sierica.
    - Numero delle competenze acquisite alla Settimana 52 in base ai singoli elementi della NSAA.
    - Numero delle competenze potenziate o mantenute alla Settimana 52 in base ai singoli elementi della NSAA.
    - Variazione dal basale alla Settimana 52 nella velocità di 10 metri di corsa/camminata.
    - Variazione dal basale alla Settimana 52 nella velocità in cui ci si alza da terra.
    - Variazione dal basale alla Settimana 52 nello strumento di raccolta dati per i risultati pediatrici modificati (PODCI). Scala di valutazione della mobilità di base e delle capacità motorie (pediatragenitore)
    - Variazione dal basale alla Settimana 52 nel PODCI modificato: Scala di valutazione della funzionalità fisica e sportiva (pediatra genitore).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 52
    Settimana 52
    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 No
    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.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 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) 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 EEA16
    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
    Belgium
    Canada
    China
    France
    Germany
    Israel
    Italy
    Japan
    Korea, Republic of
    Russian Federation
    Spain
    Switzerland
    Taiwan
    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 the date of the last visit of the last participant in the study.
    La fine dello studio è definita come la data dell'ultima visita dell'ultimo partecipante allo studio.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months0
    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.1.1.1In 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) Yes
    F.1.1.5.1Number of subjects for this age range: 99
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 23
    F.4.2.2In the whole clinical trial 99
    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)
    No difference from the expected normal treatment of that condition
    Nessuna differenza rispetto al trattamento normale previsto per tale condizione
    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 Decision2020-12-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-09-10
    P. End of Trial
    P.End of Trial StatusOngoing
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