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    Summary
    EudraCT Number:2015-001611-12
    Sponsor's Protocol Code Number:56022473AML2002
    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:2021-06-17
    Trial results View 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 number2015-001611-12
    A.3Full title of the trial
    A Randomized Phase 2/3 Study of DACOGEN¿ (Decitabine) Plus JNJ-56022473 (Anti-CD123) Versus DACOGEN (Decitabine) Alone in Patients with AML who are not Candidates for Intensive Chemotherapy
    Uno studio randomizzato di fase 2/3 su DACOGEN¿ (decitabina) + JNJ-56022473 (anti-CD123) rispetto a DACOGEN (decitabina) in monoterapia in pazienti con leucemia mieloide acuta (AML) non candidati alla chemioterapia intensiva
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Efficacy and Safety Study of Decitabine (DACOGEN) Plus JNJ-56022473 (Anti CD123) Versus Decitabine (DACOGEN) alone in Participants with acute myeloid leukemia (AML) Ineligible for Intensive Chemotherapy.
    Uno studio di efficacia e sicurezza di DACOGEN¿ (decitabina) + JNJ-56022473 (anti-CD123) rispetto a DACOGEN (decitabina) in monoterapia in pazienti con leucemia mieloide acuta (AML) non candidati alla chemioterapia intensiva
    A.3.2Name or abbreviated title of the trial where available
    An Efficacy and Safety Study of Decitabine (DACOGEN) Plus JNJ-56022473 (Anti CD123) Versus Decitabin
    Uno studio di efficacia e sicurezza di DACOGEN¿ (decitabina) + JNJ-56022473 (anti-CD123) rispetto a
    A.4.1Sponsor's protocol code number56022473AML2002
    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 SponsorJANSSEN CILAG INTERNATIONAL NV
    B.1.3.4CountryBelgium
    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 supportJANSSEN CILAG SPA
    B.4.2CountryItaly
    B.4.1Name of organisation providing supportJanssen-Cilag International NV
    B.4.2CountryBelgium
    B.4.1Name of organisation providing supportJanssen Research & Development; LLC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationJanssen-Cilag International NV
    B.5.2Functional name of contact pointClinical Registry Group
    B.5.3 Address:
    B.5.3.1Street AddressArchimedesweg 29
    B.5.3.2Town/ cityLEIDEN
    B.5.3.3Post code2333 CM
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31715242166
    B.5.5Fax number+31715242110
    B.5.6E-mailClinicalTrialsEU@its.jnj.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameTalacotuzumab
    D.3.2Product code [JNJ-56022473]
    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 INNTalacotuzumab
    D.3.9.2Current sponsor codeNJ-56022473
    D.3.9.4EV Substance CodeSUB176319
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 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.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.1Trade name DACOGEN
    D.2.1.1.2Name of the Marketing Authorisation holderJanssen-Cilag International NV
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameDACOGEN
    D.3.2Product code [DACOGEN]
    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 INNDECITABINA
    D.3.9.1CAS number 2353-33-5
    D.3.9.2Current sponsor codeDACOGEN
    D.3.9.3Other descriptive nameDECITABINA
    D.3.9.4EV Substance CodeSUB06932MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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 Yes
    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) 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Acute Myeloid Leukemia (AML)
    leucemia mieloide acuta
    E.1.1.1Medical condition in easily understood language
    Acute Myeloid Leukemia (AML)
    leucemia mieloide acuta
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10000886
    E.1.2Term Acute myeloid leukemia
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part A: The primary objectives of Part A of the study are to assess safety and to confirm the recommended Phase 2 dose (RP2D) of JNJ-56022473 monotherapy.
    Part B: The primary objectives of Part B of the study are to assess complete response (CR) rate and overall survival (OS) in patients with previously untreated AML who are not eligible for intense induction chemotherapy and who are randomly assigned to receive decitabine plus JNJ-56022473 at the RP2D or decitabine alone.
    Gli obiettivi primari della Parte A dello studio sono la valutazione della sicurezza e la conferma della dose di JNJ 56022473 in monoterapia raccomandata nella fase 2 (RP2D).
    Gli obiettivi primari della Parte B dello studio sono la valutazione della percentuale di risposta completa (CR) e la valutazione della sopravvivenza globale (OS) nei pazienti con AML non precedentemente trattata non idonei alla chemioterapia intensiva di induzione e assegnati mediante randomizzazione al trattamento con decitabina + JNJ 56022473 alla dose RP2D o con decitabina in monoterapia.
    E.2.2Secondary objectives of the trial
    The secondary objectives are to assess the event-free survival (EFS), the overall response rate (ORR, defined as the rate of CR + complete response with incomplete blood count recovery [CRi]), the rate of CR plus MRD negative CRi, relapse-free survival (RFS), time to and duration of response, and the safety profile of study treatments; to assess the pharmacokinetics (PK) of decitabine and JNJ-56022473 when used alone or in combination; to assess the immunogenicity of JNJ-56022473 in
    combination with decitabine, to evaluate minimal residual disease (MRD) and to evaluate patient-reported outcomes measures (PRO) using the FACT LEU and EQ-5D-5L instruments.
    Gli obiettivi secondari sono i seguenti: la valutazione della sopravvivenza libera da eventi (EFS), della percentuale di risposta globale (ORR, definita come la percentuale di CR + risposta completa con recupero incompleto delle conte delle cellule ematiche [CRi]), percentuale di CR piu¿ malattia residua minima (MRD) negativa a CRi, della sopravvivenza libera da recidiva (RFS), del tempo alla risposta, della durata della risposta e del profilo di sicurezza dei trattamenti dello studio; la valutazione della farmacocinetica (PK) della decitabina e di JNJ-56022473 utilizzati in monoterapia o in associazione; la valutazione dell¿immunogenicit¿ di JNJ-56022473 in associazione alla decitabina; la valutazione della malattia residua minima (MRD) e la valutazione delle misure degli esiti riferiti dal paziente (PRO) registrate con gli strumenti FACT LEU ed EQ 5D-5L.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    De novo or secondary acute myeloid leukemia (AML) according to WHO 2008 criteria
    For Part A: -Participants With AML: treatment naive or relapsed for whom experimental therapy is appropriate (as assessed by their treating physician)
    For Part B: =75 years of age or 65 up to 75 years of age and have at least one of the following:
    -Congestive heart failure or ejection fraction =50%
    -Creatinine >2 mg/dL, dialysis or prior renal transplant
    -Documented pulmonary disease with lung diffusing capacity for carbon monoxide (DLCO) =65% of expected, or forced expiratory volume in 1 second (FEV1) =65% of expected or dyspnea at rest requiring oxygen
    -ECOG performance status of 2
    -Prior or current malignancy that does not require concurrent treatment
    -Unresolved infection
    -Comorbidity that, in the Investigator's opinion, makes the patient unsuitable for intensive chemotherapy and must be documented and approved by the Sponsor before randomization
    -De novo or secondary AML (post myelodysplastic syndrome [MDS] or myeloproliferative neoplasm [MPN] or after leukemogenic chemotherapy)
    -Previously untreated AML (except: emergency leukapheresis and/or hydroxyurea during the screening phase to control hyperleukocytosis
    but must be discontinued at least one day prior to start of study therapy)
    -Not eligible for an allogeneic hematopoietic stem cell transplantation
    -ECOG Performance Status score of 0, 1 or 2
    -A woman must be either: Not of childbearing potential: postmenopausal
    (more than [>] 45 years of age with amenorrhea for at least 12 months;
    If,
    of childbearing potential must be practicing a highly effective method of birth control
    -A woman of childbearing potential must have a negative serum (betahuman chorionic gonadotropin [betahCG]) or urine pregnancy test at screening
    -A man who is sexually active with a woman of childbearing potential and has not had a vasectomy must agree to use a barrier method of birth
    control eg, either condom with spermicidal foam/gel/film/cream/suppository or partner with occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/suppository for at least 3 months after last study treatment
    AML de novo o secondaria in accordo con i criteri WHO del 2008.
    Per la parte A:
    - Pazienti con AML (nuovi al trattamento o con recidiva) per i quali è appropriata la terapia sperimentale (secondo la valutazione del medico curante).
    Per la parte B:
    Età = 75 anni o
    Eta’ =65 anni fino a 75 anni ed hanno almeno una delle seguenti condizioni:
    - Insufficienza cardiaca congestizia o frazione di eiezione =50%
    -Creatinina >2 mg/dL, dialisi o precedente trapianto renale
    -Malattia polmonare documentata con diffusione alveolo capillare del monossido di carbonio (DLCO) =65% dell’atteso o volume espiratorio forzato in 1 secondo (FEV1) =65% dell’atteso o dispnea a riposo con richiesta di ossigeno.
    -ECOG performance status pari a 2
    -Prior or current malignancy Tumore precedente o concomitante che non richiede trattamento concomitante
    -Infezione non risolta
    -Comorbidità che, nell’opinione dello sperimentatore, rende il paziente non adatto per la chemioterapia intensiva, il che va documentato e approvato dallo Sponsor prima della randomizzazione
    -AML de novo o secondaria, sindrome postmielodisplastica [MDS] o neoplasma mieloproliferativo [MPN] o dopo chemioterapia leuchemogenica),
    -AML non precedentemente trattata (tranne leucaferesi e/o idrossiurea di emergenza durante la fase di screening per il controllo dell’iperleucocitosi ma deve essere interrotto almeno un giorno prima dell’inizio del trattamento dello studio.
    - Soggetti non idonei al trapianto allogenico di cellule staminali ematopoietiche;
    - Performance status ECOG 0, 1 o 2;
    Le donne devono soddisfare una delle seguenti condizioni:
    - Donne non fertili: devono essere in post-menopausa (> 45 anni di età con amenorrea da almeno 12 mesi);
    - Donne potenzialmente fertili che praticano un metodo contraccettivo altamente efficace;
    - Le donne potenzialmente fertili devono presentare un test di gravidanza sul siero (¿ gonadotropina corionica umana [¿ hCG]) o sulle urine negativo allo screening.
    - Gli uomini sessualmente attivi con donne potenzialmente fertili e che non sono stati sottoposti a vasectomia devono acconsentire a usare un metodo contraccettivo a barriera, ad es. un profilattico con schiuma/gel/pellicola/crema/supposta spermicida oppure la partner deve indossare un cappuccio occlusivo (diaframma o cappuccio cervicale) con schiuma/gel/pellicola/crema/supposta spermicida per almeno 3 mesi dopo l’ultimo trattamento dello studio.
    E.4Principal exclusion criteria
    -Acute promyelocytic leukemia with t(15;17), or its molecular equivalent (PMLRARalpha)
    -For Part B only: Known leukemic involvement or clinical symptoms of leukemic involvement of the central nervous system
    -Participants who received prior treatment with a hypomethylating agent
    -ForPart A only: Participants who did not recover from all clinically significant toxicities (excluding alopecia and hematologic toxicities) of any
    previous surgery, radiotherapy, targeted therapy, or chemotherapy to less than or equal to Grade 1
    -A diagnosis of other malignancy that requires concurrent treatment -Any uncontrolled active systemic infection that requires treatment with
    IV antibiotics
    -Active systemic hepatitis infection requiring treatment or other clinically active liver disease
    - Leucemia promielocitica acuta con t(15;17) o l’equivalente molecolare (PML-RARa);
    - Solo per la Parte B: noto coinvolgimento leucemico o sintomi clinici di coinvolgimento leucemico del sistema nervoso centrale;
    - Soggetti che hanno ricevuto un precedente trattamento con un agente ipometilante;
    - Solo per la Parte A: soggetti che non si sono rimessi da tutte le tossicità clinicamente significative (escluse alopecia e tossicità ematologiche) di un precedente intervento chirurgico o ciclo di radioterapia, terapia mirata o chemioterapia raggiungendo un grado pari o inferiore a 1;
    - Diagnosi di un’altra neoplasia maligna che richiede trattamento concomitante
    - Qualsiasi infezione attiva sistemica non controllata che richieda trattamento con antibiotici IV
    -Infezione sistemica epatica attiva che richiede trattamento o altra epatopatia clinicamente attiva
    E.5 End points
    E.5.1Primary end point(s)
    1-Complete Response Rate
    2-Overall Survival
    1 Percentuale di CR
    2 Sopravvivenza globale (OS)
    E.5.1.1Timepoint(s) of evaluation of this end point
    1-Disease assessment will continue until treatment failure or relapse or clinical cutoff (Approximately up to 4 years after first participant enrollment)
    2-Approximately up to 4 years after first participant enrollment
    1-La valutazione malattia continuerà fino fallimento del trattamento o alla ricaduta o al cutoff clinico (Approssimativamente fino a 4 anni dopo l'arruolamento del primo soggetto)
    2-Approssimativamente fino a 4 anni dopo l'arruolamento del primo soggetto
    E.5.2Secondary end point(s)
    1-Overall response rate (ORR)
    2- Event Free Survival (EFS)
    3 - Rate of CR plus MRD negative CRi
    4-Relapse free survival (RFS)
    5- Number of Participants With Adverse Event
    6-Number of Participants With Anti-drug Antibody at any Visit
    7-Functional Assessment of Cancer Therapy Leukemia (FACT-Leu) Score
    8-European Quality of Life- 5 Dimension 5 Level (EQ-5D-5L) Score
    9-Area under the concentration- time curve between time t1 and t2 (AUC
    t1-t2)
    10-Total Systemic Clearance (CL)
    11- Maximum Observed Serum Concentration (Cmax)
    12- Minimum Observed Concentration (Cmin)
    13- Volume of Distribution
    14-Time to Response
    15- Duration of Response
    1-percentuale di risposta globale ORR
    2-sopravvivenza libera da eventi (EFS)
    3-percentuale di CR piu¿ malattia residua minima (MRD) negativa a CRi,
    4-Sopravvivenza libera da recidiva (RFS)
    5-Numero di soggetti con eventi avversi
    6-Numero di soggetti con anticorpi anti-farmaco a ogni visita
    7-Valutazione del punteggio del questionario FACT-LEU
    8-Valutazione del punteggio del questionario EQ-5D-5L
    9-Area sotto la curva concentrazione-tempo tra il tempo t1 e t2 (AUC T1-T2)
    10-clearance sistemica totale (CL)
    11-Massima concentrazione di siero osservata (Cmax)
    12-Minima concentrazione osservata (Cmin)
    13-Volume di distribuzione
    14-Tempo della risposta
    15-Durata della risposta
    E.5.2.1Timepoint(s) of evaluation of this end point
    1: Disease assessment will continue until treatment failure or relapse or clinical cutoff (Approx up to 4 years after first participant enrollment)
    2-6: Approximately up to 4 years after first participant enrollment
    7-8: Baseline to first followup after completion of study treatment, up to
    4 years after first participant enrollment
    9-13: Pre-specified time points during treatment through Cycle 1 (Days 1,2,3,4,8), cyc 2(Day 1), cyc 3,5 (Day 8) for part A and Cycle 1,4 (Days 1,5,8,9,22,23) cyc 2 (Days 1,8) for part B and also up to 30 days after last dose of any study drug in part A and B
    14-15: Disease assessment will continue until treatment failure or relapse or clinical cutoff (Approximately up to 4 years after first participant enrollment)
    1-La valutazione malattia continuer¿ fino fallimento del trattamento o alla ricaduta o al cutoff clinico (Approssimativamente fino a 4 anni dopo l'arruolamento del primo soggetto);
    2-6: Approssimativamente fino a 4 anni dopo l'arruolamento del primo soggetto
    7-8: Baseline al primo follow-up dopo il completamento del trattamento in studio, fino a 4 anni dopo l'arruolamento del primo soggetto
    9-13: predefiniti time points nel ciclo 1 (giorni 1,2,3,4,8), ciclo 2 (giorno 1), cicli 3,5 (giorno 8) per la parte A e nei cicli 1,4 (giorni
    1,5,8,9,22,23), ciclo 2 (giorni 1,8) per la parte B e anche fino a 30 giorni dopo l'ultima dose di ogni farmaco in studio nelle parti A e B;
    14-15: La valutazione malattia continuer¿ fino fallimento
    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 Yes
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity, Biomarker
    Immunogenetica, Biomarcatori
    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) Yes
    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 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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    DACOGEN
    DACOGEN
    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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA40
    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
    Australia
    Belgium
    European Union
    Germany
    Israel
    Italy
    Korea, Republic of
    Poland
    Russian Federation
    Spain
    Sweden
    Taiwan
    Turkey
    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 270 deaths have occurred or at 6 months after the last subject is randomized, whichever occurs later.
    At that time, follow up of subjects and data collection will end. Subjects who are continuing to derive benefit from treatment as assessed by their investigator will continue to receive treatment and be monitored for safety by the sponsor.
    EoS è definita nel momento in cui avvengono 270 decessi 6 mesi dopo la randomizzazione dell'ultimo soggetto, a patto che tutti gli eventi che siano stati raggiunti ( vedi protocollo 9.1.5 ). A quel punto si concluderanno il follow-up dei pazienti e la raccolta dei dati. I pazienti che continuano a trarre beneficio dal trattamento a giudizio dello sperimentatore continueranno a ricevere il trattamento e saranno monitorati sotto il punto di vista della sicurezza da parte dello sponsor .
    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 months3
    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 months9
    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: 6
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 400
    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 state28
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 164
    F.4.2.2In the whole clinical trial 400
    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)
    After the end of study, follow up of subjects and data collection will end. Subjects who are continuing to derive benefit from treatment as assessed by their investigator and confirmed by the study evaluation team will continue to receive treatment and be monitored for safety by the sponsor.
    Dopo la conclusione dello studio termineranno il follow-up dei pazienti e la raccolta dei dati. I pazienti che continuano a trarre beneficio dal trattamento a giudizio dello sperimentatore e per conferma dal team dello studio, continueranno a ricevere il trattamento e saranno monitorati sotto il punto di vista della sicurezza da parte dello sponsor .
    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 Decision2016-09-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-07-19
    P. End of Trial
    P.End of Trial StatusOngoing
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