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    Summary
    EudraCT Number:2017-002432-17
    Sponsor's Protocol Code Number:ALN-AS1-003
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-02-02
    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 number2017-002432-17
    A.3Full title of the trial
    ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias
    ENVISION: Studio di fase 3, multicentrico, randomizzato, in doppio cieco, controllato verso placebo, con estensione in aperto volto a valutare l¿efficacia e la sicurezza di givosiran nei pazienti affetti da porfirie epatiche acute
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias
    Studio del farmaco sperimentale, Givosiran, per il trattamento delle porfirie epatiche acute
    A.3.2Name or abbreviated title of the trial where available
    ENVISION
    ENVISION
    A.4.1Sponsor's protocol code numberALN-AS1-003
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN12345678
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03338816
    A.5.3WHO Universal Trial Reference Number (UTRN)U1234-1234-1234
    A.5.4Other Identifiers
    Name:INDNumber:126094
    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 SponsorALNYLAM PHARMACEUTICALS, 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 supportAlnylam Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMedpace
    B.5.2Functional name of contact pointRegulatory Submissions
    B.5.3 Address:
    B.5.3.1Street Address5375 Medpace Way
    B.5.3.2Town/ cityCincinnati, Ohio
    B.5.3.3Post code45227
    B.5.3.4CountryUnited States
    B.5.4Telephone number00498989557180
    B.5.5Fax number004989895571810
    B.5.6E-mailregsubmissions@medpace.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/3/16/1731
    D.3 Description of the IMP
    D.3.1Product nameGivosiran
    D.3.2Product code ALN-AS1
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNALN-60519
    D.3.9.1CAS number 1639325-44-2
    D.3.9.2Current sponsor codeALN-60519
    D.3.10 Strength
    D.3.10.1Concentration unit mg/l milligram(s)/litre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number189
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Acute Hepatic Porphyrias (AHP)
    Porfiria Epatica Acuta
    E.1.1.1Medical condition in easily understood language
    Acute hepatic porphyrias (AHP) represent a sub-group of porphyrias characterized by the occurrence of neuro-visceral attacks with or without cutaneous manifestations
    La porfiria epatica acuta (AHP) rappresenta un sottogruppo di porfiria caratterizzato dal verificarsi di attacchi neuro-viscerali con o senza manifestazioni cutanee
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10036182
    E.1.2Term Porphyria acute
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10036182
    E.1.2Term Porphyria acute
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10036184
    E.1.2Term Porphyria hepatic
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10036182
    E.1.2Term Porphyria acute
    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
    Evaluate the effect of subcutaneous (SC) givosiran, compared to placebo, on the rate of porphyria attacks requiring hospitalization, urgent healthcare visit, or intravenous (IV) hemin administration at home in patients with acute intermittent porphyria (AIP)
    Valutare l¿effetto di givosiran (ALN-AS1) somministrato per via sottocutanea, rispetto al placebo, sul tasso di attacchi di porfiria che richiedono ricovero, visita medica urgente o somministrazione di emina a domicilio in pazienti affetti da porfiria intermittente (AIP).
    E.2.2Secondary objectives of the trial
    ¿ Evaluate the effect of givosiran, compared to placebo, on urinary aminolevulinic acid (ALA) levels
    in patients with AIP
    ¿ Evaluate the effect of givosiran, compared to placebo, on urinary porphobilinogen (PBG) levels in
    patients with AIP
    ¿ Evaluate the effect of givosiran, compared to placebo, on hemin usage in patients with AIP
    ¿ Evaluate the effect of givosiran, compared to placebo, on the rate of porphyria attacks requiring
    hospitalization, urgent healthcare visit, or IV hemin administration at home in patients with any AHP
    ¿ Evaluate the effect of givosiran, compared to placebo, in patients with AIP on the symptoms of
    pain, nausea, and fatigue
    ¿ Evaluate the effect of givosiran, compared to placebo, in patients with AIP on the Physical
    Component Summary (PCS) of the 12-item Short-Form Health Survey (SF-12)
    ¿ Evaluate the safety and tolerability of givosiran in patients with any AHP
    ¿ Valutare l'effetto di givosiran, rispetto al placebo, sui livelli di acido aminolevulinico (ALA) urinario (ALA) nei pazienti con AIP
    ¿ Valutare l¿effetto di givosiran, rispetto al placebo, sui livelli di porfobilinogeno (PBG) urinario nei pazienti con AIP
    ¿ Valutare l¿effetto di givosiran, rispetto al placebo, sull¿uso di emina nei pazienti con AIP
    ¿ Valutare l¿effetto di givosiran rispetto al placebo, sul tasso di attacchi di porfiria che richiedono ricovero, visita medica urgente o somministrazione di emina a domicilio in pazienti affetti da porfiria epatica acuta (AHP)
    ¿ Valutare l'effetto di givosiran, rispetto al placebo, nei pazienti con AIP sui sintomi del dolore, della nausea e della stanchezza
    ¿ Valutare l'effetto di givosiran, rispetto al placebo, nei pazienti con AIP sul Physical Component Summary (PCS) del Questionario sullo stato di salute SF-12 [12-item Short-Form Health Survey]
    ¿ Valutare la sicurezza e la tollerabilit¿ di givosiran nei pazienti con AIP
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age =12 years
    2. Documented diagnosis of AIP, HCP, VP, or ADP based on clinical features (eg, acute
    attacks of abdominal, back, chest, extremities, and/or limb pain), at least one documented
    urinary or plasma PBG or ALA value =4× upper limit of normal (ULN) within the past
    year prior or during Screening, AND one of the following:
    ¿ Documented genetic evidence of mutation in a porphyria-related gene, defined as
    ANY of the following:
    ¿ AIP: mutation in the hydroxymethylbilane synthase gene (HMBS; also
    referred to as the porphobilinogen deaminase [PBGD] gene)
    ¿ HCP: mutation in the coproporphyrinogen oxidase (CPOX) gene
    ¿ VP: mutation in the protoporphyrinogen oxidase (PPOX) gene
    ¿ ADP: mutation in the aminolevulinic acid dehydratase (ALAD) homozygous
    or compound heterozygous genes
    ¿ OR if the results of a patient’s genetic testing do not identify a mutation in a
    porphyria-related gene (<5% of cases), a patient may be eligible for the study if
    they have both clinical features and diagnostic biochemical criteria consistent with
    AHP (Table 10)
    3. Have active disease, with at least 2 porphyria attacks requiring hospitalization, urgent
    healthcare visit or treatment with IV hemin at home within the 6 months prior to
    Screening
    4. Willing to discontinue and/or not initiate use of prophylactic hemin at the time of
    Screening and for the duration of the study
    5. Have adequate venous access for study sample collection as judged by the investigator
    6. Be willing to comply with the contraceptive requirements during the study period, as
    described in Section 6.4.
    7. Be willing and able to comply with the study requirements and to provide written
    informed consent as per local and national requirements. In the case of patients under the age of legal consent, legal guardian(s) must provide written informed consent and the patient
    should provide assent per
    local and national requirements and institutional standards
    1. Età = a 12 anni;
    2. Diagnosi documentata di AIP, HCP, VP o ADP basata su caratteristiche cliniche (es attacchi acuti di dolore addominale, dolore alla schiena, al petto alle estremità e/o alle gambe), almeno un valore PBG o ALA, urinario o plasmatico, = 4 volte superiore al normale (ULN) nell’anno precedente o durante lo screening e uno dei seguenti:
    • AIP: mutazione nel gene dell'idrossimetilbilano-sintasi (HMBS, noto anche come gene porfobilinogeno deaminasi [PBGD])
    • HCP: mutazione nel gene della coproporfirinogeno ossidasi (CPOX)
    • VP: mutazione nel gene della protoporfirinogeno ossidasi (PPOX)
    • ADP: mutazione nei geni omozigoti o eterozigoti composti dell’acido aminolevulinico deidrato (ALAD)
    • OR se i risultati dei test genetici di un paziente non identificano una mutazione in un gene correlato alla porfiria (<5% dei casi), un paziente può essere ammesso allo studio se dispongono sia di caratteristiche cliniche che di criteri diagnostici biochimici coerenti con AHP
    3. Avere malattia attiva con almeno 2 attacchi che richiedono ricovero, visita medica urgente o somministrazione di emina a domicilio prima dello screening
    4. Volontà di interrompere e/o non iniziare l’uso profilattico di emina al momento dello screening a per l’intera durata dello studio
    5. Avere un'adeguata accessibilità venosa per la raccolta di campioni di studio come giudicata dall'investigatore
    6. Essere disposti a rispettare i requisiti contraccettivi durante il periodo di studio, come descritto nella Sezione 6.4
    7. Essere di disposti e capaci di rispettare i requisiti dello studio e di fornire consenso scritto secondo i regolamenti locali e nazionali. Nel caso di pazienti minorenni, il tutore(I) legale deve fornire un consenso informato scritto e il paziente dovrebbe dare l'assesnso in accordo ai regolamenti locali e nazionali e gli standard instituzionali.
    E.4Principal exclusion criteria
    1. Any of the following laboratory parameter assessments at Screening:
    a. Alanine aminotransferase (ALT) >2×ULN
    b. Total bilirubin >1.5× ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert’s syndrome are eligible if the total bilirubin is <2×ULN
    c. International normalized ratio (INR) > 1.5 (patients on an anticoagulant [eg, warfarin] with an INR< 3.5 will be allowed)
    2. Estimated Glomerular Filtration Rate (eGFR) <30 mL/min/1.73 m2 using the Modification of Diet in Renal Disease (MDRD) formula
    3. On an active liver transplantation waiting list, or anticipated to undergo liver transplantation during the blinded study treatment period
    4. History of multiple drug allergies or history of allergic reaction to an oligonucleotide or to N-acetylgalactosamine (GalNAc)
    5. History of intolerance to subcutaneous injection(s)
    6. Known active HIV infection; or evidence of current or chronic hepatitis C virus (HCV) or hepatitis B virus (HBV) infection
    7. Currently enrolled in another investigational device or drug study, or less than 30 days or 5 half-lives (whichever is longer) since ending another investigational device or drug
    study(s), or receiving other investigational agent(s)
    8. Females who are pregnant, breast-feeding, or planning to become pregnant during the
    study
    9. Any condition (eg, medical concern or alcohol or substance abuse), which in the opinion of the Investigator, would make the patient unsuitable for dosing or which could interfere with the study compliance, the patient’s safety and/or the patient’s participation in the 6month treatment period of the study. This includes significant active and poorly
    controlled (unstable) cardiovascular, neurologic, gastrointestinal, endocrine, renal or psychiatric disorders
    unrelated to porphyria identified by key laboratory abnormalities or medical history.
    10. History of recurrent pancreatitis, or acute pancreatitis with disease activity within the past 12 months prior to Screening
    11. Has a major surgery planned during the first 6 months of the study
    12. History of serious infection within one month prior to Screening
    13. Had a malignancy within 5 years prior to Screening, except for basal or squamous cell carcinoma of the skin, cervical in-situ carcinoma, or breast ductal carcinoma, that has been successfully treated
    1. Valore di uno qualsiasi dei seguenti parametri di laboratorio al momento dello screening:
    a. Alanina aminotransferasi (ALT)> 2 x ULN
    b. Bilirubina totale> 1,5 × ULN. I pazienti con valori elevati di bilirubina totale che sono secondari alla sindrome di Gilbert documentata sono ammissibili se la bilirubina totale è < 2 x ULN
    c. Rapporto normalizzato internazionale (INR)> 1,5 (i pazienti in terapia anticoagulante [ad esempio warfarin] con un INR <3,5 saranno ammessi)
    2. Velocità di filtrazione glomerulare stimata (eGFR) <30 mL / min / 1,73 m2 utilizzando la formula di modifica della dieta nella malattia renale (MDRD)
    3. In lista di attesa attiva per il trapianto di fegato o si prevede che il soggetto si sottoponga a trapianto di fegato durante il periodo di trattamento in cieco dello studio
    4. Storia di allergie multiple ai farmaci o storia di reazione allergica ad un oligonucleotide o alla N-acetilgalattosamina (GalNAc)
    5. Storia di intolleranza alle iniezioni sottocutanee
    6. Infezione da HIV attiva nota; o indizi di infezione presente o cronica da parte del virus dell’epatite C (HCV) o dell'epatite B (HBV)
    7. Partecipazione ad un altro studio su dispositivo o farmaco sperimentale, o periodo temporale inferiore ai 30 giorni o a 5 emivite (a seconda di quale è il più esteso) dopo aver terminato un altro studio/i su dispositivo o farmaco sperimentale o dopo aver ricevuto uno o più agenti sperimentali
    8. Le donne in stato di gravidanza, in allattamento o che intendono rimanere incinte durante lo studio
    9. Qualsiasi condizione (ad esempio, controindicazioni mediche o abuso di alcol o sostanze stupefacenti) che a giudizio dello sperimentatore renderebbe il paziente inadatto alla somministrazione o che potrebbe interferire con l’aderenza alle direttive dello studio, con la sicurezza del paziente e / o con la partecipazione del paziente al periodo di trattamento di 6 mesi dello studio. Ciò include disturbi significativi che siano attivi e scarsamente controllati (instabili) a livello cardiovascolare, neurologico, gastrointestinale, endocrino, renale o psichiatrico, che non sono correlati alla porfiria e che sono stati identificati in base ad anomalie di laboratorio o in base all’anamnesi.
    10. Storia di pancreatite ricorrente o pancreatite acuta con malattia presente negli ultimi 12 mesi precedenti lo screening
    11. Avere un intervento chirurgico maggiore pianificato durante i primi 6 mesi dello studio
    12. Storia di infezione grave entro un mese prima dello screening
    13. Tumore maligno sviluppato nei 5 anni prima dello screening, ad eccezione del carcinoma cutaneo basale o a cellule squamose, del carcinoma cervicale in situ o del carcinoma duttale al seno che è stato trattato con successo.
    E.5 End points
    E.5.1Primary end point(s)
    Annualized rate of porphyria attacks requiring hospitalization, urgent healthcare visit, or IV hemin
    administration at home in patients with AIP over the 6-month treatment period
    • Tasso annualizzato di attacchi di porfiria che richiedono ricovero, visita medica urgente o somministrazione di emina a domicilio in pazienti con AIP durante un periodo di trattamento di 6 mesi.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Over the 6-month treatment period.
    Durante un periodo di trattamento di 6 mesi.
    E.5.2Secondary end point(s)
    Urinary ALA in patients with AIP at 3 months
    ¿ Urinary ALA in patients with AIP at 6 months
    ¿ Urinary PBG in patients with AIP at 6 months
    ¿ Annualized rate of administered hemin doses in patients with AIP over the 6-month treatment
    period
    ¿ Annualized rate of porphyria attacks requiring hospitalization, urgent healthcare visit, or IV hemin
    administration at home in patients with any AHP over the 6-month treatment period
    ¿ Daily worst pain score as measured by Brief Pain Inventory-Short Form (BPI-SF) numeric rating
    scale (NRS) in patients with AIP over the 6-month treatment period
    ¿ Daily worst nausea score as measured by NRS in patients with AIP over the 6-month treatment
    period
    ¿ Daily worst fatigue score as measured by Brief Fatigue Inventory-Short Form (BFI-SF) NRS in
    patients with AIP over the 6-month treatment period
    ¿ Change from baseline in the PCS of the SF-12 in patients with AIP at 6 months
    ¿ Acido aminolevulinico (ALA) urinario in pazienti con AIP a 3 mesi
    ¿ Acido aminolevulinico (ALA) urinario in pazienti con AIP a 6 mesi
    ¿ Porfobilinogeno (PBG) urinario in pazienti con AIP a 6 mesi
    ¿ Tasso annualizzato di dosi di emina somministrate in pazienti con AIP durante un periodo di trattamento di 6 mesi.
    ¿ Tasso annualizzato di attacchi di porfiria che richiedono ricovero, visita medica urgente o somministrazione di emina a domicilio in pazienti con qualsiasi forma di AHP in un periodo di trattamento di 6 mesi
    ¿ Punteggio giornaliero del dolore peggiore, misurato mediante la scala di valutazione numerica (NRS) del BPI-SF [Brief Pain Inventory-Short Form] in pazienti con AIP in un periodo di trattamento di 6 mesi.
    ¿ Punteggio giornaliero della nausea peggiore, misurato mediante la scala di valutazione numerica (NRS) in pazienti con AIP in un periodo di trattamento di 6 mesi
    ¿ Punteggio giornaliero dell¿affaticamento peggiore, misurato mediante la scala di valutazione numerica (NRS) del BPI-SF [Brief Pain Inventory-Short Form] in pazienti con AIP in un periodo di trattamento di 6 mesi
    ¿ Variazione dal valore basale del Physical Component Summary (PCS) del Questionario sullo stato di salute SF-12 [12-item Short-Form Health Survey] in pazienti con AIP a 6 mesi
    E.5.2.1Timepoint(s) of evaluation of this end point
    All secondary endpoints will be measured over the 6-month double-blind
    treatment period
    Tutti gli endpoint secondari saranno misurati durante il periodo di trattamento in doppio cieco di 6 mesi
    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 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 No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Lo studio prevede un periodo di 6 mesi in doppio cieco e un successivo periodo in aperto (30 mesi)
    The study envisages a double blind 6-month period and a subsequent open-label extension period (30m)
    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 EEA14
    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
    Brazil
    Bulgaria
    Canada
    Czechia
    Denmark
    Finland
    France
    Germany
    Italy
    Japan
    Korea, Republic of
    Mexico
    Netherlands
    Poland
    South Africa
    Spain
    Sweden
    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
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years32
    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 years3
    E.8.9.2In all countries concerned by the trial months2
    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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 1
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 58
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 15
    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 state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 36
    F.4.2.2In the whole clinical trial 74
    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)
    Consistent with pre-study treatment, if any.
    Coerente con il trattamento pre-studio, se presente
    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 Decision2018-02-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-12-12
    P. End of Trial
    P.End of Trial StatusCompleted
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