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    Summary
    EudraCT Number:2019-002835-27
    Sponsor's Protocol Code Number:ION-682884-CS2
    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:2021-08-03
    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 number2019-002835-27
    A.3Full title of the trial
    A Phase 3 Global, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
    Uno studio di fase 3 globale, in doppio cieco, randomizzato, controllato da placebo per valutare l'efficacia e la sicurezza di ION-682884 in pazienti con cardiomiopatia amiloide mediata da transtiretina (ATTR CM)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A global trial to determine the effectiveness and safety of ION-682884 in patients with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
    Uno studio globale per valutare l'efficacia e la sicurezza di di ION-682884 in pazienti con cardiomiopatia amiloide mediata da transtiretina (ATTR CM)
    A.3.2Name or abbreviated title of the trial where available
    CARDIO –TTRANSFORM
    CARDIO –TTRANSFORM
    A.4.1Sponsor's protocol code numberION-682884-CS2
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04136171
    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 SponsorIONIS 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 supportIonis Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIonis Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointIonis Clinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address2855 Gazelle Court
    B.5.3.2Town/ cityCarlsbad
    B.5.3.3Post codeCA 92010
    B.5.3.4CountryUnited States
    B.5.4Telephone number0017606033890
    B.5.5Fax number0017606032504
    B.5.6E-mailClinicalTrials@ionisph.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 nameION-682884
    D.3.2Product code [ION-682884]
    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.3.9.1CAS number 1637600-16-8
    D.3.9.2Current sponsor codeION-682884
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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 Yes
    D.3.11.13.1Other medicinal product typeAntisense Oligonucleotide
    D.IMP: 2
    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 nameMultivitaminico Adulti Teva
    D.3.2Product code [na]
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codena
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3000
    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) 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 Yes
    D.3.11.13.1Other medicinal product typeIntegratore multivitaminico
    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
    Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
    Cardiomiopatia amiloide mediata da transtiretina (ATTR CM)
    E.1.1.1Medical condition in easily understood language
    A condition that causes damage to the heart
    Una condizione che causa danno al cuore
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10002020
    E.1.2Term Amyloid cardiomyopathy
    E.1.2System Organ Class 100000004849
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the effect of treatment with ION-682884 compared to placebo for 120 weeks on the composite endpoint of cardiovascular (CV) death and frequency of CV clinical events in patients with transthyretinmediated amyloid cardiomyopathy (ATTR-CM) receiving available
    standard of care (SoC).
    To evaluate the effect of treatment with ION-682884 compared to placebo for 60 weeks (Week 61 study visit) on exercise tolerance measured by the 6-minute walk test (6MWT) in patients with ATTR-CM receiving available SoC.
    To evaluate the effect of treatment with ION-682884 compared to placebo for 120 weeks CV clinical events, CV death, and all-cause death in patients with ATTR-CM receiving available SoC.
    Valutare l'effetto del trattamento con ION-682884 rispetto al placebo per 120 settimane sull'endpoint composito della mortalità cardiovascolare (CV) e la frequenza degli eventi clinici CV in pazienti con ATTR-CM che ricevono lo standard di cura (SoC) disponibile
    Valutare l'effetto del trattamento con ION-682884 rispetto al placebo per 60 settimane (visita dello studio della settimana 61) sulla tolleranza all'esercizio misurata con il test della camminata di 6 minuti (6MWT) in pazienti con ATTR-CM che ricevono il SoC disponibile.
    Valutare l'effetto del trattamento con ION-682884 rispetto al placebo per 120 settimane, sugli eventi clinici CV, la morte CV e la morte per tutte le cause in pazienti con ATTR-CM che ricevono il SoC disponibile.
    E.2.2Secondary objectives of the trial
    To evaluate the effect of treatment with ION-682884 compared to placebo for 120 weeks on exercise tolerance and patient reported outcomes in patients with ATTR-CM receiving available SoC.
    To evaluate the effect of treatment with ION-682884 compared to placebo for 120 weeks on the rates of CV death, CV clinical events, and all-cause death in patients with ATTR-CM receiving available SoC.
    Valutare l'effetto del trattamento con ION-682884 rispetto al placebo per 120 settimane sulla tolleranza all'esercizio (valutato con 6MWT) e gli esiti riportati dal paziente (Kansas City Cardiomyopathy Questionnaire [KCCQ]) in pazienti con ATTR CM che ricevevano il SoC disponibile
    Valutare l'effetto del trattamento con ION-682884 rispetto al placebo per 120 settimane sui tassi di morte CV, eventi clinici CV e morte per tutte le cause in pazienti con ATTR-CM che ricevono il SoC disponibile
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal or abstinent. If engaged in sexual relations of child-bearing potential, agree to use 1 highly effective contraceptive method
    Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the participant or the participant's non-pregnant female partner must be using a highly effective contraceptive method
    Willing to be genetically tested for mutations in the transthyretin (TTR) gene during screening, if it was not done before
    Amyloid deposits in cardiac or non-cardiac tissue confirmed by Congo Red (or equivalent) staining OR technetium scintigraphy (99mTc -3,3-diphosphono-1,2- propanodicarboxylic acid [DPD-Tc], 99m Tc- pyrophosphate [PYP-Tc], or 99mTc-hydroxymethylene-diphosp te [HMDP-Tc]) with Grade 2 or 3 cardiac uptake in the absence of abnormal light chains ratio, centrally confirmed
    End-diastolic interventricular septum thickness of major of 12 mm on Screening echocardiogram
    Medical history of heart failure (HF) secondary to hereditary or wildtype ATTR-CM with at least: A) prior hospitalization for HF, which may include hospitalization for arrhythmia or pacemaker/ICD (implantable cardioverter defibrillator) placement, OR B) symptoms and signs of volume overload or elevated intracardiac pressure that requires treatment with diuretics other than mineralocorticoid receptor antagonists (MRA) for clinical stabilization
    Screening N-terminal prohormone of brain natriuretic peptide (NTproBNP) greater = 600 pg/mL (greater = 70 pmol/L) by central lab. For patients in atrial fibrillation at screening the NT-proBNP values greater = 1200 pg/mL (greater = 140 pmol/L)
    New York Heart Association (NYHA) class I-III6MWT greater than 150 metri
    If on medical treatment for HF on stable dosage regimen for 2 weeks prior to randomization
    Willingness to adhere to vitamin A supplement per protocol
    Soggetto di sesso femminile: non deve essere incinta né in fase di allattamento e chirurgicamente sterile o in post menopausa o astinente.d. se in età fertile e sessualmente attiva, accetta di utilizzare 1 metodo contraccettivo efficace
    I soggetti di sesso maschile devono essere chirurgicamente sterili o astinenti; se sessualmente attivi con una donna in età fertile, il partecipante o la partner non incinta deve utilizzare un metodo contraccettivi
    Disponibile a sottoporsi al test genetico per le mutazioni del gene TTR durante lo screening, se non è stato fatto prima
    Depositi amiloidi nel tessuto cardiaco o non cardiaco confermati dalla colorazione Congo Red (o equivalente) OPPURE da scintigrafia con tecnezio (99mTc -3,3-difosfono-1,2-acido propanodicarbossilico [DPD-Tc], 99m Tc-pirofosfato [PYP-Tc] o 99mTc-idrossimetilendifosfonato [HMDP-Tc]) con assorbimento cardiaco di grado 2 o 3 in assenza di un rapporto anomalo delle catene leggere, confermato centralmente
    Spessore del setto interventricolare tele-diastolico maggiore di 12 mm su ecocardiogramma di screening
    Storia clinica di insufficienza cardiaca (HF) secondaria ad ATTR CM ereditario o wild-type con almeno: A) 1 precedente ricovero ospedaliero per HF, che può includere ricovero ospedaliero per aritmia o impianto di pacemaker/ICD (defibrillatore cardioverter impiantabile), OPPURE B) sintomi e segni di sovraccarico di volume o elevata pressione intracardiaca che richiede un trattamento con diuretici per la stabilizzazione clinica
    Screening del Proormone N-terminale del peptide natriuretico cerebrale (NT-proBNP) maggiore uguale a 600pg/mL (maggiore uguale a 70 pmol/L )secondo il laboratorio centrale. Per i pazienti con fibrillazione atriale allo screening, il valore di idoneità di NT-proBNP è maggiore uguale a1200 pg/mL ( maggiore uguale 140 pmol/L)
    Classe I-III secondo la New York Heart Association (NYHA)
    6MWT maggiore uguale a 150 metri
    Se in trattamento medico per HF, regime terapeutico stabile per almeno 2 settimane prima della randomizzazione
    Disponibilità ad aderire all'integrazione di vitamina A secondo il protocollo
    E.4Principal exclusion criteria
    Acute coronary syndrome, unstable angina, stroke, transient ischemic attack (TIA), coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening
    Cardiomyopathy not primarily caused by ATTR-CM, for example,cardiomyopathy due to hypertension, valvular heart disease, or ischemic heart disease
    Monoclonal gammopathy of undetermined significance (MGUS) and/or alterations in immunoglobulin free light chain (FLC) ratio, unless fat, bone marrow, or heart biopsy confirming the absence of light chain and the presence of TTR protein by mass spectrometry or immunoelectron microscopy. For patients with CKD and without presence of monoclonal protein in blood and urine, the acceptable FLC ratio is 0.26-2.25. Results different from that may be discussed with local hematologist,Investigator and Medical Monitor if the risks associated with the biopsy outweigh the benefits
    Prior liver or heart transplant, and/or Left Ventricular Assist Device (LVAD) or anticipated liver transplant or LVAD within 1 year after randomization
    Current or previous treatment with Tegsedi™ (inotersen) or Onpattro™ (patisiran) or other oligonucleotide or RNA therapeutic (including siRNA)
    Current treatment with diflunisal, doxycycline, with or without ursodeoxycholic acid, and/or calcium-channel blocker (e.g. verapamil, diltiazem). Participants receiving any of these agents must respect a washout period of 14 days before randomization.
    Abnormal thyroid function tests with clinical significance per Investigator judgement.
    Contraindication for immunosuppressive therapy, per Investigator's discretion
    Known history of or positive test for human immunodeficiency virus (HIV) (as evidenced by positive tests for HIV antibody and HIV RNA), hepatitis C (as evidenced by positive tests for HCV antibody and HCV RNA) or hepatitis B (as evidenced by a positive test for hepatitis B surface
    antigen)
    History of bleeding, diathesis or coagulopathy (e.g., liver cirrhosis, hematologic malignancy,antiphospholipid antibody syndrome,congenital disorders such as hemophilia A, B, and Von Willebrand disease)
    Sindrome coronarica acuta, angina instabile, ictus, attacco ischemico transitorio (TIA), rivascolarizzazione coronarica, impianto di dispositivo cardiaco, riparazione della valvola cardiaca o chirurgia maggiore entro 3 mesi dallo screening
    Cardiomiopatia non causata principalmente da ATTR-CM, ad esempio cardiomiopatia da ipertensione, cardiopatia valvolare o cardiopatia ischemica
    Gammopatia monoclonale di significato indeterminato (MGUS) e/o alterazioni del rapporto della catena leggera libera (FLC) delle immunoglobuline, a meno che grasso, midollo osseo o biopsia cardiaca confermino l'assenza di catena leggera e la presenza della proteina TTR mediante spettrometria di massa o microscopia immunoelettronica. Per i pazienti con CKD e privi di proteina monoclonale nel sangue e nelle urine, il rapporto FLC accettabile è di 0,26-2,25. Risultati diversi da questo possono essere discussi con l'ematologo locale, lo Sperimentatore e il Monitor medico se i rischi associati alla biopsia superano i benefici
    Precedente trapianto di fegato o cuore e/o dispositivo di assistenza ventricolare sinistra (LVAD) o trapianto di fegato o LVAD previsti entro 1 anno dalla randomizzazione
    Trattamento attuale o precedente con Tegsedi™ (inotersen) o Onpattro™ (patisiran) o altri oligonucleotidi o RNA terapeutici (incluso siRNA)
    Trattamento attuale con diflunisale, doxiciclina, con o senza acido ursodeossicolico, e/o calcio-antagonista senza diidropiridina (es. verapamil, diltiazem). I pazienti che ricevono uno di questi agenti devono rispettare un periodo di wash-out di 14 giorni prima della randomizzazione.
    Test di funzionalità tiroidea anormali con significatività clinica secondo il giudizio dello Sperimentatore.
    Controindicazione per terapia immunosoppressiva, a discrezione dello Sperimentatore
    Storia nota o test positivo per virus dell'immunodeficienza umana (HIV) (come evidenziato da test positivi per anticorpi HIV e RNA HIV), epatite C (come evidenziato da test positivi per anticorpi HCV e RNA HCV) o epatite B (come evidenziato da un test positivo per l’antigene di superficie del virus dell’epatite B)
    Storia di sanguinamento, diatesi o coagulopatia (es. cirrosi epatica, neoplasia ematologica, sindrome da anticorpi antifosfolipidi, disturbi congeniti come emofilia A, B e malattia di Von Willebrand)
    E.5 End points
    E.5.1Primary end point(s)
    Composite endpoint of CV-mortality and recurrent CV clinical events comparing the 2 study arms at Week 120 study visit using the Andersen-Gill method.
    Cardiovascular clinical events include:
    -Hospitalization for myocardial infarction (MI)
    -Hospitalization for heart failure (HF)
    -Hospitalization for arrhythmias
    -Hospitalization for stroke/ Transient Ischemic Attack (TIA)
    -HF urgent visits to Emergency Department/Emergency Room (ED/ER) or HF clinics requiring administration of intravenous (IV) diuretics for improvement.
    All deaths and CV clinical events will be adjudicated by an independent Clinical Adjudication Committee (CAC) using the '2017 Cardiovascular and Stroke Endpoint Definitions for Clinical Trials'
    Interim Analysis Primary Endpoint at Week 61
    Change from baseline on the 6MWT comparing the first approximately
    370 patients enrolled into the 2 study arms at Week 61 study visit.
    Endpoint composito della mortalità CV e eventi clinici ricorrenti CV confrontando i 2 bracci dello studio alla settimana 120 usando il metodo di Andersen-Gill.
    Gli eventi clinici cardiovascolari includono:
    -Ricovero ospedaliero per infarto del miocardio (MI)
    -Ricovero ospedaliero per esacerbazione dell'HF
    -Ricovero ospedaliero per aritmie
    -Ricovero ospedaliero per ictus/TIA
    -Visite urgenti, non programmate per HF, presso ambulatori di pronto soccorso o HF che richiedono la somministrazione di diuretici per via endovenosa (ev) per il miglioramento
    Tutti i decessi e gli eventi clinici CV saranno giudicati da un CAC indipendente utilizzando le "2017 Cardiovascular and Stroke Endpoint Definitions for Clinical Trials" ("Definizioni endpoint cardiovascolari e di ictus 2017 per le sperimentazioni cliniche")
    Analisi ad interim dell'Endpoint primario alla settimana 61
    Variazione rispetto al basale del 6MWT confrontando i primi 370 pazienti circa arruolati nei 2 bracci dello studio alla visita dello studio della settimana 61.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Final Analysis Primary Endpoints at Week 120
    Analisi finale dell' endpoint primario alla settimana 120
    E.5.2Secondary end point(s)
    Changes from baseline in the 6MWT distance and Kansas City Cardiomyopathy Questionnaire (KCCQ) scores comparing the 2 study arms at Week 120 study visit
    CV clinical events, CV mortality, and all-cause mortality comparing the 2 study arms at Week 120 study visit
    Variazioni rispetto al basale della distanza 6MWT e dei punteggi KCCQ confrontando i 2 bracci dello studio alla settimana 120
    Eventi clinici CV, mortalità CV e mortalità per tutte le cause confrontando i 2 bracci dello studio alla visita dello studio della settimana 120
    E.5.2.1Timepoint(s) of evaluation of this end point
    Final Analysis of Secondary Endpoints at Week 120; Analisi finale dell'endpoint secondario alla settimana 120
    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 Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    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
    Brazil
    Canada
    Israel
    Japan
    United States
    Austria
    Belgium
    Denmark
    France
    Germany
    Italy
    Poland
    Portugal
    Spain
    Sweden
    United Kingdom
    Czechia
    Argentina
    Greece
    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 visit last subject (LVLS)
    Ultima visita dell'ultimo paziente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days30
    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: 187
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 563
    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 state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 260
    F.4.2.2In the whole clinical trial 750
    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 completion of the Treatment Period, eligible patients may elect to receive ION-682884 in an open-label extension (OLE) study, pending study approval by the Institutional review Board/Independent Ethics Committee (IRB/IEC) and the regulatory authority. In this case, patients will not participate in the Post-Treatment Evaluation assessments. All participating patients in the OLE study will receive ION-682884 SC once every 4 weeks.
    Dopo il completamento del periodo di trattamento, i pazienti idonei possono scegliere di ricevere ION-682884 in uno studio di estensione in aperto (OLE) in attesa dell'approvazione dello studio da parte del Comitato di revisione istituzionale/Comitato etico indipendente e dell'autorità regolatoria. In questo caso, i pazienti non parteciperanno alla valutazione post-trattamento. Tutti i pazienti partecipanti allo studio OLE continueranno a ricevere ION-682884 SC una volta ogni 4 settimane.
    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 Decision2020-03-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-01-15
    P. End of Trial
    P.End of Trial StatusOngoing
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