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    Summary
    EudraCT Number:2019-003153-28
    Sponsor's Protocol Code Number:ALN-TTRSC02-003
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2021-05-20
    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 number2019-003153-28
    A.3Full title of the trial
    HELIOS-B: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy)
    HELIOS-B: Studio di fase 3, randomizzato, in doppio cieco, controllato con placebo, multicentrico volto a valutare l’efficacia e la sicurezza di vutrisiran in pazienti con amiloidosi da transtiretina con cardiomiopatia (amiloidosi ATTR con cardiomiopatia)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy
    Studio volto a valutare vutrisiran in pazienti affetti da amiloidosi da transtiretina con cardiomiopatia
    A.3.2Name or abbreviated title of the trial where available
    Helios B
    HELIOS B
    A.4.1Sponsor's protocol code numberALN-TTRSC02-003
    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 organisationAlnylam Pharmaceuticals, Inc
    B.5.2Functional name of contact pointClinical Trials Information Line
    B.5.3 Address:
    B.5.3.1Street Address300 Third Street
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post code02142
    B.5.3.4CountryUnited States
    B.5.4Telephone number0018772569526
    B.5.6E-mailclinicaltrials@alnylam.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 Yes
    D.2.1.1.1Trade name ELEVIT PRONATAL
    D.2.1.1.2Name of the Marketing Authorisation holderUAB Bayer
    D.2.1.2Country which granted the Marketing AuthorisationLithuania
    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 nameELEVIT PRONATAL
    D.3.2Product code [ELEVIT PRONATAL]
    D.3.4Pharmaceutical form Film-coated 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.3.8INN - Proposed INNRETINOLO
    D.3.9.1CAS number 68-28-6
    D.3.9.2Current sponsor codeELEVIT PRONATAL
    D.3.9.4EV Substance CodeSUB16102MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3600
    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.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/18/2026
    D.3 Description of the IMP
    D.3.1Product namevutrisiran
    D.3.2Product code [ALN-TTRSC02]
    D.3.4Pharmaceutical form Solution for injection in pre-filled syringe
    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 INNvutrisiran
    D.3.9.1CAS number 1867157-35-4
    D.3.9.2Current sponsor codeALN-65492
    D.3.9.4EV Substance CodeSUB-182382
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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 Yes
    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 in pre-filled syringe
    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 Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy)
    Amiloidosi da transtiretina con cardiomiopatia (amiloidosi ATTR con cardiomiopatia)
    E.1.1.1Medical condition in easily understood language
    Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy)
    Amiloidosi da transtiretina con cardiomiopatia (amiloidosi ATTR con cardiomiopatia)
    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 PT
    E.1.2Classification code 10007509
    E.1.2Term Cardiac amyloidosis
    E.1.2System Organ Class 10007541 - Cardiac 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 evaluate the efficacy of vutrisiran compared to placebo on reducing all-cause mortality and Cardiovascular (CV) events
    Valutare l’efficacia di vutrisiran rispetto al placebo nel ridurre la mortalità per qualsiasi causa ed eventi cardiovascolari (CV)
    E.2.2Secondary objectives of the trial
    To evaluate the efficacy of vutrisiran compared with placebo treatment on:
    - Functional capacity
    - Patient-reported health status and healthrelated quality of life
    - Cardiac structure and function
    - Additional assessments of death, hospitalizations, and urgent heart failure (HF) visits
    - Components of the primary composite outcome endpoint (all-cause mortality, and CV events)
    - Cardiac biomarkers
    Valutare l’efficacia di vutrisiran confrontato con un trattamento placebo su:
    - capacità funzionali
    - stato di salute riferito dal paziente e qualità della vita correlata alla salute
    - struttura e funzione cardiaca
    - valutazioni aggiuntive del decesso, ricoveri e visite urgenti per insufficienza cardiaca (IC)
    - componenti degli endpoint di esito composito primario (mortalità per qualsiasi causa ed eventi CV)
    - biomarcatori cardiaci
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age 18 (or age of legal consent per local regulations, whichever is older) to 85 years, inclusive.
    2. Documented diagnosis of ATTR amyloidosis with cardiomyopathy, classified as either hATTR amyloidosis with cardiomyopathy or wtATTR amyloidosis with cardiomyopathy
    3. Medical history of HF with at least 1 prior hospitalization for HF (not due to arrhythmia or a conduction system disturbance treated with a permanent pacemaker) OR clinical evidence of HF (with or without hospitalization) manifested by signs and symptoms of volume overload or elevated intracardiac pressures (eg, elevated jugular venous pressure, shortness of breath or signs of pulmonary congestion on x-ray or auscultation, peripheral edema) that currently requires treatment with a diuretic.
    4. Patient meets one of the following criteria:
    a. Tafamidis-naïve and not actively planning to commence treatment with tafamidis during the first 12 months following randomization (per exclusion criterion #7); or
    b. On tafamidis (Note: must be on-label use of commercial tafamidis per an approved cardiomyopathy indication and dose in the country of use)
    5. Patient is clinically stable, with no CV-related hospitalizations within 6 weeks prior to randomization, as assessed by the Investigator.
    6. Screening NT-proBNP >300 ng/L and <8500 ng/L; in patients with permanent or persistent atrial fibrillation, screening NT-proBNP >600 ng/L and <8500 ng/L..
    7. Able to complete =150 meters on the 6-MWT at Screening.
    8. Have a Karnofsky performance status of =60%.
    9. Patient is able to understand and is willing and able to comply with the study requirements and to provide written informed consent.
    10. Patient agrees to sign a separate medical records release form, where allowed by local regulations, to allow for the collection of information on vital status, cardiac transplant procedures, left-ventricular assist device placement, and hospitalizations, for the duration of the DB Period of the study.
    1. Età da 18 anni (o l’età di consenso legale secondo le normative locali, a seconda di quale sia maggiore) a 85 anni, compiuti.
    2. Diagnosi documentata di amiloidosi ATTR con cardiomiopatia, classificata come amiloidosi hATTR con cardiomiopatia o amiloidosi wtATTR con cardiomiopatia
    3. Anamnesi medica di IC con almeno 1 precedente ricovero per IC (non a causa di aritmia o un disturbo del sistema di conduzione trattati con un pacemaker permanente) O evidenza clinica di IC (con o senza ricovero) manifestata da segni e sintomi di sovraccarico di volume o elevata pressione intracardiaca (per esempio, elevata pressione venosa giugulare, affanno o segni di congestione polmonare alla radiografia o auscultazione, edema periferico) che necessita attualmente un trattamento con un diuretico.
    4. Il paziente soddisfa uno dei seguenti criteri:
    a. Naïve a tafamidis e non ha attivamente intenzione di iniziare il trattamento con tafamidis durante i primi 12 mesi dopo la randomizzazione (secondo il criterio di esclusione n. 7) o per un’indicazione e una dose approvate per la cardiomiopatia nel Paese di utilizzo
    5. Il paziente è clinicamente stabile, senza ricoveri correlati a CV entro 6 settimane prima della randomizzazione, come valutato dallo sperimentatore.
    6. NT-proBNP >300 ng/L e <8500 ng/L allo screening; in pazienti con fibrillazione atriale permanente o persistente, NT-proBNP >600 ng/L e <8500 ng/L allo screening.
    7. In grado di completare =150 metri sul test del cammino in 6 minuti (6-minute walk test, [6MWT]) allo screening.
    8. Presenta una valutazione delle prestazioni secondo la scala di Karnofsky =60%.
    9. Il paziente è in grado di comprendere ed è disposto e in grado di attenersi ai requisiti dello studio e di fornire il consenso informato scritto.
    10. Il paziente accetta di firmare un modulo separato per il rilascio delle cartelle cliniche, ove consentito dalle normative locali, per consentire la raccolta di informazioni sullo stato vitale, le procedure di trapianto cardiaco, l’impianto di un dispositivo di assistenza ventricolare sinistra e ricoveri per la durata del periodo DB dello studio.
    E.4Principal exclusion criteria
    1. Has known primary amyloidosis or leptomeningeal amyloidosis
    2. NYHA Class IV heart failure; or NYHA Class III heart failure AND ATTR Amyloidosis Disease Stage 3 (defined as NT-proBNP >3000 ng/L and eGFR <45 ml/min)
    3. Has a polyneuropathy disability (PND) Score IIIa, IIIb, or IV (requires cane or stick to walk due to polyneuropathy, or is wheelchair bound) at the Screening visit
    4. Has any of the following laboratory parameter assessments at Screening: a. AST or ALT levels >2.0 × ULN; b. Total bilirubin >2.0 × 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 (unless patients were on anticoagulant therapy in which case excluded if INR >3.5)
    5. Has eGFR <30 mL/min/1.73 m2 (using the modification of diet in renal disease [MDRD] formula) at Screening
    6. Has known human immunodeficiency virus infection; or evidence of current or chronic hepatitis C virus or hepatitis B virus infection
    7. Tafamidis-naïve patients (at baseline) for whom the Investigator actively plans or anticipates commencing treatment with tafamidis during the first 12 months following randomization, taking into consideration clinical status, patient preference and/or commercial availability of tafamidis
    8. Received prior TTR-lowering treatment (including revusiran, patisiran or inotersen) or participated in a gene therapy trial for hATTR amyloidosis
    9. Currently taking diflunisal; if previously on this agent, must have at least a 30-day wash-out prior to dosing (Day 1)
    10. Currently taking doxycycline, ursodeoxycholic acid or tauroursodeoxycholic acid; if previously on any of these agents, must have completed a 30-day wash-out prior to dosing (Day 1)
    PLEASE REFER TO PROTOCOL FOR FURTHER CRITERIA
    1. Presenta amiloidosi primaria o amiloidosi leptomeningea
    2. Insufficienza cardiaca di classe IV-New York Heart Association (NYHA); o insufficienza cardiaca di classe III-NYHA E malattia da amiloidosi ATTR di Stadio 3 (definita come NT-proBNP > 3000 ng/L e velocità di filtrazione glomerulare stimata (estimated glomerular filtration rate, [eGFR])<45 ml/min)
    3. Presenta un punteggio di invalidità di polineuropatia (polyneuropathy disability, [PND]) IIIa, IIIb, o IV (necessita di bastone per camminare a causa della polineuropatia, o è costretto alla sedia a rotelle) alla visita di screening
    4. Allo screening presenta un valore relativo a una qualsiasi delle seguenti valutazioni dei parametri di laboratorio: a. livelli di AST o ALT > 2,0 × ULN; b. bilirubina totale >2,0 x ULN. I pazienti con bilirubina totale elevata che sia secondaria alla sindrome di Gilbert documentata sono idonei se la bilirubina totale è < 2 × ULN); c. Rapporto normalizzato internazionale (international normalized ratio, [INR]) > 1,5 (eccetto i pazienti che sono stati in terapia anticoagulante, nel qual caso esclusi se INR > 3,5)
    5. Presenta eGFR < 30 ml/min/1,73 m2 (utilizzando la formula della modifica della dieta nella della malattia renale [modification of diet in renal disease, [MDRD]]) allo screening
    6. Presenta infezione nota da virus dell’immunodeficienza umana o evidenza di infezione da virus dell’epatite C o da virus dell’epatite B attuale o cronica
    7. Pazienti naïve a tafamidis (al basale) per i quali lo sperimentatore pianifica o prevede attivamente di iniziare il trattamento con tafamidis durante i primi 12 mesi successivi alla randomizzazione, tenendo in considerazione lo stato clinico, la preferenza del paziente e/o la disponibilità commerciale di tafamidis
    8. Pazienti che hanno ricevuto un precedente trattamento per abbassare la TTR (compresi revusiran, patisiran o inotersen) o che hanno partecipato a una sperimentazione di terapia genica per hATTR
    9. Attuale assunzione di diflunisal; se in precedenza in trattamento con questo agente, deve avere almeno un washout di 30 giorni prima della somministrazione (Giorno 1)
    10. Attuale assunzione di doxiciclina, acido ursodesossicolico o acido tauroursodesossicolico; se in precedenza in trattamento con uno qualsiasi di questi agenti, deve aver completato un washout di 30 giorni prima della somministrazione (Giorno 1)
    SI PREGA DI FAR RIFERIMENTO AL PROTOCLLO PER GLI ULTERIORI CRITERI
    E.5 End points
    E.5.1Primary end point(s)
    Composite outcome of all-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits)
    Esito composito di mortalità per qualsiasi causa ed eventi CV ricorrenti (ricoveri per CV e visite urgenti per IC)
    E.5.1.1Timepoint(s) of evaluation of this end point
    36 months
    36 mesi
    E.5.2Secondary end point(s)
    - Change from baseline in 6-minute walk test (6-MWT)
    - Change from baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS)
    - Change from baseline in mean left ventricular (LV) wall thickness (by echocardiographic assessment)
    - Change from baseline in global longitudinal strain (by echocardiographic assessment)
    - Composite endpoint of all-cause mortality and recurrent all-cause hospitalizations and urgent HF visits.
    - All-cause mortality
    - Recurrent CV events (CV hospitalizations and urgent HF visits)
    - Change from baseline in N-terminal prohormone B-type natriuretic peptide (NT-proBNP)
    - Variazione rispetto al basale nel test del cammino in 6 minuti (6-MWT)
    - Variazione rispetto al basale nel riepilogo generale del questionario sulla cardiomiopatia di Kansas City (Kansas City Cardiomyopathy Questionnaire Overall Summary, [KCCQ-OS])
    - Variazione rispetto al basale dello spessore medio parietale del ventricolo sinistro (left ventricular, [LV]) (mediante valutazione ecocardiografica)
    - Variazione rispetto al basale nel ceppo longitudinale globale (mediante valutazione ecocardiografica)
    - Endpoint composito di mortalità per qualsiasi causa e ricoveri ricorrenti per qualsiasi causa e visite urgenti per IC.
    - Mortalità per qualsiasi causa
    - Eventi CV ricorrenti (ricoveri per CV e visite urgenti per IC)
    - Variazione rispetto al basale del pro-ormone N-terminale del peptide natriuretico di tipo B (NT-proBNP)
    E.5.2.1Timepoint(s) of evaluation of this end point
    6-MWT: Weeks 1, 24, 48, 72, 84, 108, 132
    KCCQ-OS: Weeks 1, 24, 48, 72, 84, 108, 132
    echocardiogram: Weeks 48, 72, 108, 132
    NT-proBNP: Weeks 12, 24, 36, 48, 72, 108, 132
    mortality and CV events assessed over 36 month period

    6-MWT: Settimane 1, 24, 48, 72, 84, 108, 132
    KCCQ-OS: Settimane 1, 24, 48, 72, 84, 108, 132
    ecocardiogramma: Settimane 48, 72, 108, 132
    NT-proBNP: Settimane 12, 24, 36, 48, 72, 108, 132 eventi di mortalità e CV valutati su un periodo di 36 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 Yes
    E.6.7Pharmacodynamic Yes
    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 trial1
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA80
    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
    Colombia
    Costa Rica
    Israel
    Japan
    Jordan
    Korea, Republic of
    Lebanon
    Malaysia
    Mexico
    Moldova, Republic of
    Peru
    Russian Federation
    Saudi Arabia
    Singapore
    Taiwan
    Thailand
    Turkey
    United States
    Austria
    Belgium
    Croatia
    Denmark
    France
    Germany
    Hungary
    Ireland
    Italy
    Latvia
    Lithuania
    Netherlands
    Norway
    Poland
    Portugal
    Romania
    Slovenia
    Spain
    Sweden
    United Kingdom
    Czechia
    Argentina
    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
    LPLV
    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 months0
    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 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 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: 300
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 300
    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 state16
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 408
    F.4.2.2In the whole clinical trial 700
    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 participation in the trial has ended, patients will be treated at the discretion of their physician based on local standard of care. In the case where there are no commercially available treatment options, the Sponsor will aim to provide access to vutrisiran via the means available
    Dopo la fine della partecipazione alla sperimentazione, i pazienti saranno trattati a discrezione del proprio medico in base allo standard di cura locale. Nel caso in cui non vi siano opzioni di trattamento disponibili in commercio, lo sponsor cercherà di fornire accesso a vutrisiran tramite i mezzi disponibili
    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 Decision2021-02-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-02-23
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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