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    Summary
    EudraCT Number:2014-003835-20
    Sponsor's Protocol Code Number:ALN-TTRSC-004
    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:2016-03-09
    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 number2014-003835-20
    A.3Full title of the trial
    A phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-Controlled Study to evaluate the Efficacy and Safety of ALN TTRSC in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)
    Uno studio di fase tre, multicentrico, multinazionale, randomizzato, in doppio cieco, controllato da placebo per valutare l’efficacia e la sicurezza di ALN TTRSC in pazienti affetti da cardiomiopatia amiloidotica familiare (FAC) mediata da transtiretina (TTR)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to look at the efficacy and safety of ALN TTRSC in patients with an inherited condition that causes certain protein molecules to deposit in the heart
    Uno studio mirato a valutare l'efficacia e la sicurezza di ALN TTRSC in soggetti con una condizione ereditaria che causa nel cuore il deposito di alcune molecole proteiche
    A.3.2Name or abbreviated title of the trial where available
    na
    na
    A.4.1Sponsor's protocol code numberALN-TTRSC-004
    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 Trial Hotline
    B.5.3 Address:
    B.5.3.1Street Address300 Third Street
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA 02142
    B.5.3.4CountryUnited States
    B.5.4Telephone number001 866 330 0326
    B.5.5Fax number001 617 551 8101
    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 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/3/14/1267
    D.3 Description of the IMP
    D.3.1Product nameALN-TTRSC
    D.3.2Product code ALN-TTRSC
    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.3.9.2Current sponsor codeALN-51547
    D.3.9.4EV Substance CodeSUB104164
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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
    Trasthyretin (TTR) mediated familial amyloidotic cardiomyopathy (FAC)
    Cardiomiopatia amiloidotica familiare (FAC) mediata da transtiretina (TTR)
    E.1.1.1Medical condition in easily understood language
    FAC is a hereditary disease caused by protein aggregates in the heart. It leads to heart dysfunction, conduction defects, arrhythmia, congestive heart failure and death
    FAC è una malattia ereditaria causata da proteine aggregate nel cuore. Questo comporta a una disfunzione del cuore, difetti di conduzione, aritmie, infarti, morte
    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 10016202
    E.1.2Term Familial amyloidosis
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the efficacy of ALN-TTRSC in patients with FAC
    Determinare l'efficacia di ALN-TTRSC in pazienti affetti da FAC
    E.2.2Secondary objectives of the trial
    Not applicable
    Non applicabile
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Are male or female of 18 to 90 years of age (inclusive).
    2. Have a documented TTR mutation.
    3. Amyloid deposits in cardiac or non-cardiac tissue confirmed by Congo Red (or equivalent) staining or technetium (99mTC) scintigraphy (99mTc-3,3-diphosphono-1,2-propanodicarboxylic acid [DPD-Tc] or 99mTc-yrophosphate [pyp-Tc]) with Grade 2 or 3 cardiac uptake, centrally confirmed.
    4. If patient has monoclonal gammopathy, TTR amyloidosis needs to be confirmed through TTR protein identification by immunohistochemistry or mass spectrometry.
    5. Have a medical history of heart failure (HF) with at least 1 prior hospitalization for HF, which may include hospitalization for arrhythmia or pacemaker placement, OR clinical evidence of HF (as evidenced by one or more of the following: elevated jugular venous pressure, peripheral edema, shortness of breath or signs of pulmonary congestion on x-ray or auscultation) that either requires/required treatment with diuretics or is/was associated with an N terminal prohormone of B-type natriuretic peptide (NT-proBNP) >400 ng/L or B-type natriuretic peptide (BNP) >100 ng/L.
    6. Have evidence of cardiac involvement by Screening/Baseline echocardiogram including an end-diastolic intraventricular septum thickness of =12 mm. For patients with an end-diastolic intraventricular septum thickness of <12mm, an endomyocardial biopsy showing amyloid deposition is required.
    7. Can walk =150 meters on a 6-minute walk test (6-MWT).
    8. Have a Karnofsky performance status of =50%.
    9. Symptoms of HF optimally managed with no CV hospitalizations within 4 weeks prior to consent or during Screening/Baseline.
    10. Have adequate liver function, demonstrated by an AST and ALT = 2.0 × ULN, albumin >3 g/dL (>4.35 µmol/L), and total bilirubin <2.0 mg/dL (34.2 µmol/L), unless elevation in total bilirubin is due to Gilbert’s syndrome.
    11. No active infection with hepatitis B (HBV) or hepatitis C (HCV) by serology.
    12. Women of child-bearing potential must have a negative pregnancy test, cannot be breastfeeding, and use 1 highly effective methods of contraception in combination with a barrier method throughout study participation, and for 28 days after last dose or study drug..
    13. Males with partners of child-bearing potential, must agree to use a condom, accompanied with spermicidal foam, gel, film, cream, or suppository, except in countries where spermicide is not available for use in combination with condom, throughout study participation and for 28 days after the last dose of study drug; males must also abstain from sperm donation after the first dose of study drug through study participation and for 28 days after the last dose of study drug.
    14. Must be willing and able to comply with protocol-required visit schedule and visit requirements and provide informed consent or have a legal guardian who can provide informed consent.
    1. Uomini e donne di età compresa tra 18 e 90 anni (estremi inclusi).
    2. Mutazione TTR documentata.
    3. I depositi di amiloide nel tessuto cardiaco o non cardiaco, confermati mediante colorazione con rosso Congo (o equivalente) oppure scintigrafia con tecnezio (99mTc) (99mTc-3,3-difosfono-1,2-acido propanodicarbossilico [DPD-Tc] o 99mTc-pirofosfato [PYP-Tc]) con captazione cardiaca di grado 2 o 3, confermata a livello centrale.
    4. Se il paziente soffre di gammopatia monoclonale, l’amiloidosi TTR va confermata attraverso l'identificazione della proteina TTR mediante un esame immunoistochimico o spettrometria di massa.
    5. Anamnesi di insufficienza cardiaca (HF) con almeno 1 ricovero precedente per HF, che può comprendere ricovero per aritmia o impianto di pacemaker O evidenza clinica di HF (dimostrata da uno o più dei segni seguenti: pressione venosa giugulare elevata, edema periferico, dispnea o segni di congestione polmonare all'esame radiografico o all'auscultazione) che richiede/ha richiesto trattamento con diuretici o è/era associata a un pro-ormone N-terminale del peptide natriuretico di tipo B (NT-proBNP) >400 ng/l o del peptide natriuretico di tipo B (BNP) >100 ng/l.
    6. Presenza dimostrata di coinvolgimento cardiaco tramite ecocardiogramma alla visita di screening/basale, compreso uno spessore telediastolico del setto intraventricolare =12 mm. Per i pazienti con uno spessore telediastolico del setto intraventricolare <12 mm è richiesta una biopsia endomiocardica che mostri i depositi amiloidi.
    7. Capacità di camminare =150 metri durante il test della distanza percorsa a piedi in 6 minuti (6MWD).
    8. Stato prestazionale di Karnofsky =50%.
    9. Sintomi di HF gestiti in modo ottimale senza ricoveri CV nelle 4 settimane antecedenti il consenso o durante la visita di screening/basale.
    10. Avere una funzionalità epatica adeguata, dimostrata da AST e ALT =2,0 x ULN, albumina >3 g/dl (> 4,35 µmol/l) e bilirubina totale <2,0 mg/dl (34,2 µmol/l), a meno che l'elevazione della bilirubina totale sia dovuta alla sindrome di Gilbert.
    11. Nessuna infezione attiva da epatite B (HBV) o epatite C (HCV) in base agli esami sierologici.
    12. Le donne in età fertile devono sottoporsi a un test di gravidanza con esito negativo, non possono allattare e devono utilizzare un metodo di contraccezione assolutamente efficace in combinazione con un metodo a barriera durante la partecipazione allo studio e per 28 giorni dopo l'ultima dose del farmaco in studio.
    13.I soggetti di sesso maschile con partner di età fertile devono accettare l’uso di un preservativo, accompagnato da spermicida sotto forma di schiuma, gel, film, crema o supposta, tranne nei paesi in cui lo spermicida non è disponibile per l'uso in combinazione con il preservativo, durante la partecipazione allo studio e per 28 giorni dopo l'ultima dose del farmaco in studio; i soggetti di sesso maschile devono astenersi inoltre dalla donazione di sperma dopo la prima dose del farmaco in studio per tutta la durata della partecipazione allo studio e per 28 giorni dopo l'ultima dose del farmaco in studio.
    14. Volontà e capacità di rispettare il programma di visite richiesto dal protocollo e i requisiti delle visite e fornire il consenso informato o avere un tutore legale in grado di fornire il consenso informato.
    E.4Principal exclusion criteria
    1. Has estimated Glomerular Filtration Rate (eGFR) <30 mL/min/1.73m2 (using the Modification of Diet in Renal Disease [MDRD] formula)
    2. Has known primary amyloidosis (AL), leptomeningeal amyloidosis, non-FAC hereditary cardiomyopathy, hypertensive cardiomyopathy, or cardiomyopathy due to valvular heart disease
    3. Has non -amyloid diseases affecting exercise testing (e.g. severe chronic obstructive lung disease, severe arthritis, peripheral vascular disease affecting ambulation).
    4. Has uncontrolled hypertension
    5. Has uncontrolled ischemic heart disease
    6. Has uncontrolled clinically significant cardiac arrhythmia
    7. Had acute coronary syndrome within the past 3 months
    8. Has a Polyneuropathy Disability score >2
    9. Has untreated hypo- or hyperthyroidism
    10. Has a New York Heart Association (NYHA) classification of IV
    11. Has known or suspected systemic bacterial, viral, parasitic, or fungal infection
    12. Has known human immunodeficiency virus (HIV) infection
    13. Current, heavy alcohol use, defined as regular consumption of greater than 2 to 3 units/day for women and 3 to 4 units/day for men (a unit of alcohol equals 1 glass of wine [125 mL], 1 measure of spirits, or 1/2 pint od beer), or a known history of alcohol abuse within the past 2 years.
    14. Has received an investigational agent or device within 30 days of anticipated study drug administration or 5 half-lives of the investigational drug, whichever is longer
    15. Is currently taking diflunisal, tafamidis, doxycycline, or tauroursodeoxycholic acid; if previously on any of these agents, must have completed a 14-day wash-out prior to start of study drug administration in this study
    16. Had metastatic cancer within the past 5 years
    17. History of allergic reaction to an oligonucleotide or N-acetylgalactosamine (GalNAc).
    18. Has a history of intolerance to SC injection
    19. Has other medical conditions or comorbidities which, in the opinion of the Investigator, would interfere with study compliance or data interpretation.
    20. Has had a heart or liver transplant, or is being considered for a transplant during the study period.
    21. Known history of clinically significant chronic liver disease in the opinion of the Investigator.
    1. Velocità di filtrazione glomerulare stimata (eGFR) <30 ml/min/1,73 m2 (utilizzando la formula di modifica della dieta nelle malattie renali [MDRD])
    2. Presenza nota di amiloidosi (AL) primaria, amiloidosi leptomeningea, cardiomiopatia ereditaria non FAC, cardiomiopatia ipertensiva o cardiomiopatia dovuta a cardiopatia valvolare
    3. malattie diverse dall’amiloidosi che incidono sui test da sforzo (ad es., malattia polmonare ostruttiva cronica grave, artrite grave, vasculopatia periferica che limita la deambulazione).
    4. Ipertensione non controllata
    5. Cardiopatia ischemica non controllata
    6. Aritmia cardiaca clinicamente significativa non controllata
    7. Sindrome coronarica acuta entro gli ultimi 3 mesi
    8. Punteggio della disabilità da polineuropatia >2
    9. Ipo o ipertiroidismo non trattato
    10. Livello IV nella classificazione NYHA (New York Heart Association)
    11. Infezione batterica sistemica, virale, parassitica o fungina nota o sospetta
    12. Infezione da virus dell’immunodeficienza umana (HIV) nota
    13. Consumo di alcol attuale eccessivo, definito come l’assunzione regolare di più di 2 o 3 unità/giorno per le donne e di 3 o 4 unità/giorno per gli uomini (una unità di alcol equivale a 1 bicchiere di vino [125 ml], 1 dose di superalcolici, o ½ litro di birra), o anamnesi positiva per abuso di alcol negli ultimi 2 anni.
    14. Somministrazione di agente o dispositivo sperimentale entro 30 giorni dalla somministrazione prevista del farmaco in studio o 5 emivite del farmaco sperimentale, a seconda di quale periodo sia maggiore
    15. Assunzione in corso di diflunisal, tafamidis, doxiciclina o acido tauroursodesossicolico; in caso di assunzione precedente di uno di questi agenti, deve avere completato un periodo di smaltimento di 14 giorni prima dell'inizio della somministrazione del farmaco in studio
    16. Presenza di cancro metastatico entro gli ultimi 5 anni
    17. Anamnesi di reazioni allergiche a un oligonucleotide o alla N-acetilgalattosamina (GalNAc)
    18. Anamnesi di intolleranza alle iniezioni SC
    19. Altre patologie o comorbilità che, secondo l'opinione dello sperimentatore, potrebbero interferire con la conformità allo studio o con l'interpretazione dei dati.
    20. Precedente trapianto di cuore o fegato, oppure attualmente oggetto di valutazione per un possibile trapianto durante il periodo dello studio.
    21. Anamnesi positiva per malattia epatica cronica clinicamente significativa, a giudizio dello sperimentatore.
    E.5 End points
    E.5.1Primary end point(s)
    The co-primary endpoints of the study are to evaluate the difference
    between the ALN-TTRSC and placebo groups for:
    1. Change in 6-MWD at 18 months compared to baseline
    2. Percent reduction in serum TTR burden over 18 months
    Gli endpoint co-primari dello studio mirano a valutare la differenza tra il gruppo con ALN-TTRSC e il gruppo con placebo per:
    1. Variazione in 6MWD a 18 mesi rispetto al basale
    2. Riduzione percentuale dell'impatto della TTR nel siero su 18 mesi
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. At 18 months
    2. Over 18 months
    1. A 18 mesi
    2. Su 18 mesi
    E.5.2Secondary end point(s)
    The secondary endpoints of the study are to determine the effect of ALNTTRSC
    on various clinical parameters by assessing the difference
    between the ALN-TTRSC and placebo groups at 18 months for:
    • Composite CV mortality and CV hospitalization
    • Change in New York Heart Association (NYHA) class compared to
    baseline
    • Change in Kansas City Cardiomyopathy Questionnaire (KCCQ)
    compared to baseline
    • Cardiovascular (CV) mortality
    • CV hospitalization
    • All-cause mortality
    Gli endpoint secondari dello studio mirano a determinare l'effetto di ALN-TTRSC su vari parametri clinici valutando la differenza tra il gruppo con ALN-TTRSC e il gruppo con placebo a 18 mesi per:
    • Mortalità CV composita e ricovero CV
    • Cambiamento della classe NYHA (New York Heart Association) rispetto al basale
    • Variazione nel questionario Kansas City Cardiomyopathy Questionnaire (KCCQ) rispetto al basale
    • Mortalità cardiovascolare (CV)
    • Ricovero CV
    • Mortalità generale
    ; Gli endpoint secondari dello studio mirano a determinare l'effetto di ALN-TTRSC su vari parametri clinici valutando la differenza tra il gruppo con ALN-TTRSC e il gruppo con placebo a 18 mesi per:
    • Mortalità CV composita e ricovero CV
    • Cambiamento della classe NYHA (New York Heart Association) (Appendice 1) rispetto al basale
    • Variazione nel questionario Kansas City Cardiomyopathy Questionnaire (KCCQ) rispetto al basale
    • Mortalità cardiovascolare (CV)
    • Ricovero CV
    • Mortalità generale
    ; Gli endpoint secondari dello studio mirano a determinare l'effetto di ALN-TTRSC su vari parametri clinici valutando la differenza tra il gruppo con ALN-TTRSC e il gruppo con placebo a 18 mesi per:
    • Mortalità CV composita e ricovero CV
    • Cambiamento della classe NYHA (New York Heart Association) rispetto al basale
    • Variazione nel questionario Kansas City Cardiomyopathy Questionnaire (KCCQ) rispetto al basale
    • Mortalità cardiovascolare (CV)
    • Ricovero CV
    • Mortalità generale
    E.5.2.1Timepoint(s) of evaluation of this end point
    • At 18 months
    - A 18 mesi; - A 18 mesi; - A 18 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 Yes
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic Yes
    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 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
    Belgium
    Brazil
    Canada
    France
    Germany
    Italy
    Netherlands
    Spain
    Sweden
    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 years3
    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 years3
    E.8.9.2In all countries concerned by the trial months6
    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: 100
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 100
    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 80
    F.4.2.2In the whole clinical trial 200
    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)
    Eligible patients who complete study dosing and the 18 month efficacy assessment may be eligible to participate in an ALN-TTRSC open-label extension study
    I pazienti idonei che completano il dosaggio dello studio e le valutazioni dell'efficacia a 18 mesi possono essere idonei a partecipare a uno studio di estensione in aperto su ALN-TTRSC
    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 Decision2015-03-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-05-05
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-03-31
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    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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