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    Summary
    EudraCT Number:2012-002465-35
    Sponsor's Protocol Code Number:B3461028
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-12-27
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2012-002465-35
    A.3Full title of the trial
    A multicentre, international, phase 3, double-blind, placebo-controlled, randomized study to evaluate the efficacy, safety and tolerability of daily oral dosing of tafamidis meglumine (PF-06291826) 20 mg or 80 mg in comparison to placebo in subjects diagnosed with transthyretin cardiomyopathy (TTR-CM).
    Estudio de fase III, multicéntrico, internacional, aleatorizado, doble ciego, controlado con placebo para evaluar la eficacia, seguridad y tolerabilidad de la administración diaria por vía oral de 20 mg u 80 mg de tafamidis meglumina (PF-06291826) en comparación con placebo en pacientes con un diagnóstico de miocardiopatía por transtirretina (MC-TTR)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A double-blind, randomized study looking at the efficacy, safety and tolerability of tafamidis meglumine (PF-06291826) 20 mg or 80 mg compared to placebo when taken daily by oral administration mouth in subjects diagnosed with transthyretin cardiomyopathy (TTR-CM).
    Estudio aleatorizado, doble ciego, controlado con placebo para evaluar la eficacia, seguridad y tolerabilidad de la administración diaria por vía oral de 20 mg u 80 mg de tafamidis meglumina (PF-06291826) en comparación con placebo en pacientes con un diagnóstico de miocardiopatía por transtirretina (MC-TTR)
    A.4.1Sponsor's protocol code numberB3461028
    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 SponsorFoldRx Pharmaceuticals, a Pfizer Company
    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 supportPfizer Inc, 235 42nd Street, New York, NY 10017
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPfizer Inc
    B.5.2Functional name of contact pointClinicaltrials.gov Call Center
    B.5.3 Address:
    B.5.3.1Street Address235 East 42nd Street
    B.5.3.2Town/ cityNew York
    B.5.3.3Post codeNY 10017
    B.5.3.4CountryUnited States
    B.5.4Telephone number0034914909900
    B.5.5Fax number0013037391119
    B.5.6E-mailclinicaltrials.govcallcenter@pfizer.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 Vyndaqel 20 mg soft capsule
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEMA/OD/032/06 & EMA/OD/115/12
    D.3 Description of the IMP
    D.3.1Product nameTafamidis meglumine
    D.3.2Product code PF-06291826-83, Fx-1006A
    D.3.4Pharmaceutical form Capsule, soft
    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 INNTafamidis
    D.3.9.1CAS number 951395-08-7
    D.3.9.2Current sponsor codePF-06291826, Fx-1006A
    D.3.9.3Other descriptive nameTafamidis meglumine 20mg soft gelatin capsules
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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 No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    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 placeboCapsule, soft
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Transthyretin amyloid cardiomyopathy (TTR-CM)
    Miocardiopatía por transtirretina (MC-TTR)
    E.1.1.1Medical condition in easily understood language
    A condition characterized by the buildup of abnormal deposits of a protein called amyloid in the body's organs and tissues that include the heart, which can result in worsening of its function
    Una enfermedad que se caracteriza por el depósito de una proteína denominada amiloide en varios órganos y tejidos incluido el corazón, que puede hacer empeorar su funcionamiento
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.1
    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
    The primary objective of this study is to assess the efficacy of an oral dose of 20 mg or 80 mg tafamidis meglumine soft-gel capsules based on all-cause mortality and on frequency of cardiovascular-related hospitalizations as well as to assess safety and tolerability in comparison to placebo. Tafamidis or placebo will be administered once daily, in addition to standard of care, for 30 months in subjects diagnosed with variant or wild-type TTR cardiomyopathy (TTR-CM).
    El objetivo principal es evaluar la eficacia de una dosis por vía oral de 20 mg u 80 mg de tafamidis meglumina, en forma de cápsulas de gelatina blandas, en base a la mortalidad general y a la frecuencia de hospitalizaciones relacionadas con eventos cardiovasculares, así como evaluar la seguridad y la tolerabilidad en comparación con placebo. Tafamidis o placebo se administrarán una vez al día, junto con el tratamiento habitual, durante 30 meses en pacientes diagnosticados con una variante de la TTR o la MC-TTR en su forma nativa.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects must meet all of the following inclusion criteria to be eligible for enrollment into the study:
    1. Evidence of a personally signed and dated Informed Consent Document indicating that the subject (or a legal representative) has been informed of all pertinent aspects of the study and evidence of a personally signed and dated
    separate Medical Release Form regarding access to medical records as well as vital status follow-up by the site with the subject or the subject?s caregivers 30 months after study randomization.

    2. Subjects who are willing and able to comply with scheduled visits, treatment plan,
    laboratory tests, and other study procedures,

    3. Age greater than or equal to 18 and less than or equal to 90 years old at the time
    of randomization,

    4. Medical history of Heart Failure (HF) with at least 1 prior hospitalization for HF or clinical evidence of HF (without hospitalization) manifested by signs or symptoms of volume overload or elevated intracardiac pressures (e.g., elevated jugular venous pressure, shortness of breath or signs of pulmonary congestion on x-ray or auscultation, peripheral edema) that required/requires treatment with a
    diuretic for improvement,

    5. Subject has documented TTR amyloid cardiomyopathy in accordance with institutional site standard of care, which is defined as:

    a. Variant TTR amyloid cardiomyopathy defined by all of the following:

    i. presence of a variant TTR genotype associated with cardiomyopathy and presenting with a cardiomyopathy phenotype (e.g., a history of congestive heart failure),

    ii. evidence of cardiac involvement by echocardiography with an enddiastolic interventricular septal wall thickness > 12 mm,

    iii. presence of amyloid in biopsy tissue, such as fat aspirate, salivary gland, median nerve connective tissue sheath, or cardiac (amyloid
    demonstrated per appropriate stain such as Congo red or alcin blue stain).

    b. Wild-type TTR amyloid cardiomyopathy defined by all of the following:

    i. absence of a variant TTR genotype,

    ii. evidence of cardiac involvement by echocardiography with an enddiastolic
    interventricular septal wall thickness > 12 mm,

    iii. presence of amyloid deposits in biopsy tissue, such as fat aspirate, salivary gland, median nerve connective tissue sheath, or cardiac (amyloid demonstrated per appropriate stain such as Congo red or alcin blue stain),

    iv. TTR precursor protein identification by mass spectrometry.

    6. Biopsy to determine the presence of amyloid and demonstration of TTR precursor
    protein must be done during Screening or documented as having been performed
    within 5 years prior to enrollment,

    7. Subject must be able to read in native language and complete self-administered
    questionnaires independently,

    8. Subject?s symptoms of HF are optimally managed and clinically stable with no
    cardiovascular-related hospitalizations within 2 weeks prior to Baseline, as assessed by the Principal Investigator,

    9. Male and female subjects of childbearing potential must agree to use two highly effective methods of contraception throughout the study and for at least 28 days after the last dose of assigned treatment. A subject is of childbearing potential if, in the opinion of the investigator, he/she is biologically capable of having children and is sexually active,

    10. Subject must have a Screening visit NT-proBNP concentration ? 600 pg/mL,

    11. Subjects must be able to complete > 100 m on the 6-Minute Walk Test.
    Los pacientes deben cumplir todos los criterios de inclusión siguientes para ser elegibles para participar en el estudio:
    1. Evidencia de un documento de consentimiento informado, firmado y fechado personalmente, que indique que el paciente (o un representante legal) ha sido informado de todos los aspectos pertinentes del estudio, y evidencia de un formulario de divulgación médica aparte, firmado y fechado personalmente, relativo al acceso al expediente médico así como al seguimiento del estado vital por parte del centro con el paciente o el cuidador del paciente, 30 meses después de la aleatorización del estudio.
    2. Los pacientes deben estar dispuestos a y ser capaces de cumplir con las visitas programadas, el plan de tratamiento, las pruebas de laboratorio y otros procedimientos del estudio,
    3. Edad mayor o igual a 18 y menor o igual a 90 años en el momento de la aleatorización,
    4. Antecedentes médicos de insuficiencia cardíaca (IC) con al menos 1 hospitalización previa debida a la IC o un cuadro clínico de IC (sin hospitalización) puesta de manifiesto por los signos o síntomas de sobrecarga de volumen o presiones intracardíacas elevadas (p. ej., presión venosa yugular elevada, falta de aire o signos de edema pulmonar en la radiografía o la auscultación, edema periférico) que han requerido/requieren un tratamiento con un diurético para mejorar,
    5. El paciente tiene miocardiopatía amiloide por TTR documentada de acuerdo con la práctica habitual del centro institucional, la cual se define como:
    a. Miocardiopatía amiloide por variante de la TTR definida por todo lo siguiente:
    i. presencia de un genotipo variante de la TTR asociado con miocardiopatía y que presenta un fenotipo de miocardiopatía (p. ej., antecedentes de insuficiencia cardíaca congestiva),
    ii. evidencia de afectación cardíaca mediante ecocardiografía con un grosor del septo interventricular de fin de diástole de > 12 mm,
    iii. presencia de amiloide en la biopsia de tejido como, por ejemplo, aspirado de grasa, glándula salival, vaina de tejido conectivo del nervio mediano, o tejido cardíaco (amiloide demostrado mediante una prueba de tinción adecuada como rojo Congo o azul alcián).
    b. Miocardiopatía amiloide por TTR en su forma nativa definida por todo lo siguiente:
    i. ausencia de un genotipo variante de la TTR,
    ii. evidencia de afectación cardíaca mediante ecocardiografía con un grosor del septo interventricular de fin de diástole de > 12 mm,
    iii. presencia de depósitos de amiloide en la biopsia de tejido como, por ejemplo, aspirado de grasa, glándula salival, vaina de tejido conectivo del nervio mediano, o tejido cardíaco (amiloide demostrado mediante una prueba de tinción adecuada como rojo Congo o azul alcián),
    iv. identificación de la proteína precursora TTR mediante espectrometría de masas.
    6. La biopsia para determinar la presencia de amiloide y la demostración de la proteína precursora TTR deben hacerse durante la selección o estar documentadas como que se han hecho en los 5 años anteriores a la inscripción,
    7. El paciente debe ser capaz de leer en el idioma materno y cumplimentar los cuestionarios de auto-administración de manera independiente,
    8. Los síntomas de IC del paciente están controlados de manera óptima y permanecen clínicamente estables sin hospitalizaciones relacionadas con eventos cardíacos en las 2 semanas anteriores al momento basal, según la evaluación del investigador principal,
    9. Los pacientes de sexo masculino y femenino en edad fértil deben acceder a utilizar dos métodos anticonceptivos de eficacia elevada durante todo el estudio y durante, al menos, 28 días después de la última dosis del tratamiento asignado. Se considera que un paciente está en edad fértil si, a juicio del investigador, está capacitado biológicamente para tener hijos y es sexualmente activo,
    10. El paciente debe tener una concentración de NT-proBNP en la visita de selección de > 600 pg/ml,
    11. Los pacientes deben ser capaces de completar > 100 m en la prueba de marcha de 6 minutos.
    E.4Principal exclusion criteria
    Subjects presenting with any of the following will not be included in the study:

    1. Subjects with echocardiogram assessment at Screening that is not deemed interpretable by the central echocardiogram reader for the measurement of wall thickness,

    2. Subjects using non-steroidal anti-inflammatory drugs (NSAIDs) that are not
    allowable in the protocol within 2 weeks prior to randomization

    3. Subjects with an mBMI below 600,

    4. Subjects with a history of drug or alcohol abuse within the past 5 years that in the
    opinion of the investigator would interfere with compliance with study procedures or follow-up visits,

    5. Subjects taking or have previously taken tafamidis,

    6. Subjects requiring treatment with calcium channel blockers or digitalis,

    7. Subjects with primary (light chain) or secondary (serum amyloid A) amyloidosis,

    8. Subjects who have prior liver or heart transplantation,

    9. Subject has positive serology for hepatitis B (HBsAg), hepatitis C (anti-HCV), or HIV,

    10. Subjects with renal failure requiring dialysis and/or have a creatinine clearance of
    < 25 mL/min.,

    11. Subjects with urinary retention requiring self-catheterization,

    12. Subjects who are investigational site staff members directly involved in the conduct of the trial and their family members, site staff members otherwise supervised by the Investigator, or subjects who are Pfizer employees directly involved in the conduct of the trial.

    13. Subjects who have symptoms indicative of New York Heart Association Classification IV at the Screening or Baseline visit,

    14. Subjects with liver function test abnormalities (alanine transaminase and/or
    aspartate transaminase) greater than 2 times the upper limit of normal that are considered to be due to reduced liver function or active liver disease,

    15. Subjects with participation in studies involving investigational drug(s) (Phases 1-
    4) within 60 days before the current study begins and/or during study participation. For diflunisal, this time period will be at least 2 weeks prior to enrollment and/or any time during study participation,

    16. Subjects with other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would
    make the subject inappropriate for entry into this study,

    17. Subjects who are pregnant females; breastfeeding females; males and females of
    childbearing potential; males and females of childbearing potential who are unwilling or unable to use two (2) highly effective method of contraception as outlined in this protocol for the duration of the study and for at least 28 days, after last dose of investigational product;

    18. Subjects with a history of sustained ventricular tachycardia or aborted ventricular
    fibrillation or with a history of atrioventricular (AV) nodal or sinoatrial (SA) nodal dysfunction for which a pacemaker is indicated but will not be placed,

    19. Subjects with evidence for prior myocardial infarction by documented history of cardiac enzymes and ECG changes.
    Los pacientes que presenten cualquiera de las siguientes características no serán incluidos en el estudio:
    1. Pacientes con una evaluación de ecocardiograma en la selección, la cual es considerada por el lector central de ecocardiogramas como no interpretable para la medición del grosor de la pared,
    2. Pacientes que utilizan antiinflamatorios no esteroideos (AINE) que no están permitidos en el protocolo en las 2 semanas previas a la aleatorización (ver sección 5.5.1),
    3. Pacientes con un IMC modificado por debajo de 600,
    4. Pacientes con antecedentes de alcoholismo o drogadicción en los últimos 5 años que, en opinión del investigador, podrían interferir en el cumplimiento de los procedimientos del estudio o en las visitas de seguimiento,
    5. Pacientes que estén tomando o hayan tomado previamente tafamidis,
    6. Pacientes que requieran un tratamiento con bloqueadores de los canales de calcio o digitalis,
    7. Pacientes con amiloidosis primaria (de cadena ligera) o secundaria (amiloide A sérico),
    8. Pacientes que tienen un trasplante previo de hígado o corazón,
    9. Pacientes con serología positiva para la hepatitis B (HBsAg), hepatitis C (anti-VHC), o VIH,
    10. Pacientes con insuficiencia renal que requieren diálisis y/o con un aclaramiento de creatinina de < 25 ml/min.,
    11. Pacientes con retención urinaria que requieren un autosondaje,
    12. Pacientes que sean miembros del personal del centro de investigación directamente implicados en la realización del ensayo, así como sus familiares, miembros del personal del centro supervisados de alguna otra manera por el investigador o empleados de Pfizer directamente implicados en la realización del ensayo.
    13. Pacientes que presentan síntomas indicativos de una clase IV en la clasificación de la New York Heart Association en la visita de selección o basal,
    14. Pacientes con anomalías en las pruebas de función hepática (alanina transaminasa y/o aspartato transaminasa) más de 2 veces el límite superior de la normalidad, que se consideran debidas a una función hepática reducida o una enfermedad hepática activa,
    15. Pacientes con participación en otros estudios que incluyan fármaco/s en investigación (fases I-IV) en los 60 días anteriores al inicio del estudio actual y/o durante la participación en el estudio. En el caso de diflunisal, este período de tiempo será de, al menos, 2 semanas antes de la inclusión y/o en cualquier momento durante la participación en el estudio,
    16. Pacientes con otras afecciones de grado intenso, agudo o crónico, médicas o psiquiátricas, o anomalías en los resultados de laboratorio que podrían aumentar el riesgo asociado con la participación en el estudio o con la administración del producto experimental, o que puedan interferir en la interpretación de los resultados del estudio y, a juicio del investigador, hagan que el paciente no sea apto para incorporarse a este estudio,
    17. Mujeres embarazadas; mujeres en período de lactancia; hombres y mujeres en edad fértil; hombres y mujeres en edad fértil que no desean o no pueden usar dos (2) métodos anticonceptivos de elevada eficacia tal como se describe en este protocolo durante todo el estudio y durante, al menos, 28 días después de la última dosis del producto en investigación;
    18. Pacientes con antecedentes de taquicardia ventricular sostenida o fibrilación ventricular recuperada, o con antecedentes de disfunción del nodo auriculoventricular (AV) o del nódulo sinusal (SA) para las que está indicado un marcapasos pero no está colocado,
    19. Pacientes con evidencias de infarto de miocardio previo mediante una historia documentada de enzimas cardíacas y cambios en el ECG.
    E.5 End points
    E.5.1Primary end point(s)
    1. All-cause mortality and

    2. Frequency of cardiovascular-related hospitalizations over the duration of the trial, which is defined as the number of times a subject is hospitalized (i.e., admitted to a hospital) for cardiovascular-related morbidity.
    1. Mortalidad general
    2. Frecuencia de hospitalizaciones relacionadas con eventos cardiovasculares, la cual se define como el número de veces que un paciente es hospitalizado (es decir, ingresado en el hospital) debido a una morbilidad relacionada con eventos cardiovasculares
    E.5.1.1Timepoint(s) of evaluation of this end point
    Both primary end points will be assessed throughout the study.
    Se analizarán los dos criterios de valoración principales a lo largo del estudio.
    E.5.2Secondary end point(s)
    Key Secondary Endpoints
    1. Change from Baseline to Month 30 in the distance walked during 6-Minute Walk Test (6MWT),

    2. Change from Baseline to Month 30 in the Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS).

    Other secondary endpoints
    1. Cardiovascular-related mortality,

    2. Frequency of cardiovascular-related hospitalization,

    3. All-cause mortality,

    4. TTR stabilization at Month 1.
    Criterios de valoración secundarios clave
    1. Cambio entre el momento basal y el Mes 30 de la distancia recorrida durante la prueba de marcha de 6 minutos (PM6M),
    2. Cambio entre el momento basal y el Mes 30 de la puntuación del sumario global del Cuestionario de miocardiopatía de la ciudad de Kansas (Kansas City Cardiomyopathy Questionnaire Overall Summary, KCCQ-OS)

    Criterios de valoración secundarios:
    1. Mortalidad relacionada con eventos cardiovasculares,
    2. Frecuencia de hospitalizaciones relacionadas con eventos cardiovasculares,
    3. Mortalidad general,
    4. Estabilización de la TTR en el Mes 1.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Distance walked during 6 Minute Walk Test (6MWT) at baseline (Day 1) and Months 6, 12, 18, 24 and 30 or end of treatment.

    2. Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS) at baseline (Day 1) and Months 6, 12, 18 and 30 or end of treatment.
    1. Distancia recorrida durante la prueba de marcha de 6 minutos (PM6M), en el momento basal (día 1) y los meses 6, 12, 18, 24 y 30 o fin de tratamiento
    2. Cuestionario de miocardiopatía de la ciudad de Kansas (Kansas City Cardiomyopathy Questionnaire Overall Summary, KCCQ-OS) en el momento basal (día 1) y los meses 6, 12, 18, 24 y 30 o fin de tratamiento
    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 Yes
    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 trial3
    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 EEA21
    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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Canada
    France
    Germany
    Italy
    Japan
    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
    Last subject last visit.
    Última visita del último paciente
    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 months9
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months9
    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: 40
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 360
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 132
    F.4.2.2In the whole clinical trial 400
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Upon completion of the study at Month 30 visit, subjects will be eligible for open-label treatment with tafamidis in a separate study.
    Tras finalizar la participación en el estudio en la visita del mes 30, los pacientes podrán participar en otro estudio abierto con tafamidis
    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 Decision2014-02-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-12-23
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-02-07
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
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