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    Summary
    EudraCT Number:2016-000868-42
    Sponsor's Protocol Code Number:B3461045
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2016-07-15
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2016-000868-42
    A.3Full title of the trial
    A PHASE 3 MULTICENTER, RANDOMIZED, DOUBLE-BLIND, EXTENSION STUDY TO EVALUATE THE SAFETY OF DAILY ORAL DOSING OF TAFAMIDIS MEGLUMINE (PF-06291826) 20 MG OR 80 MG IN SUBJECTS DIAGNOSED WITH TRANSTHYRETIN CARDIOMYOPATHY (TTR-CM)
    ESTUDIO DE EXTENSIÓN DE FASE 3, MULTICÉNTRICO, ALEATORIZADO Y DOBLE CIEGO PARA EVALUAR LA SEGURIDAD DE LA ADMINISTRACIÓN DIARIA POR VÍA ORAL DE 20 MG U 80 MG DE TAFAMIDIS MEGLUMINA (PF 06291826) EN PACIENTES CON UN DIAGNÓSTICO DE MIOCARDIOPATÍA POR TRANSTIRETINA (MC TTR)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 3, double-blind randomized, long-term extension safety study designed to obtain additional safety data for 20 mg and 80 mg tafamidis and to continue to provide subjects who have completed 30 months of blinded treatment on Protocol B3461028 with tafamidis for up to 60 additional months, or until subject has access to tafamidis for TTR-CM via prescription, whichever occurs first.
    Estudio de seguridad de extensión a largo plazo de fase 3, doble ciego, aleatorizado diseñado para obtener datos adicionales de seguridad para tafamidis 20 mg y 80 mg y para continuar proporcionando a los sujetos que han completado 30 meses de tratamiento enmascarado en el protocolo B3461028 con tafamidis hasta un máximo de 60 meses más, o hasta que el sujeto tenga acceso a tafamidis para MC TTR mediante prescripción, lo que ocurra antes.
    A.4.1Sponsor's protocol code numberB3461045
    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 SponsorPfizer 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 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 number+3491 490 99 00
    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 numberEU/3/06/401
    D.3 Description of the IMP
    D.3.1Product nameTafamidis meglumine
    D.3.2Product code PF-06291826-83
    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 meglumine
    D.3.9.1CAS number 951395-08-7
    D.3.9.2Current sponsor codePF-06291826
    D.3.9.3Other descriptive nameTAFAMIDIS MEGLUMINE
    D.3.9.4EV Substance CodeSUB31457
    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 amiloidea por transtiretina (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 caracterizada por la acumulación de depósitos anormales de una proteína llamada amiloide en órganos y tejidos inluido el corazón, que puede resultar en un empeoramiento de su función.
    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 19.0
    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 obtain additional, long-term, safety data for tafamidis in subjects with transthyretin amyloid cardiomyopathy (TTR-CM).
    - To provide investigational product, tafamidis, to TTR-CM subjects who complete 30 months of blinded treatment on protocol B3461028.
    - Obtener más datos de seguridad a largo plazo de tafamidis en sujetos con miocardiopatía amiloidea por transtiretina (MC TTR).
    - Proporcionar el producto en investigación, tafamidis, a sujetos con MC TTR que completen 30 meses de tratamiento enmascarado en el protocolo B3461028.
    E.2.2Secondary objectives of the trial
    Not Applicable
    No Aplica.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male and female subjects with TTR amyloid cardiomyopathy who have completed 30 months of study treatment on Protocol B3461028.
    2. Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.
    3. Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
    4. Male subjects able to father children and female subjects of childbearing potential and at risk for pregnancy must agree to use 2 highly effective methods of contraception throughout the study and for at least 28 days after the last dose of assigned treatment.
    Female subjects who are not of childbearing potential (ie, meet at least 1 of the following criteria):
    - Have undergone a documented hysterectomy and/or bilateral oophorectomy;
    - Have medically confirmed ovarian failure; or
    - Achieved postmenopausal status, defined as follows: cessation of regular menses for at least 12 consecutive months with no alternative pathological or physiological cause; status may be confirmed by having a serum folliclestimulating hormone (FSH) level confirming the post-menopausal state.
    All other female subjects (including females with tubal ligations) will be considered o be of childbearing potential.
    1. Sujetos de uno u otro sexo con miocardiopatía amiloidea por TTR que hayan completado 30 meses de tratamiento del estudio en el protocolo B3461028.
    2. Existencia de un documento de consentimiento informado, firmado y fechado personalmente, que indique que se ha informado al sujeto de todos los aspectos pertinentes del estudio.
    3. Disposición del sujeto a cumplir las visitas programadas, el plan de tratamiento, las pruebas analíticas y otros procedimientos del estudio y capacidad de hacerlo.
    4. Los varones con capacidad de engendrar hijos y las mujeres en edad fértil y con riesgo de embarazo deberán comprometerse a utilizar dos métodos anticonceptivos muy eficaces durante todo el estudio y hasta, como mínimo, 28 días después de la última dosis del tratamiento asignado.
    Se considerará que no están en edad fértil a las mujeres que cumplan al menos uno de los criterios siguientes:
    • Se han sometido a una histerectomía u ovariectomía bilateral documentada.
    • Presentan insuficiencia ovárica confirmada médicamente.
    • Han llegado al estado posmenopáusico, definido de la manera siguiente: cese de la menstruación regular durante al menos 12 meses consecutivos sin otra causa patológica o fisiológica; dicho estado puede confirmarse con una determinación de la concentración sérica de folitropina (FSH) que confirme el estado posmenopáusico.
    Se considerará que todas las demás mujeres (incluso las que se hayan sometido a una ligadura de trompas) están en edad fértil.
    E.4Principal exclusion criteria
    1. Chronic use of diflunisal, TTR stabilizer, tauroursodeoxycholate, doxycycline, digitalis, calcium channel blockers, investigational drug(s) or other experimental interventions, other than tafamidis, independently or as part of a study within 30 months prior to enrollment.
    2. Use of certain non-steroidal anti-inflammatory drugs (NSAIDs)
    3. Liver and/or heart transplant, or implanted cardiac mechanical assist device.
    4. Pregnant females (or planning to become pregnant during the study interval); breastfeeding females; male subjects with partners currently pregnant.
    5. Require initiation of treatment with calcium channel blockers.
    6. Urinary retention requiring chronic self-catheterization.
    7. Breach of compliance with treatment/significant protocol violations during conduct of B3461028 for which the subject was accountable.
    8. Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or subjects who are Pfizer employees directly involved in the conduct of he study.
    9. Other severe acute or chronic medical or psychiatric condition or laboratory bnormality that may increase the risk associated with study participation or investigational product administration, or that may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject an appropriate for entry into this study.
    1. Uso crónico de diflunisal, estabilizadores de TTR, tauroursodesoxicolato, doxiciclina, digitálicos, antagonistas del calcio, fármaco en investigación u otras intervenciones experimentales, diferente de tafamidis, independientemente o como parte de un estudio en los 30 meses previos al reclutamiento.
    2. Uso de determinados antiinflamatorios no esteroideos (AINE).
    3. Trasplante de hígado o corazón o implantación de un dispositivo de asistencia mecánica cardíaca.
    4. Mujeres embarazadas (o que tengan previsto quedarse embarazadas durante el intervalo del estudio) o lactantes y varones cuyas parejas estén embarazadas.
    5. Necesidad de iniciar un tratamiento con antagonistas del calcio.
    6. Retención urinaria con necesidad de autosondaje a largo plazo.
    7. Incumplimiento del tratamiento o transgresiones importantes del protocolo durante la realización del estudio B3461028 de las que fue responsable el sujeto.
    8. Sujetos que sean miembros del personal del centro de investigación con intervención directa en la realización del estudio y sus familiares, miembros del personal del centro supervisado por el investigador o empleados de Pfizer que intervengan directamente en la realización del estudio.
    9. Otro trastorno médico o psiquiátrico grave, agudo o crónico, o cualquier alteración analítica que pueda aumentar el riesgo asociado a la participación en el estudio o a la administración del producto en investigación o que pueda interferir en la interpretación de los resultados del estudio y que, en opinión del investigador, impida la participación del sujeto.
    E.5 End points
    E.5.1Primary end point(s)
    Safety as measured by:
     All-cause mortality.
     Incidence of treatment-emergent adverse events.
    Seguridad determinada mediante.
    • Mortalidad global.
    • Incidencia de acontecimientos adversos surgidos durante el tratamiento.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Both primary end points will be assessed throughout the study.
    Ambos criterios de valoración se valorarán a lo largo de todo el estudio.
    E.5.2Secondary end point(s)
    Cardiovascular-related mortality.

    Frequency of all-cause hospitalization.

    Frequency of cardiovascular-related hospitalization (including heart failure, arrhythmia, myocardial infarction, stroke and other cardiovascular-related events).

    Change from baseline at each visit in Kansas City Cardiomyopathy Questionnaire. Overall Score and domain scores (Physical limitation, Symptom stability, Symptoms, Self-efficacy, Social limitation, and Quality of life) and domain summary scores (Functional summary and Clinical summary).
    New York Heart Association classification at each visit.

    Change from baseline in Body Mass Index/modified Body Mass Index at each visit.

    Assessment of physical examinations, use of concomitant medications,
    electrocardiograms (ECGs), clinical laboratory testing, vital signs at each visit.
    - Mortalidad de origen cardiovascular.
    - Frecuencia de hospitalizaciones por cualquier causa.
    - Frecuencia de hospitalizaciones por motivos cardiovasculares (como insuficiencia cardíaca, arritmia, infarto de miocardio, ictus y otros episodios cardiovasculares).
    - Variación entre el momento basal y cada visita de la puntuación global en el Cuestionario de miocardiopatía de Kansas City, así como de las puntuaciones de dominios (limitación física, estabilidad de síntomas, síntomas, eficacia valorada por el propio sujeto, limitación social y calidad de vida) y las puntuaciones resumen de dominios (resumen funcional y resumen clínico).
    - Clasificación de la New York Heart Association en cada visita.
    - Variación entre el momento basal y cada visita del índice de masa corporal/índice de masa corporal modificado.
    - Evaluación de la exploración física, uso de medicamentos concomitantes, electrocardiogramas (ECG), pruebas analíticas y constantes vitales en cada visita.
    E.5.2.1Timepoint(s) of evaluation of this end point
    - 12-lead ECG: An annual basis (every 12 months) at clinic visits Months 12, 24, 36, 48 and 6at 0 month or early study termination
    - NYHA Classification: At day 0, evry 6 months, at 60 month or early study termination
    - KCCQ: At day 0, evry 6 months, at 60 month or early study termination
    - ECG de 12 derivaciones: anualmente (cada 12 meses) en las visitas de los meses 12, 24, 36, 48 y 6 en el mes 0 o en la finalización prematura del estudio.
    - Clasificacion NYHA: el día 0, cada 6 meses, en el mes 60 o en la finalización prematura del estudio.
    - KCCQ: El día 0, cada 6 meses, en el mes 60 o en la finalización prematura del estudio
    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 No
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA19
    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
    Belgium
    Brazil
    Canada
    Czech Republic
    France
    Germany
    Italy
    Japan
    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
    The time at which it is deemed that a sufficient number of subjects have been recruited and completed the study as stated in the regulatory application (ie, clinical trial application [CTA]) and ethics application in the Member State. Poor recruitment (recruiting less than the anticipated number in the CTA) by a Member State is not a reason for premature termination but is considered a normal conclusion to the study in that Member State.
    El momento en el que se considere que se ha reclutado un número suficiente de sujetos y se ha completado el estudio como se establece en la solicitud regulatoria (esto es, la solicitud de ensayo clínico) y la solicitud del comité en el Estado Miembro. Un bajo reclutamiento (reclutar menos del número previsto en la solicitud de ensayo clínico) por un Estado Miembro no es un motivo para la finalizacion prematura, pero se considera como un final normal del estudio en el Estado Miembro.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    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 years5
    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: 30
    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 state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 123
    F.4.2.2In the whole clinical trial 330
    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)
    None
    ninguno
    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 Decision2016-09-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-07-26
    P. End of Trial
    P.End of Trial StatusOngoing
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