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    Summary
    EudraCT Number:2018-004280-32
    Sponsor's Protocol Code Number:AG10-301
    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:2019-06-11
    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 number2018-004280-32
    A.3Full title of the trial
    A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of AG10 in Subjects with Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTRIBUTE-CM Trial)
    Estudio de fase III, aleatorizado, doble ciego y controlado con placebo de la eficacia y la seguridad de AG10 en pacientes con miocardiopatía amiloide por transtiretina sintomática (Ensayo ATTRIBUTE-CM)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of the Efficacy and Safety of AG10 in participants with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
    Estudio de la eficacia y la seguridad de AG10 en participantes con miocardiopatía amiloide por transtiretina sintomática (MC-ATTR)
    A.3.2Name or abbreviated title of the trial where available
    Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Cardiomyopathy
    Eficacia y seguridad de AG10 en sujetos con miocardiopatía amiloide por transtiretina
    A.4.1Sponsor's protocol code numberAG10-301
    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 SponsorEidos Therapeutics, 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 supportEidos Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationEidos Therapeutics, Inc.
    B.5.2Functional name of contact pointDirector, Clinical Operations
    B.5.3 Address:
    B.5.3.1Street Address101 Montgomery Street, Suite 2550
    B.5.3.2Town/ citySan Francisco
    B.5.3.3Post codeCA 94104
    B.5.3.4CountryUnited States
    B.5.4Telephone number0014158871471
    B.5.6E-mailvknobel@eidostx.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/3/18/2081 - EMA/OD/096/18
    D.3 Description of the IMP
    D.3.1Product nameAG10
    D.3.2Product code AG10
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAG10 HCl
    D.3.9.1CAS number 2242751-53-5
    D.3.9.3Other descriptive name3-(3-(3,5-DIMETHYL-1H-PYRAZOL-4-YL)PROPOXY)-4-FLUOROBENZOIC ACID
    D.3.9.4EV Substance CodeSUB193376
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number400
    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 placeboTablet
    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
    Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
    Miocardiopatía amiloide por transtiretina sintomática (MC-ATTR)
    E.1.1.1Medical condition in easily understood language
    Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM) is a rare disease that occurs when a protein, called transthyretin, is accumulated in the heart.
    La miocardiopatía amiloide por transtiretina sintomática (MC-ATTR) es una enfermedad rara que se produce cuando una proteína denominada transtiretina se acumula en el corazón.
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10002020
    E.1.2Term Amyloid cardiomyopathy
    E.1.2System Organ Class 100000004849
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part A: To determine the efficacy of AG10 in the treatment of subjects with symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM) by evaluating the difference between the AG10 and placebo groups in the change from baseline in the Six-Minute Walk test (6MWT)
    Part B: To determine the efficacy of AG10 in the treatment of subjects with symptomatic ATTR-CM by evaluating the difference between the AG10 and placebo groups in the combined endpoint of All-Cause Mortality and the cumulative frequency of cardiovascular (CV)-related hospitalization

    Key Secondary
    Part A: To evaluate the effects of AG10 on quality of life (QoL) in subjects with symptomatic ATTR-CM
    Part B:
    •To evaluate the effects of AG10 on 6MWT
    •To evaluate the effects of AG10 on QoL in subjects with symptomatic ATTR-CM
    Parte A: determinar la eficacia de AG10 en el tratamiento de pacientes con miocardiopatía amiloide por transtiretina (MC-ATTR) sintomática mediante la evaluación de la diferencia entre los grupos de AG10 y placebo en el cambio con respecto al inicio en la prueba de la marcha de 6 minutos (PM6M)
    Parte B: determinar la eficacia de AG10 en el tratamiento de pacientes con MC-ATTR sintomática mediante la evaluación de la diferencia entre los grupos de AG10 y placebo en el criterio de valoración combinado de mortalidad por cualquier causa y frecuencia acumulada de hospitalizaciones por causas cardiovasculares (CV)
    Secundarios clave
    Parte A: evaluar los efectos de AG10 en la calidad de vida (CdV) de pacientes con MC-ATTR sintomática
    Parte B:
    • Evaluar los efectos de AG10 en la PM6M
    • Evaluar los efectos de AG10 en la CdV de los pacientes con MC-ATTR sintomática
    E.2.2Secondary objectives of the trial
    Part A:
    •To assess safety and tolerability of AG10 in subjects with symptomatic ATTR-CM
    •To assess the PD effects of AG10 as assessed by circulating prealbumin (transthyretin, TTR) concentration as an in vivo biomarker of stabilization and established ex vivo assays of TTR stabilization

    Part B:
    •To determine the efficacy of AG10 treatment as measured by the components of the primary endpoint
    •To determine the efficacy of AG10 in reducing CV mortality in subjects with symptomatic ATTR-CM
    •To describe the PD properties and the PK-PD relationship of AG10 as assessed by circulating prealbumin (transthyretin, TTR) concentration as an in vivo biomarker of stabilization and established ex vivo assays of TTR stabilization, and correlated with AG10 PK
    •To evaluate the safety and tolerability of AG10 administered for 30 months to subjects with symptomatic ATTR-CM
    Parte A:
    • Evaluar la seguridad y la tolerabilidad de AG10 en pacientes con MC-ATTR sintomática
    • Evaluar los efectos FD de AG10 determinados mediante la concentración de prealbúmina (transtiretina, TTR) circulante como biomarcador in vivo de la estabilización y mediante análisis demostrados de la estabilización de la TTR ex vivo
    Parte B:
    • Determinar la eficacia del tratamiento con AG10 determinada a través de los componentes del criterio de valoración princip.
    • Determinar la eficacia de AG10 en la reducción de la mortalidad por causas CV en pacientes con MC-ATTR sintomática
    • Describir las propiedades FD y la relación FC-FD de AG10, evaluadas mediante la concentración de prealbúmina (transtirretina, TTR) circulante como biomarcador in vivo de la estabilización y mediante análisis demostrados de estabilización de la TTR ex vivo, y correlacionadas con la FC de AG10
    • Evaluar la seguridad y la tolerabilidad de AG10 administrado durante 30 meses a pacientes con MC-ATTR sintomática
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Have the ability to understand and sign a written informed consent form, which must be obtained prior to initiation of study procedures.
    2.Be male or female ≥18 to ≤90 years of age.
    3.Have an established diagnosis of ATTR-CM with either wild-type TTR or a variant TTR genotype (confirmed by genotyping) based on either (1) endomyocardial biopsy, or (2) positive 99mTc-pyrophosphate or bisphosphonate scan, combined with accepted laboratory criteria excluding a diagnosis of AL amyloidosis (based on both immunofixation electrophoresis (IFE) of serum and/or urine, and serum free light chain (sFLC) analysis); subjects with concurrent monoclonal gammopathy of undetermined significance (MGUS) may require confirmation of the diagnosis of ATTR-CM by endomyocardial biopsy with mass spectrometric analysis. Diagnosis of ATTR-CM will be confirmed by central review of the clinical data that were used to establish the diagnosis.
    4.Have
    a.a history of heart failure evidenced by at least one prior hospitalization for heart failure or
    b.clinical evidence of heart failure without prior heart failure 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, or peripheral edema) or
    c.heart failure symptoms that required or require ongoing treatment with a diuretic.
    5.Have NYHA Class I-III symptoms due to ATTR cardiomyopathy.
    6.Female subjects of childbearing potential who engage in heterosexual intercourse must agree to use a highly effective method of contraception beginning with randomization and continuing for 30 days after the last dose of AG10 (as described in section 3.5). A man who is sexually active with a woman of childbearing potential and has not had a vasectomy must agree to use a barrier method of birth control (as described in section 3.5).
    7.For subjects taking cardiovascular medical therapy, with the exception of diuretic dosing, must be on stable doses (defined as no greater than 50% dose adjustment and no categorical changes of medications) for at least 2 weeks prior to Screening.
    8.Have completed ≥150 m on the 6MWT on 2 consecutive tests prior to randomization.
    9.Must have NT-proBNP levels ≥300 pg/mL at Screening.
    10.Must have LV wall (interventricular septum or LV posterior wall) thickness ≥13 mm as measured by transthoracic echocardiogram or cardiac magnetic resonance (CMR) documented in medical history within 10 years of Screening or at Screening echocardiogram.
    1. Tener la capacidad de entender y firmar un formulario de consentimiento informado, que se debe obtener antes de iniciar los procedimientos del estudio.
    2. Ser un hombre o una mujer de entre ≥18 y ≤90 años.
    3. Tener un diagnóstico confirmado de MC-ATTR con TTR de genotipo natural o con una variante genotípica de la TTR (confirmada por genotipado), sobre la base de (1) la biopsia endomiocárdica o (2) de un resultado positivo en la gammagrafía con 99mTc unido a pirofosfato o a bisfosfonato, combinado con criterios analíticos aceptados que descarten un diagnóstico de amiloidosis de cadenas ligeras (basándose tanto en electroforesis de inmunofijación [EIF] en suero y/u orina como en análisis de cadenas ligeras libres [CLL] en suero); los pacientes con gammapatía monoclonal de significado incierto (GMSI) simultánea quizá requieran la confirmación del diagnóstico de MC-ATTR por biopsia endomiocárdica con espectrometría de masas. El diagnóstico de MC-ATTR se confirmará mediante revisión central de los datos clínicos que se usaron para establecer el diagnóstico.
    4. Tener
    a. antecedentes de insuficiencia cardíaca demostrada por al menos una hospitalización previa por insuficiencia cardíaca o
    b. indicios clínicos de insuficiencia cardíaca sin hospitalización previa por insuficiencia cardíaca, manifestada por signos o síntomas de sobrecarga de volumen o presión intracardíaca elevada (p. ej., elevación de la presión venosa yugular, disnea o signos de congestión pulmonar en una radiografía o auscultación, o edema periférico) o
    c. síntomas de insuficiencia cardíaca que hayan requerido o requieran tratamiento en curso con un diurético.
    5. Síntomas de clase I-III de la NYHA debido a la miocardiopatía por ATTR.
    6. Las pacientes con capacidad de concebir que mantengan relaciones heterosexuales deben aceptar el uso de un método anticonceptivo muy eficaz a partir de la aleatorización y seguir usándolo 30 días después de la última dosis de AG10 (tal como se describe en la sección 3.5). Un hombre sexualmente activo con una mujer con capacidad de concebir y que no se haya sometido a una vasectomía debe estar de acuerdo en usar un método
    anticonceptivo de barrera (tal como se describe en la sección 3.5).
    7. Los pacientes que estén recibiendo farmacoterapia cardiovascular, exceptuando el tratamiento con diuréticos, deben haber recibido dosis estables (definidas como un ajuste de la dosis no superior al 50 % y sin cambios categóricos de los medicamentos) durante un mínimo de 2 semanas antes de la selección.
    8. Haber recorrido una distancia ≥150 m en la PM6M en dos pruebas consecutivas antes de la aleatorización.
    9. Concentración de NT-proBNP ≥300 pg/ml en la selección.
    10. Con un grosor de la pared del VI (tabique interventricular o pared posterior del VI) ≥13 mm medido mediante ecocardiograma transtorácico o resonancia magnética cardíaca (RMC) documentado en el historial médico en los
    10 años previos a la selección o en el ecocardiograma de la selección.
    E.4Principal exclusion criteria
    Subjects who meet any of the following criteria at the Screening visit will not be eligible to participate in the study:
    1.Acute myocardial infarction, acute coronary syndrome or coronary revascularization within 90 days prior to Screening.
    2.Experienced stroke within 90 days prior to Screening.
    3.Has hemodynamic instability at Screening or Randomization that, in the judgment of the Investigator, would pose too great a risk for participation in the study.
    4.Is likely to undergo heart transplantation within a year of Screening.
    5.Has confirmed diagnosis of light-chain amyloidosis.
    6.Has abnormal liver function tests at Screening, defined as alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >3 × upper limit of normal (ULN) or total bilirubin >3 × ULN.
    7.Has NT-proBNP levels ≥7000 pg/mL at Screening.
    8.Has estimated glomerular filtration rate (eGFR) by MDRD formula < 15 mL/min/1.73 m2 at Screening.
    9.Known hypersensitivity to study drug (AG10 or placebo), its metabolites, or formulation excipients.
    10.Current treatment for ATTR-CM with tafamidis, or with marketed drug products lacking a labeled indication for ATTR-CM (e.g., diflunisal, doxycycline); natural products or derivatives used as unproven therapies for ATTR-CM (e.g., green tea extract, tauroursodeoxycholic acid [TUDCA]/ursodiol), patisiran, inotersen, or any investigational agent for the treatment of ATTR-CM, within 14 days or 90 days for patisiran and 180 days for inotersen prior to dosing. If, during participation in the study, tafamidis becomes commercially available and subjects have access to it, subjects will be permitted to initiate therapy with tafamidis as a concomitant medication if they have completed at least 24 months of blinded study therapy.
    11.Requires treatment with calcium channel blockers (e.g., verapamil, diltiazem) or digitalis.
    12.Females who are pregnant or breastfeeding. Lactating females must agree to discontinue nursing before study drug is administered. A negative urine pregnancy test at Screening and at Randomization are required for female subjects of childbearing potential.
    13.In the judgment of the Investigator or Medical Monitor, has any clinically important ongoing medical condition or laboratory abnormality or condition that might jeopardize the subject’s safety, increase their risk from participation, or interfere with the study.
    14.Participation in another investigational drug or investigational device study within 30 days prior to dosing with potential residual effects that might confound the results of this study.
    15.Has any condition that, in the opinion of the Investigator or Medical Monitor, would preclude compliance with the study protocol such as a history of substance abuse, alcoholism or a psychiatric condition.
    Los pacientes que cumplan alguno de los siguientes criterios en la visita de selección no serán aptos para participar en el estudio:
    1. Infarto agudo de miocardio, síndrome coronario agudo o revascularización coronaria en el plazo de 90 días antes de la selección.
    2. Accidente cerebrovascular en los 90 días previos a la selección.
    3. Inestabilidad hemodinámica en la selección o en la aleatorización que, en opinión del investigador, pudiera suponer demasiado riesgo en caso de participación en el estudio.
    4. Probabilidad de recibir un trasplante de corazón en el plazo de un año desde la selección.
    5. Diagnóstico confirmado de amiloidosis de cadenas ligeras.
    6. Anomalías en las pruebas funcionales hepáticas en la selección, definidas como valores de alanina-aminotransferasa (ALT) o aspartato-aminotransferasa (AST) >3 × límite superior de la normalidad (LSN) o de bilirrubina total >3 × LSN.
    7. Concentración de NT-proBNP ≥7000 pg/ml en la selección.
    8. Tasa de filtración glomerular estimada (TFGe) mediante la fórmula de MDRD <15 ml/min/1,73 m2 en la selección.
    9. Hipersensibilidad conocida al fármaco del estudio (AG10 o placebo), sus metabolitos o los excipientes de la formulación.
    10. Tratamiento en curso para la MC-ATTR con tafamidis o con fármacos comercializados sin indicación autorizada para la MC-ATTR (p. ej., diflunisal, doxiciclina); productos naturales o derivados utilizados como tratamientos no demostrados de la MC-ATTR (p. ej., extracto de té verde, ácido tauroursodesoxicólico [TUDCA]/ursodiol), patisirán, inotersén, o cualquier otro fármaco en investigación para el tratamiento de la MC-ATTR, en un plazo de 14 días, o 90 días en el caso de patisirán y 180 días en el caso de inotersén, antes de la administración de la dosis. Si durante la participación en el estudio se empieza a comercializar tafamidis y los pacientes tienen acceso a este, podrán iniciar el tratamiento con tafamidis como medicamento concomitante si han completado al menos 24 meses de tratamiento enmascarado del estudio.
    11. Necesidad de tratamiento con antagonistas del calcio (p. ej., verapamilo, diltiazem) o digitálicos.
    12. Mujeres que estén embarazadas o en período de lactancia. Las mujeres lactantes deben aceptar interrumpir la lactancia antes de la administración del fármaco del estudio. En las pacientes con capacidad de concebir se exige una prueba de embarazo en orina negativa en la selección y la aleatorización.
    13. Cualquier enfermedad, anomalía analítica o situación médica en curso que sea clínicamente importante y que, en opinión del investigador o del supervisor médico, podría poner en peligro la seguridad del paciente, aumentar su riesgo debido a la participación o interferir en el estudio.
    14. Participación en otro estudio con un fármaco o dispositivo en investigación en un plazo de 30 días antes de la administración de la dosis con posibles efectos residuales que podrían confundir los resultados de este estudio.
    15. Cualquier afección que, en opinión del investigador o del supervisor médico, impediría el cumplimiento del protocolo del estudio, por ejemplo, antecedentes de abuso de sustancias, alcoholismo o enfermedad psiquiátrica.
    E.5 End points
    E.5.1Primary end point(s)
    Part A: Change from baseline to Month 12 of treatment in distance walked during the 6MWT

    Part B: A hierarchical combination of All-Cause mortality and CV-related hospitalization over a 30-month period
    Parte A: Cambio desde el inicio hasta el mes 12 de tratamiento en la distancia recorrida durante la PM6M

    Parte B: una combinación jerárquica de la mortalidad por cualquier causa y la hospitalización relacionada con causas CV a lo largo de un período de 30 meses
    E.5.1.1Timepoint(s) of evaluation of this end point
    Part A
    -Distance walked during the 6MWT: Prior to randomisation, Month 6, 9, 12

    Part B
    All-cause mortality and CV-related hospitalization: Month 0-30
    Parte A
    Distancia recorrida durante la PM6M: Antes de la aleatorización, Mes 6, 9 y 12

    Parte B
    Mortalidad por cualquier causa y hospitalizaciones por causas CV: Mes 0-30
    E.5.2Secondary end point(s)
    Key secondary
    Part A
    -Change from baseline to Month 12 of treatment in Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS)
    Part B
    -Change from baseline to Month 30 of treatment in distance walked during the 6MWT
    -Change from baseline to Month 30 of treatment in KCCQ-OS
    Other secondary
    Part A
    -Change from baseline in TTR (prealbumin) level (an in vivo measure of TTR stabilization) and established ex vivo assays of TTR stabilization at Day 28 and subsequent visits
    Part B
    -All-Cause Mortality by Month 30
    -Cumulative frequency of CV-related hospitalization by Month 30
    -CV Mortality by Month 30
    -Change from baseline in TTR (prealbumin) level (an in vivo measure of TTR stabilization) and established ex vivo assays of TTR stabilization
    Secondarios claves
    Parte A
    -Cambio desde el inicio hasta el mes 12 de tratamiento en la puntuación total del cuestionario de miocardiopatía de Kansas City (Kansas City Cardiomyopathy Questionnaire Overall Score, KCCQ-OS)
    Parte B
    -Cambio desde el inicio hasta el mes 30 de tratamiento en la distancia recorrida durante la PM6M
    -Cambio desde el inicio hasta el mes 30 de tratamiento en la KCCQ-OS
    Otros secundarios
    Parte A
    -Cambio desde el inicio en el nivel de TTR (prealbúmina) (una medida in vivo de la estabilización de la TTR) y en ensayos establecidos ex vivo de estabilización de la TTR el día 28 y en visitas posteriores
    Parte B
    -Mortalidad por cualquier causa en el mes 30
    -Frecuencia acumulada de hospitalizaciones por causas CV en el mes 30
    -Mortalidad por causa CV en el mes 30
    -Cambio desde el inicio en el nivel de TTR (prealbúmina) (una medida in vivo de la estabilización de la TTR) y en ensayos establecidos ex vivo de estabilización de la TTR
    E.5.2.1Timepoint(s) of evaluation of this end point
    Key secondary
    Part A
    -KCCQ-OS: Day 1, Month 3, 6, 9, 12
    Part B
    -Distance walked during the 6MWT: Prior to randomization, Month 6, 9, 12, 18, 24, 30
    -KCCQ-OS: Day 1, Month 3, 6, 9, 12, 15, 18, 21, 24, 27, 30
    Other secondary
    Part A
    - TTR (prealbumin) level and ex vivo assays of TTR stabilization: Day 1, Day 28, Month 3, 6, 9, 12
    Part B
    - All-Cause Mortality: Month 0-30
    - Cumulative frequency of CV-related hospitalization: Month 0-30
    - CV Mortality: Month 0-30
    - TTR (prealbumin) level ex vivo assays of TTR stabilization: Day 1, Day 28, Month 3, 6, 9, 12, 15, 18, 21, 24, 27, 30
    Secundarios clave
    Parte A
    -KCCQ-OS: día 1, mes 3, 6, 9 y 12
    Parte B
    -Distancia recorrida durante la PM6M: antes de la aleatorización, mes 6, 9, 12, 18, 24 y 30
    -KCCQ-OS: día 1, mes 3, 6, 9, 12, 15, 18, 21, 24, 27 y 30
    Otros secundarios
    Parte A
    -Nivel de TTR (prealbúmina) y ensayos ex vivo de estabilización de la TTR: día 1, día 28, mes 3, 6, 9 y 12
    Parte B
    -Mortalidad por cualquier causa: mes 0-30
    -Frecuencia acumulada de hospitalización por causas CV: mes 0-30
    -Mortalidad por causas CV: mes 0-30
    -Nivel de TTR (prealbúmina) y ensayos ex vivo de estabilización de la TTR: día 1, día 28, mes 3, 6, 9, 12, 15, 18, 21, 24, 27 y 30
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group 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 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
    Argentina
    Australia
    Belgium
    Brazil
    Canada
    Denmark
    Ireland
    Israel
    Italy
    Korea, Republic of
    Mexico
    Netherlands
    New Zealand
    Portugal
    Spain
    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
    UVUS (última visita último sujeto)
    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 months10
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months2
    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: 21
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 489
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 220
    F.4.2.2In the whole clinical trial 510
    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)
    At the end of the treatment, the study doctor may continue to give the subject AG10 as an extension of this study, until AG10 is commercially available for the treatment of subjects with symptomatic Transthyretin Amyloid Cardiomyopathy, unless the study doctor believes it no longer in your best interests, or unless the Sponsor terminates development of the AG10 for the treatment of symptomatic ATTR-CM in your country.
    Al final del tratamiento, el médico del estudio podrá seguir administrando AG10 al paciente en una ampliación de este estudio hasta que AG10 se comercialice para el tratamiento de la miocardiopatía amiloide por transtiretina sintomática, a menos que el médico del estudio considere que ya no es lo mejor para el paciente, o a menos que el promotor finalice el desarrollo de AG10 para el tratamiento de la MC-ATTR en España.
    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 Decision2019-10-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-06-17
    P. End of Trial
    P.End of Trial StatusOngoing
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