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    Summary
    EudraCT Number:2020-005643-22
    Sponsor's Protocol Code Number:AG10-304
    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:2022-03-08
    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 number2020-005643-22
    A.3Full title of the trial
    An Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)
    Estudio abierto de extensión y vigilancia de la seguridad de acoramidis (AG10) en participantes con miocardiopatía amiloide por transtiretina sintomática que completaron el ensayo de fase III ATTRibute-CM (AG10-301)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Extension study and safety monitoring of Acoramidis (AG10) in participants with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
    Estudio de extensíon y monitorización de la seguridad de Acoramidis (AG10) en participantes con miocardiopatía amiloide por transtiretina (MC-ATTR)
    A.4.1Sponsor's protocol code numberAG10-304
    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 pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street Address1800 Owens Street, Suite C-1200
    B.5.3.2Town/ citySan Francisco
    B.5.3.3Post codeCA 94158
    B.5.3.4CountryUnited States
    B.5.4Telephone number0014158871471
    B.5.6E-mailinfo@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 nameAcoramidis (AG10)
    D.3.2Product code AG10
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAcoramidis
    D.3.9.1CAS number 1446711-81-4
    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 number356
    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
    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.
    Miocardiopatía amiloide por transtiretina sintomática (MC-ATTR) es una enfermedad rara que ocurre cuando una proteina, llamada 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
    To assess safety and tolerability of acoramidis in participants with symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM)
    Evaluar la seguridad y tolerabilidad de acoramidis en participantes con miocardiopatía amiloide por transtiretina (MC-ATTR) sintomática.
    E.2.2Secondary objectives of the trial
    - To evaluate the effect of acoramidis on: all-cause mortality and cardiovascular (CV) mortality; the 6-minute walk test (6MWT); health-related quality of life (QoL) Kansas City Cardiomyopathy Questionnaire (KCCQ); the frequency of CV-related hospitalization
    - To assess the pharmacodynamic (PD) effects of acoramidis as assessed by
    •circulating TTR concentration as an in vivo biomarker of stabilization and
    •established ex vivo assays of TTR stabilization.
    - Evaluar el efecto de acoramidis sobre: mortalidad por cualquier causa y mortalidad cardiovascular (CV); prueba de marcha de 6 minutos (PM6M); cuestionario de miocardiopatía de Kansas City para medir la calidad de vida (CdV) relacionada con la salud (Kansas City Cardiomyopathy Questionnaire, KCCQ); frecuencia de hospitalización por causas CV.
    - Evaluar los efectos farmacodinámicos (FD) de acoramidis mediante
    • la concentración de TTR circulante como biomarcador in vivo de estabilización y
    • en ensayos establecidos ex vivo de estabilización de la TTR.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    To be eligible to participate in this OLE study, participants must meet all the following criteria:
    1. Completed 30 months of the blinded study treatment in Study AG10-301 and the Study AG10-301 Month 30 visit including assessments and procedures.
    2. Have the ability to understand and sign a written informed consent form, which must be obtained prior to initiation of study procedures.
    3. Female participants of childbearing potential who engage in heterosexual intercourse must agree to use a highly effective method of contraception beginning with enrollment and continuing for 30 days after the last dose of acoramidis. Female participants using oral contraceptives must agree to use an additional birth control method. While not considered highly effective, a double-barrier method is acceptable. A male participant who is sexually active with a female of childbearing potential and has not had a vasectomy must agree to use a double-barrier method of birth control.
    Los participantes deben cumplir todos estos criterios con el fin de ser aptos para participar en este ensayo de extensión abierta:
    1. Deben haber completado los 30 meses de tratamiento enmascarado del estudio en el estudio AG10301 y la visita del mes 30 del estudio AG10-301, incluidas las evaluaciones y los procedimientos.
    2. Deben poder comprender y firmar un formulario de consentimiento informado por escrito, que debe obtenerse antes de iniciar los procedimientos del estudio.
    3. Las participantes con capacidad de concebir que mantengan relaciones sexuales heterosexuales deben aceptar el uso de un método anticonceptivo altamente eficaz a partir de la inscripción y seguir usándolos 30 días después de la última dosis de acoramidis. Las participantes que utilicen anticonceptivos orales deben aceptar el uso de un método anticonceptivo adicional. Aunque no se considera altamente eficaz, se acepta un método de doble barrera. Un participante varón sexualmente activo con una mujer con capacidad de concebir y que no se haya sometido a una vasectomía debe aceptar utilizar un método anticonceptivo de doble barrera.
    E.4Principal exclusion criteria
    Participants who meet any of the following criteria at the Day 1 visit will not be eligible to participate in the study:
    1. Has hemodynamic instability, that in the judgment of the Investigator, would pose too great a risk for participation in the study.
    2. Has had a heart and/or liver transplant within the year prior to Day 1.
    3. Has had implantation of a cardiac mechanical assist device (CMAD).
    4. Has confirmed diagnosis of light-chain (AL) amyloidosis at any time during Study AG10-301.
    5. Is on dialysis or has a degree of renal impairment that in the opinion of the Investigator might jeopardize the participant’s safety, increase their risk from participation, or interfere with the study.
    6. Known hypersensitivity to acoramidis, its metabolites, or formulation excipients.
    7. At the end of Study AG10-301 or at Day 1 of Study AG10-304 (or any time during the study), participant is on prohibited medication.
    8. Females who are pregnant or breastfeeding. A negative urine pregnancy test at the Day 1 visit and at each study visit are required for female participants of childbearing potential.
    9. 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 participant’s safety, increase their risk from participation, or interfere with the study.
    10. Participation in another interventional clinical trial (with the exception of Study AG10-301) within 30 days prior to dosing. Participation in observational and/or registry studies should be discussed with the Medical Monitor.
    11. 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 participantes que cumplan cualquiera de los siguientes criterios en la visita del día 1 no serán aptos para participar en el ensayo:
    1. Presentar inestabilidad hemodinámica que, en opinión del investigador, supondría un riesgo excesivo para la participación en el estudio.
    2. Haber recibido un trasplante de corazón o hígado en el año anterior al día 1.
    3. Haberse sometido a la implantación de un dispositivo de asistencia mecánica cardíaca (DACM).
    4. Tener un diagnóstico confirmado de amiloidosis de cadenas ligeras en cualquier momento durante el estudio AG10-301.
    5. Estar recibiendo diálisis o presentar un grado de insuficiencia renal que, en opinión del investigador, podría poner en peligro la seguridad del participante, aumentar el riesgo derivado de la participación o interferir en el estudio.
    6. Tener hipersensibilidad conocida a acoramidis, sus metabolitos o los excipientes de la formulación.
    7. Recibir medicación que esté prohibida al final del estudio AG10-301 o el día 1 del estudio AG10-304 (o en cualquier momento durante el estudio).
    8. Ser una mujer embarazada o en período de lactancia. Las participantes con capacidad de concebir deben tener un resultado negativo en la prueba de embarazo en orina realizada el día 1 y en cada visita del estudio.
    9. A criterio del investigador o del supervisor médico, tener cualquier afección médica, anomalía de laboratorio u cualquier situación en curso clínicamente importante que pudiera poner en peligro la seguridad del participante, aumentar su riesgo debido a la participación o interferir en el ensayo.
    10. Participar en otro ensayo clínico intervencionista (con la excepción del estudio AG10-301) en los 30 días previos a la administración. La participación en estudios observacionales o de registro debe comentarse con el supervisor médico.
    11. Tener cualquier afección que, en opinión del investigador o del supervisor médico, impediría el cumplimiento del protocolo del ensayo, como antecedentes de abuso de sustancias, alcoholismo o una enfermedad psiquiátrica.
    E.5 End points
    E.5.1Primary end point(s)
    Safety parameters to be assessed: treatment- emergent serious adverse events (SAEs) and adverse events (AEs), AEs leading to treatment discontinuation, abnormal physical exam findings of clinical relevance, abnormal vital signs of clinical relevance, abnormal electrocardiogram (ECG) parameters of clinical relevance, and changes in clinical safety laboratory parameters of clinical relevance.
    Parámetros de seguridad que se evaluarán: acontecimientos adversos graves (AAG) y acontecimientos adversos (AA) surgidos durante el tratamiento, AA que lleven a la interrupción del tratamiento, anomalías en los hallazgos de la exploración física de relevancia clínica, constantes vitales anómalas de relevancia clínica, parámetros anómalos del electrocardiograma (ECG) de relevancia clínica y cambios en los parámetros analíticos de seguridad clínica de posible relevancia clínica.
    E.5.1.1Timepoint(s) of evaluation of this end point
    AEs: Month 0-60; ET, unscheduled visit
    Physical examination, Vital signs, ECG: Day 1, Month 1, Month 6-60 every 6 months, Month 61; ET, unscheduled visit, 30 days post last dose.
    AA: Meses 0-60; RT, visita no programada
    Exploración física, constantes vitales, ECG Día 1, mes 1, meses 6-60 cada 6 meses, mes 61; RT, visita no programada, 30 días después de la última dosis.
    E.5.2Secondary end point(s)
    - All-cause mortality and CV mortality
    - Change from Baseline in distance walked during the 6MWT
    - Change from Baseline in KCCQ Overall Score (KCCQ-OS)
    - CV-related hospitalization
    - Change from baseline in TTR (prealbumin) level (an in vivo measure of TTR stabilization)
    - TTR stabilization measured in established ex vivo assays (FPE and Western blot) in the PK-PD substudy
    - Mortalidad por cualquier causa y por causas CV.
    - Cambio desde el inicio en la distancia recorrida durante la PM6M.
    - Cambio con respecto al inicio en la puntuación total de KCCQ (KCCQ-OS).
    - Hospitalización por causas CV.
    - Cambio respecto al inicio en el nivel de TTR (prealbúmina) (una medida in vivo de la estabilización de la TTR).
    - Estabilización de la TTR medida en ensayos establecidos ex vivo (FPE e inmunoelectrotransferencia) en el subestudio FC-FD
    E.5.2.1Timepoint(s) of evaluation of this end point
    - All-cause mortality and CV mortality: Month 0-61
    - Distance walked during the 6MWT: Day 1, Month 6, 12, 18, 24, 30, 36, 42, 48, 54, 60; ET, unscheduled visit
    - KCCQ will be obtained on Day 1, Month 1, Month 6, and every 6 months up to Month 60 during the study. KCCQ should be completed prior to the EQ-5D-5L and prior to conduction of the 6MWT at those visits during which a 6MWT is performed
    - CV-related hospitalization will be summarized by number of patients who have at least one CV-related hospitalization and the frequency of CV-related hospitalization; cumulative frequency of CV-related hospitalization: Month 0-61
    - Mortalidad por cualquier causa y por causas CV: meses 0-61
    - Distancia recorrida durante la PM6M: Día 1, meses 6, 12, 18, 24, 30, 36, 42, 48, 54, 60; RT, visita no programada
    - El KCCQ se obtendrá el día 1, el mes 1, el mes 6 y cada 6 meses hasta el mes 60 durante el estudio. El KCCQ debe completarse antes del EQ-5D-5L y antes de la realización de la PM6M en aquellas visitas durante las que se realice una PM6M.
    - La hospitalización por causas CV se resumirá teniendo en cuenta el número de pacientes que tengan al menos una hospitalización por causas CV y la frecuencia de la hospitalización por causas CV; frecuencia acumulada de hospitalización por causas CV: meses 0-61
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other No
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA29
    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
    Australia
    Brazil
    Canada
    Israel
    Korea, Republic of
    New Zealand
    United States
    Belgium
    Czechia
    Denmark
    Greece
    Ireland
    Italy
    Netherlands
    Poland
    Portugal
    Spain
    United Kingdom
    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
    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 months1
    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 months1
    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: 11
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 535
    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 state44
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 223
    F.4.2.2In the whole clinical trial 546
    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)
    The study is planned to continue until participants have access to acoramidis by prescription for treatment of ATTR-CM or up to approximately 60 months treatment period, followed by 1-month follow-up. Total duration of participation in the study is expected to be up to approximately 61 months.
    Está previsto que el estudio continúe hasta que los participantes puedan recibir acoramidis con receta para el tratamiento de la MC-ATTR o durante un período de tratamiento de aproximadamente 60 meses, seguido de un seguimiento de 1 mes. Se espera que la duración total de la participación en el estudio sea de aproximadamente 61 meses.
    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 Decision2022-04-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-03-31
    P. End of Trial
    P.End of Trial StatusOngoing
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