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    Summary
    EudraCT Number:2021-005329-26
    Sponsor's Protocol Code Number:CV027-031
    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:2022-11-01
    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 number2021-005329-26
    A.3Full title of the trial
    A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adults with Symptomatic Nonobstructive Hypertrophic Cardiomyopathy
    Estudio clínico aleatorizado, doble ciego y controlado con placebo para evaluar mavacamten en adultos con cardiomiopatía hipertrófica sintomática no obstructiva
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adults with Cardiomyopathy
    Estudio clínico aleatorizado, doble ciego y controlado con placebo para evaluar mavacamten en adultos con cardiomiopatía
    A.3.2Name or abbreviated title of the trial where available
    Mavacamten in nonobstructive HCM
    Mavacamten en CMH no obstructiva
    A.4.1Sponsor's protocol code numberCV027-031
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1269-8581
    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 SponsorMyokardia, 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 supportMyokardia, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBristol-Myers Squibb International Corporation
    B.5.2Functional name of contact pointHead of the GSM-CT
    B.5.3 Address:
    B.5.3.1Street AddressParc de l'Alliance - Avenue de Finlande, 4
    B.5.3.2Town/ cityBraine-l'Alleud
    B.5.3.3Post code1420
    B.5.3.4CountryBelgium
    B.5.4Telephone number900 150 160
    B.5.6E-mailclinical.trials@bms.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameMavacamten
    D.3.2Product code MYK-461
    D.3.4Pharmaceutical form Capsule, hard
    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 INNNA
    D.3.9.1CAS number NA
    D.3.9.2Current sponsor codeMYK-461
    D.3.9.3Other descriptive nameMAVACAMTEN
    D.3.9.4EV Substance CodeSUB191202
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    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.IMP: 2
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameMavacamten
    D.3.2Product code MYK-461
    D.3.4Pharmaceutical form Capsule, hard
    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 INNNA
    D.3.9.1CAS number NA
    D.3.9.2Current sponsor codeMYK-461
    D.3.9.3Other descriptive nameMAVACAMTEN
    D.3.9.4EV Substance CodeSUB191202
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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.IMP: 3
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameMavacamten
    D.3.2Product code MYK-461
    D.3.4Pharmaceutical form Capsule, hard
    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 INNNA
    D.3.9.1CAS number NA
    D.3.9.2Current sponsor codeMYK-461
    D.3.9.3Other descriptive nameMAVACAMTEN
    D.3.9.4EV Substance CodeSUB191202
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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.IMP: 4
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameMavacamten
    D.3.2Product code MYK-461
    D.3.4Pharmaceutical form Capsule, hard
    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 INNNA
    D.3.9.1CAS number NA
    D.3.9.2Current sponsor codeMYK-461
    D.3.9.3Other descriptive nameMAVACAMTEN
    D.3.9.4EV Substance CodeSUB191202
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2.5
    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.IMP: 5
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameMavacamten
    D.3.2Product code MYK-461
    D.3.4Pharmaceutical form Capsule, hard
    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 INNNA
    D.3.9.1CAS number NA
    D.3.9.2Current sponsor codeMYK-461
    D.3.9.3Other descriptive nameMAVACAMTEN
    D.3.9.4EV Substance CodeSUB191202
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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, hard
    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 Nonobstructive Hypertrophic Cardiomyopathy
    cardiomiopatía hipertrófica sintomática no obstructiva
    E.1.1.1Medical condition in easily understood language
    Symptomatic hardening of the left ventricle.
    Solidificación Sintomática del ventrículo izquierdo
    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 PT
    E.1.2Classification code 10049813
    E.1.2Term Non-obstructive cardiomyopathy
    E.1.2System Organ Class 10007541 - Cardiac disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10020871
    E.1.2Term Hypertrophic cardiomyopathy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the efficacy of a 52-week course of
    mavacamten compared to placebo on patient reported health status

    To assess the efficacy of a 52-week course of
    mavacamten compared to placebo on exercise
    capacity
    Evaluar la eficacia de un ciclo de 52 semanas de mavacamten en comparación con placebo en el estado de salud notificado por el paciente

    Evaluar la eficacia de un ciclo de 52 semanas de mavacamten en comparación con placebo en la capacidad para hacer ejercicio
    E.2.2Secondary objectives of the trial
    Evaluate the effects of mavacamten on exercise capacity as measured by VE/VCO2

    Evaluate the effects of mavacamten on NYHA classification

    Evaluate the effects of mavacamten on the composite of NYHA and pVO2

    Evaluate the effects of mavacamten on cardiac biomarkers

    Evaluate the effects of mavacamten on patient reported shortness of breath

    Evaluate the effects of mavacamten on composite of cardiovascular events
    Evaluar los efectos de mavacamten sobre la capacidad para hacer ejercicio medida mediante VE/VCO2

    Evaluar los efectos de mavacamten en la clasificación de la NYHA

    Evaluar los efectos de mavacamten en el criterio combinado de NYHA y pVO2

    Evaluar los efectos de mavacamten sobre los biomarcadores cardíacos

    Evaluar los efectos de mavacamten en la disnea notificada por el paciente

    Evaluar los efectos de mavacamten en el criterio combinado de acontecimientos cardiovasculares
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Cardiac magnetic resonance (CMR) imaging sub-study (80 participants)
    •Primary endpoint: change in measures of LV mass index
    •Exploratory endpoints: changes in myocardial fibrosis
    •CMR will be done at randomization and at week 52

    Incidence of cardiac arrhythmias sub-study (~80 participants)
    •Sub-study endpoint: Incidence of AF, ventricular tachycardia, ventricular fibrillation, and conduction abnormalities including heart block from baseline to 52 weeks
    •A wearable electrocardiogram (ECG) monitor will be applied 10 times during the study as per the Schedule of Activities
    Subestudio de imágenes por resonancia magnética cardíaca (CMR) (80 participantes)
    •Criterio de valoración primario: cambio en las medidas del índice de masa del VI
    •Criterios de valoración exploratorios: cambios en la fibrosis miocárdica
    •La CMR se realizará en la aleatorización y en la semana 52

    Subestudio de incidencia de arritmias cardíacas (~80 participantes)
    •Criterio de valoración del subestudio: Incidencia de FA, taquicardia ventricular, fibrilación ventricular y anomalías de la conducción, incluido el bloqueo de corazón desde el inicio hasta las 52 semanas
    •Se aplicará un monitor de electrocardiograma (ECG) portátil 10 veces durante el estudio según el Calendario de Actividades
    E.3Principal inclusion criteria
    1. Participants must be 18 years old or local age of majority at the time of signing the informed consent
    2. Female participants must adhere to highly effective contraceptive methods or have documented proof that they are not of childbearing potential
    3. No additional contraceptive measures are required to be used for male participants
    4. Diagnosis of HCM consistent with current American College of Cardiology Foundation/American Heart Association and European Society of Cardiology guidelines.
    5. Peak LVOT pressure gradient < 30 mmHg at rest and < 50 mmHg with provocation (Valsalva maneuver and stress echocardiography)
    6. CPET: Documented oxygen saturation at rest >90% at Screening. Able to perform an upright cardiopulmonary stress test (CPET) and has a respiratory exchange ratio (RER) ≥ 1.0 at Screening per central reading. If the RER is between 0.91 and 1.0, the participant may be enrolled if the central CPET laboratory determines that peak exercise has been achieved. Participants with subpeak performance may not be enrolled as described in the CPET Laboratory Manual.
    7. New York Heart Association (NYHA) Class II or III
    8. NT-proBNP≥200 pg/mL or BNP≥70 pg/mL
    9. LVEF ≥60 % as determined by the echocardiography central laboratory
    1. Los participantes deben tener cumplidos los 18 años o ser mayores de edad, según la normativa nacional, en el momento de firmar el consentimiento informado
    2. Las participantes femeninas deben adherirse a un sistema altamente efectivo de método anticonceptivo o disponer de pruebas documentadas de que no tienen capacidad de quedarse embarazadas
    3. No se requieren medidas anticonceptivas adicionales en el caso de los participantes varones
    4. Diagnóstico de MCH concordante con las directrices actuales de la American College of Cardiology Foundation [Fundación del Colegio Estadounidense de Cardiología]/American Heart Association [Asociación Estadounidense de Cardiología] y la European Society of Cardiology [Sociedad Europea de Cardiología]
    5. Gradiente de presión máxima del infundíbulo ventricular izquierdo <30 mmHg en reposo y <50 mmHg con provocación (maniobra de Valsalva y ecocardiografía de esfuerzo)
    6. Prueba de ejercicio cardiopulmonar (PECP): Saturación de oxígeno documentada en reposo >90 % en la selección. El participante puede realizar una PECP en posición vertical y tiene un cociente respiratorio (CR) ≥1,0 en la selección según la lectura central. Si el CR está entre 0,91 y 1,0, se podrá inscribir al participante si el laboratorio central encargado de la PECP determina que se ha alcanzado el máximo ejercicio. Los participantes que presenten un rendimiento inferior al pico no pueden inscribirse para las afecciones descritas en el manual de laboratorio de la PECP
    7. Clase II o III de la New York Heart Association (Asociación de Cardiología de Nueva York [NYHA])
    8. NT-proBNP ≥200 pg/ml o BNP ≥70 pg/ml
    9. FEVI ≥60 % según lo determinado por la ecocardiografía del laboratorio central
    E.4Principal exclusion criteria
    Medical Conditions
    - Known infiltrative or storage disorder causing cardiac hypertrophy that mimics nHCM such as Fabry disease, amyloidosis, or Noonan syndrome with LV hypertrophy
    - History of unexplained syncope within 6 months prior to screening
    - History of sustained ventricular tachyarrhythmia (> 30 seconds) within 6 months prior to Screening
    - Paroxysmal or persistent (non-permanent) AF detected at the time of screening. Permanent AF or history of paroxysmal or persistent AF is allowed if the participant is anticoagulated and the investigator considers the heart rate adequately controlled
    - CV diseases or treatments that in the opinion of the investigator increase the unpredictability of or change the participants’ clinical course.
    - Acute heart failure from 4 weeks prior to screening up to randomization
    - Myocardial infarction (increase in cardiac enzymes in combination with symptoms of ischemia or new ischemic ECG changes), coronary artery bypass graft surgery, or other major CV surgery, stroke, or transient ischemic attack in the past 90 days
    - Women who are breastfeeding or pregnant.

    Prior/Concomitant Therapy
    - Any adjustments of beta-blockers, verapamil, or diltiazem within 2 weeks prior to Screening and up to the day of randomization
    - Concomitant use of strong inhibitors of cytochrome P450 (CYP) 2C19

    Other Exclusion Criteria
    - Any other serious condition that in the opinion of the investigator could prevent participation in the study and follow-up
    - Completed a study with an investigational device or drug < 30 days or 5 half-lives prior to screening
    - Participants who have completed a study with mavacamten or aficamten
    Condiciones medicas
    • Trastorno infiltrativo o del almacenamiento conocido que cause hipertrofia cardíaca que imita a la MCHn, como enfermedad de Fabry, amiloidosis o síndrome de Noonan con hipertrofia del VI
    • Antecedentes de síncope de filiación desconocida en los 6 meses anteriores a la selección
    • Antecedentes de taquiarritmia ventricular sostenida (>30 segundos) en los 6 meses anteriores a la selección
    • FA paroxística o persistente (no permanente) detectada en el momento de la selección. Se permite la FA permanente o los antecedentes de FA paroxística o persistente si el participante está anticoagulado y el investigador considera que la frecuencia cardíaca está corregida adecuadamente
    • Enfermedades o tratamientos CV que, en opinión del investigador, aumenten la imprevisibilidad de la evolución clínica de los participantes o modifiquen dicha evolución
    • Insuficiencia cardíaca aguda desde 4 semanas antes de la selección hasta la aleatorización
    • Infarto de miocardio (aumento de las enzimas cardíacas en combinación con síntomas de isquemia o nuevos cambios isquémicos en el ECG), operación de injerto de derivación de arteria coronaria u otra intervención de cirugía CV mayor, accidente cerebrovascular o accidente isquémico transitorio en los últimos 90 días
    • Mujeres en período de lactancia o Embarazadas.

    Tratamientos previos o simultáneos
    • Cualquier ajuste en los betabloqueantes, el verapamilo o el diltiazem en las 2 semanas anteriores a la selección y hasta el día de la aleatorización
    • Uso simultáneo de inhibidores potentes del citocromo P450 (CYP) 2C19.

    Otros criterios de exclusión
    • Cualquier otra afección grave que, en opinión del investigador, pueda impedir la participación en el estudio y el seguimiento
    • Finalización por parte del candidato de un estudio llevado a cabo con un producto sanitario o fármaco en investigación <30 días o 5 semividas antes de la selección
    • Finalización por parte del candidato de un estudio con mavacamten o aficamten
    E.5 End points
    E.5.1Primary end point(s)
    1. Change from baseline in KCCQ CSS
    2. Change from baseline in pVO2
    1. Cambio desde el inicio en CSS del cuestionario KCCQ
    2. Cambio desde el inicio en pVO2
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Week 52
    2. Week 52
    1. semana 52
    2. semana 52
    E.5.2Secondary end point(s)
    1. Change from baseline in VE/VCO2 slope
    2. Proportion of participants with at least 1 class of NYHA improvement from baseline
    3. Proportion of participants with (1) pVO2 ≥ 1.5 mL/kg/min and NYHA improvement ≥ 1 from baseline ; or (2) pVO2 ≥ 3 mL/kg/min and NYHA no worsening from baseline
    4. Change from baseline in NT-pro BNP
    5. Change from baseline in cTn-T
    6. Change from baseline in HCMSQ-SoB domain
    7. First MACE-Plus events defined as any CV death, non-fatal myocardial infarction, non-fatal stroke, hospitalization for heart failure, or hospitalization for arrhythmias
    1. Cambio desde el inicio en la pendiente de EV/VCO2
    2. Proporción de participantes con mejoría de al menos 1 clase en la clasificación de la NYHA desde el inicio.
    3. Proporción de participantes con (1) pVO2 ≥1,5 ml/kg/min y mejoría en la clasificación NYHA
    ≥1; o (2) pVO2 ≥3 ml/kg/min y clasificación NYHA sin empeoramiento desde el inicio.
    4. Cambio desde el inicio en el valor de NT-proBNP
    5. Cambio desde el inicio en el valor de cTn-T
    6. Cambio desde el inicio en el cuestionario HCMSQ-SoB
    7. Los primeros acontecimientos cardiovasculares mayores (MACE-plus) se definen como cualquier muerte por causas CV, infarto de miocardio no mortal, accidente cerebrovascular no mortal, hospitalización por insuficiencia cardíaca u hospitalización por arritmias
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Week 52
    2. Week 52
    3. Week 52
    4. Week 52
    5. Week 52
    6. Week 52
    7. From the study drug initiation to occurrence of the first MACE-Plus or when the last subject reaches Week 52
    1. semana 52
    2. semana 52
    3. semana 52
    4. semana 52
    5. semana 52
    6. semana 52
    7. Desde el inicio del fármaco del estudio hasta la aparición del primer MACE-Plus o cuando el último sujeto llega a la semana 52
    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 No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Biomarkers
    Biomarcadores
    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 trial1
    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 concerned14
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA89
    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
    Japan
    Korea, Republic of
    United States
    Austria
    France
    Poland
    Netherlands
    Spain
    Czechia
    Germany
    Italy
    Belgium
    Denmark
    Hungary
    Norway
    Portugal
    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
    UVUP
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    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 years2
    E.8.9.2In all countries concerned by the trial months5
    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: 400
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 state40
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 224
    F.4.2.2In the whole clinical trial 420
    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 conclusion of the study, if the study treatment is not available as an approved and available treatment, study intervention will be provided via an extension of the study, a rollover study requiring approval by the responsible Health Authority and ethics committee, or through another mechanism at the discretion of BMS.
    Al final del estudio, si el tratamiento del estudio no está disponible como un tratamiento aprobado y disponible, la intervención del estudio se proporcionará a través de una extensión del estudio, un estudio de rollover que requiere la aprobación de la Autoridad de Salud responsable y el comité de ética, o a través de otro mecanismo a discreción de BMS.
    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 Decision2023-01-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-12-21
    P. End of Trial
    P.End of Trial StatusOngoing
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