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    Summary
    EudraCT Number:2017-004310-25
    Sponsor's Protocol Code Number:ARRAY-797-301
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-05-18
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2017-004310-25
    A.3Full title of the trial
    A Phase 3, Multinational, Randomized, Placebo-controlled Study of ARRY-371797 in Patients with Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation
    Estudio de fase 3, multinacional, aleatorizado y controlado con placebo de ARRY-371797 en pacientes con miocardiopatía dilatada sintomática por mutación del gen lamina A/C
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of ARRY-371797 in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation
    Estudio de ARRY-371797 en pacientes con miocardiopatía dilatada sintomática por mutación del gen lamina A/C
    A.4.1Sponsor's protocol code numberARRAY-797-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03439514
    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 SponsorArray BioPharma 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 supportArray Biopharma Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationArray BioPharma Inc.
    B.5.2Functional name of contact pointTeri Whisenand
    B.5.3 Address:
    B.5.3.1Street Address3200 Walnut Street
    B.5.3.2Town/ cityBoulder
    B.5.3.3Post codeCO80301
    B.5.3.4CountryUnited States
    B.5.4Telephone number34900834223
    B.5.6E-mailRegistroEspanolDeEstudiosClinicos@druginfo.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 nameARRY-371797
    D.3.2Product code ARRY-371797
    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 INNNot yet assigned
    D.3.9.1CAS number 765914-60-1
    D.3.9.2Current sponsor codeARRY-371797
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 placeboFilm-coated tablet
    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
    Dilated Cardiomyopathy (DCM) with Lamin A/C protein (LMNA) Mutation
    Miocardiopatía dilatada sintomática por mutación del gen lamina A/C
    E.1.1.1Medical condition in easily understood language
    Dilated Cardiomyopathy
    Miocardiopatía dilatada
    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 10056419
    E.1.2Term Dilated cardiomyopathy
    E.1.2System Organ Class 100000004849
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    NYHA Class II/III patients only: Evaluate the effect of ARRY-371797 on functional capacity (as measured by the
    6-minute walk test [6MWT]) compared to placebo
    Solo pacientes de clase II/III de la NYHA: Evaluar el efecto de ARRY 371797 sobre la capacidad funcional (medida con la prueba de marcha de 6 minutos [PM6M]) frente a un placebo
    E.2.2Secondary objectives of the trial
    - NYHA Class II/III patients only: Evaluate additional measures
    of efficacy of ARRY-371797 compared to placebo in the randomized period
    - Characterize the plasma pharmacokinetics (PK) of ARRY-371797 and metabolites
    - Evaluate the safety of ARRY-371797 compared to placebo
    - Solo pacientes de clase II/III de la NYHA: Evaluar otras medidas de la eficacia de ARRY-371797 frente a un placebo en el periodo aleatorizado
    - Caracterizar la farmacocinética (FC) plasmática de ARRY 371797 y sus metabolitos
    - Evaluar la seguridad de ARRY-371797 frente a un placebo
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Selected Key Inclusion Criteria:
    • Age >= 18 years at time of informed consent
    • Patients with symptomatic lamin A/C protein (LMNA)-related cardiomyopathy Class II/III/ or Class IV defined as:
    o Gene positive for a deleterious mutation in the LMNA gene as determined by the study central laboratory or by initial laboratory testing (central confirmation of initial laboratory results is required either during screening or within 30 days of initiating study treatment
    o Evidence of cardiac impairment in Serum NT-proBNP and EF
    • Patient will have an implantable cardioverter defibrillator/cardiac resynchronization therapy defibrillator (ICD/CRT-D). ICD implanted at least 4 weeks prior to initiation of study treatment or CRT-D initiated at least 6 months prior to initiation of study treatment
    • Class II/III patients must have objective functional impairment evidenced by a reduction in 6-minute walk test (6MWT);
    • Stable medical and/or device therapy consistent with American Heart Association (AHA) / American College of Cardiology (ACC) or European Society of Cardiology (ESC) guidelines
    • Patients must meet acceptable hematology, hepatic and renal laboratory values as specified
    Selección de Criterios Clave de Elegibilidad:
    - Edad >= 18 años en el momento del consentimiento informado
    - Pacientes con miocardiopatía clase II/III o IV, sintomática con mutación LMNA definida como:
    a. Mutación nociva positiva del gen LMNA, determinada por el laboratorio central del estudio o el laboratorio inicial (se requiere la confirmación de los resultados iniciales por el laboatorio central, ya sea durante la selección o en los 30 días siguientes al inicio del tratamiento del estudio);
    b. Evidencia de disfunción cardiaca en NT-proBNP sérica Y FE
    - El paciente tendrá un DCI (implantado al menos 4 semanas antes del inicio del tratamiento del estudio) o un /D-TRC (implantado al menos 6 meses antes del inicio del tratamiento del estudio)
    - Los pacientes de clase funcional II/III deben tener una disfunción funcional objetiva evidenciada por una reducción de la PM6M
    - Tratamiento médico o instrumental estable compatible con las guías de la American Heart Association(AHA)/American College of Cardiology (ACC) o de la European Society of Cardiology (ESC)
    - Valores analíticos aceptables de las pruebas hematológicas y de función hepática y renal tal y como se especifica
    E.4Principal exclusion criteria
    Selected Key Exclusion Criteria:
    • Presence of other form(s) of cardiomyopathy contributing to HF (e.g., inflammatory or infiltrative cardiomyopathy) or clinically significant cardiac anatomic abnormality (e.g., LV aneurysm).
    • Clinically significant coronary artery disease (e.g., coronary revascularization, exercise-induced angina) per Investigator judgment.
    • Uncorrected, hemodynamically significant (i.e., moderatesevere) primary structural valvular disease not due to HF.
    • Currently receiving or deemed at high risk of requiring chronic renal replacement therapy (e.g., hemodialysis or peritoneal dialysis) within 6 months.
    • Treatment with any investigational agent(s) for HF within 28 days prior to Day 1. Any treatment with an investigational agent(s) requires approval from the Medical Monitor.
    • Malignancy that is active or has been diagnosed within 3 years prior to screening, except surgically curatively resected in situ malignancies or surgically cured early breast cancer, prostate cancer, skin cancer (basal cell carcinoma, squamous cell carcinoma) or cervical cancer.
    • Non-cardiac condition that limits lifespan to < 1 year.
    • Serum positive for hepatitis B surface antigen, viremic hepatitis C, or human immunodeficiency virus (HIV) at screening.
    Selección de Criterios de Exclusión clave:
    - Presencia de otras formas de miocardiopatía que contribuyan a la IC (por ejemplo, miocardiopatía inflamatoria o infiltrativa) o de una anomalía anatómica del corazón clínicamente significativa (por ejemplo, aneurisma del VI).
    - Enfermedad coronaria clínicamente significativa (por ejemplo, revascularización coronaria, angina inducida por el ejercicio) a juicio del investigador.
    - Enfermedad valvular estructural primaria no corregida y hemodinámicamente significativa (es decir, moderada-grave) no debida a la IC.
    - Tratamiento actual o riesgo alto de precisar tratamiento crónico de sustitución renal (por ejemplo, hemodiálisis o diálisis peritoneal) en el plazo de 6 meses.
    - Tratamiento con cualquier fármaco en investigación para la IC en los 28 días previos al día 1. Cualquier tratamiento con un fármaco en investigación que requiera la aprobación del monitor médico.
    - Neoplasia maligna activa o que se haya diagnosticado en los 3 años previos a la selección, excepto los cánceres in situ quirúrgicamente resecados de forma curativa, el cáncer de mama incipiente quirúrgicamente curado, el cáncer de próstata, el cáncer de piel (carcinoma basocelular, carcinoma epidermoide) o el cáncer de cuello uterino.
    - Afección no cardiaca que limite la esperanza de vida a < 1 año.
    - Positividad sérica del antígeno de superficie del virus de la hepatitis B, hepatitis C virémica o virus de la inmunodeficiencia humana (VIH) en la selección.
    E.5 End points
    E.5.1Primary end point(s)
    NYHA Class II/III patients only: Change from baseline in
    6MWT at Week 12
    Pacientes Clase II/III según NYHA sólo: Variación de la PM6M entre el momento basal y la semana 12
    E.5.1.1Timepoint(s) of evaluation of this end point
    At week 12
    Semana 12
    E.5.2Secondary end point(s)
    1. NYHA Class II/III patients only: Change from baseline in 6MWT at Weeks 4 and 24
    2. NYHA Class II/III patients only: Change from baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Physical Limitation (PL) and Total Symptom Score (TSS) domains at Weeks 12 and 24
    3. NYHA Class II/III patients only: Change from baseline in Patient Global Impression (PGI) scores at Weeks 12 and 24
    - Patient Global Impression of Severity (PGI-S)
    - Patient Global Impression of Change (PGI-C)
    4. NYHA Class II/III patients only: Change from baseline in N-terminal pro-brain natriuretic peptide (NT-proBNP) at Weeks 4, 12 and 24
    5. Plasma concentrations of ARRY-371797 and metabolites (AR00420643, AR00428028 and AR00486705) predose and at a single time point postdose on specified visit days
    6. Safety as determined by:
    - Hospitalization-free survival (HFS): Defined as the time from randomization to heart failure (HF)-related
    hospitalization or death due to any cause
    - Overall survival (OS)
    - Incidence and severity of adverse events (AEs)
    - Changes in clinical safety laboratory tests, vital signs and 12-lead electrocardiograms (ECGs)
    - Incidence and severity of ventricular or atrial arrhythmias detected clinically using existing implantable cardioverter defibrillator (ICD)/cardiac resynchronization therapy defibrillator (CRT-D) or other applicable device interrogations at Weeks 12 and 24, if applicable
    1. Solo pacientes de clase II/III de la NYHA: Variación de la PM6M entre el momento basal y las semanas 4 y 24
    2. Solo pacientes de clase II/III de la NYHA: Variación de los dominios de limitación física (PL) y puntuación sintomática total (TSS) del Kansas City Cardiomyopathy Questionnaire (KCCQ) entre el momento basal y las semanas 12 y 24
    3. Solo pacientes de clase II/III de la NYHA: Variación de las puntuaciones del cuestionario «Impresión global del paciente» (PGI) entre el momento basal y las semanas 12 y 24.
    - Impresión global de la gravedad percibida por el paciente (PGI-S)
    - Impresión global del cambio percibido por el paciente (PGI C).
    4. Solo pacientes de clase II/III de la NYHA: Variación del propéptido natriurético cerebral N terminal (NT-proBNP) entre el momento basal y las semanas 4, 12 y 24
    5. Concentraciones plasmáticas de ARRY-371797 y sus metabolitos (AR00420643, AR00428028 y AR00486705) antes de la administración y en un único momento posterior a la administración en determinados días de visita
    6. Seguridad determinada por:
    - Supervivencia sin hospitalización (SSH): Se define como el tiempo transcurrido desde la aleatorización hasta la hospitalización por insuficiencia cardiaca (IC) o la muerte por cualquier causa
    - Supervivencia global (SG)
    - Incidencia y gravedad de los acontecimientos adversos (AA)
    - Variaciones de los análisis clínicos de seguridad, las constantes vitales y los electrocardiogramas de 12 derivaciones (ECG)
    - Incidencia y gravedad de las arritmias ventriculares o auriculares detectadas clínicamente con el desfibrilador cardioversor implantable (DCI)/desfibrilador con tratamiento de resincronización cardiaca (D-TRC) existente o con los sondeos de otros dispositivos al uso en las semanas 12 y 24, si procede
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Weeks 4 and 24
    2. Weeks 12 and 24
    3. Weeks 12 and 24
    4. Weeks 4, 12 and 24
    5. Predose and at a single time point postdose on specified visit days
    6. Safety endpoints throughout the duration of the study
    1. Semanas 4 y 24
    2. Semanas 12 y 24
    3. Semanas 12 y 24
    4. Semanas 4, 12 y 24
    5. Predosis y un único momento post dosis en los días de visita especificados
    6. Objetivos de seguridad a lo largo de la duración 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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    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 Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Periodo de tratamiento aleatorizado y doble ciego, seguido de un periodo de tratamiento en abierto
    Randomized, double-blind treatment period followed by open-label treatment period
    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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA50
    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
    Belgium
    Canada
    Denmark
    France
    Germany
    Italy
    Mexico
    Netherlands
    Norway
    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
    The end of the study is reached when all patients in the open-label treatment period have had the opportunity to complete the open-label Week 24 Visit or have discontinued from the study, whichever comes first
    El final del estudio se alcanzará cuando todos los pacientes del periodo de tratamiento abierto hayan tenido oportunidad de completar la visita de la semana 24 en régimen abierto o hayan abandonado el estudio, lo que suceda primero
    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 months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    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: 140
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 123
    F.4.2.2In the whole clinical trial 160
    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)
    Patients who remain on treatment at the end of the study and who may, in the opinion of the Investigator, derive benefit from continued treatment with ARRY-371797 will be provided the opportunity to continue treatment with ARRY-371797
    Los pacientes que estén todavía en tratamiento al final del estudio y que, en opinión del investigador, obtengan un beneficio derivado del tratamiento continuado con ARRY-371797, serán provistos de la posibilidad de seguir con el tratamiento con ARRY-371797
    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 Decision2018-07-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-06-25
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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