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    Summary
    EudraCT Number:2020-006057-21
    Sponsor's Protocol Code Number:CABL001A2302
    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:2021-07-27
    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 number2020-006057-21
    A.3Full title of the trial
    A phase 3b, multi-center, open-label, treatment optimization study of oral asciminib in patients with Chronic Myelogenous Leukemia in chronic phase (CML-CP) previously treated with 2 or more tyrosine kinase inhibitors
    Estudio de fase IIIb, multicéntrico, abierto y de optimización del tratamiento con asciminib oral en pacientes con leucemia mieloide crónica en fase crónica previamente tratados con 2 o más inhibidores tirosina quinasa
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Asciminib treatment optimization in ≥ 3rd line CML-CP
    Optimización del tratamiento con asciminib en LMC-FC en >=3.ª línea
    A.4.1Sponsor's protocol code numberCABL001A2302
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04948333
    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 SponsorNovartis Farmacéutica S.A.
    B.1.3.4CountrySpain
    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 supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Farmacéutica S.A.
    B.5.2Functional name of contact pointTrial Monitoring Organization (TMo)
    B.5.3 Address:
    B.5.3.1Street AddressGran Via de les Corts Catalanes, 764
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08013
    B.5.3.4CountrySpain
    B.5.4Telephone number+34900353036
    B.5.5Fax number+34932479903
    B.5.6E-maileecc.novartis@novartis.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/3/20/2261
    D.3 Description of the IMP
    D.3.1Product nameAsciminib
    D.3.2Product code ABL001
    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 INNASCIMINIB
    D.3.9.2Current sponsor codeABL001
    D.3.9.4EV Substance CodeSUB188597
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/20/2261
    D.3 Description of the IMP
    D.3.1Product nameAsciminib
    D.3.2Product code ABL001
    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 INNASCIMINIB
    D.3.9.2Current sponsor codeABL001
    D.3.9.4EV Substance CodeSUB188597
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Chronic Myelogenous Leukemia in chronic phase (CML-CP)
    Leucemia mieloide crónica en fase
    crónica (LMC-FC)
    E.1.1.1Medical condition in easily understood language
    CML is a bone marrow cancer caused by a gene mutation which causes over growth of white blood cells.
    LMC es un cáncer de médula ósea causado por mutación genética que causa un crecimiento excesivo de los glóbulos blancos.
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10009012
    E.1.2Term Chronic myelogenous leukemia
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to estimate the molecular response
    rate (MMR) of all the patients at week 48 with CML-CP following two or
    more prior TKI treatments and with no evidence of MMR at baseline.
    El objetivo principal del estudio es estimar la tasa de respuesta molecular mayor (RMM) de todos los pacientes con LMC-FC en la semana 48 tras dos o más tratamientos previos con ITC y sin evidencia de RMM en la basal.
    E.2.2Secondary objectives of the trial
    To evaluate the safety and tolerability of asciminib in patients with CML-CP following two or more prior TKI treatments.
    To assess the rate of MMR in patients without MMR at baseline and at alternative time points at weeks 12, 24, 36, 72, 96 and 144
    To assess the rate of MMR at week 48 for patients with MMR at baseline
    To assess the time to MMR
    To assess the rate of early responses of BCR-ABL1 ≤10% and ≤1% at weeks 12, 24, 36 and 48
    To assess the rate of deep molecular responses (MR4 and MR4.5) at weeks 12, 24, 36, 48, 72, 96 and 144
    To assess cytogenetic response (% Ph+ metaphases) at weeks 48 and EOT.
    To characterize the impact of additional cytogenetic abnormalities on efficacy
    To assess cumulative molecular responses by all-time points
    To assess duration of MMR
    To assess sustained deep molecular responses as prerequisite for Treatment Free Remission (TFR)
    To assess rate of progressions (PFS)
    (Please refer to the protocol Table 2-1 for the full list)
    Evaluar la seguridad y la tolerabilidad de asciminib en pacientes con LMC-FC tras dos o más tratamientos previos con ITC.
    Evaluar la tasa de RMM en pacientes sin RMM en la basal y adicionalmente en las semanas 12, 24, 36, 72, 96 y 144.
    Evaluar la tasa de RMM en la semana 48 para pacientes con RMM en la basal.
    Evaluar el tiempo hasta la RMM.
    Evaluar tasa de respuestas tempranas de BCR-ABL1 <=10 % y <=1 % en las semanas 12, 24, 36 y 48.
    Evaluar tasa de respuestas moleculares profundas (RM4 y RM4.5) en las semanas 12, 24, 36, 48, 72, 96 y 144.
    Evaluar la respuesta citogenética (% de metafases Ph+) en las semanas 48 y EOT.
    Caracterizar el impacto de anomalías citogenéticas adicionales en la eficacia.
    Evaluar respuestas moleculares acumuladas para todos los tiempos.
    Evaluar duración de la RMM.
    Evaluar respuestas moleculares profundas sostenidas como requisito previo para la remisión libre de tratamiento (TFR)
    (Por favor consultar la Tabla 2-1 del protocolo para la lista completa)
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    'Single cell signaling and immune profiling sub-study to CABL001A2302'
    Anticipated start date: Oct 2021
    Objectives: Identify features of single cell immune and signaling profiles early after initiating asciminib treatment that predict: BCR-ABL1 (IS) level at 48 weeks (MMR or not), the decrease of BCR-ABL (IS) level during the first 6 months, AEs and patient reported outcomes
    "Subestudio de señalización unicelular y perfil inmunológico para CABL001A2302".
    Fecha comienzo anticipada: Oct 2021
    Objetivos: Identificar las características de los perfiles unicelulares inmunes y de señalización tempranos después de iniciar tratamiento con asciminib que predicen: nivel de BCR-ABL (IS) a las 48 semanas (RMM o no), la disminución de nivel de BCR-ABL (IS) durante los primeros 6 meses, AEs y resultados informados por paciente.
    E.3Principal inclusion criteria
    Male or female patients with a diagnosis of CML-CP ≥ 18 years of age
    Treatment with a minimum of 2 or more prior TKIs (i.e. imatinib, nilotinib, dasatinib, bosutinib, radotinib or ponatinib)
    Warning or failure (adapted from the 2020 ELN Recommendations) or intolerance to the most recent TKI therapy at the time of screening
    Adequate end organ function (as per central laboratory tests)
    Other protocol defined inclusion criteria may apply.
    Pacientes de ambos sexos con diagnóstico de LMC-FC >=18 años de edad.
    Tratamiento con un mínimo de 2 o más ITC previos (es decir, imatinib, nilotinib, dasatinib, bosutinib, radotinib o ponatinib).
    Alerta o fracaso (adaptado a partir de las recomendaciones de la ELN 2020) o intolerancia al tratamiento con ITC más reciente en el momento de la selección.
    Función adecuada de los órganos (según las pruebas del laboratorio central)
    Pueden aplicar otros criterios de inclusión definidos en el protocolo
    E.4Principal exclusion criteria
    Known presence of the BCR-ABL1 T315I mutation at any time prior to study entry
    Known history of AP/BC
    Previous treatment with a hematopoietic stem-cell transplantation
    Patient planning to undergo allogeneic hematopoietic stem cell transplantation
    Uncontrolled cardiac repolarization abnormality
    Severe and/or uncontrolled concurrent medical disease that in the opinion of the investigator could cause unacceptable safety risks or compromise compliance with the protocol
    History of acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis
    History of ongoing active acute or chronic liver disease
    Other protocol defined exclusion criteria may apply
    Presencia conocida de la mutación T315I en BCR-ABL1 en cualquier
    momento antes de entrar en el estudio.
    Antecedentes conocidos de FA/CB.
    Tratamiento previo con un trasplante de células madre hematopoyéticas.
    Paciente que tenga previsto someterse a un trasplante de células
    madre hematopoyéticas.
    Anomalía de la repolarización cardíaca no controlada.
    Enfermedad recurrente grave o no controlada que, según el criterio del investigador, podría causar riesgos de seguridad inaceptables o comprometer el cumplimiento del protocolo.
    Antecedentes de pancreatitis aguda durante el año anterior a la inclusión en el estudio o antecedentes de pancreatitis crónica.
    Antecedentes de enfermedad hepática aguda activa en curso o crónica.
    Pueden aplicar otros criterios de exclusión definidos en el protocolo
    E.5 End points
    E.5.1Primary end point(s)
    Major Molecular Response (MMR) rate at week 48.
    tasa de respuesta molecular mayor (RMM) en la semana 48.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 48
    Semana 48
    E.5.2Secondary end point(s)
    MMR rate at weeks 12, 24, 36, 72, 96 and 144.
    MMR rate at week 48 for patients with MMR at baseline.
    Time from the date of randomization to the date of first documented MMR
    Rate of BCR-ABL1 ≤ 10% and ≤1% at weeks 12, 24, 36 and 48
    Rate of MR4 and MR4.5 at weeks 12, 24, 36, 48, 72, 96 and 144.
    Rate of complete cytogenetic response (CCyR) at weeks 48 and EOT.
    Additional chromosomal abnormalities and occurrence of high-risk additional chromosomal abnormalities (ACAs)
    Rate of BCR-ABL1 ≤ 10%, BCR-ABL1 ≤1%, MMR, MR4 and MR4.5 by all-time points.
    Duration of MMR.
    Duration of MR4 without loss of MMR.
    Time from the date of randomization to the earliest occurrence of documented disease progression to AP/BC or the date of death from any cause.
    Time from randomization to death.
    Time from randomization to treatment failure define as BCR-ABL1 > 1%.
    Change in symptom burden and interference from baseline over time according to the MDASI-CML PRO instrument.
    Tasa de RMM en las semanas 12, 24, 36, 72, 96 y 144.
    Tasa de RMM en la semana 48 para pacientes con RMM en la basal.
    Tiempo desde la randomización hasta la fecha de la primera RMM.
    Tasa de BCR-ABL1 <=10 % y <=1 % en las semanas 12, 24, 36 y 48.
    Tasa de RM4 y RM4.5 en las semanas 12, 24, 36, 48, 72, 96 y 144.
    Tasa de respuesta citogenética completa (% de metafases Ph+) en las semanas 48 y EOT.
    Anomalías cromosómicas adicionales y aparición de anomalías cromosómicas adicionales de alto riesgo.
    Tasa de BCR-ABL1 <= 10%, BCR-ABL1 <=1%, MMR, MR4 and MR4.5 para todos los tiempos.
    Duración de la RMM.
    Duración de MR4 sin pérdida de MMR.
    Tiempo desde la fecha de aleatorización hasta la aparición de progresión de la enfermedad a FA/CB documentada o la fecha de muerte por cualquier causa.
    Tiempo desde la randomización a la muerte
    Tiempo hasta el fracaso del tratamiento (TTF) definido como BCR-ABL1 > 1%.
    Cambio en la carga de síntomas y la interferencia desde el inicio a lo largo de tiempo según el instrumento MDASI-CML PRO.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144/EOT.
    Semanas 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144/EOT.
    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 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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability, cytogenetics
    Tolerabilidad, citogenética
    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 Yes
    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 Yes
    E.8.1.7.1Other trial design description
    Régimen diferente
    Different regimen
    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 concerned5
    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
    Brazil
    Canada
    Korea, Republic of
    Malaysia
    Oman
    Russian Federation
    Singapore
    Vietnam
    Austria
    France
    Germany
    Italy
    Poland
    Spain
    United Kingdom
    Argentina
    Greece
    E.8.7Trial has a data monitoring committee No
    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
    LPLV
    último paciente última visita
    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 months7
    E.8.9.1In the Member State concerned days14
    E.8.9.2In all countries concerned by the trial years5
    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: 124
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 62
    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 95
    F.4.2.2In the whole clinical trial 186
    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)
    Please refer to the clinical protocol Section 6.1.4
    Por favor consultar la sección 6.1.4 del protocolo clínico
    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 Decision2021-11-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-09-13
    P. End of Trial
    P.End of Trial StatusOngoing
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