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    Summary
    EudraCT Number:2015-000344-42
    Sponsor's Protocol Code Number:AG-221-AML-004
    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:2015-10-29
    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 number2015-000344-42
    A.3Full title of the trial
    A Phase 3, Multicenter, Open-label, Randomized Study Comparing the Efficacy and Safety of AG-221 (CC-90007) Versus Conventional Care Regimens in Older Subjects with Late Stage Acute Myeloid Leukemia Harboring an Isocitrate Dehydrogenase 2 Mutation.
    Estudio de fase 3, multicéntrico, abierto, aleatorizado para comparar la eficacia y la seguridad de AG-221 (CC-90007) en comparación con la asistencia habitual en pacientes de edad avanzada con leucemia mieloide aguda terminal que llevan una mutación de la isocitrato deshidrogenasa 2.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial to compare the efficacy and safety of AG-221 (CC-90007) versus conventional treatment on older subjects with late stage acute myeloid leukemia harboring an Isocitrate Dehydrogenase 2 Mutation
    Ensayo Clínico para comparar la eficacia y la seguridad de AG-221 (CC-90007) en comparación con la asistencia habitual en pacientes de edad avanzada con leucemia mieloide aguda terminal que llevan una mutación de la isocitrato deshidrogenasa 2.
    A.3.2Name or abbreviated title of the trial where available
    The ?IDHENTIFY? Trial
    Ensayo "IDHENTIFY"
    A.4.1Sponsor's protocol code numberAG-221-AML-004
    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 SponsorCelgene Corporation
    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 supportCelgene Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCelgene Corporation
    B.5.2Functional name of contact pointClinicalTrialDisclosure
    B.5.3 Address:
    B.5.3.1Street Address9225 Indian Creek Parkway, Suite 900
    B.5.3.2Town/ cityOverland Park, Kansas
    B.5.3.3Post code66210
    B.5.3.4CountryUnited States
    B.5.4Telephone number+34914229000
    B.5.5Fax number+1913-266-0394
    B.5.6E-mailClinicalTrialDisclosure@celgene.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 nameAG-221
    D.3.2Product code AG-221
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAG-221 mesylate
    D.3.9.2Current sponsor codeAG-221
    D.3.9.3Other descriptive nameAG-221
    D.3.9.4EV Substance CodeSUB123982
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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 nameAG-221
    D.3.2Product code AG-221
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAG-221 mesylate
    D.3.9.2Current sponsor codeAG-221
    D.3.9.3Other descriptive nameAG-221
    D.3.9.4EV Substance CodeSUB123982
    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.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 nameAG-221
    D.3.2Product code AG-221
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAG-221 mesylate
    D.3.9.2Current sponsor codeAG-221
    D.3.9.3Other descriptive nameAG-221
    D.3.9.4EV Substance CodeSUB123982
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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 nameAG-221
    D.3.2Product code AG-221
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAG-221 mesylate
    D.3.9.2Current sponsor codeAG-221
    D.3.9.3Other descriptive nameAG-221
    D.3.9.4EV Substance CodeSUB123982
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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 RoleComparator
    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 Yes
    D.2.1.1.1Trade name CYTARABINE ACCORD 100 mg/ml Solution for Injection or Infusion
    D.2.1.1.2Name of the Marketing Authorisation holderACCORD HEALTHCARE FRANCE SAS
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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.3.2Product code Cytarabine
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCytarabine
    D.3.9.2Current sponsor codeCytarabine
    D.3.9.3Other descriptive nameCYTARABINE
    D.3.9.4EV Substance CodeSUB06880MIG
    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.IMP: 6
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.1Trade name Cytarabine 100 mg/ml Solution for Injection or Infusion
    D.2.1.1.2Name of the Marketing Authorisation holderAccord Healthcare Limited
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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.3.2Product code Cytarabine
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCytarabine
    D.3.9.2Current sponsor codeCytarabine
    D.3.9.3Other descriptive nameCYTARABINE
    D.3.9.4EV Substance CodeSUB06880MIG
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(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.IMP: 7
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.1Trade name Cytarabine 100 mg/ml Solution for Injection or Infusion
    D.2.1.1.2Name of the Marketing Authorisation holderAccord Healthcare Limited
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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.3.2Product code Cytarabine
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCytarabine
    D.3.9.2Current sponsor codeCytarabine
    D.3.9.3Other descriptive nameCYTARABINE
    D.3.9.4EV Substance CodeSUB06880MIG
    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.IMP: 8
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.1Trade name Cytarabine Accord 100 mg/ml solution for injection or infusion
    D.2.1.1.2Name of the Marketing Authorisation holderAccord Healthcare Limited
    D.2.1.2Country which granted the Marketing AuthorisationHungary
    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.3.2Product code Cytarabine
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCytarabine
    D.3.9.2Current sponsor codeCytarabine
    D.3.9.3Other descriptive nameCYTARABINE
    D.3.9.4EV Substance CodeSUB06880MIG
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(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.IMP: 9
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.1Trade name Vidaza 25mg/ml powder for suspension for injection
    D.2.1.1.2Name of the Marketing Authorisation holderCelgene Europe Ltd
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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/3/07/509
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Powder for suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAZACITIDINE
    D.3.9.1CAS number 320-67-2
    D.3.9.4EV Substance CodeSUB05624MIG
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Treatment of subjects 60 years or older with acute myeloid leukemia (AML) refractory to or relapsed after second- or third-line AML therapy and positive for an isocitrate dehydrogenase 2 (IDH2) mutation
    Tratamiento de pacientes mayores de 60 años con leucemia mieloide aguda (LMA) recidivante o resistente al tratamiento de segunda o tercera línea y con mutación del gen de la isocitrato deshidrogenasa 2
    (IDH2).
    E.1.1.1Medical condition in easily understood language
    Treatment of subjects 60 years or older with AML that does not respond to or that came back after 2 or 3 different AML therapies and that presents a mutation on isocitrate dehydrogenase 2 (IDH2)
    Tratamiento de pacientes mayores de 60 años con LMA recidivante o resistente a 2 o 3 tratamientos para la LMA diferentes y que presenten mutación de la isocitrato deshidrogenasa 2 (IDH2).
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    E.1.2Level LLT
    E.1.2Classification code 10060558
    E.1.2Term Acute myeloid leukemia recurrent
    E.1.2System Organ Class 100000004864
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    E.1.2Level LLT
    E.1.2Classification code 10000886
    E.1.2Term Acute myeloid 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
    To determine the primary efficacy, measured as overall survival (OS), of AG-221 compared with conventional care regimens (CCRs) in subjects 60 years or older with AML refractory to or relapsed after second- or third-line AML therapy and positive for an IDH2 mutation
    Analizar la eficacia principal, determinada por la supervivencia global (SG), de AG-221 en comparación con los regímenes de tratamiento habituales (RTH) en
    pacientes mayores de 60 años con LMA recidivante o resistente al tratamiento de segunda o tercera línea y con mutación de IDH2.
    E.2.2Secondary objectives of the trial
    -To determine the supporting efficacy of AG-221 compared with CCRs
    -To determine the safety and tolerability of AG-221 compared with CCRs
    -To determine the effect of AG-221 compared with CCRs on Health-related Quality-of-Life
    - Determinar la eficacia complementaria de AG-221 en comparación con los RTH.
    - Determinar la seguridad y tolerabilidad de AG-221 en comparación con los RTH.
    - Determinar el efecto de AG-221 sobre la calidad de vida relacionada con la salud (CVDRS) en comparación con los RTH.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject is ? 60 years of age at the time of signing the ICF
    2. Subject has primary (ie, de novo) or secondary (progression of MDS or
    myeloproliferative neoplasms ([MPN], or therapy-related) AML according to WHO
    classification
    3. Subject has received second- or third-line/regimen of AML therapy
    4. Subject has the following disease status:
    a. Refractory to or relapsed after second- or third-line/regimen of intensive therapy for AML (eg, the ?7 + 3? regimen): at least 5% leukemic blasts in bone marrow; or
    b. Refractory to or relapsed after second- or third-line low-intensity AML therapy
    (eg, LDAC, azacitidine or decitabine): at least 5% leukemic blasts in bone marrow after at least 2 treatment cycles
    5. Subject is eligible for and willing to receive the pre-selected CCR treatment option, according to the investigator's assessment
    6. Subject has Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
    7. Subject has IDH2 gene mutations tested centrally (using the ?investigational use only? PCR assay, Abbott RealTime IDH2) in samples of bone marrow aspirate and peripheral blood, and confirmed positive in bone marrow aspirate and/or peripheral blood
    8. Subject has adequate organ function defined as:
    - Aspartate aminotransferase (AST)/serum glutamic oxaloacetic transaminase (SGOT) and alanine aminotransferase (ALT)/serum glutamic pyruvic transaminase (SGPT) ? 3 x upper limit of normal (ULN), unless considered due to leukemic organ
    involvement, following review by the Medical Monitor; and
    - Serum total bilirubin ? 1.5 x ULN, unless considered due to Gilbert?s syndrome (eg, a gene mutation in UGT1A1) or leukemic organ involvement, following review by the Medical Monitor; and
    - Creatinine clearance >30 mL/min based on the Cockcroft-Gault glomerular filtration rate estimation: (140 - Age)x(weight in kg)x(0.85 if female)/72xserum creatinine
    1. Edad mínima de 60 años en el momento de firmar el DCI.
    2. LMA primaria (es decir, de novo) o secundaria (progresión de un SMD o una neoplasia
    mieloproliferativa [NMP], o debida a un tratamiento previo) conforme a la clasificación de la OMS.
    3. Haber recibido un régimen de segunda o tercera línea para el tratamiento de la LMA.
    4. Uno de los estados de enfermedad siguientes:
    a. Resistencia o recidiva tras un régimen de segunda o tercera línea de tratamiento intensivo para la LMA (por ejemplo, el régimen "7 + 3"): al menos un 5 % de blastos leucémicos en la médula ósea.
    b. Resistencia o recidiva tras un régimen de segunda o tercera línea de tratamiento de baja intensidad para la LMA (por ejemplo, LDAC, azacitidina o decitabina):
    al menos un 5 % de blastos leucémicos en la médula ósea después de al menos dos ciclos de tratamiento.
    5. Cumplir los requisitos para recibir la opción de RTH elegida conforme a la evaluación del investigador, y estar dispuesto a ello.
    6. Estado funcional de 0, 1 ó 2 según el Eastern Cooperative Oncology Group (ECOG).
    7. Haberse realizado un análisis centralizado de las mutaciones de IDH2 (mediante el
    análisis de PCR RealTime IDH2 de Abbott, "de uso exclusivamente experimental") en muestras de aspirado de médula ósea y sangre periférica que confirme la positividad en el aspirado de médula ósea y/o en sangre periférica.
    8. Función orgánica adecuada, definida como:
    - Aspartato aminotransferasa (AST)/transaminasa glutámicooxaloacética sérica (SGOT)/ y alanina aminotransferasa
    (ALT)/transaminasa glutámico-pirúvica sérica (SGPT) ? 3 veces el límite superior de la normalidad (LSN), a menos que se considere debido a una afectación orgánica de la leucemia, previa evaluación por parte del
    monitor médico; y Bilirrubina total en suero ? 1,5 veces el LSN, a menos que se considere debido a un síndrome de Gilbert (por ejemplo, mutación de UGT1A1) o una afectación orgánica de la leucemia, previa evaluación por parte del monitor médico; y
    - Aclaramiento de creatinina 30 ml/min calculado según la ecuación de filtración glomerular de Cockcroft-Gault: (140 ? edad) x (peso en kg) x (0,85 en las
    mujeres)/72 x creatinina sérica.
    E.4Principal exclusion criteria
    1. Subject is suspected or proven to have acute promyelocytic leukemia based on
    morphology, immunophenotype, molecular assay, or karyotype
    2. Subject has AML secondary to chronic myelogenous leukemia
    3. Subject has received a targeted agent against an IDH2 mutation
    4. Subject has received systemic anticancer therapy or radiotherapy < 14 days prior to the start of study treatment. Note that hydroxyurea is allowed prior to the start of study treatment for the control of leukocytosis in subjects with white blood cell (WBC) counts> 30 x 10^9/L (however, hydroxyurea should not be given within 72 hours prior to and after administration of azacitidine)
    5. Subject has received non-cytotoxic or investigational agents < 14 days or 5 half-lives, whichever is longer, prior to the start of study treatment
    6. Subject has undergone HSCT within 60 days prior to the start of study treatment, or on immunosuppressive therapy post HSCT at the time of screening, or with clinically significant graft-versus-host disease (GVHD). The use of a stable dose of oral steroid post-HSCT and/or topical steroids for ongoing skin GVHD is permitted.
    7. Subject has persistent, clinically significant non-hematologic toxicities from prior
    therapies
    8. Subject has or is suspected of having central nervous system (CNS) leukemia.
    Evaluation of cerebrospinal fluid is only required if CNS involvement by leukemia is
    suspected during screening.
    1. Leucemia promeliocítica aguda presunta o confirmada por la morfología, el inmunofenotipo, el análisis molecular o el cariotipo
    2. LMA secundaria a una leucemia mielógena crónica
    3. Haber recibido un fármaco dirigido contra la IDH2 mutada.
    4. Haber recibido tratamiento antineoplásico sistémico o radioterapia menos de 14 días antes del comienzo del tratamiento del estudio. Se permite la administración de hidroxiurea antes de empezar el tratamiento del estudio para controlar la leucocitosis en pacientes con
    cifras de leucocitos > 30 x 109/l (sin embargo, no debe administrarse en las 72 horas previas y posteriores a la administración de azacitidina).
    5. Haber recibido fármacos no citototóxicos o experimentales menos de 14 días o 5 semividas antes del comienzo del tratamiento del estudio, lo
    que suponga más tiempo
    6. Haberse sometido a un TCPH en los 60 días anteriores al comienzo del tratamiento del estudio, estar en tratamiento inmunodepresor posterior al TCPH en el momento de la selección o presentar una enfermedad del injerto contra el huésped (EICH) de importancia clínica. Se permite el tratamiento con esteroides orales en dosis estables después de un TCPH
    o con esteroides tópicos para la EICH cutánea.
    7. Toxicidad no hematológica persistente y de importancia clínica debida a tratamientos anteriores.
    8. Leucemia del sistema nervioso central (SNC) presunta o confirmada. Durante la selección se hará un análisis del líquido cefalorraquídeo únicamente si se sospecha afectación del SNC.
    E.5 End points
    E.5.1Primary end point(s)
    Overall survival
    Supervivencia Global
    E.5.1.1Timepoint(s) of evaluation of this end point
    Time from randomization to death due to any cause (up to approximately 28 months)
    Tiempo transcurrido desde la aleatorización hasta la fecha de muerte por cualquier causa (Aproximadamente 28 meses).
    E.5.2Secondary end point(s)
    1.Overall response rate
    2.Event-free survival
    3.Duration of response
    4.Time to response
    5.Treatment mortality at 30 and 60 days
    6.One-year survival
    7.Overall remission rate
    8.Complete remission rate
    9.Hematologic improvement rate
    10.Rate of HSCT
    11.Time to treatment failure
    12.Safety and tolerability
    13.HRQoL
    1. Tasa de respuesta global
    2. Supervivencia sin acontecimientos
    3. Duración de la respuesta
    4. Tiempo hasta la respuesta
    5. Mortalidad del tratamiento a los 30 y 60 días
    6. Supervivencia a un año
    7. Tasa de remisión global
    8. Tasa de remisión completa
    9. Tasa de mejoría hematológica
    10. Tasa de TCPH
    11. Tiempotranscurrido hasta
    el fracaso del tratamiento
    12. Seguridad y tolerabilidad
    13. CVRS
    E.5.2.1Timepoint(s) of evaluation of this end point
    1.up to approximately 28 months
    2.up to approximately 28 months
    3.up to approximately 28 months
    4.up to approximately 28 months
    5.At 30 and 60 days after treatment start
    6.up to approximately 28 months
    7.up to approximately 28 months
    8.up to approximately 28 months
    9.up to approximately 28 months
    10.up to approximately 28 months
    11.up to approximately 28 months
    12.up to approximately 28 months
    13.up to approximately 28 months
    1. Hasta aproximadamente 28 meses
    2. Hasta aproximadamente 28 meses
    3. Hasta aproximadamente 28 meses
    4. Hasta aproximadamente 28 meses
    5. En los días 30 y 60 despues del comienzo del tratamiento.
    6. Hasta aproximadamente 28 meses
    7. Hasta aproximadamente 28 meses
    8. Hasta aproximadamente 28 meses
    9. Hasta aproximadamente 28 meses
    10. Hasta aproximadamente 28 meses
    11. Hasta aproximadamente 28 meses
    12. Hasta aproximadamente 28 meses
    13. Hasta aproximadamente 28 meses
    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 Yes
    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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    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) Yes
    E.8.2.2Placebo No
    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 concerned9
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA74
    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
    Austria
    Belgium
    Brazil
    Canada
    Denmark
    France
    Germany
    Italy
    Japan
    Korea, Republic of
    Russian Federation
    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 Trial is defined as either the date of the last visit of the last subject to complete the post-treatment follow-up, or the date of receipt of the last data point from the last subject that is required for primary, secondary and/or exploratory analysis, as prespecified in the protocol, whichever is the later date.
    El fin del estudio está definido como la fecha de la última visita del último sujeto en completar el seguimiento de post-tratamiento, o la fecha de recogida del último punto de datos del último paciente que se requiera para un análisis primario, secundario y / o análisis exploratorio, como está especificado previamente en el protocolo, lo
    que suceda más tarde.
    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 months11
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months11
    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: 56
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 224
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2015-10-29. Yes
    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 state90
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 175
    F.4.2.2In the whole clinical trial 280
    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 subject's treatment after ending participation in the trial will be at the discretion of the treating physician.
    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 Decision2016-01-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-12-11
    P. End of Trial
    P.End of Trial StatusOngoing
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