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    The EU Clinical Trials Register currently displays   37700   clinical trials with a EudraCT protocol, of which   6177   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2013-004890-28
    Sponsor's Protocol Code Number:AC220-007
    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:2014-04-25
    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 number2013-004890-28
    A.3Full title of the trial
    A Phase 3 Open-Label Randomized Study of Quizartinib (AC220) Monotherapy Versus Salvage Chemotherapy in Subjects with FLT3-ITD Positive Acute Myeloid Leukemia (AML) Refractory To or Relapsed After First-line Treatment With or Without Hematopoietic Stem Cell Transplant (HSCT) Consolidation.
    Estudio abierto y aleatorizado de fase III sobre quizartinib (AC220) en monoterapia frente a la quimioterapia de rescate en pacientes con leucemia mieloide aguda (LMA) FLT3-ITD positiva resistente o en recaída tras el tratamiento de primera línea con o sin consolidación con trasplante de células madre hematopoyéticas (TCMH)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 Open-Label clinical trial of the drug Quizartinib (AC220) compared to recovery treatment in patients with Acute Myeloid Leukemia (AML), a cancer of the blood.
    Estudio abierto de fase III sobre quizartinib (AC220) comparado con quimioterapia de rescate en pacientes con leucemia mieloide aguda (LMA), un cáncer de la sangre.
    A.4.1Sponsor's protocol code numberAC220-007
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02039726
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1151-8078
    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 SponsorAmbit Biosciences 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 supportAmbit Biosciences Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAmbit Biosciences Corporation
    B.5.2Functional name of contact pointPivotal
    B.5.3 Address:
    B.5.3.1Street Address11080 Roselle Street
    B.5.3.2Town/ citySan Diego CA
    B.5.3.3Post code92121
    B.5.3.4CountryUnited States
    B.5.4Telephone number0034917081250
    B.5.5Fax number0034917081301
    B.5.6E-maildfrano@ambitbio.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/3/09/622
    D.3 Description of the IMP
    D.3.1Product nameQuizartinib
    D.3.2Product code AC220
    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 INNQuizartinib
    D.3.9.1CAS number 1132827-21-4
    D.3.9.2Current sponsor codeAC220
    D.3.9.3Other descriptive nameQUIZARTINIB
    D.3.9.4EV Substance CodeSUB89721
    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.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/3/09/622
    D.3 Description of the IMP
    D.3.1Product nameQuizartinib
    D.3.2Product code AC220
    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 INNQuizartinib
    D.3.9.1CAS number 1132827-21-4
    D.3.9.2Current sponsor codeAC220
    D.3.9.3Other descriptive nameQUIZARTINIB
    D.3.9.4EV Substance CodeSUB89721
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    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 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.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameCytarabine
    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.3.9.1CAS number 147-94-4
    D.3.9.3Other descriptive nameCYTARABINE
    D.3.9.4EV Substance CodeSUB06880MIG
    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 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.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameMitoxantrone
    D.3.4Pharmaceutical form Concentrate for solution for 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 INNMitoxantrone
    D.3.9.1CAS number 65271-80-9
    D.3.9.3Other descriptive nameMITOXANTRONE
    D.3.9.4EV Substance CodeSUB09012MIG
    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.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameEtoposide
    D.3.4Pharmaceutical form Concentrate for solution for 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 INNETOPOSIDE
    D.3.9.3Other descriptive nameETOPOSIDE
    D.3.9.4EV Substance CodeSUB07337MIG
    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.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameCytarabine
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCytarabine
    D.3.9.3Other descriptive nameCYTARABINE
    D.3.9.4EV Substance CodeSUB06880MIG
    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.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 G-CSF
    D.3.4Pharmaceutical form Solution for injection
    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 INNG-CSF
    D.3.9.1CAS number 143011-72-7
    D.3.9.3Other descriptive nameGRANULOCYTE COLONY-STIMULATING FACTOR
    D.3.9.4EV Substance CodeSUB14018MIG
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameFludarabine
    D.3.4Pharmaceutical form Powder for 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 INNFLUDARABINE
    D.3.9.1CAS number 21679-14-1
    D.3.9.4EV Substance CodeSUB07678MIG
    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.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameCytarabine
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCytarabine
    D.3.9.3Other descriptive nameCYTARABINE
    D.3.9.4EV Substance CodeSUB06880MIG
    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: 10
    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.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameIdarubicin
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous drip use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNIdarubicin
    D.3.9.3Other descriptive nameIDARUBICIN
    D.3.9.4EV Substance CodeSUB08111MIG
    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
    Refractory or Relapsed FLT3-ITD Positive Acute Myeloid Leukemia (AML)
    Leucemia Mieloide Aguda (LMA) FLT3-ITD(+) resistente al tratamiento o en recaída.
    E.1.1.1Medical condition in easily understood language
    Acute Myeloid Leukemia (AML) a cancer of the blood.
    Leucemia Mieloide Aguda (LMA), un cáncer de la sangre.
    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 16.1
    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
    The primary objective of the study is to determine whether quizartinib monotherapy prolongs overall survival (OS) compared to salvage chemotherapy in subjects with FLT3-ITD positive AML who are refractory to or have relapsed within 6 months, after first-line AML therapy.
    El objetivo principal del estudio es determinar si quizartinib en monoterapia prolonga la supervivencia global (SG) en comparación con la quimioterapia de rescate en pacientes con LMA FLT3-ITD positiva que presentan resistencia al tratamiento o recaída en un plazo de 6 meses, después del tratamiento de primera línea para la LMA.
    E.2.2Secondary objectives of the trial
    The secondary objective is to determine event-free survival (EFS) with quizartinib versus salvage chemotherapy.
    El objetivo secundario es determinar la supervivencia libre de acontecimientos (SLA) con quizartinib frente a la quimioterapia de rescate.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Provision of written informed consent approved by the Institutional Review Board (IRB) or Independent Ethics Committee (IEC) with privacy language in accordance with national regulations (e.g., HIPAA authorization for US sites) prior to any study-related procedures, including withdrawal of prohibited medications if applicable.
    2. Age ?18 years at the time of informed consent.
    3. Morphologically documented primary AML or AML secondary to myelodysplastic syndrome (MDS), as defined by World Health Organization criteria, as determined by pathology review at the study site.
    4. Refractory or relapsed AML after first-line therapy, with or without HSCT. First-line therapy can consist of 1 or 2 induction blocks, and must have included at least 1 cycle of an anthracycline/mitoxantrone-containing induction block at a standard dose.
    ? Refractory to first-line therapy is defined as:
    After 1 cycle, lack of achievement of CR, CRp, or CRi and a reduction in bone marrow blasts of less than 50%.
    After 2 cycles, lack of achievement of CR, CRp, or CRi.
    ? Relapse within 6 months or less after first-line therapy is defined as (all criteria must be met):
    Achievement of CR, CRi, or CRp, as defined by 2003 International Working Group criteria (Section 8.2) after initial AML therapy with or without consolidation or maintenance, and with or without HSCT as consolidation
    Duration of CR, CRi or CRp is measured from the date of the bone marrow assessment which confirmed response to the date of the bone marrow assessment that identified relapse or the appearance of peripheral blasts
    5. Presence of the FLT3-ITD activating mutation in bone marrow or peripheral blood (allelic ratio as determined by a central laboratory with a cutoff of >3% FLT3ITD/total FLT3).
    6. Eligibility for pre-selected salvage chemotherapy, according to the Investigator?s assessment.
    7. ECOG performance score 0-2.
    8. Discontinuation of prior AML treatment before the start of study treatment (except hydroxyurea, which is permitted for blast control up to the day of starting study treatment) for at least 2 weeks for cytotoxic agents, or for at least 5 half-lives for non cytotoxic agents.
    9. Serum creatinine ?1.5×upper limit of normal (ULN), or glomerular filtration rate >25 mL/min/1.73m2, as calculated with the modified Cockcroft-Gault formula.
    10. Serum potassium, magnesium, and calcium (serum calcium corrected for hypoalbuminemia) within institutional normal limits. Subjects with electrolytes outside the normal range will be eligible if these values are corrected upon retesting following any necessary supplementation.
    11. Total serum bilirubin ?1.5×ULN.
    12. Serum aspartate transaminase (AST) and/or alanine transaminase (ALT) ?2.5×ULN.
    1. Otorgamiento del consentimiento informado por escrito aprobado por el Comité Ético de Investigación Clínica (CEIC) con referencia a la privacidad, según lo requerido por la normativa nacional (p. ej., autorización de la HIPAA para los centros en EE. UU.), antes de realizar cualquier procedimiento relacionado con el estudio, incluida la retirada de los medicamentos prohibidos, si procede.
    2. Edad ? 18 años en el momento de la firma del consentimiento informado.
    3. LMA primaria diagnosticada morfológicamente o LMA secundaria a síndrome mielodisplásico (SMD), conforme a la definición de los criterios de la Organización Mundial de la Salud, determinada mediante estudio anatomopatológico en el centro del estudio.
    4. LMA resistente al tratamiento o en recaída después del tratamiento de primera línea, con o sin TCMH. El tratamiento de primera línea puede consistir en 1 ó 2 bloques de inducción, con o sin consolidación. El tratamiento de inducción debe haber incluido un mínimo de 1 ciclo de una antraciclina/mitoxantrona con un bloque de inducción a una dosis habitual.
    ? La resistencia al tratamiento de primera línea se define como:
    o No haber alcanzado RC, RCp o RCi y una reducción de la cifra de blastos en la médula ósea inferior al 50 %, después de un ciclo de tratamiento.
    o No haber alcanzado RC, RCp o RCi después de 2 ciclos de tratamiento.
    ? La recaída en un plazo de 6 meses o menos después del tratamiento de primera línea se define como (se deben cumplir todos los criterios):
    o Consecución de RC, RCi o RCp, definidas según los criterios del Grupo Internacional de Trabajo de 2003, después del tratamiento inicial para la LMA con o sin consolidación o mantenimiento, y con o sin consolidación del TCMH.
    o La duración de la RC, RCi o RCp se mide desde la fecha del análisis de la médula ósea que confirma la respuesta hasta la fecha del análisis de la médula ósea que identifica la recaída o la aparición de blastos periféricos.
    5. Presencia de la mutación activadora FLT3-ITD en la médula ósea o en sangre periférica (índice alélico determinado por un laboratorio central con un valor de corte > 3 % de FLT3-ITD/FLT3 total).
    6. Elegibilidad para recibir la quimioterapia de rescate preseleccionada, según la evaluación del investigador.
    7. Estado funcional del ECOG de 0 - 2.
    8. Suspensión del tratamiento previo para la LMA antes del inicio del tratamiento del estudio (salvo en el caso de hidroxiurea, permitida para el control de los blastos hasta el día del inicio del tratamiento del estudio) durante un mínimo de 2 semanas en el caso de los agentes citotóxicos o durante un mínimo de 5 semividas en el caso de los agentes no citotóxicos.
    9. Creatinina sérica ? 1,5 veces el límite superior de la normalidad (LSN) o filtración glomerular > 25 ml/min/1,73 m2, calculada con la fórmula de Cockcroft-Gault modificada.
    10. Niveles séricos de potasio, magnesio y calcio y (calcio sérico corregido para hipoalbuminemia) dentro de los límites de la normalidad del centro. Los pacientes con niveles de electrólitos fuera de los límites normales serán elegibles para el estudio si estos valores se han corregido en una nueva determinación realizada después de la administración de cualquier aporte complementario necesario.
    11. Bilirrubina sérica total ? 1,5 veces el LSN.
    12. Aspartato-aminotransferasa (AST) y/o alanina-aminotransferasa (ALT) ? 2,5 veces el LSN.
    E.4Principal exclusion criteria
    1. Acute promyelocytic leukemia (AML subtype M3).
    2. AML secondary to prior chemotherapy for other neoplasms, except AML secondary to prior MDS.
    3. History of another malignancy, unless the candidate has been disease-free for at least 5 years.
    ? Candidates with treated non-melanoma skin cancer, carcinoma in situ, or cervical intraepithelial neoplasia are eligible regardless of the time spent disease-free, if they have completed definitive treatment.
    ? Candidates with organ-confined prostate cancer, with no evidence of recurrent or progressive disease, are eligible if hormonal therapy has been begun, or if thetumor has been surgically removed or treated with definitive radiotherapy.
    4. Persistent, clinically significant > Grade 1 non-hematologic toxicity from prior AML therapy.
    5. Clinically significant GVHD or GVHD requiring initiation of treatment or treatment escalation within 21 days, and/or > Grade 1 persistent or clinically significant nonhematologic toxicity related to HSCT.
    6. History of, or current, central nervous system involvement with AML.
    7. Clinically significant coagulation abnormality, such as disseminated intravascular coagulation.
    8. Prior treatment with quizartinib or participated in a prior quizartinib study.
    9. Known presence of a FLT3-D835 mutation at study enrollment. For a candidate who has received prior FLT3-targeted therapy (with the exception of midostaurin), the absence of a FLT3-D835 mutation at study enrollment must be documented.
    10. Major surgery within 4 weeks prior to screening.
    11. Radiation therapy within 4 weeks prior to screening.
    12. Uncontrolled or significant cardiovascular disease, including:
    ? QTcF interval >450 ms (average of triplicate determinations).
    ? Diagnosed or suspected long QT syndrome, or known family history of long QT syndrome.
    ? History of clinically relevant ventricular arrhythmias, such as ventricular tachycardia, ventricular fibrillation, or torsade de pointes.
    ? History of second or third degree heart block. Candidates with a history of heart block may be eligible if they currently have pacemakers, and have no history of fainting or clinically relevant arrhythmia with pacemakers.
    ? Myocardial infarction within 6 months prior to screening.
    ? Uncontrolled angina pectoris within 6 months prior to screening.
    ? New York Heart Association (NYHA) Class 3 or 4 congestive heart failure.
    ? Left ventricular ejection fraction (LVEF) =45% or institutional lower limit of normal.
    ? Uncontrolled hypertension.
    ? Complete left or right bundle branch block.
    13. Active infection not well controlled by antibacterial or antiviral therapy.
    14. Known infection with human immunodeficiency virus, or active hepatitis B or C, or other active clinically relevant liver disease.
    15. Unwillingness to receive infusion of blood products according to the protocol.
    16. In a man whose sexual partner is a woman of childbearing potential, unwillingness or inability to use an acceptable contraceptive method for the entire study period and for at least 3 months after study completion.
    17. In a woman of childbearing potential, unwillingness or inability to use an acceptable contraceptive method for the entire study period and for at least 3 months after study completion.
    ? Women are not regarded as of childbearing potential if they are post-menopausal (at least 2 years without menses) or are surgically sterile (at least 1 month before study).
    ? Acceptable contraception comprises two forms of birth control (one of which must be a barrier method).
    18. Positive pregnancy test result.
    19. Breastfeeding.
    20. Medical condition, serious intercurrent illness, or other circumstance that, in the Investigator?s judgment, could jeopardize the candidate?s safety as a study subject, or that could interfere with study objectives.
    1. Leucemia promielocítica aguda (subtipo M3 de LMA).
    2. LMA secundaria a quimioterapia previa por otras neoplasias, excepto LMA secundaria a SMD previo.
    3. Antecedentes de otras neoplasias malignas, a menos que el candidato se haya mantenido libre de la enfermedad durante un mínimo de 5 años.
    ? Los candidatos con cáncer de piel no melanoma tratado, carcinoma in situ o neoplasia intraepitelial cervical son elegibles para participar en el estudio, con independencia del tiempo transcurrido sin enfermedad, si han completado el tratamiento definitivo.
    ? Los candidatos con cáncer de próstata localizado, sin signos de recaída o de progresión de la enfermedad, son elegibles si se ha iniciado hormonoterapia, o si el tumor se ha extirpado de forma quirúrgica o tratado con radioterapia radical.
    4. Toxicidad no hematológica de grado > 1, persistente y clínicamente significativa, debida al tratamiento previo para la LMA.
    5. EICH clínicamente significativa o EICH que requiere el inicio de tratamiento o el aumento de su dosis en un plazo de 21 días, y/o toxicidad no hematológica de grado > 1, persistente o clínicamente significativa, relacionada con la EICH.
    6. Antecedentes o presencia de afectación del sistema nervioso central por la LMA.
    7. Anomalía en la coagulación clínicamente significativa, como coagulación intravascular diseminada.
    8. Tratamiento previo con quizartinib o participación en un estudio previo con quizartinib.
    9. Presencia diagnosticada de una mutación FLT3-D835 en el momento de inclusión en el estudio. En el caso de un candidato que ha recibido tratamiento previo dirigido a FLT3 (excepto midostaurina), se debe registrar la ausencia de una mutación FLT3-D835 basal en el momento de la inclusión en el estudio.
    10. Cirugía mayor en las 4 semanas previas a la selección.
    11. Radioterapia en las 4 semanas previas a la selección.
    12. Enfermedad cardiovascular no controlada o significativa, incluidas:
    ? Intervalo QTcF > 450 ms (promedio de tres determinaciones).
    ? Diagnóstico o sospecha de síndrome de QT largo, o antecedentes familiares de síndrome de QT largo.
    ? Antecedentes de arritmias ventriculares clínicamente relevantes, como taquicardia ventricular, fibrilación ventricular o torsade de pointes.
    ? Antecedentes de bloqueo auriculoventricular de segundo o tercer grado. Los candidatos con antecedentes de bloqueo auriculoventricular pueden ser elegibles si en la actualidad llevan un marcapasos y no tienen antecedentes de desmayo o arritmia clínicamente relevante con marcapasos.
    ? Infarto de miocardio en los 6 meses anteriores a la selección.
    ? Angina de pecho no controlada en los 6 meses anteriores a la selección.
    ? Insuficiencia cardíaca congestiva de clase III o IV de la New York Heart Association (NYHA).
    ? Fracción de eyección del ventrículo izquierdo (FEVI) ? 45 % o en el límite inferior de la normalidad del centro.
    ? Hipertensión no controlada.
    ? Bloqueo de rama izquierdo o derecho completo.
    13. Infección activa no controlada satisfactoriamente mediante tratamiento antibacteriano o antivírico.
    14. Infección diagnosticada por el virus de la inmunodeficiencia humana o hepatitis B o C activa, u otra hepatopatía activa clínicamente relevante.
    15. Poca predisposición a recibir las infusiones de hemoderivados establecidas en el protocolo.
    16. En el caso de varones cuya pareja sexual sea una mujer con capacidad reproductora, falta de voluntad o incapacidad para utilizar un método anticonceptivo aceptable durante todo el periodo del estudio y como mínimo los 3 meses siguientes a la finalización del estudio.
    17. En el caso de una mujer en edad fértil, falta de voluntad o incapacidad para utilizar un método anticonceptivo aceptable durante todo el periodo del estudio y al menos 3 meses después de la finalización del estudio:
    ? Se considerará que una mujer no tiene capacidad reproductora si es posmenopáusica (un mínimo de 2 años sin menstruación) o se ha sometido a esterilización quirúrgica (como mínimo, 1 mes antes del estudio).
    ? Solo se considerará aceptable la utilización conjunta de dos métodos anticonceptivos (uno de los cuales debe ser un método de barrera).
    18. Resultado positivo en una prueba de embarazo.
    19. Mujeres en periodo de lactancia.
    20. Proceso patológico, enfermedad intercurrente grave u otra circunstancia que, en opinión del investigador, pueda poner en peligro la seguridad del candidato como paciente del estudio, o interferir en los objetivos del estudio.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy variable is overall survival, the time from randomization until death from any cause.
    La variable principal de eficacia es la supervivencia global, el tiempo desde la aleatorización hasta la muerte por cualquier causa.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Time from enrollment of first subject to the 280th event (death) will be approximately 22 months.
    El tiempo transcurrido desde la inclusión del primer paciente hasta que se produzca el acontecimiento número 280 (muerte) será de aproximadamente 22 meses.
    E.5.2Secondary end point(s)
    The secondary variable is event-free survival, the time from randomization until documented disease progression/treatment failure, relapse, or death from any cause.
    La variable secundaria es la supervivencia libre de acontecimientos, el tiempo desde la aleatorización hasta que se registra progresión de la enfermedad/fracaso del tratamiento, recaída o muerte por cualquier causa.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Time from enrollment of first subject to the 280th event (death) will be approximately 22 months.
    El tiempo transcurrido desde la inclusión del primer paciente hasta que se produzca el acontecimiento número 280 (muerte) será de aproximadamente 22 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 No
    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 Yes
    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 No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) 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 EEA68
    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
    Canada
    France
    Germany
    Italy
    Netherlands
    New Zealand
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months36
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months36
    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: 245
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 81
    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 state32
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 272
    F.4.2.2In the whole clinical trial 326
    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)
    None
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation National Institute for Health Research Cancer Research Network (NCRN)
    G.4.3.4Network Country United Kingdom
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2014-06-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-05-09
    P. End of Trial
    P.End of Trial StatusOngoing
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