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    Summary
    EudraCT Number:2018-004878-99
    Sponsor's Protocol Code Number:INCB84344-102
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-01-20
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2018-004878-99
    A.3Full title of the trial
    An Open-Label, Single-Arm, Phase 1/2 Study Evaluating the Safety and Efficacy of Ponatinib for the Treatment of Recurrent or Refractory Leukemias or Solid Tumors in Pediatric Participants
    Studio in aperto, a braccio singolo, di Fase 1/2 per valutare la sicurezza e l'efficacia di ponatinib per il trattamento di leucemie o tumori solidi ricorrenti o refrattari in partecipanti pediatrici
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study evaluating the safety and efficacy of ponatinib for the treatment of recurrent or refractory leukemias or solid tumors in children
    Studio di valutazione della sicurezza e dell’efficacia di ponatinib per il trattamento di leucemie o tumori solidi recidivanti o refrattari nei bambini
    A.3.2Name or abbreviated title of the trial where available
    NA
    NA
    A.4.1Sponsor's protocol code numberINCB84344-102
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/293/2018
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorINCYTE BIOSCIENCES INTERNATIONAL SàRL
    B.1.3.4CountrySwitzerland
    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 supportIncyte Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIncyte Corporation
    B.5.2Functional name of contact pointInformazione sulla Sperimentazione
    B.5.3 Address:
    B.5.3.1Street Address1801 Augustine Cut-Off
    B.5.3.2Town/ cityWilmington
    B.5.3.3Post codeDE 19803
    B.5.3.4CountryUnited States
    B.5.4Telephone number13024986700
    B.5.5Fax number13024252734
    B.5.6E-mailRA@incyte.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.2.1.1.1Trade name Iclusig 15 mg compresse rivestite con film
    D.2.1.1.2Name of the Marketing Authorisation holderIncyte Biosciences Distribution B.V.
    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/3/09/716 e EU/3/09/715
    D.3 Description of the IMP
    D.3.1Product nameNA
    D.3.2Product code [NA]
    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 INNponatinib
    D.3.9.1CAS number 1114544-31-8
    D.3.9.2Current sponsor codeINCB084344
    D.3.9.3Other descriptive namePONATINIB HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB121686
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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/3/09/716 e EU/3/09/715
    D.3 Description of the IMP
    D.3.1Product nameponatinib capsula mini-compressa da 5 mg
    D.3.2Product code [INCB084344]
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation Yes
    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 INNponatinib
    D.3.9.1CAS number 1114544-31-8
    D.3.9.2Current sponsor codeINCB084344
    D.3.9.3Other descriptive namePONATINIB HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB121686
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 Yes
    D.2.1.1.1Trade name Iclusig 30 mg compresse rivestite con film
    D.2.1.1.2Name of the Marketing Authorisation holderIncyte Biosciences Distribution B.V.
    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/3/09/716 e EU/3/09/715
    D.3 Description of the IMP
    D.3.1Product name-
    D.3.2Product code [-]
    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 INNponatinib
    D.3.9.1CAS number 1114544-31-8
    D.3.9.2Current sponsor codeINCB084344
    D.3.9.3Other descriptive namePONATINIB HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB121686
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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.2.1.1.1Trade name Iclusig 45 mg compresse rivestite con film
    D.2.1.1.2Name of the Marketing Authorisation holderIncyte Biosciences Distribution B.V.
    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/3/09/716 and EU/3/09/715
    D.3 Description of the IMP
    D.3.1Product name-
    D.3.2Product code [-]
    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 INNponatinib
    D.3.9.1CAS number 1114544-31-8
    D.3.9.2Current sponsor codeINCB084344
    D.3.9.3Other descriptive namePONATINIB HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB121686
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number45
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 5
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.1.1.1Trade name -
    D.2.1.1.2Name of the Marketing Authorisation holder-
    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/3/09/716 e EU/3/09/715
    D.3 Description of the IMP
    D.3.1Product nameponatinib 5 mg in capsule contenenti polvere
    D.3.2Product code [INCB084344]
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation Yes
    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 INNponatinib
    D.3.9.1CAS number 1114544-31-8
    D.3.9.2Current sponsor codeINCB084344
    D.3.9.3Other descriptive namePONATINIB HYDROCHLORIDE
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 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.2.1.1.1Trade name -
    D.2.1.1.2Name of the Marketing Authorisation holder-
    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/3/09/716 e EU/3/09/715
    D.3 Description of the IMP
    D.3.1Product nameponatinib 10 mg compressa rivestita con film
    D.3.2Product code [INCB084344]
    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 INNponatinib
    D.3.9.1CAS number 1114544-31-8
    D.3.9.2Current sponsor codeINCB084344
    D.3.9.3Other descriptive namePONATINIB HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB121686
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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
    Recurrent or Refractory Leukemias and Solid Tumors
    Leucemie e tumori solidi recidivanti o refrattari
    E.1.1.1Medical condition in easily understood language
    Recurrent or Refractory Leukemias and Solid Tumors
    Leucemie e tumori solidi recidivanti o refrattari
    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 21.0
    E.1.2Level PT
    E.1.2Classification code 10000830
    E.1.2Term Acute leukaemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10000880
    E.1.2Term Acute myeloid leukaemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10009013
    E.1.2Term Chronic myeloid leukaemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level PT
    E.1.2Classification code 10028549
    E.1.2Term Myeloid leukaemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10028553
    E.1.2Term Myeloid leukaemia, chronic
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10028552
    E.1.2Term Myeloid leukaemia, acute
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level HLGT
    E.1.2Classification code 10027655
    E.1.2Term Miscellaneous and site unspecified neoplasms malignant and unspecified
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Phase 1 Dose Escalation:
    To determine the MTD and/or RP2D of oral ponatinib administered QD in
    pediatric participants with selected advanced hematologic malignancies
    or solid tumors.
    Phase 2 Expansion:
    Group A (CP-CML):
    To determine the efficacy of oral ponatinib administered QD in pediatric
    participants with CP-CML who are resistant or intolerant to at least 1
    prior BCR-ABL–targeted TKI therapy or who have the T315I mutation. Group B (Other Tumors):
    To determine the efficacy of oral ponatinib administered QD in pediatric
    participants with other selected advanced hematologic malignancies or
    solid tumors.
    Fase 1 Incremento graduale della dose:
    Determinare la MTD e/o la RP2D di ponatinib orale somministrato QD in partecipanti pediatrici con specifici tumori maligni ematologici o tumori solidi in stadio avanzato.
    Fase 2 Espansione
    Gruppo A (CP-CML):
    Stabilire l'efficacia di ponatinib orale somministrato QD in partecipanti pediatrici con CP-CML che sono resistenti o intolleranti ad almeno 1 terapia precedente con TKI mirata a BCR-ABL o che presentano la mutazione T315I
    Gruppo B (altri tumori):
    Stabilire l'efficacia di ponatinib orale somministrato QD in partecipanti pediatrici con altri specifici tumori maligni ematologici o tumori solidi in stadio avanzato.
    E.2.2Secondary objectives of the trial
    Phase 1 Dose Escalation:
    . To examine the safety and tolerability of ponatinib in pediatric
    participants with selected advanced hematologic malignancies or solid
    tumors.
    . To evaluate the PK properties of ponatinib in pediatric participants.
    . To evaluate preliminary anticancer activity of ponatinib in pediatric
    participants.
    Phase 2 Expansion
    . Group A (CP-CML):
    • To determine the antileukemia activity of ponatinib participants with
    CP-CML.
    • To determine the cytogenetic and molecular response.
    Group B (Other Tumors):
    To determine anticancer activity of ponatinib in pediatric participants
    with selected advanced hematologic malignancies or solid tumors.
    Cohorts A and B:
    To examine the safety and tolerability of ponatinib in pediatric
    participants.
    To obtain PK data on participants dosed with a new pediatric-friendly
    formulation (when available).
    - Fase 1 Incremento graduale della dose:
    . Esaminare la sicurezza e la tollerabilità di ponatinib in partecipanti pediatrici con specifici tumori maligni ematologici o tumori solidi in stadio avanzato.
    . Valutare le proprietà di PK di ponatinib nei partecipanti pediatrici.
    . Valutare l’attività antitumorale preliminare di ponatinib nei partecipanti pediatrici.
    - Fase 2 - Espansione
    . Gruppo A (CP-CML):
    • Determinare l'attività antileucemica di ponatinib nei partecipanti con CP-CML.
    • Determinare la citogenetica e la risposta molecolare.
    Gruppo B (altri tumori):
    Determinare l'attività antitumorale di ponatinib in partecipanti pediatrici con specifici tumori maligni ematologici o tumori solidi in stadio avanzato.
    Coorti A e B:
    Esaminare la sicurezza e la tollerabilità di ponatinib in partecipanti pediatrici
    Ottenere i dati di PK nei partecipanti che hanno ricevuto trattamento con una nuova formulazione adatta ai pazienti pediatrici (se disponibile).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Histologically or cytologically confirmed diagnosis of the following
    malignancies:
    a. Phase 1:
    - CP-CML, BP-CML, AP-CML (relapse).
    - ALL.
    - AML.
    - Other leukemias.
    - Lymphoma.
    - Any other tumors, including tumors of the CNS, for which standard
    therapy is not available or is not indicated.
    b. Phase 2, Group A with CP-CML:
    - CP-CML at the time of study entry and must be resistant to or
    intolerant of at least 1 prior BCR-ABL–targeted TKI therapy or have the
    T315I kinase domain mutation.
    - Must have 1 bone marrow aspirate with documentation of BCR-ABL
    translocation by conventional cytogenetics, metaphase FISH, or q-PCR
    performed within 42 days before the first dose of ponatinib.
    c. Phase 2, Group B with other leukemias or solid tumors:
    - ALL.
    - AML.
    - Other leukemias.
    - Lymphoma.
    - Any other tumors, including tumors of the CNS, with mutations of RET,
    KIT, FGFR, PGFR, VEGFR, or any other mutations where ponatinib may
    have biological activity on fresh or archived tumor tissue.
    - Participants with solid tumors or with lymphoma must have
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    measurable disease by CT or MRI based on RECIST v1.1 or the Lugano
    lymphoma guidelines (Cheson et al 2014) as determined by site
    radiology.
    Note: Lesions situated in a previously irradiated area are considered
    measurable if progression has been demonstrated in such lesions.
    2. Prior therapies as follows:
    a. Phase 1:
    - Participants with CML who are resistant to or intolerant of to at least 1
    prior BCR-ABL–targeted TKI therapy.
    - Participants with ALL who have failed all available or indicated
    therapies, which may have included 1 prior BCR-ABL–targeted TKI
    therapy.
    - Participants with AML or other leukemias who have failed at least 1
    prior induction attempt or for whom no effective standard therapy is
    available or indicated.
    - Participants with solid tumors (including tumors of the CNS) or
    lymphomas who have progressed despite standard therapy or for whom
    no effective standard therapy is available or indicated.
    b. Phase 2, Group A with CP-CML:
    - Participants who are resistant to or intolerant of at least 1 prior BCRABL–
    targeted TKI therapy.
    c. Phase 2, Group B with other leukemias or solid tumors:
    - Participants with ALL who have failed all available or indicated
    therapies, which must have included 1 prior BCR-ABL–targeted TKI
    therapy.
    - Participants with AML or other leukemias who have failed at least 1
    prior induction attempt or for whom no effective standard therapy is
    available or indicated.
    - Participants with solid tumors (including tumors of the CNS) or
    lymphomas who progressed despite standard therapy or for whom no
    effective standard therapy is available or indicated.
    3. Male and female participants = 1 to < 18 years old, inclusive, at the
    time of signing the informed consent.
    4. Karnofsky performance status = 40% for participants > 16 years old
    or Lansky Play Scale = 40 for pediatric participants = 16 years old.
    1. Diagnosi confermata da esame istologico o citologico dei seguenti tumori maligni:
    a. Fase 1:
    CP-CML, BP-CML, AP-CML (recidiva).
    - ALL.
    - AML.
    - Altre leucemie.
    - Linfoma.
    - Qualsiasi altro tumore, inclusi tumori del SNC, per i quali la terapia standard non è disponibile o non è indicata.
    b. Fase 2, Gruppo A con CP-CML:
    - CP-CML al momento dell'ingresso nello studio e deve essere resistente o intollerante ad almeno 1 terapia precedente con TKI mirata a BCR-ABL o presentare la mutazione T315I del dominio chinasico.
    - Devono presentare 1 aspirato di midollo osseo con documentazione di traslocazione BCR-ABL secondo citogenetica convenzionale, FISH in metafase o q-PCR eseguita nei 42 giorni precedenti la prima dose di ponatinib.
    c. Fase 2, Gruppo B con altre leucemie o tumori solidi:
    - ALL.
    - AML.
    - Altre leucemie.
    - Linfoma.
    - Qualsiasi altro tumore, inclusi tumori del SNC, con mutazioni di RET, KIT, FGFR, PGFR, VEGFR, o qualsiasi altra mutazione in cui ponatinib possa avere attività biologica su tessuto tumorale recente o d'archivio.
    - I partecipanti con tumori solidi o con linfoma devono presentare malattia misurabile mediante TAC o RMI secondo RECIST v1.1 o linee guida di Lugano per i linfomi (Cheson et al 2014) come stabilito dalla radiologia del centro.
    Nota: le lesioni situate in un'area precedentemente sottoposta a radioterapia sono considerate misurabili se in tali lesioni è stata dimostrata la progressione.
    2. Terapie precedenti come di seguito indicato:
    a. Fase 1:
    - Partecipanti con CML che sono resistenti o intolleranti ad almeno 1 terapia precedente con TKI mirata a BCR-ABL.
    - Partecipanti con ALL che hanno avuto insuccesso con tutte le terapie disponibili o indicate, tra cui potrebbe essere compresa 1 terapia precedente con TKI mirata a BCR-ABL.
    - Partecipanti con AML o altre leucemie che hanno avuto insuccesso con almeno 1 tentativo di induzione precedente o per i quali non è disponibile o indicata alcuna terapia standard efficace.
    - Partecipanti con tumori solidi (compresi tumori del SNC) o linfomi che sono progrediti nonostante la terapia standard o per i quali non è disponibile o indicata alcuna terapia standard efficace.
    b. Fase 2, Gruppo A con CP-CML:
    - Partecipanti che sono resistenti o intolleranti ad almeno 1 terapia precedente con TKI mirata a BCR-ABL.
    c. Fase 2, Gruppo B con altre leucemie o tumori solidi:
    - Partecipanti con ALL che hanno avuto insuccesso con tutte le terapie disponibili o indicate, tra cui deve essere compresa 1 terapia precedente con TKI mirata a BCR-ABL.
    - Partecipanti con AML o altre leucemie che hanno avuto insuccesso con almeno 1 tentativo di induzione precedente o per i quali non è disponibile o indicata alcuna terapia standard efficace.
    - Partecipanti con tumori solidi (compresi tumori del SNC) o linfomi che sono progrediti nonostante la terapia standard o per i quali non è disponibile o indicata alcuna terapia standard efficace.
    3. Partecipanti di sesso maschile e femminile, di età da = 1 a < 18 anni inclusi, al momento della firma del consenso informato.
    4. Stato di performance di Karnofsky = 40% per partecipanti > 16 anni o scala di Lansky relativa alla capacità di gioco = 40 per partecipanti pediatrici = 16 anni.
    E.4Principal exclusion criteria
    1. Participants with CP-CML who are in MCyR or better.
    2. Prior therapies:
    a. Participants with BP-CML, ALL, or AML who have received any of the
    following:
    - Corticosteroids or hydroxyurea within 24 hours before the first dose of
    ponatinib.
    - Vincristine within 7 days before the first dose of ponatinib.
    - Other chemotherapy (excluding intrathecal chemotherapy) within 14
    days before the first dose of ponatinib.
    b. Participants (except the BP-CML, ALL, and AML participants described
    above) who:
    - Have had cytotoxic chemotherapy or radiotherapy within 21 days (or
    42 days for nitrosoureas or mitomycin C) before the first dose of
    ponatinib.
    c. Prior radiation therapy within 6 weeks or radio-isotope therapy within
    6 weeks before the first dose of ponatinib.
    d. Autologous or allogeneic stem cell transplant < 3 months before the
    first dose of ponatinib.
    e. Major surgery within 14 days before the first dose of ponatinib.
    Note: Minor surgical procedures, such as central venous catheter
    placement or bone marrow aspirate/biopsy, are permitted.
    f. Inadequate recovery and/or complications from a major surgery
    before starting therapy.
    g. Prior treatment with any of the following:
    - Immunosuppressive therapy (including post stem cell transplant
    regimens) within 14 days before the first dose of ponatinib.
    - Any targeted cancer therapy (including TKIs) within 7 days before the
    first dose of ponatinib.
    - Any other investigational anticancer agents within 30 days or 5 halflives,
    whichever is longer, before randomization.
    - Any monoclonal antibody–directed anticancer therapy within 5 halflives
    of the first dose of ponatinib.
    - Any chimeric antigen receptor therapy within 28 days before the first
    dose of ponatinib
    1. Partecipanti con CP-CML che presentano MCyR o una risposta migliore.
    2. Terapie precedenti:
    a. Partecipanti con BP-CML, ALL o AML che hanno ricevuto uno dei seguenti farmaci:
    - Corticosteroidi o idrossiurea nelle 24 ore precedenti la prima dose di ponatinib.
    - Vincristina entro i 7 giorni antecedenti la somministrazione della prima dose di ponatinib.
    - Altra chemioterapia (esclusa chemioterapia intratecale) nei 14 giorni precedenti la prima dose di ponatinib.
    b. Partecipanti (eccetto i partecipanti con BP-CML, ALL e AML sopra descritti) che:
    - Sono stati sottoposti a chemioterapia citotossica o radioterapia nei 21 giorni (o 42 giorni per nitrosourea o mitomicina C) precedenti la prima dose di ponatinib.
    c. Precedente radioterapia nelle 6 settimane o terapia con radioisotopi nelle 6 settimane precedenti la prima dose di ponatinib.
    d. Trapianto di cellule staminali autologo o allogenico < 3 mesi prima della prima dose di ponatinib.
    e. Intervento chirurgico importante entro i 14 giorni antecedenti la somministrazione della prima dose di ponatinib.
    Nota: sono consentite procedure chirurgiche di minore entità, come il posizionamento di catetere venoso centrale o aspirato/biopsia del midollo osseo.
    f. Recupero inadeguato e/o complicanze a seguito di intervento chirurgico importante prima di iniziare la terapia.
    g. Precedente trattamento con uno dei farmaci seguenti:
    - Terapia immunosoppressiva (compresi regimi successivi a trapianto di cellule staminali) nei 14 giorni precedenti la prima dose di ponatinib.
    - Qualsiasi terapia antitumorale mirata (compresi TKI) nei 7 giorni precedenti la prima dose di ponatinib.
    - Qualsiasi altro agente antitumorale sperimentale nei 30 giorni o nelle 5 emivite, a seconda di quale sia il periodo più lungo, precedenti la randomizzazione.
    - Qualsiasi terapia antitumorale mirata con anticorpi monoclonali nelle 5 emivite precedenti la prima dose di ponatinib.
    - Qualsiasi terapia con recettori chimerici per l'antigene nei 28 giorni precedenti la prima dose di ponatinib
    E.5 End points
    E.5.1Primary end point(s)
    - Phase 1 Dose Escalation
    Determination of DLTs during the DLT evaluation period (first 28 days of
    treatment).
    - Phase 2 Expansion
    Group A (CP-CML):
    MCyR, defined as CCyR or PCyR by 12 months, assessed by conventional
    cytogenetics or FISH.
    Group B (Other Tumors):
    Hematologic malignancies:
    • BCR-ABL–positive leukemias (CML in AP or BP; Ph+ ALL):
    - MaHR or MMR assessed by q-PCR by 3 months.
    • Other leukemias:
    - CR.
    - CRi, as assessed by conventional cytogenetics, FISH, or q-PCR.
    • Lymphoma:
    - CR according to Lugano criteria (Cheson et al 2014) based on CT or
    MRI (or PET).
    Solid tumors:
    • ORR, defined as the percentage of participants having CR or PR, as
    determined by investigator assessment of radiographic disease per
    tumors per RANO for CNS tumors or RECIST v1.1 for other solid tumors
    based on CT or MRI (or PET).
    - Fase 1 Incremento graduale della dose
    Determinare le DLT durante il periodo di valutazione delle DLT (primi 28 giorni di trattamento).
    - Fase 2 Gruppo di Espansione A (CP- CML):
    MCyR, definita come CCyR o PCyR entro 12 mesi, valutata mediante citogenetica convenzionale o FISH.
    Gruppo B (altri tumori): Tumori maligni ematologici:
    • Leucemie BCR-ABL positive (CML in AP o BP; Ph+ ALL):
    - MaHR o MMR valutate mediante q-PCR entro 3 mesi.
    • Altre leucemie:
    - CR.
    - CRi valutata mediante citogenetica convenzionale, FISH o q-PCR.
    • Linfoma:
    - CR secondo i criteri di Lugano (Cheson et al 2014) in base a TAC o RMI (o PET).
    Tumori solidi:
    • ORR, definito come la percentuale di partecipanti con CR o PR, come stabilito dalla valutazione dello sperimentatore della malattia da esame radiografico per i tumori secondo i criteri RANO per i tumori del SNC o RECIST v1.1 per altri tumori solidi sulla base di TAC o RMI (o PET).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Phase 1 Dose Escalation:
    first 28 days
    Phase 2 Expansion:
    by 3 months, by 12 months,
    Fase 1 Incremento graduale della dose: primi 28 giorni
    Fase 2 Espansione: entro 3 mesi, entro 12 mesi.
    E.5.2Secondary end point(s)
    - Phase 1 Dose Escalation:
    • Frequency, duration, and severity of AEs and SAEs.
    • Changes in vital signs and clinical evaluations.
    • Changes in clinical laboratory blood samples.
    • PK parameters: Tmax, AUCss,0-24, t½, CLss/F, Vz/F.
    Hematologic malignancies:
    • BCR-ABL–positive leukemias (CML in AP or BP; Ph+ ALL):
    - MCyR by cytogenetics or FISH and MMR by q-PCR at 3 months.
    • CP-CML:
    - CHR at 6 months.
    - CCyR at 12 months. MMR at 12 months.
    - TTR, defined as the interval from the date of the first dose of study
    treatment to first response.
    - DOR, defined as defined as the interval between the first assessment
    at which the criteria for response are met until the criteria for
    progression are met. PFS, defined as the interval from the date of the first dose of study
    treatment until the date of progression of disease or the date of death
    from any cause, whichever is earlier.
    For additional secondary objectives, please refers to the correspondent section in the protocol.
    - Fase 1 Incremento graduale della dose:
    • Frequenza, durata e gravità degli AE e dei SAE.
    • Variazioni nelle funzioni vitali e nelle valutazioni cliniche.
    • Variazioni nei campioni di sangue per gli esami clinici di laboratorio.
    • Parametri PK: Tmax, AUCss,0-24, t½, CLss/F, Vz/F. Tumori maligni ematologici:
    • Leucemie BCR-ABL positive (CML in AP o BP; Ph+ ALL):
    - MCyR valutata mediante citogenetica o FISH e MMR valutata mediante q-PCR a 3 mesi.
    • CP-CML:
    - CHR a 6 mesi.
    - CCyR a 12 mesi. MMR a 12 mesi.
    - TTR, definito come l'intervallo tra la data della prima dose di trattamento in studio e la prima risposta.
    - DOR, definita come l'intervallo tra la prima valutazione nella quale sono soddisfatti i criteri per la risposta e il momento in cui sono soddisfatti i criteri per la progressione.
    - PFS, definita come l'intervallo tra la data della prima dose di trattamento in studio e la data della progressione della malattia o la data del decesso per qualsiasi causa, a seconda dell'evento che si verifica prima.
    - OS, definita come l’intervallo tra la data della prima dose di trattamento in studio e il decesso per qualsiasi causa.
    • Altre leucemie:
    - CR.
    - CRi valutata mediante citogenetica convenzionale, FISH o q-PCR.
    • Linfoma:
    - CR secondo i criteri di Lugano (Cheson et al 2014) in base a TAC o RMI (o PET).
    Tumori solidi:
    • ORR, definito come la percentuale di partecipanti con CR o PR, come stabilito dalla valutazione dello sperimentatore della malattia da esame radiografico per i tumori secondo i criteri RANO per i tumori del SNC o RECIST v1.1 per altri tumori solidi.
    sulla base di TAC o RMI (o PET).
    - Fase 2 - Espansione
    Gruppo A (CP-CML):
    • CHR a 6 mesi.
    • CCyR a 12 mesi.
    • MMR a 12 mesi.
    • TTR, definito come l'intervallo tra la data della prima dose di trattamento in studio e la prima risposta.
    • DOR, definita come l'intervallo tra la prima valutazione nella quale sono soddisfatti i criteri per la risposta e il momento in cui sono soddisfatti i criteri per la progressione.
    • PFS, definita come l'intervallo tra la prima dose di trattamento in studio e la data della progressione della malattia o del decesso per qualsiasi causa, a seconda dell'evento che si verifica prima.
    • OS, definita come l’intervallo tra la data della prima dose di trattamento in studio e il decesso per qualsiasi causa.
    Gruppo B (altri tumori):
    Tumori maligni ematologici:
    • Leucemie BCR-ABL positive (CML in AP o BP; Ph+ ALL):
    - MHR o MMR entro 3 mesi.
    • Altre leucemie:
    - CR.
    - CRi valutata mediante citogenetica convenzionale, FISH o q-PCR.
    • Linfoma:
    - CR secondo i criteri di Lugano (Cheson et al 2014) in base a TAC o RMI (o PET).

    Tumori solidi:
    • ORR, definito come la percentuale di partecipanti con CR o PR, come stabilito dalla valutazione dello sperimentatore della malattia da esame radiografico per i tumori secondo i criteri RANO per i tumori del SNC o RECIST v1.1 per altri tumori solidi sulla base di TAC o RMI (o PET).
    • OS, definita come l’intervallo tra la data della prima dose di trattamento in studio e la data del decesso per qualsiasi causa.
    • DOR, definita come l'intervallo tra la prima valutazione nella quale sono soddisfatti i criteri per la risposta e il momento in cui sono soddisfatti i criteri per la progressione. PFS, definita come l'intervallo tra la data della prima dose di trattamento in studio e la data della progressione della malattia o del decesso per qualsiasi causa, a seconda dell'evento che si verifica prima. PFS, definita come l'intervallo tra la prima dose di trattamento in studio e la data della progressione della malattia o del decesso per qualsiasi causa, a seconda dell'evento che si verifica prima.

    per gli altri obiettivi secondari si rimanda alla relativa sezione del protocollo
    E.5.2.1Timepoint(s) of evaluation of this end point
    During all study
    At 3, 6 and 12 months
    Durante tutto lo studio
    ai mesi 3, 6 e 12
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Phase 1/2 study
    studio di fase 1/2
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    NA
    NA
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA13
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    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
    The end of the study is defined as the date of the last visit for the last participant on study when participants are still on study at the time of the primary endpoint analysis and are continuing to receive study treatment until treatment withdrawal criteria are met.
    La conclusione dello studio si definisce come la data dell’ultima visita dell’ultimo partecipante in studio quando i partecipanti sono ancora coinvolti nello studio al momento dell’analisi dell’endpoint primario e continuano a ricevere il trattamento in studio finché i criteri di ritiro dal trattamento non vengono soddisfatti.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months8
    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 Yes
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 28
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 28
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 60
    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)
    Provisions will be made to ensure access to treatment for any remaining participants benefitting from treatment at time of cutoff.
    Saranno prese misure per garantire l’accesso al trattamento per eventuali partecipanti rimanenti che stiano ottenendo benefici dal trattamento al momento del cut-off.
    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 Decision2020-01-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-10-09
    P. End of Trial
    P.End of Trial StatusOngoing
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