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    Summary
    EudraCT Number:2016-002903-25
    Sponsor's Protocol Code Number:CC-4047-BRN-001
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-05-31
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2016-002903-25
    A.3Full title of the trial
    A Phase 2 clinical study of pomalidomide (CC-4047) monotherapy for children and young adults with recurrent or progressive primary brain tumors.
    Estudio clínico de fase 2 de pomalidomida (CC-4047) en monoterapia para niños y adultos jóvenes con tumores cerebrales primarios recurrentes o progresivos
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of CC-4047 in Patients with Brain Malignancies
    Estudio de CC-4047 en pacientes con tumores cerebrales
    A.4.1Sponsor's protocol code numberCC-4047-BRN-001
    A.5.4Other Identifiers
    Name:INDNumber:123692
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCelgene Corporation
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportCelgene Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCelgene Corporation
    B.5.2Functional name of contact pointClinicalTrialDisclosure
    B.5.3 Address:
    B.5.3.1Street Address9225 Indian Creek Parkway, Suite 90
    B.5.3.2Town/ cityOverland Park, Kansas
    B.5.3.3Post code66210
    B.5.3.4CountryUnited States
    B.5.4Telephone number+34914229000
    B.5.5Fax number+349132660394
    B.5.6E-mailClinicalTrialDisclosure@celgene.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Imnovid 1 mg hard capsules
    D.2.1.1.2Name of the Marketing Authorisation holderCelgene Europe Ltd
    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.3.4Pharmaceutical form Capsule, hard
    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 INNPomalidomide
    D.3.9.1CAS number 19171-19-8
    D.3.9.2Current sponsor codeCC-4047
    D.3.9.3Other descriptive namePOMALIDOMIDE
    D.3.9.4EV Substance CodeSUB33379
    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 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 Yes
    D.2.1.1.1Trade name Imnovid 2 mg hard capsules
    D.2.1.1.2Name of the Marketing Authorisation holderCelgene Europe Ltd
    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.3.4Pharmaceutical form Capsule, hard
    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 INNPomalidomide
    D.3.9.1CAS number 19171-19-8
    D.3.9.2Current sponsor codeCC-4047
    D.3.9.3Other descriptive namePOMALIDOMIDE
    D.3.9.4EV Substance CodeSUB33379
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 3
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Imnovid 3 mg hard capsules
    D.2.1.1.2Name of the Marketing Authorisation holderCelgene Europe Ltd
    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.3.4Pharmaceutical form Capsule, hard
    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 INNPomalidomide
    D.3.9.1CAS number 19171-19-8
    D.3.9.2Current sponsor codeCC-4047
    D.3.9.3Other descriptive namePOMALIDOMIDE
    D.3.9.4EV Substance CodeSUB33379
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 4
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Imnovid 4 mg hard capsules
    D.2.1.1.2Name of the Marketing Authorisation holderCelgene Europe Ltd
    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.3.4Pharmaceutical form Capsule, hard
    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 INNPomalidomide
    D.3.9.1CAS number 19171-19-8
    D.3.9.2Current sponsor codeCC-4047
    D.3.9.3Other descriptive namePOMALIDOMIDE
    D.3.9.4EV Substance CodeSUB33379
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number4
    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 RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namePomalidomide
    D.3.4Pharmaceutical form Capsule, hard
    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 INNPomalidomide
    D.3.9.1CAS number 19171-19-8
    D.3.9.2Current sponsor codeCC-4047
    D.3.9.3Other descriptive namePOMALIDOMIDE
    D.3.9.4EV Substance CodeSUB33379
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.5
    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 RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namePomalidomide
    D.3.4Pharmaceutical form Powder for oral suspension
    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 INNPomalidomide
    D.3.9.1CAS number 19171-19-8
    D.3.9.2Current sponsor codeCC-4047
    D.3.9.3Other descriptive namePOMALIDOMIDE
    D.3.9.4EV Substance CodeSUB33379
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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
    Recurrent or progressive primary brain tumors in one of four primary brain tumor types: high-grade glioma, medulloblastoma, ependymoma and diffuse intrinsic pontine glioma (DIPG
    Tumores cerebrales primarios recurrentes o progresivos, correspondientes a uno de cuatro tipos de tumores cerebrales primarios: glioma de alto grado, meduloblastoma, ependimoma y glioma pontino intrínseco difuso (GPID).
    E.1.1.1Medical condition in easily understood language
    Brain Malignancies
    Tumores cerebrales
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To identify potential tumor type(s) for further development by establishing the preliminary efficacy of pomalidomide in children and young adults with recurrent or progressive primary brain tumors within four distinct tumor types.
    Identificar tipos potenciales de tumores para posterior desarrollo determinando la eficacia preliminar de pomalidomida en niños y adultos jóvenes con tumores cerebrales primarios recurrentes o progresivos entre cuatro tipos diferentes de tumores.
    E.2.2Secondary objectives of the trial
    To evaluate the safety of pomalidomide within the study populations.
    To estimate the long-term efficacy of pomalidomide treatment.
    Evaluar la seguridad de pomalidomida en las poblaciones del estudio.
    Evaluar la eficacia a largo plazo del tratamiento con pomalidomida.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject is 1 to < 21 years of age at the time of signing the ICF/IAF.
    2. Subject must understand and voluntarily sign an ICF/IAF prior to any study-related assessments/procedures being conducted.
    3. Subject has received at least one prior standard therapy (or generally accepted upfront therapy if no standard exists) and have no known curative therapy.
    4. Subject has a diagnosis of high-grade glioma, medulloblastoma, ependymoma or DIPG that is recurrent or progressive.
    5. Subject has histological verification of tumor either at the time of diagnosis or recurrence.
    6. Subject has measurable disease, defined as a tumor that is measurable in two perpendicular diameters on MRI.
    7. To document the degree of tumor through scan;
    8. Subject has Karnofsky (age ≥ 16 years) or Lansky (age < 16 years) performance status score ≥ 50 at screening (Appendix D)
    9. Subject has adequate bone marrow function;
    10. Subject has adequate renal function;
    11. Subject has adequate liver function;
    12. Subject has adequate pulmonary function;
    13. Subject has recovered from clinically significant acute treatment related toxicities from all prior therapies.
    14. Subject has no significant worsening in clinical status for a minimum of 7 days prior to first dose of study drug;
    15. Subject (and when applicable, with parental/legal representative) is willing and able to adhere to the study visit schedule and other protocol requirements.
    16. Females of Childbearing Potential (FCBP), Female Children of Childbearing Potential (FCCBP), and male subjects who have reached puberty (and when applicable, with parental/legal representative) must agree to undergo physician-approved reproductive education and discuss the side effects of the study therapy on reproduction.
    17. All subjects and/or parents/legal representative must have an understanding that pomalidomide could have a potential teratogenic risk. Female children of childbearing potential, defined as females who have achieved menarche and/or breast development in Tanner Stage 2 or greater and have not undergone a hysterectomy or bilateral oophorectomy and FCBP, defined as a sexually mature woman who has not undergone a hysterectomy or bilateral oophorectomy and has not been naturally postmenopausal for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months) must agree and meet the following conditions below:
    -Medically supervised pregnancy tests with a sensitivity of at least 25 mIU/mL must be conducted in FCCBP/FCBP, including those who commit to true abstinence*.
    Two pregnancy tests must be conducted prior to starting pomalidomide. The first pregnancy test must be performed 10 to 14 days prior to the start of pomalidomide and the second pregnancy test must be performed within 24 hours prior to starting pomalidomide. The subject may not receive pomalidomide until the Investigator has verified that the results of these pregnancy tests performed on Cycle 1, Day 1 are negative. Female children of childbearing potential/females of childbearing potential with regular or no menstrual cycles must agree to have pregnancy tests weekly for the first 28 days study participation, every 28 days while on study, at study treatment discontinuation visit, and at Day 28 following pomalidomide discontinuation. If menstrual cycles are irregular, the pregnancy testing must occur weekly for the first 28 days of study participation and then every 14 days while on study, at study treatment discontinuation visit, and at Days 14 and 28 following pomalidomide discontinuation.
    -Female subjects must, as appropriate to age and at the discretion of the study Investigator, either commit to true abstinence* from heterosexual contact (which must be reviewed on a monthly basis) and/or agree to the use of two reliable forms of approved and effective contraceptive methods simultaneously. The two methods of reliable contraception must include one highly effective method (ie, oral, injectable, or implantable hormonal contraceptive; tubal ligation; intra-uterine device; vasectomized partner) and one additional effective barrier method (ie, male condom, diaphragm, cervical cap) 28 days prior to starting pomalidomide, throughout the entire duration of study treatment including dose interruptions and 28 days after discontinuation of pomalidomide.
    -All male and female subjects must follow all requirements defined in the pomalidomide Pregnancy Prevention Program.
    18. Male subjects must, as appropriate to age and the discretion of the study physician:
    -Practice true abstinence* (which must be reviewed on a monthly basis) or agree to use a condom during sexual contact with a pregnant female or a FCBP while participating in the study, during dose interruptions and for at least 28 days following pomalidomide discontinuation, even if he has undergone a successful vasectomy or practices complete abstinence.
    1.Tener edad entre 1 y 21 años en el momento de firmar el DCI/DAI.
    2.Comprender y firmar voluntariamente un DCI/DAI antes de someterse a las evaluaciones y procedimientos relacionados con el estudio.
    3.Haber recibido, al menos, un tratamiento de referencia previo (o tratamiento de primera línea aceptado de manera general cuando no exista tratamiento de referencia) y ausencia de tratamiento curativo conocido.
    4.Tener un diagnóstico de glioma de alto grado, meduloblastoma, ependimoma o GPID que es recurrente o progresivo.
    5.Contar con una verificación histológica dl tumor, ya sea en el momento del diagnóstico o de la recidiva.
    6.Tener enfermedad mensurable (incluidos los sujetos que se hayan sometido a una resección quirúrgica antes de su inclusión), definida como un tumor mensurable en dos diámetros perpendiculares en la RM.
    7.Documentar el grado del tumor a través de escáner.
    8.Tener una puntuación de estado funcional de Karnofsky (edad ≥ 16 años) o de Lansky (edad < 16 años) ≥ 50 en el momento de selección.
    9.Presentar una función medular adecuada.
    10.Presentar una función renal adecuada
    11.Presentar una función hepática adecuada
    12.Presentar una función pulmonar adecuada
    13.Haberse recuperado de la toxicidad aguda clínicamente significativa relacionada con todos los tratamientos previos.
    14.No mostrar un empeoramiento significativo del estado clínico durante un mínimo de 7 días antes de la primera dosis del fármaco del estudio.
    15.Estar dispuesto y tener capacidad de cumplir el calendario de visitas del estudio y los demás requisitos del protocolo.
    16.Las mujeres en edad fértil (MEF), las niñas en edad fértil (NEF) y los varones que hayan alcanzado la pubertad deberán acceder a recibir información sobre la reproducción aprobada por el médico y a comentar los efectos secundarios del tratamiento del estudio sobre la reproducción.
    17.Todos los sujetos y sus progenitores o representantes legales deberán saber que la pomalidomida entraña un posible riesgo teratógeno. Las niñas en edad fértil, definidas como aquellas que hayan tenido la menarquia o muestren un desarrollo mamario en estadio 2 o superior de Tanner y no se hayan sometido a una histerectomía u ovariectomía bilateral, y las mujeres en edad fértil, deberán aceptar y cumplir las condiciones siguientes:*Deberán hacerse pruebas de embarazo supervisadas por un médico con una sensibilidad mínima de 25 mUI/ml a las NEF/MEF, incluidas las que se comprometan a practicar abstinencia real. Deberán hacerse dos pruebas de embarazo antes de iniciar la administración de pomalidomida. La primera prueba de embarazo se hará entre 10 y 14 días antes del inicio del tratamiento con pomalidomida y la segunda, en un plazo de 24 horas antes del comienzo del tratamiento con pomalidomida. La participante no podrá recibir pomalidomida hasta que el investigador haya verificado que los resultados de las pruebas de embarazo realizadas el día 1 del ciclo 1 son negativas. Las NEF/MEF con ciclos menstruales regulares o sin menstruación deberán aceptar someterse a pruebas de embarazo semanalmente durante los primeros 28 días de participación en el estudio, cada 28 días mientras permanezcan en el estudio, en la visita de suspensión del tratamiento del estudio y 28 días después de suspender el tratamiento con pomalidomida. Si una participante presenta ciclos menstruales irregulares, deberá someterse a pruebas d embarazo semanalmente durante los primeros 28 días de participación en el estudio, cada 14 días mientras permanezcan en el estudio, en la visita de suspensión del tratamiento del estudio y 14 y 28 días después de suspender el tratamiento con pomalidomida. *Las mujeres deberán comprometerse, según proceda para la edad y a criterio del investigador del estudio, a practicar abstinencia real* de relaciones heterosexuales o a utilizar simultáneamente dos métodos anticonceptivos fiables, aprobados y eficaces. Los dos métodos anticonceptivos fiables deberán incluir un método muy eficaz y un método de barrera eficaz adicional 28 días antes de comenzar la administración de pomalidomida, durante todo el tratamiento del estudio, incluidas las interrupciones de la administración, y 28 días después de suspender el tratamiento con pomalidomida.*Todos los sujetos d ambos sexos deberán cumplir todos los requisitos definidos en el Programa de prevención de embarazos con pomalidomida.
    18.Los varones deberán, según proceda para la edad y a criterio del médico del estudio:*Practicar abstinencia real o comprometerse a utilizar preservativo durante ls relaciones sexuales con 1 mujer embarazada o MEF mientras participe en el estudio, durante ls interrupciones de la administración y hasta al menos 28 meses después d suspender el tratamiento con pomalidomida, aunque se haya sometido a una vasectomía con éxito o practique abstinencia sexual completa.
    E.4Principal exclusion criteria
    1. Subject has a history of non-central line related thrombosis (arterial or venous), more than one prior central-line related thrombosis or known coagulopathy.
    2. Subject has first degree family member with a known hereditary coagulopathy.
    3. Subject is actively on anticoagulation therapy.
    4. Subject has had major (per Investigator discretion) surgery, with the exception of tumor resection, within 21 days from first dose of study drug.
    5. Subject has previously received (presence of any of the following will exclude a subject from enrollment):
    -Any prior treatment with pomalidomide. Subjects who have prior treatment with other IMiDs (thalidomide, lenalidomide) ARE eligible if they meet all other eligibility criteria and did not have allergic reactions or other “significant toxicity” per Investigator discretion associated with lenalidomide or thalidomide use.
    -Myelosuppressive chemotherapy, immunotherapy, or any investigational agent: ≤ 28 days (≤ 42 days if a nitrosourea) prior to screening.
    -Biological (anti-neoplastic) therapy: ≤ 7 days prior to screening.
    -Immunomodulatory therapy: ≤ 28 days prior to screening.
    -Monoclonal antibody treatment and agents with known prolonged half-lives: < 3 half-lives have elapsed or ≤ 28 days prior to screening, whichever is longer.
    -Prior radiation:
    .Cranial irradiation, total body irradiation (TBI), or ≥ 50% radiation of pelvis: ≤ 3 months prior to screening.
    .Focal irradiation: ≤ 6 weeks prior to screening.
    .Local palliative radiation therapy (small port): < 4 weeks prior to screening.
    -Bone marrow transplant:
    .Presence of graft versus host disease (GVHD)
    . < 6 months since allogeneic bone marrow transplant prior to screening.
    . < 3 months since autologous bone marrow/stem cell transplant prior to screening.
    . < 3 months since stem cell transplant (SCT) or Rescue without TBI with no evidence of GVHD prior to screening.
    - Radioisotopes: fluorothymidine (18FLT) ≤ 72 hours prior to first dose of study drug
    8. Subject is pregnant, breast-feeding or lactating.
    1. Antecedentes de trombosis relacionada con una vía no central (arterial o venosa), más de una trombosis previa relacionada con una vía central o coagulopatía.
    2. Familiar de primer grado con coagulopatía hereditaria conocida.
    3. Recepción activa de tratamiento anticoagulante.
    4. Intervención de cirugía mayor (a criterio del investigador), a excepción de resección del tumor, en los 21 días previos a la primera dosis del fármaco del estudio.
    5. Recepción previa (la presencia de cualquiera de las circunstancias siguientes excluirá a un sujeto de participar en el estudio) de:
    • Cualquier tratamiento previo con pomalidomida. Los sujetos que hayan recibido tratamiento previo con otros inmunomoduladores (talidomida, lenalidomida) PODRÁN participar siempre que cumplan todos los demás criterios de elegibilidad y no hayan presentado reacciones alérgicas ni otra “toxicidad significativa”, a criterio del investigador, asociada al uso de lenalidomida o talidomida.
    • Quimioterapia mielodepresora, inmunoterapia o cualquier fármaco en investigación: ≤ 28 días (≤ 42 días en caso de una nitrosourea) antes de la selección.
    • Tratamiento biológico (antineoplásico): ≤ 7 días antes de la selección.
    • Tratamiento inmunomodulador: ≤ 28 días antes de la selección.
    • Tratamiento con anticuerpos monoclonales y fármacos con semividas prolongadas conocidas: equivalente a < 3 semividas o ≤ 28 días antes de la selección, lo que suponga más tiempo.
    • Radioterapia previa:
     Radioterapia craneal, radioterapia corporal total (RCT) o irradiación ≥ 50 % de la pelvis: ≤ 3 meses antes de la selección.
     Radioterapia focal: ≤ 6 semanas antes de la selección.
     Radioterapia paliativa local (puerto pequeño): < 4 semanas antes de la selección.
    • Trasplante de médula ósea:
     Presencia de enfermedad del injerto contra el huésped (EICH).
     < 6 meses desde el alotrasplante de médula ósea antes de la selección.
     < 3 meses desde el autotrasplante de médula ósea/células madre antes de la selección.
     < 3 meses desde el trasplante de células madre (TCM) o rescate sin RCT sin signos de EICH antes de la selección.
    • Radioisótopos: fluorotimidina (18FLT) ≤ 72 horas previas a la primera dosis del fármaco del estudio.
    8.Embarazo o período de lactancia.
    E.5 End points
    E.5.1Primary end point(s)
    Objective response and long-term stable disease rate
    Respuesta objetiva y tasa de enfermedad estable a largo plazo
    E.5.1.1Timepoint(s) of evaluation of this end point
    Cycle 1, Day 1 to completion of Cycle 6 (completion of Cycle 3 for DIPG)
    Ciclo 1, Día 1 hasta la finalización del Ciclo 6 (finalización del Ciclo 3 para GPID).
    E.5.2Secondary end point(s)
    1. Objective response rate
    2. Long-term stable disease rate
    3. Duration of response
    4. Progression-free survival
    5. Overall Survival
    6. Safety
    1. Tasa de respuesta objetiva
    2. Tasa de enfermedad estable a largo plazo
    3. Duración de la respuesta
    4. Supervivencia libre de progresión
    5. Supervivencia general
    6. Seguridad
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Cycle 1, Day 1 of Treatment Period to end of Follow-up Period
    2. Cycle 1, Day 1 to completion of Cycle 6 (completion of Cycle 3 for DIPG)
    3. Cycle 1, Day 1 of Treatment Period to end of Follow-up Period
    4. Cycle 1, Day 1 of Treatment Period to end of Follow-up Period
    5. Cycle 1, Day 1 of Treatment Period to end of Follow-up Period
    6. Signing of informed consent form (ICF)/informed assent form (IAF) until the end of Follow-up Period
    1. Ciclo 1, Día 1 del Período de Tratamiento hasta el final del Período de Seguimiento
    2. Ciclo 1, Día 1 hasta la finalización del Ciclo 6 (finalización del Ciclo 3 para GPID)
    3. Ciclo 1, Día 1 del Período de Tratamiento hasta el final del Período de Seguimiento
    4. Ciclo 1, Día 1 del Período de Tratamiento hasta fel inal del Período de Seguimiento
    5. Ciclo 1, Día 1 del Período de Tratamiento hasta el final del Período de Seguimiento
    6. Firma del formulario de consentimiento informado (FCI) / formulario de asentimiento informado (FAI) hasta el final del Período de Seguimiento
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    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 No
    E.8.2.4Number of treatment arms in the trial4
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA17
    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
    France
    Italy
    Spain
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    The End of Trial is defined as either the date of the last visit of the last subject to complete the post-treatment follow-up, or the date of receipt of the last data point from the last subject that is required for primary, secondary and/or exploratory analysis, as prespecified in the protocol, whichever is the later date.
    El final del ensayo se define como la fecha de la última visita del último sujeto que complete el seguimiento posterior al tratamiento, o la fecha de recepción de los últimos datos correspondientes al último sujeto que sean necesarios para los análisis principales, secundarios o exploratorios, tal como se establece en el protocolo, la que sea más tardía.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days28
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days28
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 60
    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: 20
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 20
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 20
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 No
    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 No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Subjects under age incapable of giving consent personally.
    Personas menores de edad incapaces de dar su consentimiento personalmente.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 44
    F.4.2.2In the whole clinical trial 80
    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
    Ninguno
    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 Decision2017-07-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-06-27
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-05-04
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