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    Summary
    EudraCT Number:2013-000144-26
    Sponsor's Protocol Code Number:ABI-007-PST-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:2013-09-05
    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 number2013-000144-26
    A.3Full title of the trial
    A Phase 1/2, multicenter, open-label, dose-finding study to assess the safety, tolerability, and preliminary efficacy of weekly nab-paclitaxel in pediatric patients with recurrent or refractory
    solid tumors.
    Estudio de fase 1/2 multicéntrico, abierto, de búsqueda de dosis para evaluar la seguridad, tolerabilidad y eficacia preliminar de nab®-paclitaxel semanal, en pacientes pediátricos con tumores sólidos en recaída o refractarios.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    clinical study for pediatric patients with solid tumors that have come back after disappearing or that don't respond to any treatment, designed to find the safest and most tolerable dose of nab®-paclitaxel and to asses the preliminary efficacy of the treatment.
    Estudio clínico de pacientes pediátricos con tumores sólidos que han recaído después de desaparecer la enfermedad o que no responden a ningún tratamiento, diseñado para encontrar la dosis más segura y más tolerable de nab-paclitaxel ® y para evaluar la eficacia preliminar del tratamiento
    A.4.1Sponsor's protocol code numberABI-007-PST-001
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/116/2013
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAbraxis BioScience, LLC, a wholly-owned subsidiary of Celgene Corporation
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportCelgene Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCelgene Corporation
    B.5.2Functional name of contact pointClinicalTrialDisclosure
    B.5.3 Address:
    B.5.3.1Street Address9225 Indian Creek Parkway, Suite 900
    B.5.3.2Town/ cityOverland Park, Kansas
    B.5.3.3Post code66210
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1-888-260-1599
    B.5.5Fax number+1 913-266-0394
    B.5.6E-mailClinicalTrialDisclosure@celgene.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Abraxane
    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.3.2Product code nab-paclitaxel, ABI-007
    D.3.4Pharmaceutical form Powder for suspension 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 INNPACLITAXEL
    D.3.9.1CAS number 33069-62-4
    D.3.9.2Current sponsor codeABI-007
    D.3.9.4EV Substance CodeSUB09583MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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 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 Yes
    D.3.11.13.1Other medicinal product typeIMP contiene un excipiente de origen biológico,albúmina,agente estabilizador no activo.Derivado de sangre humana de sujeto para selección de donantes aprobados y procesos de fabricación de productos
    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 solid tumor
    tumores sólidos en recaída o refractarios
    E.1.1.1Medical condition in easily understood language
    subjects with solid tumors that have come back after disappearing or that don't respond to any treatment
    personas con tumores sólidos en recaída o que no responden a ningún tratamiento
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level LLT
    E.1.2Classification code 10065147
    E.1.2Term Malignant solid tumor
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Phase I:
    determine the pediatric MTD/RP2D, safety and tolerability of nab-paclitaxel administered intravenously over 30min on Days 1, 8, and 15 of a 28-day cycle in patients ? 6 months and < 18 years old with recurrent or refractory solid tumors

    Phase II:
    assess the antitumor activity by the overall response rate (ORR) of nab-paclitaxel given at the RP2D in patients ? 6 months and ? 21 years old with several discrete recurrent or refractory solid tumor types such as
    neuroblastoma, rhabdomyosarcoma, and a mixed solid tumor group (e.g., nonrhabdomyosarcoma soft tissue sarcomas, melanoma, or tumors in which the drug showed activity during the Phase 1
    portion of the study and/or in preclinical studies).
    Parte de fase 1:
    Determinar la dosis pediátrica máxima tolerada (DMT)/dosis
    pediátrica recomendada para la fase 2 (DRF2), la seguridad y la tolerabilidad.

    Parte de fase 2:
    Determinar la actividad antitumoral de la DRF2 mediante la tasa de
    respuesta global (TRG).
    E.2.2Secondary objectives of the trial
    Phase I:
    - To evaluate pharmacokinetics (PK).
    - To characterize the ORR

    Phase II:
    - To characterize duration of response (DOR).
    - To characterize the disease control rate (DCR).
    - To characterize progression-free survival (PFS).
    - To characterize 1-year survival.
    - To confirm safety.
    - To evaluate PK.
    Parte de fase 1:
    Evaluar la farmacocinética (FC) y determinar la TRG.

    Parte de fase 2:
    Determinar la duración de la respuesta (DR), la tasa de control de la enfermedad (TCE), la supervivencia sin progresión (SSP) y la supervivencia al cabo de 1 año. Confirmar la seguridad y evaluar la FC.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patient is male or female, meeting the following age requirements at the time the informed consent document (and assent form, if applicable) is signed.
    a. Phase 1: patient is >= 6 months to < 18 years of age
    b. Phase 2: patient is >= 6 months to <= 21 years of age

    2. Patient has a confirmed solid tumor diagnosis according to the following:
    a. Phase 1: patient has a recurrent or refractory solid tumor that has progressed or did not respond to standard therapy, or for which no standard anticancer therapy exists
    b. Phase 2: patient has one of the following tumor types and has failed first or second-line treatment or has evidence of refractory disease
    i. Group 1: neuroblastoma
    ii. Group 2: rhabdomyosarcoma
    iii. Group 3: mixed solid tumors, including nonrhabdomyosarcoma soft tissue sarcoma, melanoma, or other tumor types identified during the Phase 1 portion or in preclinical studies

    3. The patient has a Lansky/ Karnofsky performance status score of >= 70%

    4. The patient has adequate serum chemistry levels, evidenced by the following laboratory values
    a. AST (SGOT), ALT (SGPT) <= 2.5 × upper limit of normal range (ULN)
    b. Total bilirubin <= 1.5 × ULN
    c. Creatinine <= 1.5 × ULN

    5. The patient has adequate bone marrow function, evidenced by the following:
    a. Absolute neutrophil count >= 1.0 × 10^9 cells/L
    b. Platelets >= 80 × 10^9 cells/L (transfusion independent, defined as not receiving platelet transfusions within 7 days prior to laboratory sample)
    c. Hemoglobin >= 8 g/dL (transfusion is permitted to fulfill this criterion)

    6. The patient (when applicable) or patient's parent(s) or legal guardian(s) understand(s) and voluntarily signed an informed consent document prior to any study-related assessments/ procedures being conducted. Where locally applicable, the patient also understands and voluntarily provides his/her assent prior to any study-related assessments/procedures being conducted.

    7. Male patients of childbearing potential must use a condom during sexual intercourse and shall not father a child during the study and for 6 months after the last dose of study medication.

    8. Female patients of childbearing potential [defined as all female patients >= 12 years old or who have reached menarche, whichever occurs first] must have both of the following:
    a. Agree to the use of two physician-approved contraceptive methods simultaneously or practice complete abstinence while on study medication and for 3 months following the last dose of study medication
    i. True abstinence: When this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception.
    ii. Acceptable contraceptive methods include: oral, injectable, or implantable hormonal contraceptive; tubal ligation; intra-uterine device; barrier contraceptive with spermicide; or vasectomized partner) including at least one barrier method.

    b. Have negative serum pregnancy test result at screening confirmed by negative urine pregnancy dipstick within 72 hours prior to first dose of IP (if serum test occurred > 72 hours from first dose); pregnancy test with sensitivity of at least 25 mIU/mL
    1. Pacientes de ambos sexos, que cumplan los siguientes requisitos de edad en el momento
    de la firma del documento de consentimiento informado (y asentimiento, si procede).
    a. Fase 1: el paciente es ? 6 meses y < 18 años de edad.
    b. Fase 2: el paciente es ? 6 meses y ? 21 años de edad.

    2. El paciente tiene un diagnóstico de tumor sólido confirmado con arreglo a lo siguiente:
    a. Fase 1: el paciente tiene un tumor sólido en recaída o refractario que ha
    progresado o que no respondió al tratamiento habitual, o para el que no existe
    ningún tratamiento anticanceroso de referencia.
    b. Fase 2: el paciente tiene uno de los siguientes tipos de tumor con fracaso del
    tratamiento de primera o segunda línea, o presenta evidencias de enfermedad
    refractaria.
    i. Grupo 1: neuroblastoma
    ii. Grupo 2: rabdomiosarcoma
    iii. Grupo 3: tumores sólidos misceláneos como sarcomas de tejidos blandos
    distintos del rabdomiosarcoma, melanoma, u otros tipos de tumores
    identificados en la parte de fase 1 o en los estudios preclínicos

    3. El paciente tiene una puntuación en el estado funcional de Lansky/Karnofsky ? 70 %.

    4. El paciente tiene valores de bioquímica sérica adecuados, definidos por lo siguiente:
    a. AST (SGOT) y ALT (SGPT) ? 2,5 × límite superior de la normalidad (LSN)
    b. Bilirrubina total ? 1,5 x LSN
    c. Creatinina ? 1,5 × LSN

    5. El paciente tiene una función medular adecuada, evidenciada por lo siguiente:
    a. Recuento absoluto de neutrófilos ? 1,0 × 10^9/l
    b. Plaquetas ? 80 × 10^9/l (no dependiente de transfusiones, definido como ausencia
    de transfusiones de plaquetas en los 7 días previos a la obtención de la muestra)
    c. Hemoglobina ? 8 g/dl (se permite la transfusión para cumplir este criterio)

    6. El paciente (si procede) o su(s) progenitor(es) o representante legal ha entendido y
    firmado voluntariamente el documento de consentimiento informado antes de que se
    realice cualquier evaluación o procedimiento relacionado con el estudio. Si lo exige la
    normativa local, el paciente también ha entendido y otorgado voluntariamente su
    asentimiento antes de que se realice cualquier evaluación o procedimiento relacionado
    con el estudio.

    7. Los varones en edad fértil deberán utilizar preservativo durante las relaciones sexuales y
    no podrán engendrar durante el estudio y hasta 6 meses después de la última dosis de la
    medicación del estudio.

    8. Las pacientes en edad fértil [definidas como todas las pacientes ? 12 años o que hayan
    tenido la menarquia, lo que ocurra antes] deben cumplir las dos condiciones siguientes:
    a. Comprometerse a utilizar de forma simultánea dos métodos anticonceptivos
    aprobados por el médico o practicar la abstinencia sexual completa mientras estén
    recibiendo la medicación del estudio y hasta 3 meses después de la última dosis
    de la medicación del estudio.
    i. Abstinencia completa: si se adecua al tipo de vida preferido y habitual de
    la paciente. La abstinencia periódica (p. ej., métodos del calendario, de la
    ovulación, sintotérmico y de postovulación) y el método de retirada no son
    métodos anticonceptivos aceptables.
    ii. Son métodos anticonceptivos aceptables los siguientes: anticonceptivos
    hormonales orales, inyectables o en implantes, ligadura de trompas,
    dispositivo intrauterino, anticonceptivo de barrera con espermicida o
    vasectomía de la pareja, con al menos un método de barrera.
    b. Tener un resultado negativo en la prueba de embarazo en suero de la selección,
    que se confirmará con una prueba de embarazo negativa con tira reactiva en orina
    en las 72 horas previas a la primera administración del PEI si la prueba en suero
    se realizó más de 72 horas antes de la misma; la prueba de embarazo debe tener
    una sensibilidad de al menos 25 mUI/ml.
    E.4Principal exclusion criteria
    1. The patient has a primary brain tumor(s) or brain metastasis (unless metastasis is treated and stable for > 28 days). In patients who are symptomatic, a brain scan is required to exclude metastasis.

    2. The patient has received therapeutic dose chemotherapy or radiotherapy <= 21 days prior to start of IP.

    3. The patient has received maintenance dose chemotherapy (e.g., low dose cyclophosphamide) <= 7 days from the first dose of IP.

    4. The patient has received any investigational therapy <= 28 days prior to start of IP.
    Investigational therapy is defined as any medicinal product that is not approved in the country of treatment for any indication, adult or pediatric

    5. The patient has received any biological therapy <= 7 days prior to the start of IP, or monoclonal antibody <= 3 half-lives or 28 days, whichever is shorter, prior to the first dose of IP.

    6. The patient has had major surgery or significant trauma <= 14 days prior to start of IP.

    7. The patient has not recovered from the acute toxic effects of prior chemotherapy, radiation, or major surgery/significant trauma.

    8. The patient has had minor surgery <= 7 days from the start of study treatment (excluding the placement of central/
    peripheral lines, skin biopsy).

    9. The patient has a known history of stroke, myocardial infarction, peripheral vascular disease, or recent (within 3 months) uncontrolled deep venous thrombosis.

    10. The patient has a known history or current diagnosis of HIV infection, regardless of treatment status.

    11. The patient has an uncontrolled intercurrent illness including but not limited to ongoing or active infection requiring antibiotic, antifungal, or antiviral therapy, symptomatic heart failure, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.

    12. The patient has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the patient from participating in the study.

    13. The patient has any condition, including the presence of laboratory abnormalities, that places him/her at unacceptable risk if he/she were to participate in the study.

    14. The patient has any condition that confounds the ability to interpret data from the study.

    15. The patient or parent(s)/guardian(s) is/are unable to comply with the study visit schedule and other protocol requirements, in the opinion of the investigator.
    1. El paciente tiene un tumor cerebral primario o metástasis cerebral (a menos que la
    metástasis se haya tratado y se haya mantenido estable durante > 28 días). En los
    pacientes que presenten síntomas, se requiere una prueba de imagen cerebral para
    descartar metástasis.

    2. El paciente ha recibido quimioterapia en dosis terapéuticas o radioterapia ? 21 días antes
    del inicio del PEI.

    3. El paciente ha recibido quimioterapia en dosis de mantenimiento (por ejemplo,
    ciclofosfamida en dosis bajas) ? 7 días antes de la primera administración del PEI.

    4. El paciente ha recibido cualquier tratamiento en investigación ? 28 días antes del inicio
    del PEI. Tratamiento en investigación se define como cualquier medicamento que no esté
    aprobado en ese país para ninguna indicación, pediátrica o en adultos.

    5. El paciente ha recibido cualquier tratamiento biológico ? 7 días antes del inicio del PEI, o
    anticuerpo monoclonal ? 3 semividas o 28 días, lo que sea más corto, antes de la primera
    administración del PEI.

    6. El paciente se ha sometido a una intervención de cirugía mayor o ha sufrido un
    traumatismo importante ?14 días antes del inicio del PEI.

    7. El paciente no se ha recuperado de los efectos tóxicos agudos de una quimioterapia o
    radioterapia previa, o de una intervención de cirugía mayor o un traumatismo importante

    8. El paciente se ha sometido a una intervención de cirugía menor ? 7 días antes del inicio
    del tratamiento del estudio (excepto colocación de vías centrales/periféricas o biopsia
    cutánea).

    9. El paciente tiene antecedentes conocidos de ictus, infarto de miocardio, vasculopatía
    periférica o trombosis venosa profunda no controlada reciente (en los 3 meses previos).

    10. El paciente tiene antecedentes o un diagnóstico actual de infección por el VIH,
    independientemente de la situación de tratamiento.

    11. El paciente presenta una enfermedad intercurrente no controlada, como por ejemplo,
    infección activa o en curso que requiera antibióticos, antimicóticos o antivirales,
    insuficiencia cardiaca sintomática, arritmia cardiaca o enfermedad psiquiátrica o
    condiciones sociales que podrían limitar el cumplimiento de los requisitos del estudio.

    12. Cualquier enfermedad, anomalía analítica o trastorno psiquiátrico que impediría al
    paciente participar en el estudio.

    13. El paciente presenta cualquier trastorno, incluida la presencia de anomalías analíticas,
    que entrañe un riesgo inaceptable para el paciente en caso de participar en el estudio.

    14. El paciente presenta cualquier trastorno que altere la capacidad de interpretar los datos
    del estudio.

    15. El paciente o su(s) progenitor(es)/representante legal no puede cumplir el calendario de
    visitas del estudio u otros requisitos del protocolo, en opinión del investigador.
    E.5 End points
    E.5.1Primary end point(s)
    Phase I:
    Incidence of DLTs and the incidence of treatment-emergent adverse events (TEAEs).

    Phase II:
    ORR (CR + PR) confirmed no less than 4 weeks after the criteria for response are first met, based on RECIST version 1.1 criteria
    Parte de fase 1:
    incidencia de TLD y de acontecimientos adversos aparecidos durante el tratamiento (AAAT).

    Parte de fase 2:
    TRG (RC + RP) confirmada no menos de 4 semanas después de que los criterios de respuesta se cumplan por primera vez, según los criterios RECIST, versión 1.1.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Phase I:
    Initial 1-2 cycles of treatment

    Phase II:
    Expected 12-16 weeks after starting treatment; longer depending on the patient
    Parte de fase 1:
    en los ciclos 1 y 2 iniciales

    Parte de fase 2:
    Entre 12 y 16 semanas después del comienzo del tratamiento; esto puede ser más tarde dependiendo del paciente
    E.5.2Secondary end point(s)
    Phase I:
    1) PK parameters including the maximum observed concentration in blood (Cmax), area under the blood concentration-time curve (AUC), clearance and volume of distribution (Vss).
    2) ORR

    Phase II:
    1) DoR in patients with a confirmed objective CR or partial response (PR). DCR is the percentage of patients with a confirmed objective CR or PR, or stable disease for at least 16 weeks.
    2) PFS based on investigator assessment of response using RECIST 1.1 guidelines
    3) Survival at 1 year
    4) The incidence of TEAEs
    5) Population PK parameters
    Parte de fase 1:
    1) Parámetros farmacocinéticos (FC) como concentración máxima observada en sangre (Cmáx), área bajo la curva concentración-tiempo (AUC), aclaramiento y volumen de distribución (Vee).
    2) TRG.

    Parte de fase 2:
    1) DR en pacientes con una respuesta completa (RC) o respuesta parcial (RP) objetiva confirmada. TCE es el porcentaje de pacientes con RC o RP objetiva confirmada o con enfermedad estable durante al menos 16 semanas.
    2) SSP basada en la evaluación de la respuesta por el investigador según los RECIST 1.1.
    3) Supervivencia al cabo de 1 año.
    4) Incidencia de AAAT.
    5) Parámetros de FC poblacional.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Phase I:
    1) and 2) Up to 4 days after the first treatment.

    Phase II:
    1)Until progression according to RECIST 1.
    Expected 24 weeks after treatment starts; longer depending on the patient.
    2)Until PFS event
    3)1 Year after starting treatment
    4)Up to 28 days after stopping study treatment
    5)Up to 4 days after the first treatment.
    Parte de fase 1:
    1) y 2) hasta 4 dias después del primer tratamiento

    Parte de fase 2:
    1) hasta la progression de la enfermedad según los RECIST 1.1. Unas 24 semanas después del inicio del tratamiento; o más tarde dependiendo del paciente.
    2) Hasta SSP
    3) 1 año después del inicio del tratamiento
    4) hasta 28 días después de la interrupción del tratamiento
    5) hasta 4 días después del primer tratamiento
    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 No
    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) 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 I / II
    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 No
    E.8.1.1Randomised Information not present in EudraCT
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    fase 1: diseño "rolling-6" de aumento escalonado de la dosis; fase 2: tratamiento con la DMT/DRF2
    phI: rolling-6 dose escalation; phII: treatment with MTD/RP2D
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    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
    Canada
    France
    Italy
    Spain
    Switzerland
    United Kingdom
    United States
    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
    After the EOT visit, all patients will be followed for survival until death, lost to follow-up, or one year after EOT, whichever occurs first.
    The study will end when all patients have completed the posttreatment follow-up (max 1 year) or have died, whichever occurs first
    Después de la visita de final del tratamiento (FDT), se hará un seguimiento de la supervivencia de todos los pacientes hasta su muerte o su pérdida para el seguimiento o hasta un año después del FDT, lo que ocurra antes.
    El ensayo acabará cuando todos los pacientes hayan completado el seguimiento después de la FDT (máx 1 año) o hayan muerto, lo que ocurra antes.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months1
    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.1.1Number of subjects for this age range: 100
    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: 11
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 45
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 44
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 5
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Minors (< 18 years old) will not sign an informed consent but they will be asked for assent
    los menores de < 18 años no firmará un consentimiento informado pero se les pedirá su asentimiento
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state27
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 81
    F.4.2.2In the whole clinical trial 105
    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)
    Patients may remain on treatment until disease progression, unacceptable toxicity, until they begin a new anticancer therapy, withdrawal of consent, parent/guardian/patient refusal, physician decision or death Upon treatment discontinuation from any of these reasons, patients will be treated at the investigator decision
    Los pacientes seguirán en tratamiento hasta que su enfermedad progrese, que sufran una toxicidad considerada inaceptable, que empiezen otro tratamiento anticanceroso, que retiren su consentimiento, que rechacen el tratamiento (o que lo rechacen los padres o tutores en el caso de los menores), que lo decida el médico o que mueran. Una vez que se interrumpa el tratamiento por una de esas razones, los pacientes serán tratados de acuerdo con el criterio del investigador principal.
    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 Decision2013-10-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-10-04
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-11-06
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