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    EudraCT Number:2010-019340-40
    Sponsor's Protocol Code Number:MOZ15609
    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:2013-10-18
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2010-019340-40
    A.3Full title of the trial
    A Phase 1/2 Combined Dose Ranging and Randomised, Open-label, Comparative Study of the Efficacy and Safety of Plerixafor in Addition to Standard Regimens for Mobilisation of Haematopoietic Stem Cells into Peripheral Blood, and Subsequent Collection by Apheresis, Versus Standard Mobilisation Regimens Alone in Paediatric Patients, Aged 2 to <18 Years, with Solid Tumours Eligible for Autologous Transplants
    Estudio combinado de fase 1/2 de búsqueda de dosis y comparativo, abierto, aleatorizado, para evaluar la eficacia y seguridad de plerixafor junto con regímenes estándar para la movilización de células madre hematopoyéticas a sangre periférica, y posterior recolección por aféresis, frente a solo regímenes estándar para la movilización en pacientes pediátricos, de 2 a <18 años, con tumores sólidos que reúnen los requisitos para trasplantes autólogos
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Combined Study in Pediatric Cancer Patients for Dose Ranging and Efficacy/Safety of Plerixafor Plus Standard Regimens for Mobilization Versus Standard Regimens Alone
    Estudio combinado en pacientes pediátricos con cáncer para evaluar la eficacia y seguridad de plerixafor junto con regímenes estándar para la movilización de células madre frente regímenes estándar solos
    A.4.1Sponsor's protocol code numberMOZ15609
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01288573
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/48/2010
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorGenzyme Europe B.V.
    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 supportGenzyme Europe B.V.
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationsanofi-aventis, s.a.
    B.5.2Functional name of contact pointUnidad de Estudios Clínicos
    B.5.3 Address:
    B.5.3.1Street Addressc/ Josep Pla nº2
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08019
    B.5.4Telephone numberNA
    B.5.5Fax numberNA
    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. name Mozobil
    D. of the Marketing Authorisation holderGenzyme Europe BV
    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 Yes
    D.2.5.1Orphan drug designation numberEu/3/04/227
    D.3 Description of the IMP
    D.3.1Product namePlerixafor
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNplerixafor
    D.3.9.1CAS number 110078-46-1
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    Paediatric cancer patients (aged 2 to <18 years) with Ewing?s sarcoma/soft tissue sarcoma, neuroblastoma, brain tumours, and all other malignancies (excluding leukaemia) who are planned to undergo high dose chemotherapy followed by autologous haematopoietic stem cell transplantation (HSCT) rescue
    Pacientes pediátricos, de 2 a <18 años, con Sarcoma de Ewing/sarcoma de tejidos blandos, neuroblastoma, tumores cerebrales, y todos los otros tumores malignos (con exclusión de la leucemia) que tienen previsto someter a altas dosis de quimioterapia seguido de un transplante autólogo de células madre hematopoyéticas
    E.1.1.1Medical condition in easily understood language
    Solid tumours (excluding leukaemia) which will be treated with high dose chemotherapy followed by transplantation of the child's own stem cells.
    Tumores sólidos (con exclusión de leucemia) que serán tratados con altas dosis de quimioterapia seguido de un transplante de células madre propias del niño.
    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
    The primary objective of this study is to confirm the appropriate dose and efficacy, and to characterise the safety, pharmacokinetics and pharmacodynamics of plerixafor across age and size in paediatric cancer patients when given in addition to standard mobilisation of HSCs into peripheral blood.
    El objetivo principal de este estudio es confirmar la dosis adecuada y la eficacia, y caracterizar la seguridad, farmacocinética y farmacodinamia de plerixafor según la edad y el tamaño en pacientes pediátricos con cáncer, cuando se administra junto con el tratamiento habitual para la movilización de células madre hematopoyéticas (haematopoietic stem cells, HSC) a sangre periférica.
    E.2.2Secondary objectives of the trial
    The secondary objective of this study is to confirm the efficacy and safety of plerixafor in addition to standard mobilisation of HSCs into peripheral blood, and subsequent collection by apheresis, in paediatric cancer patients.
    El objetivo secundario de este estudio es confirmar la eficacia y seguridad de plerixafor añadidoal tratamiento habitual para la movilización de HSC a sangre periférica, y la posterior recolección por aféresis, en pacientes pediátricos con cáncer.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age 2 to <18 years
    2. Ewing?s sarcoma, soft tissue sarcoma, neuroblastoma, brain tumours or other
    malignancy (excluding any form of leukaemia) requiring treatment with high dose
    chemotherapy and autologous transplant as rescue therapy
    3. Eligible for autologous transplantation
    4. Recovered from all acute significant toxic effects of prior chemotherapy
    5. Adequate performance status
    ? for patients ?16 years of age, defined as Karnofsky score >60
    ? for patients <16 years of age, defined as Lansky score >60
    6. Absolute neutrophil count >1.0 × 10P9/L
    7. Platelet count >75 × 10P9/L
    8. Calculated creatinine clearance (using the Schwartz method):
    ? during study Stage 1, >80 mL/min/1.73mP2
    ? during study Stage 2, >60 mL/min/1.73mP2
    9. Aspartate aminotransferase/serum glutamic oxaloacetic transaminase
    (AST/SGOT), alanine aminotransferase/serum glutamic pyruvic transaminase
    (ALT/SGPT) and total bilirubin <3 × upper limit of normal
    10. The patient and/or their parent/legal guardian is willing and able to provide signed informed consent
    1. Edad de 2 a <18 años
    2. Sarcoma de Ewing, sarcoma de tejidos blandos, neuroblastoma, tumores cerebrales u otras neoplasias malignas (excluyendo cualquier forma de leucemia) que requieran tratamiento con quimioterapia a altas dosis y trasplante autólogo como terapia de rescate
    3. Apto para trasplante autólogo
    4. Recuperado de todos los efectos tóxicos agudos significativos de la quimioterapia previa
    5. Estado funcional adecuado
    ? para pacientes ?16 años, definido como índice de Karnofsky >60
    ? para pacientes <16 años, definido como índice de Lansky >60
    6. Recuento absoluto de neutrófilos >1,0 × 109/l
    7. Recuento plaquetario >75 × 109/l
    8. Aclaramiento de creatinina calculado (utilizando el método Schwartz):
    ? durante la Etapa 1 del estudio, >80 ml/min/1,73m2
    ? durante la Etapa 2 del estudio, >60 ml/min/1,73m2
    9. Aspartato aminotransferasa/transaminasa glutámica oxalacética sérica (AST/SGOT), alanina aminotransferasa/transaminasa glutámica pirúvica sérica (ALT/SGPT) y bilirrubina total <3 × límite superior de la normalidad
    10. El paciente y/o su padre, madre o tutor legal deberán estar dispuestos y capacitados para proporcionar el consentimiento informado firmado
    11. Los pacientes sexualmente activos deben aceptar abstenerse de mantener relaciones sexuales o aceptar utilizar un método anticonceptivo aprobado mientras reciban plerixafor o el tratamiento de movilización estándar y durante al menos 3 meses después de cualquier tratamiento con plerixafor
    E.4Principal exclusion criteria
    1. Any form of leukaemia
    2. A co-morbid condition which, in the view of the Investigator, renders the patient
    at high-risk from treatment complications
    3. Previous stem cell transplantation
    4. Patients with tumours frequently involving bone marrow (e.g., lymphomas and
    neuroblastoma) will be expected to have had evaluation of their marrow as part of
    their standard staging evaluations. Persistent high percentage marrow
    involvement prior to mobilisation will be prohibited. (Specific guidelines for
    different indications are provided in Section 9.2.1 with details of bone marrow
    examination requirements at Screening)
    5. A residual acute medical condition resulting from prior chemotherapy
    6. Acute infection
    7. Fever (temperature >38.5°C) - if fever is between 37°C and 38.5°C, infection
    must be excluded as a cause
    8. Pulse oximetry ?92%
    9. Known HIV positive
    10. Positive pregnancy test in post pubertal girls
    11. History of clinically significant cardiac abnormality or arrhythmia
    12. Use of an investigational drug which is not approved in any indication either in
    adults or paediatrics within 4 weeks prior to the first dose of G-CSF to be
    administered as part of the patient?s planned standard mobilisation regimen,
    and/or during the study up until engraftment of the transplant. Drugs approved
    for other indications that are being used in a manner considered standard of care
    for this transplant procedure are allowed
    13. The patient (and/or their parent/legal guardian), in the opinion of the Investigator,
    is unable to adhere to the requirements of the study
    1. Cualquier forma de leucemia
    2. Una enfermedad comórbida que, desde el punto de vista del Investigador, pone al paciente en alto riesgo de sufrir complicaciones derivadas del tratamiento
    3. Trasplante de células madre previo
    4. Se espera que los pacientes con tumores que suelen afectar a la médula ósea (p. ej., linfomas y neuroblastoma) hayan efectuado una evaluación de la misma como parte de sus evaluaciones de estadificación. La afectación persistente de un alto porcentaje de la médula ósea previo a la movilización estará prohibida.
    5. Una afección médica aguda residual como resultado de la quimioterapia previa
    6. Infección aguda
    7. Fiebre (temperatura >38,5 °C); si la fiebre se sitúa entre 37 °C y 38,5 °C, debe excluirse la infección como causa de la misma
    8. Saturación de oxígeno por pulsioximetría ?92 %
    9. VIH positivo conocido previamente
    10. Prueba de embarazo positiva en niñas postpuberales
    11. Antecedentes de arritmia o anomalía cardíaca clínicamente significativas
    12. Uso de un fármaco en investigación que no ha sido aprobado para ninguna indicación en adultos o en pacientes pediátricos en las 4 semanas previas a la primera dosis de G-CSF administrada como parte del tratamiento de movilización estándar previsto para el paciente o durante el estudio hasta el injerto del trasplante. Se permite el uso de fármacos aprobados para otras indicaciones que se utilicen de forma estándar para este tipo de trasplante
    13. En opinión del Investigador, el paciente (o su padre, madre o tutor legal) no es capaz de cumplir los requisitos del estudio
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint will be the difference between the 2 treatment arms in Stage 2 (comparative part of the study) in the proportion of patients achieving at least a doubling of peripheral blood CD34+ count from the morning of the day preceding the first apheresis day to the morning prior to apheresis.
    El criterio de valoración principal de la eficacia será la diferencia entre los 2 brazos de tratamiento en la Etapa 2 (parte comparativa del estudio) en la proporción de pacientes que alcancen por lo menos el doble del recuento de células CD34+ en sangre periférica desde la mañana del día anterior a la primera aféresis hasta la mañana antes de la aféresis.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to 5 days
    Hasta 5 días
    E.5.2Secondary end point(s)
    ?Number of days of apheresis required to reach ?2 × 10^6 CD34+ cells/kg [ Time Frame: Up to 5 days ] During Stage 1 and Stage 2
    ?Yield of CD34+ cells for each apheresis [ Time Frame: Up to 5 days ]
    During Stage 1 and Stage 2
    ?Total CD34+ cell yield [ Time Frame: Up to 5 days ] During Stage 1 and Stage 2
    ?Percentage of patients proceeding to transplant [ Time Frame: Within 6 months of last apheresis ] During Stage 1 and Stage 2
    ?Percentage of patients successfully engrafting [ Time Frame: 3, 6, 12 and 24 months post-transplant ] During Stage 1 and Stage 2
    ?Percentage of patients with durable engraftment [ Time Frame: 3, 6, 12 and 24 months post-transplant ] During Stage 1 and Stage 2
    ?Summary of adverse events (AEs) [ Time Frame: Up to 24 months after last transplant or 24 months after last dose (for patients that do not transplant within 6 months of last apheresis) ] During Stage 1 and Stage
    ?Duration of hospitalizations (planned or unplanned) [ Time Frame: Throughout the duration of the study ] During Stage 1 and Stage 2
    ?Mobilization of tumor cells into peripheral blood [ Time Frame: Up to 5 days ] During Stage 1 and Stage 2
    ?Relapse rates [ Time Frame: 3, 6, 12 and 24 months post-transplant ]
    During Stage 1 and Stage 2
    ?Occurrence of secondary malignancies [ Time Frame: 3, 6, 12 and 24 months post-transplant ] During Stage 1 and Stage 2
    ?Incidence of primary and secondary graft failure [ Time Frame: 3, 6, 12 and 24 months post-transplant ] During Stage 1 and Stage 2
    ?Time to secondary graft failure [ Time Frame: Up to 24 months posttransplant] During Stage 1 and Stage 2
    ?Survival rates [ Time Frame: 3, 6, 12 and 24 months post-transplant ]
    During Stage 1 and Stage 2
    1. Número de días de aféresis necesarios para alcanzar ?2 × 106 células CD34+/kg
    2. Producción de células CD34+ por cada aféresis
    3. Producción total de células CD34+
    4. Porcentaje de pacientes a los que se realiza el trasplante
    5. Porcentaje de pacientes con éxito del injerto
    6. Porcentaje de pacientes con injerto duradero a los 3, 6, 12 y 24 meses tras el trasplante
    7. Incidencia de AA, incluyendo
    8. Incidencia de fiebre, neutropenia febril e infecciones
    9. Incidencia e intensidad de las reacciones en el sitio de la inyección
    10. Duración de las hospitalizaciones (previstas o no) durante el estudio
    11. Movilización de las células tumorales a sangre periférica la mañana previa a la aféresis y la mañana de la primera aféresis, y en el producto de la aféresis en la recogida, y justo antes de la infusión (cuando sea posible analizarlo)
    12. Tasas de recidivas
    13. Frecuencia de neoplasias malignas secundarias
    14. Incidencia de fracaso primario y secundario del injerto
    15. Tiempo hasta el fracaso secundario del injerto
    16. Tasas de supervivencia a los 3, 6, 12 y 24 meses tras el trasplante
    17. Recuento de células CD34+ trasplantadas (infundidas)
    18. Porcentaje de pacientes con un aumento de 2 veces en el recuento de células CD34+ en sangre periférica Y que alcancen el nivel umbral específico del centro requerido para iniciar la aféresis
    19. Aumento en los recuentos de células CD34+ en sangre periférica
    20. Volumen de sangre total procesada
    21. Tiempo hasta el prendimiento del injerto de neutrófilos (en días)
    22. Tiempo hasta el prendimiento del injerto de plaquetas (en días)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Up to 5 days
    Hasta 5 días
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Stage 1 only: dose ranging
    Etapa 1 (estudio de escalado de dosis)
    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. trial type description
    Stage 1 only: dose ranging
    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 Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E. trial design description
    Stage 2 only: a comparative randomised study
    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. description
    Stage 2 only: standard mobilisation treatment
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    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 No
    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
    Please refer to Protocol
    Definida en el Protocolo
    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 months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years6
    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.1.1Number of subjects for this age range: 67
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F. of subjects for this age range: 44
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 23
    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 Yes
    F. of subjects incapable of giving consent
    Children age 2-18 Parents sign ICF
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 67
    F.4.2.2In the whole clinical trial 67
    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)
    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 Decision2013-12-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-11-15
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-05-09
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