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    Summary
    EudraCT Number:2011-006197-88
    Sponsor's Protocol Code Number:FANCOSTEM-1
    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:2012-01-27
    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 number2011-006197-88
    A.3Full title of the trial
    Clinical Phase II Trial to evaluate efficacy and safety of CD34+ cells mobilization and collection after treatment with plerixafor and filgrastim in patients with Fanconi anemia for subsequent transduction with a lentiviral vector carring FANCA gene and reinfusion in the patient
    Ensayo clínico Fase II para evaluar la seguridad y eficacia de la movilización y colecta de células CD34+ tras tratamiento con plerixafor y filgrastim en pacientes con Anemia de Fanconi para su posterior transducción con un vector lentiviral portador del gen FANCA y reinfusión en el paciente
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical Phase II Trial to evaluate efficacy and safety of CD34+ cells mobilization and collection after treatment with plerixafor and filgrastim in patients with Fanconi anemia for subsequent transduction with a lentiviral vector carring FANCA gene and reinfusion in the patient
    Ensayo clínico Fase II para evaluar la seguridad y eficacia de la movilización y colecta de células CD34+ tras tratamiento con plerixafor y filgrastim en pacientes con Anemia de Fanconi para su posterior transducción con un vector lentiviral portador del gen FANCA y reinfusión en el paciente
    A.3.2Name or abbreviated title of the trial where available
    FANCOSTEM-1
    FANCOSTEM-1
    A.4.1Sponsor's protocol code numberFANCOSTEM-1
    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 SponsorCristina Díaz de Heredia Rubio
    B.1.3.4CountrySpain
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportCristina Diaz de Heredia Rubio
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCristina Díaz de Heredia Rubio
    B.5.2Functional name of contact pointCristina Díaz de Heredia Rubio
    B.5.3 Address:
    B.5.3.1Street AddressPaseo Vall d?Hebron 119-129
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08035
    B.5.3.4CountrySpain
    B.5.4Telephone number0034934893093
    B.5.5Fax number0034934893883
    B.5.6E-mailcrdiaz@vhebron.net
    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 Mozobil
    D.2.1.1.2Name of the Marketing Authorisation holderGenzyme Europe B.V.
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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 namemozobil
    D.3.4Pharmaceutical form Injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 110078-46-1
    D.3.9.3Other descriptive namePLERIXAFOR
    D.3.9.4EV Substance CodeSUB28849
    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.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 Neupogen
    D.2.1.1.2Name of the Marketing Authorisation holderAmgen Europe B.V.
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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 nameNeupogen
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNFILGRASTIM
    D.3.9.1CAS number 121181-53-1
    D.3.9.4EV Substance CodeSUB07627MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.6
    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
    Fanconi anemia
    Anemia de Fanconi
    E.1.1.1Medical condition in easily understood language
    Fanconi anemia
    Anemia de Fanconi
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level LLT
    E.1.2Classification code 10055206
    E.1.2Term Fanconi's anemia
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The main objective is to assess the safety of cell mobilization after treatment with filgrastim and plerixafor in patients with Fanconi Anemia.
    El objetivo principal es determinar la seguridad de la movilización con filgrastim y plerixafor en pacientes diagnosticados de Anemia de Fanconi.
    E.2.2Secondary objectives of the trial
    - To assess the efficacy of CD34+ cell mobilization after treatment with filgrastim and plerixafor in patients with Fanconi Anemia.
    - To assess the CD34+ collection eficacy after treatment with high doses of filgrastim and plerixafor in patients with Fanconi Anemia.
    - To assess the efficacy of the immunomagnetic haematopoietic progenitors selection (CD34+ cells) from the apheresis product
    - Determinar la eficacia de movilización de células CD34+ a sangre periférica en pacientes diagnosticados de Anemia de Fanconi tras el tratamiento con filgrastim y plerixafor
    - Determinar la eficacia de la colecta de células CD34+ en pacientes diagnosticados de Anemia de Fanconi tras el tratamiento con filgrastim y plerixafor
    - Determinar la eficacia de la selección inmunomagnética de progenitores hematopoyéticos (células CD34+) del producto de aféresis
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - male or female > 1 year
    - diagnosed of Fanconi's anemia confirmed by instability chromosomal test with diepoxy-butane or mitomycin C
    - At least one of the following parameters must be higher than these values: Hemoglobin:8,0 g/dL; neutrophils: 750/mm3; platelets: 30.000/mm3
    - Lansky index> 60%.
    - Left ventricular ejection fraction >50%.
    - To grant informed consent in agreement with current law norms
    - Women in childbearing age must obtain a negative result in the pregnancy test in serum or urine in the visit of selection and accept the use of suitable contraceptive methods since at least 14 days prior to the first dose of mobilizing treatment until the 14 days following the last
    - Pacientes con diagnóstico de AF confirmado por un test de inestabilidad cromosómica con diepoxibutano o mitomicina C
    - Edad> 1 año.
    - Al menos uno de los siguientes parámetros debe superar los valores indicados: Hemoglobina:8,0 g/dL; Número de neutrófilos: 750/mm3; Número de plaquetas: 30.000/mm3.
    - Índice de Lansky > 60%.
    - Fracción de eyección del ventrículo izquierdo >50%.
    - Otorgar consentimiento informado de acuerdo con la normativa legal vigente.
    - Las mujeres en edad fértil deberán obtener un resultado negativo en la prueba de embarazo en suero o en orina en la visita de selección , y aceptar el empleo de métodos anticonceptivos adecuados al menos desde los 14 días previos a la primera dosis del tratamiento movilizador hasta los 14 días siguientes a la última.
    E.4Principal exclusion criteria
    - Evidence of myelodysplastic syndromes or leukemia, or cytogenetic abnormalities predicted of these syndromes in bone marrow aspiration. Cytogenetic analyses performed 2 months before starting study are accepted
    - Patients with active infection process or any other underlaying severe medical process
    - Severe Functional alteration of organs (hepatic, renal, respiratory)(?3), according to National Cancer Institute (NCI CTCAE v3) criteria
    - Haematopoietic transplant
    - Any disease or concomitant process that is not compatible with the study as per investigator opinion
    - Patients not elegible because of an psico-social evaluation
    - Patients that received transfusional support during the last 3 months.
    - Pregnant or breastfeeding women
    - Evidencias de síndrome mielodisplásico o leucemia, o anomalías citogenéticas predictivas de las mismas en aspirados de médula ósea Estudios citogenéticos realizados con dos meses de antelación se considerarán aceptables.
    - Pacientes con proceso infeccioso activo u otro estado médico subyacente grave.
    - Alteración funcional de órganos (hepática, renal, respiratorio) grave (?3),según los criterios del National Cancer Institute (NCI CTCAE v3).
    - Haber recibido trasplante hematopoyético.
    - Toda enfermedad o proceso concomitante que, en opinión del investigador, incapacite al sujeto para su participación en el estudio.
    - Pacientes que tras una evaluación psico-social se censuran como no aptos para el procedimiento.
    - Haber recibido soporte transfusional en los tres meses previos.
    - Mujeres embarazadas o en período de lactancia
    E.5 End points
    E.5.1Primary end point(s)
    Toxicity of the mobilization procedure according to National Cancer Institute (CTC NCI, versión 3.0)
    Toxicidad del protocolo de movilización según los criterios comunes de toxicidad del National Cancer Institute (CTC NCI, versión 3.0)
    E.5.1.1Timepoint(s) of evaluation of this end point
    It is anticipated that the patient will receive the medical discharged at the end of the last apheresis. Once the patient is given a medical discharge, hematologic controls are performed at 2 weeks after the last apheresis (in the center where the collection was made), at 2 months, at 6 months and 9 months (in the center of patient referral if your physician is a research trial, or of the collection).

    At a year (± 30 days) after the last apheresis, a complete physical examination, blood cell count, basic biochemistry and bone marrow aspirate will be done to the patient in order to control their general health status.
    Se prevé que el paciente será dado de alta al finalizar la última aféresis. Una vez dado el paciente de alta médica, se realizarán controles hematológicos a las 2 semanas de la última aféresis (en el centro donde se realizó la colecta), a los 2 meses, a los 6 meses y a los 9 meses (en el centro de referencia del paciente si su médico es investigador del ensayo, o en el de la colecta).

    Al año (±30 dias) de realizada la última aféresis, se realizará un examen físico completo, recuento celular sanguíneo, bioquímica básica y un aspirado de la médula ósea del paciente para control.
    E.5.2Secondary end point(s)
    - The effectiveness of the mobilization protocol will be determined by the percentage of patients who achieve peripheral blood counts exceeding 5 CD34+ cells /microliter.
    - The effectiveness of the CD34+ cell collection protocol will be determined by the percentage of patients who reach at least one million CD34 + cells per kilogram of body weight projected to 5 years after the mobilization process.
    - The effectiveness of the of CD34+ cell selection process will be determined by the proportion of immunoselected samples where the recovery of CD34 + cells is at least 50%, and where the final percentage of CD34+ cells is at least 50%.
    - The effectiveness of the procedure will be determined by the percentage of patients who reach at least one million CD34 + cells per kilo of weight projected to 5 years after the immunomagnetic selection process of all the collected cells.
    - Se determinará la eficacia de la movilización por el porcentaje de pacientes que alcancen en sangre periférica recuentos superiores a 5 células CD34+/microlitro.
    - Se determinará la eficacia de la recolección de células CD34+ por el porcentaje de pacientes que alcancen tras las aféresis más de un millón de células CD34+ por kilo de peso proyectado a 5 años tras la movilización.
    - Se determinará la eficacia de la selección de células CD34+ del producto obtenido tras aféresis, por el porcentaje de muestras en donde la recuperación de células CD34+ sea superior al 50%, y por el porcentaje final de células CD34+.
    -Se determinará la eficacia del procedimiento global por el porcentaje de pacientes que alcancen más de un millón de células CD34+ por kilo de peso proyectado a 5 años, tras la selección inmunomagnética de todas las aféresis realizadas.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Última aferesis
    Last apheresis
    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 No
    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 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 No
    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 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 concerned2
    E.8.5The trial involves multiple Member States No
    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 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
    Patients will be excluded or abandon their participation in the study for any of the following reasons:
    - Withdrawal of consent by the patient.
    - Evidence of disease progression.
    - Development of unacceptable toxicity in the investigator's opinion.
    - If it is consider relevant to the patient a change in treatment due to loss of clinical benefit, at the discretion of the investigator
    Los pacientes serán excluidos ó abandonarán su participación en el estudio por cualquiera de las siguientes razones:
    - Retirada del consentimiento por parte del paciente.
    - Evidencia de progresión de la enfermedad.
    - Desarrollo de toxicidad inaceptable a juicio del investigador.
    - Si se considera de interés para el paciente un cambio en el tratamiento por pérdida del beneficio clínico, según el criterio del investigador.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months0
    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: 8
    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.1.1.5.1Number of subjects for this age range: 6
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 2
    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
    children
    Niños
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    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)
    Preventional traeatments for Fanconi anemia patients. Probable treatment by gene therapy, genetically correcting their CD34+ cells
    Tratamientos preventivos para pacientes con Anemia de Fanconi. Posible tratamiento con terapia génica, corrigiendo genéticamente us células CDE34+
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Red Española de Investigación en Anemia de Fanconi
    G.4 Investigator Network to be involved in the Trial: 2
    G.4.1Name of Organisation Sociedad Española de Hematología y Oncología Pediátrica
    G.4 Investigator Network to be involved in the Trial: 3
    G.4.1Name of Organisation Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER)
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2012-03-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-03-09
    P. End of Trial
    P.End of Trial StatusCompleted
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