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    Summary
    EudraCT Number:2011-006100-12
    Sponsor's Protocol Code Number:FANCOLEN-1
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    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 number2011-006100-12
    A.3Full title of the trial
    Clinical Trial Phase I / II to evaluate the safety and efficacy of the infusion of autologous CD34+ cells transduced with a lentiviral vector carrying the FANCA gene (orphan drug) for patients with Fanconi Anemia Subtype A.
    Ensayo clínico Fase I/II para evaluar la seguridad y eficacia de la infusión de células CD34+ autólogas transducidas con un vector lentiviral portador del gen FANCA (medicamento huérfano) para pacientes con Anemia de Fanconi del Subtipo A.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical Trial Phase I / II to evaluate the safety and efficacy of the infusion of cells transduced with a therapeutic lentiviral vector for patients with Fanconi Anemia Subtype A.
    Ensayo clínico Fase I/II para evaluar la seguridad y eficacia de la infusión de células transducidas con un vector lentiviral terapeutico para pacientes con Anemia de Fanconi del Subtipo A.
    A.3.2Name or abbreviated title of the trial where available
    Fancolen-1
    Fancolen-1
    A.4.1Sponsor's protocol code numberFANCOLEN-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 SponsorFUNDACION PARA LA INVESTIGACION BIOMEDICA DEL HOSPITAL UNIVERSITARIO NIÑO JESUS
    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 supportFundacion para la investigacion Biomédica del Hospital Universitario Niño Jesus
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationHospital Niño Jesus
    B.5.2Functional name of contact pointServicio Oncohematologia
    B.5.3 Address:
    B.5.3.1Street AddressMenendez Pelayo, 65
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28009
    B.5.3.4CountrySpain
    B.5.4Telephone number+34915035938
    B.5.5Fax number+34915744669
    B.5.6E-mailjsevilla.hnjs@salud.madrid.org
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/10/822
    D.3 Description of the IMP
    D.3.1Product nameCD34+ Cells
    D.3.4Pharmaceutical form 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 INNCD34+ CELLS
    D.3.9.3Other descriptive nameCD34+ CELLS
    D.3.10 Strength
    D.3.10.1Concentration unit IU/kg international unit(s)/kilogram
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number100.000
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product Yes
    D.3.11.3.2Gene therapy medical product Yes
    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 typeSe van a inyectar una suspension de Células CD34+ procedentes de pacientes con anemia de Fanconi del Subtipo A (AF-A) transducidas ex vivo con vector lentiviral portador del gen FANCA.
    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 (Subtype A)
    Anemia de Fanconi (Subtipo A)
    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 15.1
    E.1.2Level LLT
    E.1.2Classification code 10055206
    E.1.2Term Fanconi's anemia
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The main objective of this Phase I/II clinical trial is to evaluate the safety and the therapeutic efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene in patients with Fanconi Anemia Subtype A.
    El objetivo principal de este ensayo clínico abierto de Fase I/II es el de evaluar la seguridad y la eficacia terapéutica de un procedimiento de terapia génica hematopoyética con un medicamento huérfano consistente en un vector lentiviral portador del gen FANCA para pacientes con Anemia de Fanconi del Subtipo A.
    E.2.2Secondary objectives of the trial
    Not applicable
    No aplica
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Patients diagnosed with the Fanconi anemia complementation group-A
    - At least one of the following parameters must be less than the indicated values?: hemoglobin 8.0 g / dL, neutrophil count: 750/mm3, platelets: 30.000/mm3
    - Age> 1 year
    - Lansky Index> 60%
    - Mild functional impairment of organs
    - To provide informed consent in accordance with current legislation
    - Number of transduced CD34+ cells: At least 3x100000 purified CD34 + / kg body weight
    - Pacientes del grupo de complementación AF-A
    - Al menos uno de los siguientes parámetros debe ser inferior a los valores indicados: Hemoglobina:8,0 g/dL; Número de neutrófilos: 750/mm3; Número de plaquetas: 30.000/mm3
    - Edad> 1 año
    - Índice de Lansky > 60%.
    - Alteración funcional de órganos leve.
    - Otorgar consentimiento informado de acuerdo con la normativa legal vigente.
    - Número de células a transducir: Al menos 3x100000 CD34+ purificadas/Kg de peso.
    E.4Principal exclusion criteria
    - Patients with HLA-identical family donor.
    - Evidence of leukemia or myelodysplastic syndrome, or cytogenetic abnormalities in bone marrow aspirates predictive of those. In this case studies performed two months before the entry of the patient in the clinical trial will be considered as valid
    - Evidence that the patient has signs of somatic mosaicism associated to hematologic improvement.
    - Any concomitant disease or condition in the investigator's opinion incapacitate the subject for their participation in the study.
    - Effect on pre-existing sensory or motor> = grade 2 according to the criteria of the National Cancer Institute (NCI).
    - Pacientes con donante familiar HLA idéntico.
    - Evidencias de síndrome mielodisplásico o leucemia, o anomalías citogenéticas
    predictivas de las mismas en aspirados de médula ósea. En este caso se darán por válidos los estudios realizados con dos meses de antelación a la entrada del paciente en el ensayo clínico.
    - Evidencias de que el paciente a infundir tenga signos de mosaicismo somático, con mejoría hematológica.
    - Toda enfermedad o proceso concomitante que en opinión del investigador incapacite al sujeto para su participación en el estudio.
    - Afectación sensorial o motora preexistente >= grado 2 según los criterios del National Cancer Institute (NCl)
    E.5 End points
    E.5.1Primary end point(s)
    - Determine the toxicity associated with infusion of CD34 + cells transduced with lentiviral vector therapy in FA-A patients.
    - Determining the degree of graft associated to infusion o fautologous CD34 + cells transduced with lentiviral vector therapy in FA-A patients.
    - Determinar la toxicidad asociada a la infusión de células CD34+ autólogas transducidas con el vector lentiviral terapéutico en pacientes con anemia de Fanconi del subtipo A.
    - Determinar el grado de injerto asociado a la infusión de células CD34+ autólogas transducidas con el vector lentiviral terapéutico en pacientes con anemia de Fanconi del subtipo A.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to 2 years after infusion
    Hasta 2 años despues de la infusion
    E.5.2Secondary end point(s)
    - To determine the clinical response associated to infusion pof autologous CD34 + cells transduced with therapeutic lentiviral vector in FA-A patients
    - Determinar la respuesta clínica asociada a la infusión de células CD34+ autólogas transducidas con el vector lentiviral terapéutico en pacientes con anemia de Fanconi del subtipo A
    E.5.2.1Timepoint(s) of evaluation of this end point
    Up to 2 years after infusion
    Hasta 2años despues de la infusion
    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 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 Yes
    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
    Last visit last patient included
    Ultima visita del ultimo paciente incluido en el estudio
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years10
    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 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: 5
    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: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 2
    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: 5
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 5
    F.4.2.2In the whole clinical trial 5
    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)
    Follow-up
    Seguimiento del estado del paciente
    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 investigacion en anemia de Fanconi
    G.4 Investigator Network to be involved in the Trial: 2
    G.4.1Name of Organisation Sociedad Española de hematología pediatrica
    G.4 Investigator Network to be involved in the Trial: 3
    G.4.1Name of Organisation centro de investigacion en red de enfermedades raras
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2013-04-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-01-29
    P. End of Trial
    P.End of Trial StatusOngoing
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