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    The EU Clinical Trials Register currently displays   43857   clinical trials with a EudraCT protocol, of which   7284   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2014-005264-14
    Sponsor's Protocol Code Number:P130103
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2015-06-19
    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 ConcernedFrance - ANSM
    A.2EudraCT number2014-005264-14
    A.3Full title of the trial
    NA
    FancoMob: Etude pilote évaluant la faisabilité de la mobilisation sanguine et de la collection des cellules CD34+ après traitement par G-CSF et plérixafor chez des patients atteints d'Anémie de Fanconi en vue d'un traitement ulterieur par thérapie génique_FancoMob
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    FancoMob: Pilote study for a combined treatment helping to collect stem cells in patient suffering Fanconi anemia
    FancoMob: Pilote study for a combined treatment helping to collect stem cells in patient suffering Fanconi anemia
    A.3.2Name or abbreviated title of the trial where available
    EUROFANCOLEN
    A.4.1Sponsor's protocol code numberP130103
    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 SponsorASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    B.1.3.4CountryFrance
    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 supportEuropean Commission
    B.4.2CountryEuropean Union
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    B.5.2Functional name of contact pointProject manager
    B.5.3 Address:
    B.5.3.1Street Address1 rue Claude Vellefaux
    B.5.3.2Town/ cityParis
    B.5.3.3Post code75010
    B.5.3.4CountryFrance
    B.5.4Telephone number33144.84.17.44
    B.5.5Fax number33144.84.17.01
    B.5.6E-maildidier.bouton@aphp.fr
    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 Zarzio
    D.2.1.1.2Name of the Marketing Authorisation holderSandoz GmbH
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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 nameZarzio
    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 INNfilgrastim
    D.3.9.3Other descriptive nameG-CSF
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number30MU/0,5ml to 48MU/0,5ml
    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) 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 Yes
    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 Mozobil
    D.2.1.1.2Name of the Marketing Authorisation holderGenzime Europe BV
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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 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 INNplérixafor
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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
    Anémie de Fanconi
    E.1.1.1Medical condition in easily understood language
    Fanconi Anemia
    Anémie 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 18.1
    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 Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    NA
    L'objectif principal de cette étude consiste en l'évaluation de la faisabilité de l'administration conjointe de G-CSF et de plérixafor à 5 patients atteints d'anémie de Fanconi en vue de la mobilisation et du recueil de cellules souches hématopoïétiques sanguines pour une potentielle utilisation dans un protocole de thérapie génique.
    E.2.2Secondary objectives of the trial
    Evaluation détaillée de la cinétique de mobilisation des cellules souches.
    Evaluer les caractéristiques biologiques des cellules recueillies.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    NA
    - Diagnostic d'une anémie de Fanconi de type Fanc A.
    - Indication potentielle d'une greffe de moelle osseuse allogénique et absence de fratrie HLA-identique disponible.
    - Enfant âgé de plus de 4 ans et de moins de 18 ans - Enfant de plus de 20 kg
    - pour les patientes en âge de procréer, ne pas être enceinte et utiliser des contraceptifs efficaces durant toute la durée de sa participation à la recherche
    - patient suivi durant au moins les deux années précédentes dans un centre spécialisé où ils ont bénéficié d'une évaluation complète de leur maladie.
    - patient affilié ou bénéficiaire d'un régime de sécurité sociale
    - Consentement écrit et éclairé signé le(s) titulaire(s) de l'autorité parentale
    E.4Principal exclusion criteria
    NA
    - patient ne pouvant, à sa connaissance et celle de l'investigateur, effectuer les visites de l'essai exigées par le protocole
    - Sérologie positive pour HIV-1/2 HTLV-1/2 VHC et Antigène HbS
    - Infection active bactérienne, virale, fungique ou parasitaire avec signes cliniques.
    - Antécédent personnel de cancer, maladie myélodysplasique ou leucémique
    - Insuffisance cardiaque et/ou troubles du rythme cardiaque
    - Antécédent d'allogreffe de cellules souches hématopoïétiques
    - Patient ayant un donneur familial HLA identique
    - Existence d'une myélodysplasie diagnostiquée sur le myélogramme
    - Existence d'anomalie cytogénétique sur le caryotype
    - Existence d'une tumeur solide maligne
    - Processus médullaire de réversion génétique spontanée documenté.
    - Diagnostic d'une pathologie psychiatrique compromettant la capacité à participer à l'étude
    - toute forme de trouble qui, selon l'investigateur, pourrait compromettre la capacité du patient à donner son consentement éclairé écrit et/ou à se conformer à toutes les procédures requises de l'étude.
    - Grossesse ou allaitement en cours
    - Insuffisance cardiaque, rénale ou hépatique sévère
    - Participation active à un autre essai
    - Patient sous Aide Médicale d'Etat
    - Hypersensibilité connue au plérixafor ou tout excipient contenu dans Mozobil®
    - Hypersensibilité connue au filgrastim ou à l'un des excipients (en particulier intolérance au fructose)
    E.5 End points
    E.5.1Primary end point(s)
    NA
    critère d'évaluation principal
    la stratégie associant G-CSF et plérixafor sera jugée satisfaisante si :
    - elle permet d'obtenir soit une mobilisation sanguine minimale d'au moins 10 cellules CD34+/µl soit la mobilisation de 5 à 10 cellules CD34+/µl mais présentant un aspect " groupé " et non dispersé en cytométrie de flux.
    . cette mobilisation sanguine peut être soutenue plusieurs jours de suite si cela s'avère nécessaire
    . Permet de recueillir l'ensemble des cellules souhaités tant pour la constitution du greffon de secours que celui destiné à la thérapie génique, soit un montant minimum de 5 x 106 CD34+/kg qui correspond d'une part à un premier greffon de minimum 3 x 106 CD34+/kg (en poids " projeté " à 5 ans) pour la procédure de thérapie génique et d'autre part, un " parachute minimum "de 2 x 106 CD34+/kg (en poids " projeté " à 5 ans si il s'agit d'un enfant) pour constituer un greffon non manipulé en cas d'échec de la thérapie génique.
    E.5.1.1Timepoint(s) of evaluation of this end point
    after visit of each patient
    après la dernière visite de chaque patient
    E.5.2Secondary end point(s)
    NA
    - survenue d'effets indésirables liés à l'administration conjointe de G-CSF et de plérixafor durant la période d'administration des drogues et la réalisation des cytaphérèses mais également au-delà (jusqu'à 30 jours après).
    - Nombre d'injection de G-CSF et de plérixafor nécessaire à la réalisation des objectifs ainsi que le nombre de cytaphérèse requis.
    - Profil cinétique de mobilisation sanguine des cellules CD34+ tout au long de la procédure de mobilisation afin de déterminer le tempo adapté pour le monitoring sanguin des cellules souches et la décision temporelle d'initiation des cytaphérèses.
    - Profil phénotypique des cellules souches recueillies.
    E.5.2.1Timepoint(s) of evaluation of this end point
    up to 30 days after last injection
    jusqu'à 30 jours après la dernière injection
    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) No
    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
    II
    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 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 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 Information not present in EudraCT
    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
    last site last visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned 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: 8
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 8
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2015-06-19. Yes
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    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
    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 Decision2016-01-05
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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