Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43846   clinical trials with a EudraCT protocol, of which   7282   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).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2019-003555-11
    Sponsor's Protocol Code Number:PD20180302
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2020-03-25
    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 number2019-003555-11
    A.3Full title of the trial
    A Phase 1/2 Open Label non randomized Study, multicentric, single arm
    evaluating the Safety and Efficacy of Gene Therapy of the severe combined
    immunodeficiency (SCID) caused by mutations in the human DCLRE1C
    gene (Artemis) by transplantation of a single dose of autologous CD34+
    cells transduced ex vivo with the G2ARTE lentiviral vector expressing the
    DCLRE1C cDNA
    A Phase 1/2 Open Label non randomized Study, multicentric, single arm
    evaluating the Safety and Efficacy of Gene Therapy of the severe combined
    immunodeficiency (SCID) caused by mutations in the human DCLRE1C
    gene (Artemis) by transplantation of a single dose of autologous CD34+
    cells transduced ex vivo with the G2ARTE lentiviral vector expressing the
    DCLRE1C cDNA

    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    safety and efficacity of gene thérapie of the severe combined
    immunodeficiency (SCID)
    Sécurité et efficacité d'une therapie génique d'une immunodéfiscience sévére combinée (SCID)
    A.3.2Name or abbreviated title of the trial where available
    ARTEGENE
    ARTEGENE
    A.4.1Sponsor's protocol code numberPD20180302
    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-HOPÏTAUX DE PARIS
    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 support
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameCD34+modifiées et transduites avec le vecteur lentiviral G2ARTE exprimant le DCLRE1CcDNA
    D.3.4Pharmaceutical form Concentrate and solvent for concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    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 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
    immunodeficiency (SCID) caused by mutations in the human DCLRE1C
    gene (Artemis)
    Immuno-défiscience (SCID) causé par la mutation DCLRE1C du géne
    ARTEMIS
    E.1.1.1Medical condition in easily understood language
    immunodeficiency (SCID) caused by mutations in the human DCLRE1C
    gene (Artemis)
    Immuno-défiscience (SCID) causé par la mutation DCLRE1C du géne
    ARTEMIS
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study consists in assessing the initial safety
    and efficacy of treatment with ARTEGENE drug product, including the
    mobilization procedure, conditioning regimen and transplantation with
    ARTEGENE lentiviral vector gene modified autologous hematopoietic
    stem cells in in up to 5 Artemis deficient patients.
    L'objectif principal de l'étude consiste à évaluer l'innocuité et l'efficacité
    initiales du traitement avec le produit médicamenteux ARTEGENE, y
    compris la procédure de mobilisation, le régime de conditionnement et la
    transplantation avec les cellules souches hématopoïétiques autologues
    modifiées par un gène vecteur lentiviral ARTEGENE chez jusqu'à 5
    patients déficients en artémis.
    E.2.2Secondary objectives of the trial
    • The clearing of on going infections present before the transplantation
    • .
    • The evaluation of the functional performance of this novel lentiviral
    vector
    • The evaluation of the molecular characteristics of vector integration.
    Absence d infections en cours présentes avant la transplantation
    •.
    • L'évaluation des performances fonctionnelles de ce nouveau vecteur
    lentiviral
    • L'évaluation des caractéristiques moléculaires de l'intégration
    vectorielle.
    E.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patient with a RS-SCID caused by mutation of the Artemis gene and a
    clinical diagnosis of SCID documented in the medical record
    - Absence of an HLA genoidentical donor
    - Age ≤ 1 year
    - The patient can be treated by gene therapy without delay in case of
    life threatening infections compromising the short-term prognosis.
    Active life threatening infections are defined as: viral respiratory
    infection, CMV infection, adenovirus infection, disseminated BCGitis or
    other infections grade ≥ 4 according to CTCAE scale .
    - Parental, guardian's patient signed informed consent
    Patient avec un RS-SCID provoqué par une mutation du gène Artemis et
    un diagnostic clinique de SCID documenté dans le dossier médical
    - Absence d'un donneur géno-identique HLA
    - Âge ≤ 1 an
    - Le patient peut être traité par thérapie génique sans délai en cas
    d'infections mettant la vie en danger compromettant le pronostic à court
    terme. Les infections potentiellement mortelles sont définies comme:
    infection respiratoire virale, infection à CMV, infection à adénovirus,
    BCGitis disséminé ou autres infections de grade ≥ 4 selon l'échelle
    CTCAE.
    - Consentement éclairé Parental, patient, du tuteur signé
    E.4Principal exclusion criteria
    Individuals who meet any of the following exclusion criteria will not be
    eligible to participate in the study:
    - Unwillingness to return for follow-up during the 2 year study and
    lifelong for off study review
    -
    - HIV-1 or 2 or HTLV1 infection
    French
    Les personnes qui répondent à l'un des critères d'exclusion suivants ne
    seront pas admissibles à participer à l'étude:
    - Impossibilité pour un suivi au cours de l'étude de 2 ans et à vie pour un
    examen hors étude
    -
    - Infection par le VIH-1 ou 2 ou HTLV1
    infections de grade ≥ 4 selon l'échelle CTCAE.
    - Absence consentement éclairé signé
    E.5 End points
    E.5.1Primary end point(s)
    (repeat as necessary)26
    English Safety of ARTEGENE drug product, is assessed by:
    • Incidence of transplant related mortality up to 100 days post
    treatment
    • Frequency and severity of clinical AEs and laboratory parameters
    throughout the whole period of the research
    • Incidence of RCL measured at 3 months
    The efficacy of successful transduction of CD34+ by the vector and the
    injection of autologous gene-modified hematopoietic stem cells is
    evaluated by the assessment of T and B cells reconstitution in Artemis
    deficient patients after transplantation.
    L'innocuité du médicament ARTEGENE est évaluée par:
    • Incidence de la mortalité liée à la greffe jusqu'à 100 jours après le
    traitement
    • Fréquence et gravité des EI cliniques et des paramètres de laboratoire
    pendant toute la durée de la recherche
    • Incidence de RCL mesurée à 3 mois
    L'efficacité d'une transduction réussie de CD34 + par le vecteur et
    l'injection de cellules souches hématopoïétiques génétiquement
    modifiées autologues est évaluée par l'évaluation de la reconstitution
    des cellules T et B chez des patients déficients en Artemis après
    transplantation.
    E.5.1.1
    E.5.1.1Timepoint(s) of evaluation of this end point
    3 months
    3 mois
    E.5.2Secondary end point(s)
    The assessment of the long-term safety and efficacy of treatment with
    the ARTEGENE drug product
    L'évaluation de l'innocuité et de l'efficacité à long terme du traitement
    avec le médicament ARTEGENE
    E.5.2.1Timepoint(s) of evaluation of this end point
    100 days
    100 jours
    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) Yes
    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.8.3 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 Yes
    E.8.5.1Number of sites anticipated in the EEA2
    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
    LVLS
    lvls
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months60
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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: 3
    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) No
    F.1.1.6.1Number of subjects for this age range: 0
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    CHILDRENS
    MINEURS
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    childrens
    mineurs
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    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)
    After the 2 years of participation in the protocole,the patients will be followed-up for 13 years
    Après 2 ans de suivi dans le protocole,les patients seront suivis pendant 13 ans
    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 Decision2021-04-19
    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
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Fri Apr 19 11:06:21 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA