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    Summary
    EudraCT Number:2018-001029-14
    Sponsor's Protocol Code Number:P150949J
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2018-07-02
    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 number2018-001029-14
    A.3Full title of the trial
    A phase I/II Study evaluating the safety and the efficacy of Human T Lymphoid Progenitor (HTLP) injection to accelerate immune reconstitution after partially HLA compatible allogeneic hematopoietic stem cell transplantation in SCID patients
    Essai de Phase I/II évaluant la tolérance et l'efficacité de l'injection de précurseurs de lymphocytes T humains (HTLP) pour accélérer la reconstitution immunitaire après une greffe allogénique partiellement HLA compatible de cellules souches hématopoïétiques chez les patients SCID partiellement HLA compatibles chez les patients SCID
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    HTLP
    HTLP
    A.3.2Name or abbreviated title of the trial where available
    HTLP NECKER
    A.4.1Sponsor's protocol code numberP150949J
    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 supportDGOS
    B.4.2CountryFrance
    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 pointDRCI Hôpital St Louis
    B.5.3 Address:
    B.5.3.1Street Address 1 av. Claude Vellefaux
    B.5.3.2Town/ cityPARIS
    B.5.3.3Post code75010
    B.5.3.4CountryFrance
    B.5.6E-mailcoralie.villeret@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 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 nameHTLP
    D.3.2Product code HTLP
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.11 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 Yes
    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
    SCID pediatric patients (n=12 for analysis) requiring an HLA partially compatible allogeneic HSCT.
    patients SCID
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10010099
    E.1.2Term Combined immunodeficiency
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    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 is to assess the procedure's efficacy and safety:

    1. (≥ 300/µL total CD3+ TCRαβ+ T cells at 3 months)
    2. Dose-limiting toxicity (DLT), including graft versus host disease (GVHD) grade II-IV and grade III or higher common terminology criteria adverse events (CTCAEs).
    évaluer la sécurité et l'efficacité de la geffe de HTLP
    - Efficacité : ≥ 300/µl total de cellules CD3+ TCRαβ+ à 3 mois
    - Dose limiting toxicity (DLT), y compris la maladie du greffon contre l'hôte (GvH) de grade III-IV, et les événements indésirables de critères de terminologie communs de grade III ou supérieur (CTCAE) à 3 mois.
    E.2.2Secondary objectives of the trial
    " Time course of reconstitution of the different T cell subpopulations (numbers, functions and repertoires), thymic output and diversity of the T cell receptor repertoire by immunophenotyping, proliferation assays in the presence of mitogens and antigens, TCR repertoire analysis and TRECs values. A complete immunological assessment will be performed at 3, 6, 12 and 24 months and as soon as the number of CD3+ TCR αβ cells in the blood reaches > 300/µl with particular attention to the time necessary to reach a normal number of naïve CD4 and CD8 T cells for the patient according to age
    " Cumulative incidence of opportunistic infections at 3, 6 and 12 months
    " Cumulative incidence of acute and chronic episodes of GVHD and their grade (at 3, 6, 12 and 24 months post-transplantation) according to Glucksgber GVHD staging
    " Overall survival at 2 years and EFS
    • Evaluation de la durée de la reconstitution des différentes sous-populations de lymphocytes T (nombres, fonctions, et répertoires), production thymique et diversité des récepteurs des lymphocytes T par immunophénotypage, essais de prolifération en présence de mitogènes et d’antigènes, analyse du répertoire TCR et valeurs TRECs. Une évaluation immunologique complète sera effectuée à 3, 6, 12 et 24 mois et dès que le nombre de cellules CD3+ TCRαβ+ dans le sang atteindra > 300µl, en portant une attention particulière au temps nécessaire pour atteindre un niveau normal de cellules T CD4 et CD8 αβ naïves chez le patient en fonction de son âge.
    • Incidence cumulative d’infections opportunistes à 6, et 12 mois
    • Incidence cumulative d’épisodes aigus et chroniques de GvHet leur grade (à 3, 6, 12 and 24 mois après la transplantation) selon la classification du Glucksgberb
    • Survie globale à 2 ans et survie sans évènements
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Pediatric patients affected by any type of SCID confirmed by clinical, immunological and/or molecular diagnosis and eligible for an allogeneic HSCT
    • Absence of a matched sibling donor or a matched unrelated donor (MUD) 10/10
    • Clinical conditions incompatible with the search of a MUD
    • Written, informed consent of parents/ legal representative (child)
    • Age ≤ 2 years at the time of screening
    • No prior therapy with allogeneic stem cell transplantation
    • No treatment with another investigational drug within one month before inclusion
    • Injection occurs 7 days prior to HSCT with CD34+ selected graft if all inclusion criteria are met
    • Patient affiliated to social security
    • Age ≤2 ans au moment de la sélection
    • Patients pédiatriques atteints de tout type de SCID confirmé par un diagnostic clinique, immunologique et/ou moléculaire, et éligibles à une greffe haplo-identique intrafamiliale. Absence d’un donneur compatible apparenté ou d’un donneur compatible non-apparenté (MUD) 10 /10
    • Conditions cliniques incompatibles avec la recherche d’un MUD
    • absence d’antécédent d’allogreffe
    • absence de traitement par un autre médicament expérimental dans le mois qui précède l’inclusion
    • Consentement éclairé et écrit des parents/représentant légal (enfant)
    • Patient affilié ou bénéficiaire d’un régime de sécurité sociale
    E.4Principal exclusion criteria
    " Presence of an HLA genoidentical donor
    " Absence of written parental consent
    " Treatment with another investigational drug within one month before inclusion
    " Positive for HIV infection by genome PCR
    " Contra-indication to allogeneic transplantation or conditioning therapy (except SCID patients with DNA repair deficiency)

    " Présence d'un donneur HLA géno-identique
    " Absence d'un consentement parental _signé
    " Traitement par un autre médicament expérimental dans le mois qui précède l'inclusion
    " PCR VIH positive
    " Contre-indication à la greffe de cellules souches allogéniques ou à la thérapie de conditionnement (sauf pour les patients SCID présentant un déficit de réparation de l'ADN)
    E.5 End points
    E.5.1Primary end point(s)
    na
    na
    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 No
    E.6.5Efficacy No
    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 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.8.1.7.1Other trial design description
    The protocol is designed as a dose-escalation study comprising 6 doses of the HTLP cellular product
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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
    LVLS
    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
    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: 12
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 12
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 12
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    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 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 state12
    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 Decision2018-10-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-10-12
    P. End of Trial
    P.End of Trial StatusOngoing
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