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    Summary
    EudraCT Number:2018-001157-27
    Sponsor's Protocol Code Number:2017_52-REGALIA
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-10-09
    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 number2018-001157-27
    A.3Full title of the trial
    Reversing poor graft function with eltrombopag after allogeneIc hematopoietic cell transplantation : a prospective, phase II study by the SFGM-TC.
    Étude multicentrique, prospective, de phase II évaluant l'efficacité d'eltombopag dans la prise en charge du dysfonctionnement du greffon après une transplantation allogénique de cellules souches hématopoïétiques.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Eltrombopag in treatment of poor graft function.
    Eltrombopag pour le traitement du dysfonctionnement du greffon.
    A.3.2Name or abbreviated title of the trial where available
    REGALIA
    REGALIA
    A.4.1Sponsor's protocol code number2017_52-REGALIA
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCHRU of Lille
    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 supportCHRU de Lille
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationClinical Research Federation
    B.5.2Functional name of contact pointKarim DAHACHE (CRA)
    B.5.3 Address:
    B.5.3.1Street Address2, Avenue Oscar Lambret
    B.5.3.2Town/ cityLille
    B.5.3.3Post code59037
    B.5.3.4CountryFrance
    B.5.4Telephone number+33320444145
    B.5.5Fax number+33320445711
    B.5.6E-mailfrc@chru-lille.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 Revolade
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    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.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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
    Poor Graft Function (PGF) after allogeneic hematopoietic cell transplantation (allo-HCT). PGF is commonly defined as follows: one or several significant cytopenias after allo-HCT persisting or developing after allo-HCT despite full donor chimerism and in the absence of relapse or other causes (in particular graft-versus-host disease [GVHD]).
    Mauvais fonctionnement du greffon après une greffe allogénique de cellules souches hematopoietiques. Le mauvais fonctionnement du greffon se défini par une ou plusieurs cytopénies significatives après allo-HCT persistantes ou en développement après allo-HCT malgré le chimérisme complet du donneur et en l'absence de rechute ou d'autres causes (en particulier le greffon versus maladie de l'hôte [GVHD]).défini
    E.1.1.1Medical condition in easily understood language
    Poor Graft Function (PGF).
    Mauvais fonctionnement du greffon.
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    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
    To demonstrate that eltrombopag improves PGF.
    Démontrer que l’eltrombopag améliore le mauvais fonctionnement du greffon.
    E.2.2Secondary objectives of the trial
    - To decrease the need for transfusions,
    - To decrease the rate of bleeding events,
    - To decrease the rate of infectious events,
    - To improve quality of life parameters,
    - To demonstrate that eltrombopag improves bone marrow cellularity,
    - To evaluate overall survival and non-relapse mortality,
    - To evaluate adverse events.
    - Diminuer les besoins transfusionnels,
    - Diminuer le taux d’événements hémorragiques,
    - Diminuer les risques infectieux,
    - Evaluer la survie globale et la mortalité sans rechute,
    - Améliorer les paramètres de qualité de vie,
    - Recueil des événements indésirables graves.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Patient age ≥ 6 years old
    - Diagnosis of poor graft function defined as:
    • Patient ≥ day+60 after allo-HCT,
    • Persisting thrombocytopenia on two different samples over at least two weeks (platelet < 20G/L with transfusion requirement) +/- neutropenia (ANC <1G/L) +/- anemia (Hb <8g/dL or transfusion requirement),
    • Full donor chimerism on whole blood (≥ 95%),
    • Biopsy proven hypocellular marrow without evidence of myelodysplasia
    • No evidence for relapse,
    • No evidence for active acute or chronic graft versus host disease,
    • Absence of active viral infection (EBV, CMV, ADENOVIRUS, PARVOVIRUS B19),
    • Absence of B9/B12 deficiency,
    • Absence of hypothyroidism,
    • Absence of hypogonadism,
    • Absence of dialysis,
    • Absence of thrombotic microangiopathy,
    • Absence of macrophage activation syndrome,
    • No other known causes of poor graft function.
    Diagnostic du mauvais fonctionnement du greffon défini comme :
    - Patient au, ou au-delà, jour + 60 après l’allogreffe,
    - Thrombopénie persistante sur deux échantillons différents pendant au moins deux semaines (plaquettes <20 G / L ou besoins transfusionnels) +/- neutropénie (diminution de PNN <1 G / L ou dépendance au G-CSF) +/- anémie (Hb <8g / dL ou besoins transfusionnels en CGR),
    - Chimérisme complet de type donneur sur sang total (≥ 95%),
    - Une moelle hypocellulaire prouvée par biopsie sans évidence de myélodysplasie,
    - Aucune preuve de rechute,
    - Aucune preuve de maladie du greffon contre l'hôte active,
    - Absence d'infection virale active (EBV, CMV, ADENOVIRUS, PARVOVIRUS B19),
    - Absence de déficit en B9 / B12,
    - Absence d'hypothyroïdie,
    - Absence d'hypogonadisme,
    - Absence de dialyse,
    - Absence de microangiopathie thrombotique,
    - Absence de syndrome d'activation des macrophages,
    - Aucune autre cause connue de mauvais fonctionnement du greffon,
    - Qualité d’assuré social ou ayant droit d’assuré social.
    E.4Principal exclusion criteria
    • Criteria for poor graft function not fulfilled (see above),
    • Patients aged less than 6 years old (or unable to swallow),
    • Hepatic impairment (Child-Pugh ≥ 5),
    • Hypersensitivity to eltrombopag or to any of the excipients,
    • Patients with any contra-indication to eltrombopag, filgrastim,
    • Unable to understand the investigational nature of the study or give informed consent,
    • History of congestive heart failure, arrhythmia requiring chronic treatment, arterial or venous,
    • Thrombosis (not excluding line thrombosis) within the last 1 year, or myocardial infarction within 3 months before enrollment,
    • ECOG Performance Status of 3 or greater,
    • Pregnant and/or lactating women,
    • Freedom privacy.
    Les critères de mauvais fonctionnement du greffon ne sont pas remplis (voir ci-dessus) :

    - Patients âgés de moins de 6 ans (ou incapables d'avaler),
    - Insuffisance hépatique (Child-Pugh ≥ 5),
    - Hypersensibilité à l'eltrombopag ou à l'un des excipients,
    - Les patients présentant une contre-indication à l'eltrombopag ou au filgrastim,
    - Impossibilité de comprendre la nature expérimentale de l'étude ou de donner un consentement éclairé,
    - Antécédents d'insuffisance cardiaque congestive, arythmie nécessitant un traitement chronique, artériel ou veineux,
    - Thrombose (excluant la thrombose sur cathéter veino-central) au cours de la dernière année, ou infarctus myocardique dans les 3 mois précédant l'évaluation,
    - Statut de performance ECOG de 3 ou plus,
    - Femmes enceintes et/ou allaitantes,
    - Personnes privées de liberté.
    E.5 End points
    E.5.1Primary end point(s)
    Platelet response defined as a platelet ≥20G/L at 12 weeks measured on at least two serial measurements performed 1 week apart and sustained for 1 month or more without support of platelet transfusions.
    Réponse plaquettaire définie comme une numération plaquettaire ≥ 20G / L à 12 semaines mesurée sur au moins deux mesures effectuées à 1 semaine d'intervalle et maintenue pendant 1 mois ou plus sans transfusion des plaquettes.
    E.5.1.1Timepoint(s) of evaluation of this end point
    As specified in the primary end point : platelet ≥20G/L at 12 weeks of treatment.
    Comme spécifié dans le critère principal: plaquettes ≥ 20G / L à 12 semaines de traitement.
    E.5.2Secondary end point(s)
    - Time toerythroid response defined as an increase of at least 1.5g/dL without transfusion that is sustained for at least 2 weeks,
    - Time to neutrophil response defined as an increase of ANC above 1G/L, that is sustained for at least 7 days,
    - Marrow best response at 12 and 24 weeks,
    - Transfusion requirements at 12 and 24 weeks for BRC and platelets as compared with transfusions requirements during the eight weeks preceding study entry,
    - Severe adverse events,
    - Quality of life parameters at 12 and 24 weeks,
    - Immune function (T/B/NK cells counts) at 12 and 24 weeks,
    - 6-month overall survival,
    - 6-month relapse-free survival,
    - 6-month non relapse mortality,
    - 6-month cumulative incidence of underlying disease relapse.
    - Temps de réponse érythroïde défini comme une augmentation d'au moins 1,5 g / dL sans transfusion des globules rouges (CGR), maintenue pendant au moins 2 semaines,
    - Temps de réponse des polynucléaires neutrophiles défini comme une augmentation des PNN supérieure à 1G / L, qui est maintenue pendant au moins 7 jours,
    - Meilleure réponse médullaire à 12 et 24 semaines,
    - Besoins transfusionnels à 12 et 24 semaines pour les CGR et les plaquettes par rapport aux besoins transfusionnels pendant les huit semaines précédant l'entrée dans l'étude,
    - Recueil de l’ensemble des évènements indésirables graves (EIGs) depuis le début du traitement jusqu’à l’arrêt de traitement ou jusqu’à l’arrêt prématuré (selon NCI-CTC V4.0).
    - Qualité de vie évaluée par le questionnaire (SF-36 qualité de vie à l’inclusion, 12 et 24 semaines de traitement),
    - Fonction immunitaire (nombre de cellules T / B / NK) à 12 et 24 semaines,
    - Survie globale, survie sans rechute et mortalité sans récidive à 6 mois,
    - Incidence cumulative de la rechute de maladie sous-jacente sur 6 mois.
    E.5.2.1Timepoint(s) of evaluation of this end point
    As spicified for each end point.
    Comme spécifié pour chaque critère d'évaluation secondaire.
    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) No
    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 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 concerned11
    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 of the last patient included in the study
    Dernière visite du dernier patient inclus dans l'étude
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months36
    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: 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) No
    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: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 15
    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 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 state25
    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
    Aucun
    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 Decision2018-11-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-11-06
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2023-10-23
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