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    Summary
    EudraCT Number:2017-000812-41
    Sponsor's Protocol Code Number:GFM-EPO-PRETAR
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2017-10-23
    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 number2017-000812-41
    A.3Full title of the trial
    A RANDOMIZED TRIAL TESTING EARLY VERSUS LATE ONSET OF EPO ALFA TREATMENT IN LOWER RISK MDS WITH NON RBC TRANSFUSION DEPENDENT ANEMIA AND WITHOUT DEL 5Q
    Etude randomisée évaluant l’introduction précoce versus tardive de l’Epoétine Alfa (EPREX®) chez les patients atteints de syndromes myélodysplasiques de bas risque
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A RANDOMIZED TRIAL TESTING EARLY VERSUS LATE ONSET OF EPO ALFA TREATMENT IN LOWER RISK MDS WITH NON RBC TRANSFUSION DEPENDENT ANEMIA AND WITHOUT DEL 5Q
    Etude randomisée évaluant l’introduction précoce versus tardive de l’Epoétine Alfa (EPREX®) chez les patients atteints de syndromes myélodysplasiques de bas risque
    A.4.1Sponsor's protocol code numberGFM-EPO-PRETAR
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03223961
    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 SponsorGFM-Groupe Francophone des Myélodysplasies
    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 supportJanssen
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGFM-Groupe Francophone des Myélodysplasies
    B.5.2Functional name of contact pointFatiha Chermat
    B.5.3 Address:
    B.5.3.1Street AddressService Hématologie Séniors - Hôpital Saint Louis - 1, av. Claude Vellefaux
    B.5.3.2Town/ cityParis
    B.5.3.3Post code75010
    B.5.3.4CountryFrance
    B.5.4Telephone number33171207059
    B.5.5Fax number33171207038
    B.5.6E-mailfatiha.chermat-ext@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 EPREX
    D.2.1.1.2Name of the Marketing Authorisation holderJanssen-Cilag
    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 nameEprex
    D.3.4Pharmaceutical form 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 INNEPOETIN ALFA
    D.3.9.1CAS number 113427-24-0
    D.3.9.2Current sponsor codeeprex
    D.3.9.3Other descriptive nameEprex
    D.3.9.4EV Substance CodeSUB06575MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40000
    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.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
    Adult subject (18 years of age older) with low or intermediate-1 IPSS risk MDS with transfusions independent anemia
    patients adultes (18 ans ou plus) avec SMD de risque IPPS faible ou intérmédiaire-1 présentant une anémie non-dépendante des transfusions
    E.1.1.1Medical condition in easily understood language
    Adult subject (18 years of age older) with low or intermediate-1 IPSS risk MDS with transfusions independent anemia
    patients adultes (18 ans ou plus) avec SMD de risque IPPS faible ou intérmédiaire-1 présentant une anémie non-dépendante des transfusions
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level HLT
    E.1.2Classification code 10028536
    E.1.2Term Myelodysplastic syndromes
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Randomly compare, in non RBC transfusion dependent lower risk MDS with anemia, the time to RBC transfusion dependence in patients with early onset of EPO ALFA (at inclusion) and patients with delayed onset of EPO ALFA ( at the threshold chosen for RBC transfusions)
    Déterminer si l’introduction de l’Erythropoiétine alfa, précocement ou tardivement dans le cours de SMD de faible risque non-dépendant des transfusions en culots globulaires et présentant une anémie, va influencer la réponse et retarder plus ou moins l’échéance des transfusions en globules rouges.
    E.2.2Secondary objectives of the trial
    Compare in those 2 randomized groups:
    1. erythroid response (according to IWG 2006 criteria) after 12 weeks of EPO ALFA treatment
    2. response duration to EPO ALFA
    3. progression to higher risk MDS and /or AML
    4. the incidence of cardiovascular events
    5. QoL (assessed based on usual scales)
    6.Overall survival.
    7.We will also analyse biological correlates and compare in those 2 groups the ex vivo effect of EPO ALFA on erythropoiesis, in particular on the disease clonal architecture by repeated next generation sequencing (NGS) analysis of somatic mutations. One hypothesis is that by beginning EPO ALFA early in the disease course, no or less mutated erythropoiesis may be amplified by EPO ALFA at the expense of more mutated clones, thereby potentially delaying the disease course
    Comparer dans les deux groupes (EPO précoce ou tardif) :
    1. La réponse érythroide ;
    2. La durée de la réponse au traitement l’EPO alfa ;
    3. La progression vers un SMD de risque plus élevé et/ou LAM ;
    4. La survenue des évènements cardio-vasculaires ;
    5. La qualité de vie ;
    6. La corrélation biologique et effet ex vivo du traitement par EPO alfa sur l’érythoropoièse et plus particulièrement sur l’architecture clonale et l’analyse des mutations somatiques
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age ≥ 18 years at the time of signing the informed consent form
    2. Must understand and voluntarily sign the informed consent form
    3. Diagnosis of MDS according to WHO 2016 criteria, and low or int 1 classical IPSS, including CMML with WBC <13G/l
    4. No 5q deletion
    5. Non-RBC transfusion dependent anemia with Hb level between 9.0 and 10.5 g/dl at the center’s lab, and at least 1g/dl higher than the Hb threshold chosen to start RBC transfusions for the patient based on age, comorbidities and anticipated clinical tolerance of anemia (this transfusion threshold is generally set at 8g/dl but can be increased up to 9g/dl in case of comorbidity, etc…(as an example, a patient with a transfusion threshold estimated to be 8.5g/dl can be entered only if the baseline Hb level is at least 9.5 g/dl)
    6. serum EPO level <500U/l, no other cause of anemia (iron deficiency, vitamin B12 or B9 deficiency, hemolysis, hypothyroidism…)
    7. Performance status <=2
    1. Age ≥ 18 ans au moment de la signature du formulaire de consentement éclairé
    2. Doit comprendre et signer volontairement le formulaire de consentement éclairé
    3. SMD, de score IPSS faible rou intermédiaire 1 selon les critères d’OMS 2016 ; y compris les LMMC avec GB<13G/L
    4. Sans délétion 5q
    5. Anémie non-dépendante des transfusions ;
    - Avec un Taux d’hémoglobine entre 9 et 10.5 g/dl au laboratoire du centre) ;
    - Et un taux d’hémoglobine inférieur d’au moins 1 g/dl par rapport au seuil transfusionnel défini pour débuter les transfusions en culots globulaires en prenant en compte l’âge, les comorbidités et la tolérance clinique de l’anémie pressentie (le seuil transfusionnel doit se situer entre 8 et 9 g/dl) ;
    6. EPO sérique <500U/L en absence d’autres cause de l’anémie (carence en fer, carence en vitamine B12 ou B9, hémolyse, hypothyroïdie, …) ;
    7. ECOG ≤2
    E.4Principal exclusion criteria
    1. Higher risk MDS (IPSS intermediate-2 or high)
    2. Del 5q
    3. Baseline Hemoglobin level > 10.5 g/dl or <9g/dl
    4. Transfusion threshold (based on age , comorbidities…) >9g/dl
    5. Transfusion threshold less than 1 g/dl below baseline Hb level
    6. RBC transfusion dependence. Patients may have received only one transfusion series for MDS prior to inclusion
    7. CMML , if >10 % BM blasts or WBC>13.000/mm3
    8. Uncontrolled hypertension
    9. Uncontrolled cardiovascular disease including angina pectoris or cardiac failure
    10. Renal failure: Creatinine clearance<40ml/min (using MDRD formula)
    11. Pregnancy (positive bettaHCG) or nursing
    1. SMD de haut risque (IPSS intermédiaire 2 ou élevé)
    2. Délétion 5q
    3. Hémoglobine >10.5 g/dl ou <9g/dl
    4. Seuil transfusionnel (en fonction de l’âge, des comorbidités, …) >9g/dl
    5. Moins de 1g/dl de différence entre le seuil transfusionnel et le taux d’hémoglobine à l’inclusion
    6. Dépendance en transfusion des culots globulaires. Les patients peuvent recevoir une seule transfusion pour le SMD avant l’inclusion ;
    7. LMMC, si >10% de blastes médullaires ou leucocytes >13000/mm3
    8. Hypertension artérielle non-contrôlée
    9. Maladie cardio-vasculaire non-contrôlée, y compris l’angine de poitrine ou insuffisance cardiaque ;
    10. Insuffisance rénale : clairance de créatinine <40 ml/min (selon MDRD)
    11. Grossesse (βHCG) ou allaitement
    E.5 End points
    E.5.1Primary end point(s)
    Comparison of the time to RBC transfusion dependence between patients with early onset of EPO ALFA (at inclusion ) and patients with delayed onset of EPO ALFA (at the threshold chosen for RBC transfusion)
    Comparaison du moment de l’apparition de la dépendance transfusionnelle entre les patients ayant commencé le traitement par EPO-Alfa précocement et les patients ayant débuté l’EPO alfa tardivement (au seuil défini pour le début des transfusions en culots globulaires)
    E.5.1.1Timepoint(s) of evaluation of this end point
    through the study
    durant l'étude
    E.5.2Secondary end point(s)
    1. IWG 2006 erythroid response and duration
    2. QoL,
    3. OS
    4. molecular biology corrélations
    1. Durée de la réponse érythroide selon IWG 2006
    2. Qualité de vie,
    3. Survie globale
    4. Correlations de biologie moléculaire
    E.5.2.1Timepoint(s) of evaluation of this end point
    through the study
    durant l'étude
    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 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) No
    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 Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial2
    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 concerned28
    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
    dernière visite du dernier patient
    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
    E.8.9.2In all countries concerned by the trial years5
    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 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 124
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 124
    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 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 state124
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 124
    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)
    best available treatment
    meilleur traitement disponible
    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 Decision2017-10-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-12-29
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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