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    The EU Clinical Trials Register currently displays   43976   clinical trials with a EudraCT protocol, of which   7312   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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    EudraCT Number:2010-022884-36
    Sponsor's Protocol Code Number:EPOANE3021
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-01-23
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2010-022884-36
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study Evaluating Epoetin Alfa Versus Placebo in Anemic Patients With IPSS Low- or Intermediate 1 Risk Myelodysplastic Syndromes
    Uno studio randomizzato, in doppio cieco, multicentrico, controllato da placebo, per valutare l'effetto di epoetina alfa rispetto al placebo in pazienti anemici con sindromi mielodisplastiche con rischio IPSS basso o intermedio-1
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study comparing Epoetin Alfa with Placebo in Anaemic patients with MDS
    Studio clinico per confrontare l'effetto di Epoetina Alfa verso l'effetto del placebo in pazienti anemici con sindromi mielodisplastiche
    A.4.1Sponsor's protocol code numberEPOANE3021
    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.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportJANSSEN CILAG SPA
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationJANSSEN CILAG SPA
    B.5.2Functional name of contact pointCGO
    B.5.3 Address:
    B.5.3.1Street AddressVIA M. BUONARROTI 23
    B.5.3.2Town/ cityCOLOGNO MONZESE
    B.5.3.3Post code20093
    B.5.4Telephone number+39 022510 569
    B.5.5Fax number+39 02 2510 537
    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. name EPREX*1SIR 40000UI/ML 1ML
    D. of the Marketing Authorisation holderJANSSEN CILAG SpA
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 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 INNEPOETIN ALFA
    D.3.9.1CAS number 113427-24-0
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Anemia due to IPSS Low- or Intermediate-1-Risk Myelodysplastic Syndromes
    Anemia causata da sindromi mielodisplastiche con rischio IPSS basso o intermedio-1
    E.1.1.1Medical condition in easily understood language
    Anemia due to Myelodysplastic Syndromes
    Anemia dovuta a malattie mielodisplastiche
    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 14.1
    E.1.2Level LLT
    E.1.2Classification code 10002272
    E.1.2Term Anemia
    E.1.2System Organ Class 10005329 - Blood and lymphatic system 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 primary objective is:  To demonstrate that epoetin alfa treatment is better at improving anemia outcome (as evaluated by Erythroid response – International Working Group [IWG] 2006) in subjects with International Prognostic Scoring Systems (IPSS) Low- or Intermediate-1-risk myelodysplastic syndromes (MDS), compared with placebo, through Week 24.
    L'obiettivo principale è il seguente: • Dimostrare che, nell’arco di 24 settimane, il trattamento con epoetina alfa, rispetto al placebo, è migliore nel correggere l'anemia (valutazione che verrà effettuata in base alla risposta eritroide - International Working Group [IWG] 2006) in soggetti con sindromi mielodisplastiche (SMD) con rischio IPSS (International Prognostic Scoring Systems, sistema internazionale di punteggio prognostico) Basso o Intermedio-1.
    E.2.2Secondary objectives of the trial
    The secondary objectives include: • For responders at Week 24, observe the duration of the response through Week 48. • To assess the proportion of responders at Week 24 maintaining response through Week 48 (as measured by Erythroid response – IWG 2006). • To compare time to first RBC transfusion, transfusion-free intervals, and number of RBC units transfused. • To measure and compare changes in patient-reported outcome (PRO)/quality of life from baseline via the Functional Assessment of Cancer Therapy  Anemia/Fatigue (FACT-An) and EuroQol 5 dimension (EQ-5D) questionnaires. • To collect medical resource utilization data that may be used in future economic modeling (the construction and reporting of the economic model will be conducted separately from this study). Overall safety will also be assessed.
    • Per i responder alla settimana 24, osservazione della durata della risposta fino alla settimana 48. • Valutazione della proporzione di responder alla settimana 24 che mantengono la risposta fino alla settimana 48 (la valutazione verrà effettuata in base alla risposta eritroide - IWG 2006). • Confronto dei tempi alla prima trasfusione di globuli rossi (RBC), intervallo libero da trasfusioni, e numero di unità di RBC trasfuse. • Misura e confronto delle variazioni, dalla visita basale, dei risultati riportati dal paziente (PRO, patient-reported outcome) e della qualità della vita mediante i questionari Functional Assessment of Cancer Therapy  Anemia/Fatigue (FACT-An) e EuroQol 5 dimension (EQ-5D). • Raccolta di dati sull'utilizzo delle risorse mediche, da impiegare per la creazione di futuri modelli economici. Verrà valutata anche la sicurezza generale.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Male or female subjects at least18 years of age; • diagnosis of MDS at screening, according to World Health Organization (WHO) or French-American-British (FAB) Cooperative Group pathologic classification and confirmed via bone marrow aspirate; • documentation of an IPSS score indicating Low- or Intermediate 1 risk disease; • hemoglobin concentration at screening and baseline (before the first dose of study drug) of 10.0 g/dL or less, based on untransfused hemoglobin (ie, the subject did not receive an RBC transfusion within the previous week); • screening serum erythropoietin concentration of less than 500 mU/mL; • RBC transfusion requirement of less than 4 RBC units over the last 8 weeks before randomization; • screening Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2; • adequate iron stores, defined as serum ferritin greater than 100 ng/mL, transferrin saturation greater than 20%, or both, measured within the 2-week screening period, or adequate iron stores as demonstrated by recent (within 12 weeks) bone marrow examination with iron stain; • documentation of adequate vitamin B12 and folate levels (above the lower limit of the reference range for the laboratory performing the assay), measured at screening and baseline.
    • soggetti maschi o femmine di almeno 18 anni d'età; • diagnosi di SMD allo screening, secondo la classificazione patologica dell'Organizzazione Mondiale della Sanità (WHO) o del gruppo cooperativo Franco-Americano-Britannico (FAB) e confermato tramite agoaspirato del midollo osseo; • documentazione di un punteggio IPSS che indica una malattia a rischio basso o intermedio-1; • concentrazione di emoglobina alla visita di screening e basale (precedentemente alla prima dose del farmaco dello studio) di 10,0 g/dL o inferiore, sulla base dell'emoglobina non trasfusa (ovvero il soggetto non ha ricevuto una trasfusione di RBC nella precedente settimana); • concentrazione di eritropoietina nel siero alla visita di screening inferiore a 500 mU/mL; • necessità trasfusionale di RBC inferiore a 4 unità di RBC nelle 8 settimane precedenti la randomizzazione; • performance status ECOG (Eastern Cooperative Oncology Group) allo screening pari a 0, 1 o 2; • riserve di ferro adeguate, definite come ferritina nel siero superiore a 100 mg/mL o saturazione della transferrina superiore al 20% o entrambe, con valutazione effettuata nel periodo di 2 settimane dello screening oppure riserve di ferro adeguate come dimostrato da un recente esame del midollo osseo (entro 12 settimane) con colorazione per il ferro; • documentazione di livelli adeguati di vitamina B12 e folato (al di sopra del limite inferiore del range di riferimento per il laboratorio, che esegue la valutazione), in base alla valutazione effettuata allo screening e alla visita basale.
    E.4Principal exclusion criteria
    • Anemia attributed to factors other than MDS; • secondary MDS (ie, MDS arising after chemotherapy, immunotherapy, or radiation therapy/exposure; • active malignancy within the past year, except basal cell or carcinoma in situ of the cervix or breast; • prior therapy with epoetin alfa or any other approved or experimental ESA for more than 8 weeks before randomization; • prior use of approved or experimental agents for the treatment of MDS; • history (within 6 months) of deep vein thrombosis (including proximal and distal), pulmonary embolism, or other venous thrombosis.
    • anemia attribuita a fattori diversi da SMD; • SMD secondaria (ovvero SMD che si manifesta in seguito a chemioterapia, immunoterapia o radioterapia/esposizione a radiazioni); • patologia maligna attiva nell’anno precedente, esclusi il carcinoma basocellulare o il carcinoma in situ della cervice o del seno; • precedente terapia con epoetina alfa o qualsiasi altro ESA, approvato o sperimentale, per più di 8 settimane prima della randomizzazione; • precedente utilizzo di agenti approvati o sperimentali per il trattamento della SMD; • storia (nei 6 mesi precedenti) di trombosi venosa profonda (sia prossimale che distale), embolia polmonare o altra trombosi venosa.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is the proportion of subjects at Week 24 with improved anemia outcome, defined as Erythroid response according to the IWG 2006 criteria.
    L'endpoint primario di efficacia è la proporzione di soggetti che alla settimana 24 registrano un miglioramento dell'anemia, definito come risposta eritroide secondo i criteri IWG 2006.
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 weeks
    24 settimane
    E.5.2Secondary end point(s)
    • For responders at Week 24, the duration of the response through Week 48 • The proportion of responders at Week 24 maintaining response through Week 48 (as measured by Erythroid response – IWG 2006). • Time to first RBC transfusion, transfusion-free intervals, and number of RBC units transfused. • Change from baseline in PRO/quality of life (as assessed with the FACT-An and EQ-5D).
    • Per i responder alla settimana 24, la durata della risposta fino alla settimana 48. • La proporzione di responder alla settimana 24 che mantengono la risposta fino alla settimana 48 (valutato in base alla risposta eritroide - IWG 2006). • Il tempo alla prima trasfusione di globuli rossi (RBC), gli intervalli liberi da trasfusioni ed il numero di unità di RBC trasfuse. • La variazione dalla visita basale nei questionari PRO/qualità della vita (valutata con i questionari FACT-An e EQ-5D).
    E.5.2.1Timepoint(s) of evaluation of this end point
    48 weeks
    48 settimane
    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 No
    E. 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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 Yes
    E.8.2.3Other No
    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 concerned14
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA32
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Russian Federation
    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 visit of last subject
    ultima visita dell'ultimo paziente arruolato
    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 months23
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months24
    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.1.1Number of subjects for this age range: 0
    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: 48
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 111
    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 state60
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 147
    F.4.2.2In the whole clinical trial 159
    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)
    standard care
    pratica clinica standard
    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 Decision2011-08-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-06-13
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-01-06
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