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    Summary
    EudraCT Number:2010-022884-36
    Sponsor's Protocol Code Number:EPOANE3021
    National Competent Authority:Bulgarian Drug Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-04-26
    Trial results View 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 ConcernedBulgarian Drug 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
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Efficacy Study for Epoetin alfa in Anemic Patients with Myelodysplastic Syndromes
    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.1.1Name of SponsorJanssen-Cilag International NV
    B.1.3.4CountryBelgium
    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 Medical Affairs, Turnhoutseweg 30, B-2340 Beerse
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationJanssen-Cilag International NV
    B.5.2Functional name of contact pointClinical Registry Group
    B.5.3 Address:
    B.5.3.1Street AddressJanssen Biologics BV- Clinical Registry Group Archimdesweg 29
    B.5.3.2Town/ city2333CM Leiden
    B.5.3.4CountryNetherlands
    B.5.4Telephone number(+)31 (0)71 524 21 66
    B.5.5Fax number(+) 31 (0)71 524 21 10
    B.5.6E-mailClinicalTrialsEU@its.jnj.com
    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 ERYPO
    D.2.1.1.2Name of the Marketing Authorisation holderJANSSEN-CILAG GmbH
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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.3.9.4EV Substance CodeSUB06575MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40,000
    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 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
    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
    Anemic Patients With IPSS Low- or Intermediate-1-Risk Myelodysplastic Syndromes
    E.1.1.1Medical condition in easily understood language
    Anemic Patients With Myelodysplastic Syndromes
    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 16.0
    E.1.2Level LLT
    E.1.2Classification code 10002272
    E.1.2Term Anemia
    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
    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.
    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.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female subject at least 18 years of age;

    2. Criterion modified per amendment;
    2.1. Criterion modified per amendment;
    2.2. Diagnosis of MDS according to WHO or FAB pathologic classification (confirmed via bone marrow aspirate/biopsy) within 12 weeks prior to screening/during screening phase;
    Note: If there is any sign of disease progression, a bone marrow
    aspirate/biopsy should be repeated during the screening phase.

    3. Criterion modified per amendment;
    3.1. Criterion modified per amendment.
    3.2. Documentation of an IPSS score indicating Low- or Intermediate-1-risk disease within 12 weeks prior to screening/during screening phase;
    Note: If there is no sign of disease progression, an IPSS score based on bone marrow sample obtained up to 12 weeks before screening can be used for documentation.

    4. Criterion modified per amendment;
    4.1. Criterion modified per amendment;
    4.2. Hemoglobin concentration at screening and baseline (before the first dose of study drug) of 10.0 g/dL or less;
    Note: Assessment should be based on untransfused hemoglobin, ie, the subject did not receive a RBC transfusion within the previous 2 weeks (if the subject was transfused within 2 weeks before screening or between screening and baseline a maximum hemoglobin concentration of 10.5 g/dL at screening
    or at baseline is acceptable).

    5. Criterion modified per amendment
    5.1 Screening serum erythropoietin concentration of less than 500 mU/mL; Note: Any subject with screening serum erythropoietin concentration >500 mU/mL can be retested once if the investigator has reason to believe that this is an unusual erythropoietin concentration for the subject.


    6. Criterion modified per amendment;
    6.1. RBC transfusion requirement of less than or equal to 4 RBC units over the last 8 weeks before randomization;

    7. Screening Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2;

    8. 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;

    9. Criterion modified per amendment;
    9.1 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/or baseline.
    Note: If the levels are below the lower limit at screening, and the subject has a confirmed diagnosis of MDS based on the bone marrow sample, subjects may be resupplied with vitamin B12 and/or folate during the screening period (resupply should be started without delay) and have levels retested so that the
    results would be available at baseline (before randomization); such subjects with adequate levels from results available at baseline will be eligible for enrollment.

    10. Female subjects with reproductive potential must be surgically sterile, abstinent, or practicing an effective method of birth control (eg, prescription oral contraceptives, contraceptive injections, intrauterine device, double-barrier method [spermicidal jelly or foam with condoms or diaphragm], contraceptive patch, male partner sterilization) before entry and throughout the study and have a negative urine or serum beta-human chorionic gonadotropin (hCG) pregnancy test at screening (all female subjects with reproductive potential) and at Week 24 (female subjects with reproductive potential continuing in the treatment extension phase);

    11. Willingness to adhere to the prohibitions and restrictions specified in this protocol;

    12. Subjects (or their legally acceptable representatives) must sign an informed consent document indicating that they understand the purpose of and procedures required for
    the study and are willing to participate in the study.
    E.4Principal exclusion criteria
    1. Anemia attributed to factors other than MDS (including hemolysis, chronic renal failure, hepatitis, gastrointestinal bleeding);

    2. Secondary MDS (ie, MDS arising after chemotherapy, immunotherapy or radiation therapy/exposure);

    3. Criterion modified per amendment;
    3.1. History of malignancy except in situ skin basal cell carcinoma or carcinoma in situ of the cervix or breast curatively treated;

    4. Criterion modified per amendment;
    4.1 Prior therapy with any ESA (including innovative ESAs and biosimilar ESAs for approved indications or for investigational use) in the last 8 weeks before randomization;

    5. Criterion modified per amendment;
    5.1 Prior use of approved or experimental agents for the treatment of MDS;
    Note: This restriction pertains to agents that are intended to directly influence the proliferation of the malignant clone in the bone marrow and altering the course of the disease (eg, hypomethylating agents, lenalidomide, chemotherapy etc). Different treatments that aim to treat the cytopenias
    caused by MDS are not exclusionary in the cases where exclusion criteria 4 and 10 are not met.

    6. History (within 6 months) of DVT (including proximal and distal), pulmonary embolism, or other venous thrombosis;Note: prior superficial thrombophlebitis is not an exclusion criterion.

    7. Clinically significant, uncontrolled disease or dysfunction of the pulmonary, cardiovascular, endocrine, neurologic, gastrointestinal, or genitourinary systems not attributable to MDS;

    8. Criterion modified per amendment;
    8.1 Uncontrolled hypertension (defined as a systolic pressure of 160 mmHg or higher, or a diastolic pressure of 110 mmHg or higher, or both); Note: Pertains to a history of uncontrolled hypertension, as reported by the subject or their physician (eg, general practitioner, cardiologist). A single high blood pressure measurement at screening or baseline, which can be
    indicative of white coat syndrome, will not be exclusionary.


    9. Criterion modified per amendment;
    9.1. Use of androgens or corticosteroids for the treatment of MDS within the 2 weeks before screening; androgen or corticosteroid use is allowed for other underlying concomitant diseases (eg, rheumatoid arthritis)

    10. Criterion modified per amendment;
    10.1. Prior (within 2 weeks before screening) treatment with G-CSF or granulocyte-macrophage colony-stimulating factor (GM-CSF) for the treatment of neutropenia;
    Note: in case of neutropenic fever, use of these agents is not an exclusion criterion.

    11. Known history of human immunodeficiency virus (HIV) seropositivity;

    12. Criterion modified per amendment;
    12.1 History (within 6 months before screening) of cerebrovascular accident (including ischemic, embolic, and hemorrhagic cerebrovascular accident), transient ischemic attack, acute coronary syndrome (including myocardial ischemia, unstable angina, Q-wave myocardial infarction, non-Q-wave
    myocardial infarction), or other arterial thrombosis;
    Note: Subjects with stable angina should be asymptomatic under normal daily effort with or without treatment, there should be no ischemic changes on resting ECG and the onset of the angina should not be within 6months before screening. Subjects who had ischemic heart disease (IHD) but underwent percutaneous transluminal coronary angioplasty (PTCA) are allowed in the
    study. It is strongly recommended, when indicated, that subjects who have IHD or have had PTCA be receiving antiaggregant treatment as prophylaxis.

    13. Known history of inherited conditions that predispose the subject to thrombosis (eg, AT3 deficiency, Factor V Leiden);

    14. New-onset seizures (within 3 months before randomization) or poorly controlled seizures;

    15. Pregnancy or breastfeeding;

    16. Known hypersensitivity to mammalian cell-derived products or to human albumin;

    17. Use of experimental agents or devices for any reason within 4 weeks before randomization;

    18. Major infection requiring hospitalization and antibiotic use within 2 weeks before randomization;

    19. Major surgery within 4 weeks before randomization or planned major surgery during the study period;

    20. Planned stem cell harvest of bone marrow or high dose chemotherapy with stem cell transplantation during the study period;

    21. Previous bone marrow or stem cell transplantation;

    22. History of PRCA and/or antibody against erythropoietin;

    23. Employee of the investigator or study center, with direct involvement in the proposed study or other studies under the direction of that investigator or study center and family members of the employees or the investigator

    24. Have been receiving iron chelation therapy for 6 months or more at screening for iron overload caused by blood transfusion.
    E.5 End points
    E.5.1Primary end point(s)
    Erythroid response
    E.5.1.1Timepoint(s) of evaluation of this end point
    At week 24
    E.5.2Secondary end point(s)
    The secondary efficacy endpoints are:

    1/ Maintenance of Erythroid response

    2/ Duration of response

    3/ Time to first Red Blood Cell transfusion

    4/ Transfusion-free intervals

    5/ Number of Red Blood Cell units transfused

    6/ Quality of life as measured by Functional Assessment of Cancer Therapy-Anemia/Fatigue (FACT-An) questionnaire

    7/ Quality of life as measured by EuroQol 5-dimension (EQ-5D) questionnaire

    8/ Drug Consumption

    9/ Duration of Hospitalization

    10/ Number and duration of medical care encounters
    E.5.2.1Timepoint(s) of evaluation of this end point
    1/ every 4 weeks from week 24 to week 48

    2/ every 4 weeks after week 24

    3/ from baseline to study end (week 28 for non
    responders, week 54 for responders or 4 weeks after early withdrawal)

    4/ from baseline to study end (week 28 for non
    responders, week 54 for responders or 4 weeks after early withdrawal)

    5/ from baseline to study end (week 28 for non
    responders, week 54 for responders or 4 weeks after early withdrawal)

    6/ at baseline, week 24 and week 48

    7/ at baseline, week 24 and week 48

    8/ every 4 weeks from baseline to week 48

    9/ every 4 weeks from baseline to week 48

    10/ every 4 weeks from baseline to week 48
    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.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 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.8.5The 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
    France
    Germany
    Greece
    Italy
    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
    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 months24
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months24
    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: 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 state20
    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)
    No different than normal care
    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 Decision2013-06-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-05-03
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-01-06
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