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    Summary
    EudraCT Number:2015-003454-41
    Sponsor's Protocol Code Number:ACE-536-MDS-001
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-11-17
    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 ConcernedGermany - PEI
    A.2EudraCT number2015-003454-41
    A.3Full title of the trial
    A Phase 3, Double-Blind, Randomized Study To Compare The Efficacy And Safety Of Luspatercept (ACE-536) Versus Placebo For The Treatment Of Anemia Due To IPSS-R Very Low, Low, Or Intermediate Risk Myelodysplastic Syndromes In Subjects With Ring Syderoblasts Who Require Red Blood Cell Transfusions
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study To Compare The Benefit And Safety Of Luspatercept (ACE-536) Versus Placebo in subjects with lack of normal Red Blood Cells Requiring Transfusion due to myelodysplastic syndromes (MDS) with ring sideroblasts
    A.3.2Name or abbreviated title of the trial where available
    MEDALIST
    A.4.1Sponsor's protocol code numberACE-536-MDS-001
    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 SponsorCelgene Corporation
    B.1.3.4CountryUnited States
    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 supportCelgene Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCelgene Corporation
    B.5.2Functional name of contact pointClinicalTrialDisclosure
    B.5.3 Address:
    B.5.3.1Street Address86 Morris Avenue
    B.5.3.2Town/ citySummit, New Jersey
    B.5.3.3Post code07901
    B.5.3.4CountryUnited States
    B.5.4Telephone number+19137096862
    B.5.6E-mailClinicalTrialDisclosure@celgene.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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/14/1331
    D.3 Description of the IMP
    D.3.1Product nameLuspatercept
    D.3.2Product code ACE-536
    D.3.4Pharmaceutical form Lyophilisate for 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 INNLuspatercept
    D.3.9.1CAS number 1373715-00-4
    D.3.9.2Current sponsor codeACE-536
    D.3.9.3Other descriptive nameLuspatercept
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number37.5
    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.IMP: 2
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/14/1331
    D.3 Description of the IMP
    D.3.1Product nameLuspatercept
    D.3.2Product code ACE-536
    D.3.4Pharmaceutical form Lyophilisate for 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 INNLuspatercept
    D.3.9.1CAS number 1373715-00-4
    D.3.9.2Current sponsor codeACE-536
    D.3.9.3Other descriptive nameLuspatercept
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number87.5
    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
    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
    Subjects with ring sideroblasts who require regular Red Blood Cell (RBC) Transfusions due to anemia due to Myelodysplastic Syndromes (MDS)
    E.1.1.1Medical condition in easily understood language
    Subjects who require regular Red Blood Cell (RBC) Transfusions due to red blood cell count being lower than normal lead by a blood disorder due to damage in the blood-forming cells in the bone marrow
    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 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 Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate RBC transfusion independence (RBC-TI) of luspatercept compared with placebo for the treatment of anemia due to IPSS-R very low, low, or intermediate risk MDS in subjects with ring sideroblasts (≥ 15%) who require RBC transfusions
    E.2.2Secondary objectives of the trial
    1. To assess the safety and tolerability of luspatercept compared with placebo
    2. To evaluate the effect of luspatercept on reduction in RBC transfusions, increase in hemoglobin, duration of RBC-TI, improvement in health-related quality of life (HRQoL) (ie, European Organization for Research and Treatment of Cancer Quality of Life Questionnaire [EORTC QLQ-C30]), increase in neutrophils, increase in platelets, decrease in serum ferritin, decrease in iron chelation therapy use, and time to RBC-TI
    compared with placebo
    3. To evaluate population pharmacokinetics and exposure-response relationships for luspatercept in MDS subjects
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject is ≥ 18 years of age the time of signing the informed consent form (ICF).
    2. Subject must understand and voluntarily sign an ICF prior to any study-related assessments/procedures being conducted.
    3. Documented diagnosis of MDS according to WHO 2008 classification (Appendix B) that meets IPSS-R classification (Greenberg, 2012; Appendix D) of very low, low, or intermediate risk disease, and: Ring sideroblast ≥ 15% of erythroid precursors in bone marrow or ≥ 5% (but < 15%) if SF3B1 mutation is present,
    - < 5% blasts in bone marrow
    - Peripheral blood WBC count < 13,000/microL
    4. Refractory or intolerant to, or ineligible for, prior ESA treatment, as defined by any one of the following:
    - Refractory to prior ESA treatment - documentation of non-response or response that is no longer maintained to prior ESA-containing regimen, either as single agent or combination (eg, with G-CSF); ESA regimen must have been either:
    a) recombinant human erythropoietin (rHu EPO) ≥ 40,000 IU/wk for at least 8 doses or equivalent;
    OR
    b) darbepoetin alpha ≥ 500 μg Q3W for at least 4 doses or equivalent;
    - Intolerant to prior ESA treatment - documentation of discontinuation of prior ESAcontaining regimen, either as single agent or combination (eg, with G-CSF), at any time after introduction due to intolerance or an adverse event
    - ESA ineligible - Low chance of response to ESA based on endogenous serum erythropoietin level > 200 U/L for subjects not previously treated with ESAs
    5. If previously treated with ESAs or granulocyte colony-stimulating factor (G-CSF), granulocyte-macrophage colony-stimulating factor (GM-CSF), both agents must have been discontinued ≥ 4 weeks prior to date of randomization.
    6. Requires RBC transfusions, as documented by the following criteria:
    - average transfusion requirement of ≥ 2 units/8 weeks of pRBCs confirmed for a minimum of 16 weeks immediately preceding randomization.
    - Hemoglobin levels at the time of or within 7 days prior to
    administration of a RBC transfusion must have been ≤ 10.0 g/dL in order
    for the transfusion to be counted towards meeting eligibility criteria. Red
    blood cell transfusions administered when Hgb levels were > 10.0 g/dL
    and/or RBC transfusions administered for elective surgery will not
    qualify as a required transfusion for the purpose of meeting eligibility
    criteria.
    - no consecutive 56-day period that was RBC transfusion-free during the 16 weeks immediately preceding randomization
    7. Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2
    8. Females of childbearing potential (FCBP), defined as a sexually
    mature woman who: 1) has not undergone a hysterectomy or bilateral
    oophorectomy or 2) has not been naturally postmenopausal
    (amenorrhea following cancer therapy does not rule out childbearing
    potential) for at least 24 consecutive months (ie, has had menses at any
    time in the preceding 24 consecutive months), must:
    a) Have two negative pregnancy tests as verified by the Investigator prior to starting study therapy (unless the screening pregnancy test was
    done within 72 hours of C1D1). She must agree to ongoing pregnancy testing during the course of the study, and after end of study treatment. This applies even if the subject practices true abstinence from heterosexual contact.
    b) If sexually active, agree to use, and be able to comply with, highly effective contraception without interruption, 5 weeks prior to starting investigational product, during the study therapy (including dose interruptions), and for 12 weeks after discontinuation of study therapy.
    9. Male subjects must agree to use a condom, defined as a male latex condom or nonlatex condom NOT made out of natural (animal) membrane (for example, polyurethane), during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions and for at least 12 weeks following investigational product discontinuation, even if he has undergone a successful vasectomy.
    10. Subject is willing and able to adhere to the study visit schedule and other protocol requirements.
    E.4Principal exclusion criteria
    1. Prior therapy with disease modifying agents for underlying MDS disease
    a) subjects who previously received hypomethylating agents (HMA) or lenalidomide may be enrolled at the investigator's discretion contingent that the subject received no more than 2 doses of HMA or no more than 1 calendar week of treatment with lenalidomide The last dose must be ≥ 5 weeks from the date of randomization
    2. Previously treated with either luspatercept (ACE-536) or sotatercept (ACE-011)
    3. MDS associated with del 5q cytogenetic abnormality
    4. Secondary MDS, ie, MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases
    5. Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
    6. Prior allogeneic or autologous stem cell transplant
    7. Known history of diagnosis of AML
    8. Use of any of the following within 5 weeks prior to randomization:
    - Anticancer cytotoxic chemotherapeutic agent or treatment
    - Corticosteroid, except for subjects on a stable or decreasing dose for ≥ 1 week prior to randomization for medical conditions other than MDS
    - Iron-chelating agents, except for subjects on a stable or decreasing dose for at least 8 weeks prior to randomization
    - Other RBC hematopoietic growth factors (eg, Interleukin-3)
    - investigational drug or device, or approved therapy for investigational
    use. If the half-life of the previous investigational product is known, use within 5 times the halflife prior to randomization or within 5 weeks, whichever is longer is excluded
    9. Uncontrolled hypertension, defined as repeated elevations of diastolic blood pressure (DBP) ≥ 100 mmHg despite adequate treatment
    10. Absolute neutrophil count (ANC) < 500/μL (0.5 x 10^9/L)
    11. Platelet count < 50,000/μL (50 x 10^9/L)
    12. Estimated glomerular filtration rate (eGFR) or creatinine clearance < 40 mL/min
    13. Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) or alanine aminotransferase/serum glutamic pyruvic transaminase (ALT/SGPT) ≥ 3.0 x upper limit of normal (ULN)
    14. Total bilirubin ≥ 2.0 x ULN
    - Higher levels are acceptable if these can be attributed to active red blood cell precursor destruction within the bone marrow (ie, ineffective erythropoiesis) or in the presence of known history of Gilbert Syndrome
    - Subjects are excluded if there is evidence of autoimmune hemolytic anemia manifested as a corrected reticulocyte count of > 2% with either a positive Coombs’ test or over 50% indirect bilirubin
    15. Prior history of malignancies, other than MDS, unless the subject has been free of the disease (including completion of any active or adjuvant treatment for prior malignancy) for ≥ 5 years. However, subjects with the following history/concurrent conditions are allowed:
    - Basal or squamous cell carcinoma of the skin
    - Carcinoma in situ of the cervix
    -Carcinoma in situ of the breast
    - Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, nodes, metastasis [TNM] clinical staging system)
    16. Major surgery within 8 weeks prior to randomization. Subjects must have completely recovered from any previous surgery prior to randomization
    17. History of stroke, deep venous thrombosis (DVT), pulmonary or arterial embolism within 6 months prior to randomization
    18. Pregnant or breastfeeding females
    19. Myocardial infarction, uncontrolled angina, uncontrolled heart failure, or uncontrolled cardiac arrhythmia as determined by the investigator within 6 months prior to randomization. Subjects with a known ejection fraction < 35%, confirmed by a local ECHO or MUGA performed within 6 months prior to randomization are excluded
    20. Uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment), known Human Immunodeficiency Virus (HIV), known evidence of active infectious Hepatitis B, and/or known evidence of active Hepatitis C. Local testing confirming HIV, Hepatitis B, and Hepatitis C status should not have been performed earlier than 4 weeks from the date of ICF signature
    21. History of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the investigational product
    22. Subject has any significant medical condition, laboratory abnormality, or psychiatric illness, or is considered vulnerable by local regulations (eg, imprisioned or institutionalized) that would prevent the subject from participating in the study
    23. Subject has any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study
    24. Subject has any condition or concomitant medication that confounds the ability to interpret data from the study
    E.5 End points
    E.5.1Primary end point(s)
    Red Blood Cell Transfusion Independence (RBC-TI) ≥ 8 weeks - Proportion of subjects who are RBC transfusion free over any consecutive 56-day period
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 1 through Week 24
    E.5.2Secondary end point(s)
    Key secondary endpoint
    1. RBC-TI ≥ 12 weeks - Proportion of subjects who are RBC transfusion free over any consecutive 84-day period
    2. RBC-TI ≥ 8 weeks - Proportion of subjects who are RBC transfusion free over any consecutive 56-day period
    3. Reduction in RBC units transfused over 16 weeks - Mean change in total RBC units transfused over a fixed 16-week period
    4. Modified hematologic improvement - erythroid (mHI-E) per IWG - Proportion of subjects achieving modified HI-E over any consecutive 56-day period
    5. Mean hemoglobin increase ≥ 1.0 g/dL - Proportion of subjects achieving hemoglobin (Hgb) increase from baseline ≥ 1.0 g/dL over any consecutive 56-day period in absence of RBC transfusions
    6. Duration of RBC-TI - Maximum duration of RBC transfusion independence for
    subjects who achieve RBC TI ≥ 8 weeks
    7. Health-related quality of life (HRQoL) - Change in EORTC QLQC30 score
    8. Hematologic improvement - platelets (HI-P) per IWG; hematologic improvement-neutrophils (HI-N) per IWG - Proportion of subjects achieving HI-N / HI-P over any consecutive 56-day period
    9. Mean decrease in serum ferritin - Change in serum ferritin
    10. Mean decrease in iron chelation therapy (ICT) use - Change in mean daily dose
    of ICT
    11. Time to RBC-TI - Time from first dose to first onset of transfusion independence ≥ 8 weeks
    12. Progression to AML - Number and percentage of subjects progressing to
    AML; time to AML progression
    13. Overall survival - Time from date of randomization to death due to any cause
    14. Safety - Type, frequency, severity of AEs and relationship of AEs to luspatercept/placebo
    15. A population PK model - A Population PK model that describes the PK exposure data of luspatercept and associated variability.
    16. Exposure-response relationship for the primary efficacy endpoint, AEs of interest, and selected secondary endpoints.
    17. Anti-drug antibodies (ADA) - Frequency of anti-drug antibodies and effects on efficacy, or safety, or PK
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Week 1 to Week 24; Week 1 to Week 48
    2. Week 1 to Week 48
    3. Week 9 to 24; Week 33 to 48
    4 + 5. Week 1 to Week 24; Week 1 to Week 48
    6. Week 1 to Week 24; Week 1 to end of treatment
    7. Week 1 to Week 24; Baseline to end of treatment
    8. Week 1 to Week 24; Week 1 to Week 48
    9 + 10. Week 9 to 24; Week 33 to 48
    11. Week 1 to Week 24; Week 1 to Week 48
    12 + 13. Randomization to at least 3 years post last dose; Week 1 to Week 48
    14. Screening to 42 days post last dose; Week 1 to Week 48
    15 + 16. Randomization through 1- year post first dose
    17. Randomization through 1- year post first dose
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Yes
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    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 Yes
    E.6.13.1Other scope of the trial description
    Biomarkers
    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 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) 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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA58
    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
    Belgium
    Canada
    France
    Germany
    Israel
    Italy
    Netherlands
    Russian Federation
    Spain
    Sweden
    Turkey
    United Kingdom
    United States
    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
    Either the date of the last visit of the last subject to complete the post-treatment follow-up, or the date of receipt of the last data point from the last subject that is required for primary, secondary and/or exploratory analysis, as prespecified in the protocol and/or SAP, whichever is the later date.
    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: 44
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 185
    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 state14
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 193
    F.4.2.2In the whole clinical trial 229
    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)
    Subjects who discontinue from treatment for any reason will be followed via telephone contact by the site or subject site visit for collection of data on survival, cause(s) of death, progression to AML, other malignancies/pre-malignancies, posttreatment therapy(ies) for MDS at the 42-Day and 12-W Follow-up assessments and then every 3 months after the 12 Week Follow-up assessment for at least 3 years after last dose of IP or until death, lost to follow-up or withdrawal of consent from the study.
    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 Decision2016-05-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-04-07
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-11-26
    For support, visit the EMA Service Desk , log in using your EMA account and open a ticket specifying "EU CTR" in your request.
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