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    Summary
    EudraCT Number:2010-018965-42
    Sponsor's Protocol Code Number:CC-4047-MF-002
    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:2010-11-05
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2010-018965-42
    A.3Full title of the trial
    A PHASE-3, MULTI-CENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED,
    PARALLEL-GROUP STUDY TO COMPARE EFFICACY AND SAFETY OF POMALIDOMIDE IN
    SUBJECTS WITH MYELOPROLIFERATIVE NEOPLASM -ASSOCIATED MYELOFIBROSIS AND
    RED BLOOD CELL-TRANSFUSION-DEPENDENCE
    A.3.2Name or abbreviated title of the trial where available
    ND
    A.4.1Sponsor's protocol code numberCC-4047-MF-002
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberND
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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/3/10/757
    D.3 Description of the IMP
    D.3.1Product namePomalidomide
    D.3.2Product code CC-4047
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPomalidomide
    D.3.9.1CAS number 19171-19-8
    D.3.9.2Current sponsor codeCC-4047
    D.3.10 Strength
    D.3.10.1Concentration unit mg/g milligram(s)/gram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number.5
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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 placeboCapsule, hard
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    subjects with myeloproliferative neoplasm (MPN)-associated myelofibrosis. and red blood cell (RBC)-transfusion-dependence
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10028537
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Compare efficacy of pomalidomide versus placebo in achieving Red Blood Cell (RBC)-transfusion-independence
    in subjects with Myeloproliferative neoplasm-associated myelofibrosis with RBC-transfusion-dependence.
    E.2.2Secondary objectives of the trial
    Evaluate safety of pomalidomide in subjects with MPN-associated myelofibrosis with
    RBC-transfusion-dependence.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age ≥18 years of age at the time of signing the informed consent document.
    2. MPN-associated myelofibrosis (primary myelofibrosis (PMF), post-polycythemia vera
    myelofibrosis (post-PV MF) and/or post-essential thrombocythemia myelofibrosis (post-ET MF)
    3. RBC-transfusion-dependence:
    - Average RBC-transfusion frequency of ≥2 U/28 days over at least the 84 days
    immediately prior to randomization. There must be no interval >42 days without
    ≥1 RBC-transfusion.
    - Only RBC-transfusions given when the hemoglobin ≤90 g/L^3 are scored in
    determining eligibility.
    - RBC-transfusions due to bleeding are not scored in determining eligibility
    - RBC-transfusions due to chemotherapy-induced anemia are not scored in
    determining eligibility.
    4. Hemoglobin ≤130 g/L at randomization.
    5. Bone marrow slides that meet defined criteria for central histological review will be
    submitted to a central reviewer.
    6. A blood cell or bone marrow allo-transplant should not be an appropriate therapy at this time.
    7. Erythropoietin should not be an appropriate therapy at this time.
    8. Androgenic steroids should not be an appropriate therapy at this time.
    9. Treatment with systemic corticosteroids is permitted for non-hematological conditions providing the subject
    is receiving a stable or decreasing dose for ≥84 days immediately prior to randomization and are receiving a
    constant dose equivalent to ≤10 mg prednisone for the 28 days immediately prior to randomization.
    10. Eastern Cooperative Oncology Group (ECOG) performance score ≤2.
    11. Females of childbearing potential (FCBP) must undergo pregnancy testing based on the frequency outlined in
    protocol Appendix 18.1 and pregnancy results must be negative.
    12. Unless practicing complete abstinence from heterosexual intercourse, sexually active FCBP must agree to use
    adequate contraceptive methods as specified in protocol Appendix 18.1.
    13. Males (including those who have had a vasectomy) must use barrier contraception (condoms) when engaging
    in sexual activity with FCBP as specified in Appendix 18.1.
    14. Males must agree not to donate semen or sperm during the duration specified in Appendix 18.1.
    15. All subjects must:
    - Understand that the investigational product could have a potential teratogenic risk.
    - Agree to abstain from donating blood while taking investigational product and
    following discontinuation of investigational product
    - Agree not to share study medication with another person.
    - Be counseled about pregnancy precautions and risks of fetal exposure.
    16. Understand and voluntarily sign an informed consent document before any study related assessments/
    procedures are conducted.
    17. Able to adhere to the study visit schedule and other protocol requirements.
    E.4Principal exclusion criteria
    1. Prior bone marrow or blood cell transplant.
    2. Use of drugs to treat MPN-associated myelofibrosis ≤30 days pre-randomization(42 days for hydroxyurea) or
    ongoing adverse events from previous treatment.
    3. Use of an erythropoietin or androgenic steroids ≤84 days pre- randomization.
    4. Anemia from other proved causes other than MPN-associated myelofibrosis.
    5. Pregnant or lactating females.
    6. More than 10% blasts at any time during the 8 weeks before randomization unless there is at least one
    subsequent determination of ≤10% blasts before randomization.
    7. Prior history of other cancer unless cancer-free for ≥5 years. Subjects with the following history/concurrent
    conditions may enroll at any time:
    -Basal cell carcinoma of the skin,
    -Squamous cell carcinoma of the skin
    -Prostate cancer stage-1
    8. Proved human immunodeficiency virus-1 (HIV-1) infection
    9. Active hepatitis B virus (HBV) or active hepatitis C Virus (HCV) infection.
    10. Prior therapy with pomalidomide.
    11. Any of the following adverse reactions to prior therapy with thalidomide or lenalidomide:
    - Prior ≥grade-2 National Cancer Institute (NCI) Common Terminology Criteria
    for Adverse Events (CTCAE) allergic reaction to thalidomide and/or lenalidomide
    - Prior desquamating (blistering) rash while taking thalidomide and/or lenalidomide
    - Hypersensitivity to thalidomide and lenalidomide
    12. Any of the following laboratory abnormalities:
    -Neutrophils <0.5x10e9/L
    - Platelets <25 x 10e9/L
    - Estimated glomerular filtration rate <30 mL/min
    - Aspartate aminotransferase (AST),and alanine transaminase (ALT) >3.0 x upper limit of normal (ULN)
    - Direct bilirubin ≥3 x ULN;
    - Uncontrolled hyperthyroidism or hypothyroidism.
    13. Deep venous thrombosis (DVT) or pulmonary embolus (PE) <6 months pre-randomization.
    14. Heart disease ≤6 months pre-randomization including:
    - New York Heart Association class >II, congestive heart failure
    - Unstable angina
    - Myocardial infarction within 6 months
    15. Any significant medical condition, laboratory abnormality or psychiatric illness that
    would prevent the subject from participating in the study.
    16. Any condition including the presence of laboratory abnormalities, which places the subject at unacceptable
    risk if he/she were to participate in the study.
    17. Any condition that confounds the ability to interpret data from the study.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of subjects achieving ≥ 84 consecutive days of RBC-transfusion-independence
    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 Yes
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic Yes
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Qualita` della vita (EQ-5D, FACT-An)
    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.8.3 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 EEA46
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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
    Alla fine del periodo di follow-up, minimo 5 anni dopo che l`ultimo paziente e` stato randomizzato, o fino alla morte dell`ultimo paziente.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months6
    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.1.3Elderly (>=65 years) Yes
    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 state50
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 127
    F.4.2.2In the whole clinical trial 210
    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 Decision2010-11-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2010-10-18
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-05-15
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