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The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
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    The EU Clinical Trials Register currently displays   41039   clinical trials with a EudraCT protocol, of which   6717   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).


    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .
     
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    Summary
    EudraCT Number:2010-018965-42
    Sponsor's Protocol Code Number:CC-4047-MF-002
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2011-02-14
    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 ConcernedPoland - Office for Medicinal Products
    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
    Wieloośrodkowe, randomizowane, badanie kliniczne III fazy, podwójnie zaślepione, kontrolowane placebo, prowadzone w równoległych grupach, mające na celu ocenę skuteczności i bezpieczeństwa pomalidomidu u pacjentów z mielofibrozą w przebiegu nowotworów mieloproliferacyjnych i wymagających przetaczania czerwonych krwinek.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to compare the safety and efficacy of pomalidomide versus a nonactive pill in patients who need frequent red blood cell transfusions due to bone marrow scaring (fibrosis) associated with specific bone marrow disorders (MPNs – polycythemia vera, essential thrombocythemia, and primary myelofibrosis)
    A.3.2Name or abbreviated title of the trial where available
    RESUME
    A.4.1Sponsor's protocol code numberCC-4047-MF-002
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01178281
    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 Address9900 W. 109th Street, Suite 300, Building 70,
    B.5.3.2Town/ cityKansas
    B.5.3.3Post code66210
    B.5.3.4CountryUnited States
    B.5.4Telephone number0018882601599
    B.5.5Fax number001913451 3459
    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/3/10/757
    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.3.9.2Current sponsor codeCC-4047
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.5
    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
    Myeloproliferative Neoplasm (MPN) Associated Myelofibrosis defined as primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (post-PV MF) and post-essential thrombocythemia myelofibrosis (post-ET MF)
    E.1.1.1Medical condition in easily understood language
    Cancer of the bone marrow where normal blood cell growth is affected
    and collagen fibers begin to develop in the 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 14.1
    E.1.2Level PT
    E.1.2Classification code 10028537
    E.1.2Term Myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    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
    The presence of any of the following will exclude a subject from enrollment:
    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 by bone marrow examination or more than 10% blasts in blood in consecutive measurements spanning at least 8 weeks.

    7. Prior history of malignancies, other than the disease being studied, unless the subject has been free of the malignancy for ≥ 5 years with the following exceptions:
    -Basal cell carcinoma of the skin,
    -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 TNM (tumor, nodes, metastatic) clinical staging system)
    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/1.732m2
    - Aspartate aminotransferase (AST),and alanine transaminase (ALT) >3.0 x upper limit of normal (ULN)
    - Total bilirubin ≥4 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 Yes
    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
    QoL (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.8.2.4Number 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 concerned3
    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
    Australia
    Canada
    China
    Japan
    Russian Federation
    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
    At the end of the follow up phase, minimum 5 years after the last subject is randomized in the study, or until the last subject dies.
    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.1.2.1Number of subjects for this age range: 96
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 114
    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 state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 127
    F.4.2.2In the whole clinical trial 210
    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)
    See protocol
    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 Decision2011-06-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-04-13
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-05-15
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