Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43871   clinical trials with a EudraCT protocol, of which   7290   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 on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2010-019501-41
    Sponsor's Protocol Code Number:MPD-RC112
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-11-14
    Trial 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 ConcernedUK - MHRA
    A.2EudraCT number2010-019501-41
    A.3Full title of the trial
    Randomized Trial of Pegylated Interferon Alfa-2a versus Hydroxyurea Therapy in the Treatment of High Risk Polycythemia Vera and High Risk Essential Thrombocythemia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A randomized study comparing two drugs: hydroxyurea and Pegylated Interferon Alfa-2a, in an open label clinical trial in two independent diseases: (1) high risk polycythaemia vera and (2) high risk essential thrombocythaemia.
    A.3.2Name or abbreviated title of the trial where available
    MPD-RC 112 (Pegasys)
    A.4.1Sponsor's protocol code numberMPD-RC112
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01259856
    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 SponsorConsorzio Mario Negri Sud
    B.1.3.4CountryItaly
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportNational Cancer Institute
    B.4.2CountryUnited States
    B.4.1Name of organisation providing supportF.Hoffmann-La Roche
    B.4.2CountrySwitzerland
    B.4.1Name of organisation providing supportCancer Research UK
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationConsorzio Mario Negri Sud
    B.5.2Functional name of contact pointEleonora Prete
    B.5.3 Address:
    B.5.3.1Street AddressVia Nazionale 8\A
    B.5.3.2Town/ citySanta Maria Imbaro
    B.5.3.3Post code66030
    B.5.3.4CountryItaly
    B.5.4Telephone number0872570322
    B.5.5Fax number0872570206
    B.5.6E-mailprete@negrisud.it
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP Role
    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 Pegasys
    D.2.1.1.2Name of the Marketing Authorisation holderRoche Registration Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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.3.1Product namepeginterferon alfa-2a
    D.3.2Product code Pegasys
    D.3.4Pharmaceutical form 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 INNInterferon pegylated 2a
    D.3.9.3Other descriptive namePEG-IFN alfa-2a
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    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 RoleComparator
    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 Hydroxyurea
    D.2.1.1.2Name of the Marketing Authorisation holderHexal AG
    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.3.1Product nameHydroxyurea
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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) 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    The myeloproliferative disorders essential thrombocythaemia (ET) and polycythaemia vera (PV).
    E.1.1.1Medical condition in easily understood language
    PV and ET are chronic diseases. ET causes people to produce too many blood cells called platelets and PV causes too many platelets and red cells to be made.
    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 10043550
    E.1.2Term Thrombocythemia
    E.1.2System Organ Class 100000004851
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level LLT
    E.1.2Classification code 10036058
    E.1.2Term Polycythemia
    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
    We wish to compare the ability of two different treatments to control abnormalities of blood counts in patients with essentail thrombocythaemia and polycythaemia vera who are classed as at high risk of blood clots. The two treatments are hydroxyurea and Pegylated Interferon Alfa-2a (PEGASYS).
    E.2.2Secondary objectives of the trial
    Secondary objectives of the trial are: • To compare the toxicity, safety and tolerability of therapy in patients • To compare the rates of partial control of blood counts • To compare specific pre-defined toxicity and tolerance of therapy and use a tool to assess these and patient symptoms. • To compare the impact of therapy on key biomarkers of the disease activity and outcomes including JAK2-V617F a mutation or genetic change commonly found, the proportion of diseased cells, the appearance of the bone marrow, and cytogenetic or chromosomal abnormalities • To measure the occurence of serious late ccomplications of these diseases such as the development of leukaemia • To estimate incidence of major blood clotting (defined as cardiovascular death, myocardial infarction, stroke, transient ischemic attack, pulmonary embolism, Budd Chiari syndrome, deep vein thrombosis, and any other clinically relevant thrombotic event) during therapy.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - A diagnosis (<3 years prior to entry in the sudy) of Essential Thrombocythaemia (ET) or Polycythaemia Vera (PV) made in accordance with the WHO (2008) criteria. - Never treated with cytoreductive drugs except hydroxyurea for up to 3 months maximum (phlebotomy, aspirin allowed, anagrelide allowed). - Age: > 18 years (no upper limit). - Ability and willingness to comply with all study requirements. - Signed informed consent to participate in this study. - Willing to participate in associated correlative science biomarker study. - Serum creatinine < 1.5 x upper limit of normal. - AST and ALT < 2 x upper limit of normal. - Total Bilirubin within normal limits. - No known PNH (paroxysmal nocturnal hemoglobinuria) clone. - No concurrent hormonal oral contraceptive use. - Patients must have high risk disease as defined below: High risk PV ANY ONE of the following: 1)age >60 years; 2)Previous documented thrombosis, erythromelalgia or migraine (severe, recurrent, requiring medications, and felt to be secondary to the MPN) either after diagnosis or within 10 years before diagnosis and considered to be disease related; 3)significant splenomegaly (>5 cm below the left costal margin) or symptomatic splenomegaly (splenic infarcts or requiring analgesia); 4)platelets > 1000 x 109/L; 5)diabetes or hypertension requiring pharmacological therapy for > 6 months. High risk ET ANY ONE of the following: 1)age > 60 years; 2)platelet count > 1500 x 109/L; 3)previous documented thrombosis, erythromelalgia or migraine headaches (severe, recurrent, requiring medications, and felt to be secondary to the MPN) either after diagnosis or within 10 years before diagnosis and considered to be disease related; 4)previous hemorrhage related to ET; 5)Diabetes or hypertension requiring pharmacological therapy for > 6 months.
    E.4Principal exclusion criteria
    - Any contraindications to Pegylated interferon or Hydroxyurea (listed below). - Presence of any life-threatening co-morbidity. - History of active substance or alcohol abuse within the last year. - Subjects who are pregnant, lactating or of reproductive potential and not practicing an effective means of contraception. - History of psychiatric disorder (e.g. depression). - History of autoimmune disorder (e.g. hepatitis). - Hypersensitivity to IFN-α HIV, HBV, or systemic infection. - Evidence of severe retinopathy (e.g. CMV retinitis, macular degeneration) or clinically relevant ophthalmological disorder (e.g. due to diabetes mellitus or hypertension). - History or other evidence of decompensated liver disease. - Splanchnic vein thrombosis (includes Budd-Chiari, portal vein, splenic and mesenteric thrombosis). - History or other evidence of chronic pulmonary disease associated with functional limitation. - Thyroid dysfunction not adequately controlled. - Any investigational drug <6 weeks prior to the first dose of study drug. - Neutrophil count <1.5 x 109/L. - JAK2 exon 12 mutation. - Patients should not meet criteria for post PV or post ET-MF - No previous exposure to any formulation of pegylated interferon. - Subjects with any other medical condition, which in the opinion of the investigator would compromise the results of the study by deleterious effects of treatment.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the achievement of complete haematological response (CR) as defined by the European LeukemiaNet (ELN) criteria listed below: Criteria for Response in Essential thrombocythemia (ET): o Platelet count </= 400 x 109/L AND o No disease-related symptoms* AND o Normal spleen size on imaging AND o WBC </= 10 x 109/L Criteria for Response in Polycythemia Vera (PV) o Hematocrit <0.45 without phlebotomy AND o Platelet count < 400 x 109/L AND o WBC > 10 x 109/L AND o Normal spleen size on imaging AND o No disease related symptoms*
    E.5.1.1Timepoint(s) of evaluation of this end point
    Patients will be treated for 12 months (with a 3 month confirmation period for a total of up to 15 months) to achieve Complete Response (CR) or Partial Response (PR). Those not achieving at least a confirmed PR will stop trial therapy. Length of therapy for responders will be determined by subject’s entrance onto the study.
    E.5.2Secondary end point(s)
    The secondary end points are: o Evaluate the toxicity and tolerability of therapy in the study populations. o Ability of therapy to achieve a partial response (PR) by LeukemiaNet criteria. o Evaluate specific pre-defined toxicity and tolerance of therapy through a sequential structured symptom assessment package of patient reported outcome instruments. o Observed survival and incidence of development of a myelodysplastic disorder, myelofibrosis, or leukemic transformation. o Observed incidence of major cardiovascular events (defined as cardiovascular death, myocardial infarction, stroke, transient ischemic attack, pulmonary embolism, Budd Chiari syndrome, deep vein thrombosis, and any other clinically relevant thrombotic event) after therapy. o Measure the impact of therapy on key biomarkers of the disease(s) – JAK2-V617F, hematopoietic cell clonality in platelets and granulocytes in females, bone marrow histopathology, and cytogenetic abnormalities.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Patients will be treated for 12 months and evaluated regularly during this time. Those not achieving at least a confirmed PR will stop therapy at the end of 12 months of treatment. Length of therapy for responders will be determined by subject’s entrance onto the study. The last subjects to enter the study will continue for at least 24 months and the first subjects entered onto the study could participate for up to 48 months (assuming toxicity or loss of response does not intervene).
    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 Yes
    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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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) Yes
    E.8.2.2Placebo No
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    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
    Italy
    Netherlands
    Sweden
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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 years4
    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.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 32
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 32
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state64
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 315
    F.4.2.2In the whole clinical trial 612
    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)
    Patients will be treated for 12 months (with a 3 month confirmation period for a total of up to 15 months) to achieve complete or partial response. Those not achieving at least a confirmed partial response will stop therapy at the end of 15 months of treatment with pegylated interferon alfa-2a or hydroxyurea. The maximum length of treatment for responders will be determined by subject’s entrance onto the study.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Myeloproliferative Disorders Research Consortium
    G.4.3.4Network Country United States
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2012-11-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-01-10
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-01-01
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Fri May 03 11:03:03 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA