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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:2013-002132-25
    Sponsor's Protocol Code Number:12-181
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2015-02-03
    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 ConcernedGermany - BfArM
    A.2EudraCT number2013-002132-25
    A.3Full title of the trial
    RUXOlitinib versus BEst Available Therapy in patients with high-risk polycythemia vera or high-risk essential thrombocythemia – The RUXO-BEAT Trial
    Ruxolitinib im Vergleich zur besten verfügbaren Therapie in Patienten mit Hoch-Risiko Polycythemia vera oder Hoch-Risiko Essentieller Thrombozythämie – Die RUXO-BEAT Studie
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    RUXOlitinib versus BEst Available Therapy in patients with high-risk polycythemia vera or high-risk essential thrombocythemia – The RUXO-BEAT Trial
    Ruxolitinib im Vergleich zur besten verfügbaren Therapie in Patienten mit Hoch-Risiko Polycythemia vera oder Hoch-Risiko Essentieller Thrombozythämie – Die RUXO-BEAT Studie
    A.3.2Name or abbreviated title of the trial where available
    Ruxo-BEAT
    Ruxo-BEAT
    A.4.1Sponsor's protocol code number12-181
    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 SponsorRWTH Aachen University, represented by the Rector, himself represented by the Dean of the Medical Faculty
    B.1.3.4CountryGermany
    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 supportNovartis Pharma GmbH
    B.4.2CountryGermany
    B.4.1Name of organisation providing supportDepartment of Oncology, Haematology and Stem Cell Transplantation (Medical Clinic IV), University Hospital RWTH Aachen
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCenter for Translational & Clinical Research Aachen (CTC-A)
    B.5.2Functional name of contact pointCTC-A
    B.5.3 Address:
    B.5.3.1Street AddressPauwelsstrasse 30
    B.5.3.2Town/ cityAachen
    B.5.3.3Post code52074
    B.5.3.4CountryGermany
    B.5.4Telephone number004902418080092
    B.5.5Fax number00490241803380092
    B.5.6E-mailctc-a-spoqs@ukaachen.de
    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 Jakavi® 5 mg tablets
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Ltd.
    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 nameRuxolitinib
    D.3.2Product code INC424
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    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 INNRuxolitinib
    D.3.9.1CAS number 1092939-17-7
    D.3.9.2Current sponsor codeINC424
    D.3.9.3Other descriptive nameRUXOLITINIB-Phosphate
    D.3.9.4EV Substance CodeSUB32273
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5 to 20
    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.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 Yes
    D.2.1.1.1Trade name Jakavi® 20 mg tablets
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Ltd.
    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 nameRuxolitinib
    D.3.2Product code INC424
    D.3.4Pharmaceutical form Tablet
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Myeloproliferative Neoplasms (MPN): high-risk Polycythemia Vera (PV) and high-risk Essential Thrombocythemia (ET)
    E.1.1.1Medical condition in easily understood language
    Patients with myeloproliferative blood disorders may suffer an overproduction of red blood cells, white blood cells or platelets.
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The objective of the Ruxo-BEAT trial is to assess the feasibility, safety, and efficacy of administration of ruxolitinib vs. best available therapy in patients with high-risk PV or high-risk ET.

    Primary Endpoint
    • The rate of complete clinicohematologic response rate (CHR) at month 6 as defined by Barosi et al Blood 2009.
    E.2.2Secondary objectives of the trial
    • Safety of both regimens
    • To determine the complete response rate (CR) at month 6 as defined by Barosi et al Blood 2013 (revised ELN response criteria)
    • The rate of complete responses (CHR) at month 12 as defined by Barosi et al Blood 2009
    • The efficacy as assessed by both the absence of phlebotomy (Hct <45%) and reduction in spleen size (palpable spleen that is reduced by > 50% from baseline measured by palpation) OR platelet count < 600 x 109/l (ET)
    • The proportion of subjects achieving both durable absence of phlebotomy eligibility and durable spleen volume reduction measured by palpation (PV) OR durable platelet count < 600 x 109/l (ET) (durable defined as > 3 months) {Barosi et al 2013)
    • The rate of overall clinicohematologic remissions (CR + PR) according to both guidelines (Barosi et al 2009 and 2013)



    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Substudy to establish and prospectively validate cold-shock protein Ybx1 as diagnostic marker in MPN (August 10, 2016)

    Concept and Specific Aims:
    We aim to prospectively validate Ybx1 as a diagnostic and putative prognostic marker for MPN (PV and ET) within the Ruxo-BEAT trial.
    Specific Aims (SA):
    SA1: Assessment of Ybx1 expression in diagnostic samples (bone marrow biopsies) from patients registered and screened for the Ruxo-BEAT trial.
    SA2: Analysis of Ybx1 mRNA expression in erythro-lysed peripheral blood samples derived from patients registered and screened for the Ruxo-BEAT trial.
    SA3: Correlation of immunohistochemical stainings (multiplied M-score) and mRNA expression with mutational status, disease burden, response to therapy and prognostic & clinical data.
    E.3Principal inclusion criteria
    1. Subjects must provide written informed consent prior to performance of study-specific procedures or assessments which are not routinely performed for diagnosis or monitoring of PV or ET, and the subjects must be willing to comply with treatment and to follow up assessments and procedures
    2. Patient must be 18 years of age or older
    3. Patient´s ECOG performance status must be 0-2
    4. Patient must fulfill WHO 2008 diagnostic criteria for either polycythemia vera (PV) or essential thrombocythemia (ET). Moreover, PV- and ET-patients have to be classified as high risk according to defined criteria. For patients with high risk PV OR PV with indication for cytoreductive therapy due to progressive myeloproliferation, AT LEAST ONE of the following must be fulfilled (according to DGHO onkopedia, Barbui 2011, Passamonti 2009) :
    • Age >60 years
    • Previous documented thrombosis or thromboembolism
    • Platelet count > 1500 x 109/L
    • Poor tolerance of phlebotomy or frequent phlebotomy requirement
    • Symptomatic or progressive splenomegaly
    • Severe disease-related symptoms (according to the investigators definition)
    • Progressive leukocytosis with leukocyte count > 20 x 109/L
    For patients with high risk ET, AT LEAST ONE of the following must be fulfilled (according to DGHO guidelines):
    • Age > 60 years
    • Platelet count> 1500 x 109/L
    • Previous thrombosis or thromboembolism
    • Previous severe hemorrhage related to ET (defined as decrease of Hgb of at least 2 g/dl)

    5. Patients must fulfill the following criteria regarding prior therapy:
    PV patients:
    Never treated with cytoreductive drugs except hydroxyurea, anagrelide, or interferon for up to 6 weeks maximum (phlebotomy and/or aspirin are allowed)
    ET patients:
    Naïve and pretreated patients may be entered in this trial.
    6. Patient must have a life expectancy of more than 12 weeks
    7. Patient must have adequate liver function as indicated by a total bilirubin, AST, and ALT ≤ 2 the institutional ULN value, unless directly attributable to the patient’s MPN
    8. Patient must have a creatinine clearance >40ml/min calculated according to the modified formula of Cockcroft and Gault, eGFR, or directly measured after 24h-urine collection
    9. Able to swallow and retain oral medication
    E.4Principal exclusion criteria
    1. Patients who meet criteria for post PV-MF or post ET-MF (IWG-MRT)
    2. Patients who have received previous ruxolitinib treatment
    3. Patients who have a history of anaphylaxis following exposure to the BAT drug of choice
    4. Patients who have an inadequate bone marrow reserve as demonstrated by ANC ≤ 1 x 109/l OR platelet count <50 x 109/l
    5. Patients who have known hepatitis B or C or HIV infection
    6. Patients who suffer from other severe, concurrent diseases, including tuberculosis, serious cardiac functional dysfunction (class III or IV as defined by the New York Heart Association Classification), uncontrolled diabetes, uncontrolled hypertension, severe pulmonary disease (i.e. COPD with hypoxemia), or major organ malfunction that could interfere with the patient’s ability to participate in the study
    7. Patients who have history of active substance or alcohol abuse within the last year
    8. Female patients who are pregnant or nursing
    9. Patients who have participated in another interventional trial and/or used investigational agents or concurrent anticancer treatment for concomitant disease within the last 4 weeks of registration
    10. Any circumstance at the time of study entry that would preclude completion of the study or the required follow-up prohibits inclusion into this study
    11. Subjects who have had an active malignancy during the previous 3 years except for treated cervical intraepithelial neoplasia, basal cell carcinoma of the skin, or squamous cell carcinoma of the skin, each with no evidence for recurrence in the past 3 years
    12. Patients who have uncontrolled bacterial, viral, or fungal infection
    13. Patients who have any medical condition requiring prolonged use of oral corticosteroids with a dose of more than 20 mg per day (> 1 month)
    14. Patients who have severe cerebral dysfunction and/or legal incapacity
    15. Patients who have had active splanchnic vein thrombosis within the last 3 months (includes Budd-Chiari, portal vein, splenic and mesenteric thrombosis)
    16. Patients who have thyroid dysfunction which is not adequately controlled
    17. Fertile men or women of childbearing potential cannot be included unless they are:
    -surgically sterile or > 2 years after the onset of menopause and/or
    -willing to use a highly effective contraceptive method (Pearl Index <1) such as oral contraceptives, intrauterine device, sexual abstinence, or barrier method of contraception (i.e. condoms) in conjunction with spermicidal jelly during study treatment
    18. Patients who are taking any of the following prohibited medication:
    -clarithromycin, telithromycin, troleandomycin (antibiotics)
    -ritonavir, indinavir, saquinavir, nelfinavir, amprenavir, lopinavir (HIV protease inhibitors)
    -itraconazole, ketoconazole, voriconazole, fluconazole (antifungals)
    -nefazodone (antidepressant)
    E.5 End points
    E.5.1Primary end point(s)
    • The rate of complete clinicohematologic responses (CHR) at month 6 as defined by Barosi et al 2009
    E.5.1.1Timepoint(s) of evaluation of this end point
    EOT
    E.5.2Secondary end point(s)
    • Safety of both regimens
    • To determine the complete response rate (CR) at month 6 as defined by Barosi et al Blood 2013 (revised ELN response criteria)
    • The rate of complete responses (CHR) at month 12 as defined by Barosi et al Blood 2009
    • The efficacy as assessed by both the absence of phlebotomy (Hct <45%) and reduction in spleen size (palpable spleen that is reduced by > 50% from baseline measured by palpation) OR platelet count < 600 x 109/l (ET)
    • The proportion of subjects achieving both durable absence of phlebotomy eligibility and durable spleen volume reduction measured by palpation (PV) OR durable platelet count < 600 x 109/l (ET) (durable defined as > 3 months) {Barosi et al 2013)
    • The rate of overall clinicohematologic remissions (CR + PR) according to both guidelines (Barosi et al 2009 and 2013)
    E.5.2.1Timepoint(s) of evaluation of this end point
    EOT
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 Yes
    E.8.1.6Cross over Yes
    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 Yes
    E.8.2.3.1Comparator description
    Hydroxyurea, Interferon, Anagrelide, Pipobroman, Busulfan
    Control group will receive Best Available Therapy based on the investigator’s best judgment.
    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 concerned30
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    LVLS
    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 months
    E.8.9.1In the Member State concerned days
    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: 380
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 380
    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 state380
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 380
    F.4.2.2In the whole clinical trial 380
    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)
    For patients failing BAT at the 6 month time point, crossover is possible (see section 8.20 for crossover requirements).
    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 Decision2015-03-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-06-29
    P. End of Trial
    P.End of Trial StatusOngoing
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