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    Summary
    EudraCT Number:2013-003583-31
    Sponsor's Protocol Code Number:CINC424B2401
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-02-28
    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 ConcernedFrance - ANSM
    A.2EudraCT number2013-003583-31
    A.3Full title of the trial
    Randomized, open label, multicenter phase IIIb study evaluating the efficacy and safety of ruxolitinib versus best available therapy in patients with polycythemia vera who are hydroxyurea resistant or intolerant (Response 2)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Ruxolitinib efficacy and safety in patients with HU resistant or intolerant polycythemia vera vs best available therapy
    A.3.2Name or abbreviated title of the trial where available
    Response 2
    A.4.1Sponsor's protocol code numberCINC424B2401
    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 SponsorNovartis Pharma Services AG
    B.1.3.4CountrySwitzerland
    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 supportNovartis Pharma Services AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Pharma S.A.S.
    B.5.2Functional name of contact pointInformationetCommunicationMédicales
    B.5.3 Address:
    B.5.3.1Street Address2-4 rue Lionel Terray
    B.5.3.2Town/ cityRueil Malmaison
    B.5.3.3Post code92500
    B.5.3.4CountryFrance
    B.5.4Telephone number33155476600
    B.5.5Fax number33155476100
    B.5.6E-mailicm.phfr@novartis.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 Yes
    D.2.1.1.1Trade name Jakavi
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm 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 Yes
    D.2.5.1Orphan drug designation numberEU/3/08/572 and EU/3/09/620
    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 CodeSUB32897
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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
    Polycythemia vera resistant or intolerant to hydroxyurea
    E.1.1.1Medical condition in easily understood language
    Polycythemia vera
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level LLT
    E.1.2Classification code 10036061
    E.1.2Term Polycythemia vera
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    efficacy of ruxolitinib to BAT as assessed by Hct control at Week 28
    E.2.2Secondary objectives of the trial
    Key secondary:
    1. efficacy of ruxolitinib to BAT as assessed by peripheral blood count remission at Week 28

    Other secondary:
    2. efficacy of ruxolitinib to BAT as assessed by durable Hct control at Week 52
    3. efficacy of ruxolitinib to BAT as assessed by durable peripheral blood count remission at Week 52
    4. change in ECOG status
    5. change in spleen length
    6. efficacy of ruxolitinib to BAT as assessed by partial remission based on the ELN and IWG-MRT criteria at Week 28
    7. efficacy of ruxolitinib to BAT as assessed by durable partial remission based on the ELN and IWG-MRT criteria at Week 52
    8. efficacy of BAT patients after they cross over to ruxolitinib
    9. changes in MPN-SAF TSS
    10. resolution of disease related symptoms in MPN-SAF TSS at Week 28
    11. changes in patient reported outcomes
    12. safety of ruxolitinib and Best Available Therapy
    13. safety of BAT patients after they cross over
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Confirmed diagnosis of PV according to the 2008 World Health Organization criteria
    - Non-palpable spleen
    - Phlebotomy dependent, Resistant to or intolerant of hydroxyurea
    - ECOG performance status of 0, 1 or 2.

    Other inclusion criteria as defined by protocol may apply
    E.4Principal exclusion criteria
    - Inadequate liver or renal function
    - Significant bacterial, fungal, parasitic, or viral infection requiring treatment
    - Active malignancy within the past 5 years, excluding specific skin cancers
    - Previously received treatment with a JAK inhibitor
    - Being treated with any investigational agent
    - Women who are pregnant or nursing.

    Other exclusion criteria as defined by protocol may apply.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of patients achieving Hct control at Week 28 as defined by:
    • Hct < 45% at Week 16 and maintained until Week 28, and
    • No phlebotomy from Week 4 to Week 28, with no more than one phlebotomy occurring post randomization and prior to the Week 4 visit.
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 28
    E.5.2Secondary end point(s)
    Key secondary:
    1. Proportion of patients achieving a peripheral blood count remission at Week 28 as defined by:
    • Hct < 45% at Week 16 and maintained until Week 28, and
    • WBC < 10 x109/L at Week 16 and maintained until Week 28, and
    • Platelets ≤ 400 x 109/L at Week 16 and maintained until Week 28, and
    • No phlebotomy from Week 4 to Week 28, with no more than one phlebotomy occurring post randomization and prior to the Week 4 visit.

    Other secondary:
    2. Proportion of patients achieving a Hct control at Week 52 as defined by:
    • Hct < 45% at Week 16 and maintained until Week 52, and
    • No phlebotomy from Week 4 to Week 28, with no more than one phlebotomy occurring post randomization and prior to the Week 4 visit, and
    • In the BAT arm, no change in the treatment regimen or crossover to the ruxolitinib arm
    3. Proportion of patients achieving a peripheral blood count remission at Week 52 as defined by:
    • Hct < 45% at Week 16 and maintained until Week 52, and
    • WBC < 10 x109/L at Week 16 and maintained until Week 52, and
    • Platelets ≤ 400 x 109/L at Week 16 and maintained until Week 52, and
    • No phlebotomy from Week 4 to Week 52, with no more than one phlebotomy occurring post randomization and prior to the Week 4 visit, and
    • In the BAT arm, no change in the treatment regimen or crossover to the ruxolitinib arm.
    4. Change in ECOG status from baseline to Week 28.
    5. Change from Baseline in spleen length at each scheduled visit
    6. Proportion of patients achieving a partial remission at Week 28, based
    on the ELN and IWG-MRT criteria, as defined by:
    • MPN-SAF TSS score reduction of greater than or equal to 10 points from baseline to Week 16 and maintained until Week 28 (for patients with a baseline score of 20 or more), and
    • Hct < 45% at Week 16 and maintained until Week 28, and
    • No phlebotomy from Week 4 to Week 28, with no more than one phlebotomy occurring post randomization and prior to the Week 4 visit, and
    • WBC < 10 x109/L at Week 16 and maintained until Week 28, and
    • Platelets ≤ 400 x 109/L at Week 16 and maintained until Week 28, and
    • No palpable spleen at Week 16 and maintained until Week 28, and
    • No hemorrhagic or thrombotic events, and
    • No transformation into post-PV myelofibrosis, myelodysplastic syndrome (IWG-MRT criteria) or acute leukemia.
    7. Proportion of patients who achieved partial remission at Week 52, based on the ELN and IWG-MRT criteria, as defined by:
    • MPN-SAF TSS score reduction of greater than or equal to 10 points from baseline to Week 16 and maintained until Week 52 (for patients with a baseline score of 20 or more), and
    • Hct < 45% at Week 16 and maintained until Week 52, and
    • No phlebotomy from Week 4 to Week 52, with no more than one phlebotomy occurring post randomization and prior to the Week 4 visit, and
    • WBC < 10 x109/L at Week 16 and maintained until Week 52, and
    • Platelets ≤ 400 x 109/L at Week 16 and maintained until Week 52, and
    • No palpable spleen at Week 16 and maintained until Week 52, and
    • No hemorrhagic or thrombotic events, and
    • No transformation into post-PV myelofibrosis, myelodysplastic syndrome (IWG-MRT criteria) or acute leukemia (WHO criteria), and
    • In the BAT arm, no change in the treatment regimen or crossover to the ruxolitinib arm.
    8. Among patients randomized to BAT who cross over to ruxolitinib, proportion of patients achieving Hct < 45% at each visit after cross over
    9. Proportion of patients achieving at least a 50% reduction from baseline in MPN-SAF TSS score at each visit where it is measured.
    10. Proportion of patients achieving a resolution of disease related symptoms in MPN-SAF TSS at Week 28 defined as:
    • MPN-SAF TSS score reduction of greater than or equal to 10 points from baseline to Week 16 and maintained until Week 28 (for patients with a baseline score of 20 or more).
    11. Change in MPN-SAF TSS from baseline to each visit where measured
    • PSIS at each visit where measured.
    • Change in EQ-5D-5L score from baseline to each visit where
    measured.
    • Change in WPAI from baseline to each visit where measured.
    • PGIC at each visit where measured.
    12. Safety will be assessed by monitoring the frequency, duration and severity of Adverse Events, and evaluating changes in vital signs, electrocardiograms (ECGs), serum chemistry, hematology and urinalysis results.
    13. Safety will be assessed by monitoring the frequency, duration and severity of Adverse Events, and evaluating changes in vital signs, electrocardiograms (ECGs), serum chemistry, hematology and urinalysis results.
    E.5.2.1Timepoint(s) of evaluation of this end point
    For key secondary,
    1. week 28

    For other secondary:
    2. week 52
    3. week 52
    4. week 28
    5. week 4, 8, 12, 16, 20, 24, 28, 40, 52/End of Treatment
    6. week 28
    7. week 52
    8. +4, +8, +12, +16, +20, +24 weeks after crossover
    9. week 4, 8, 16, 28, 40, 52/End of Treatment
    10. week 28
    11. week 4, 8, 16, 28, 40, 52/End of Treatment
    12. week 28, 52/End of Treatment
    13. +52/End of Treatment
    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 Yes
    E.6.13.1Other scope of the trial description
    document medical resource utilization (MRU)
    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 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
    Best available therapy (BAT)
    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 EEA45
    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
    Australia
    Belgium
    Canada
    France
    Germany
    Hungary
    India
    Israel
    Italy
    Korea, Republic of
    Spain
    Switzerland
    Turkey
    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
    Last Patient Last Visit defined as after all patients have either discontinued or completed the Week 52 visit or the applicable cross-over visit plus the 30 day follow up visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months5
    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: 84
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 60
    F.4.2.2In the whole clinical trial 104
    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)
    Following the last visit, patients who are still receiving benefit in the opinion of the investigator may continue to receive ruxolitinib. Patients will be transitioned to a local supply of ruxolitinib, off study, which will be reimbursed by Novartis where necessary. If local supply is not available to a patient at that time, s/he may be able to receive ruxolitinib under a separate protocol if approved by the country regulatory authority.
    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 Decision2014-07-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-03-05
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-04-07
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