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    Summary
    EudraCT Number:2014-003527-22
    Sponsor's Protocol Code Number:CINC424A2X01B
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-11-10
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2014-003527-22
    A.3Full title of the trial
    An open label, multi-center, Phase IV roll-over protocol for patients who
    have completed a prior global Novartis or Incyte sponsored ruxolitinib
    (INC424) study or a study of ruxolitinib in combination with panobinostat
    (LBH589) or siremadlin (HDM201) or rineterkib (LTT462) and are judged
    by the investigator to benefit from continued treatment
    Wieloośrodkowe, otwarte badanie fazy IV dotyczące kontynuacji leczenia z udziałem pacjentów, którzy ukończyli wcześniejsze globalne badanie sponsorowane przez firmę Novartis lub Incyte dotyczące ruksolitynibu (INC424) lub skojarzenia ruksolitynibu i panobinostatu (LBH589), w przypadku których, zdaniem badacza, kontynuacja leczenia przyniesie korzyści
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study for patients who have completed a prior global Novartis or Incyte
    sponsored ruxolitinib (INC424) study or a study of ruxolitinib in
    combination with panobinostat (LBH589) or siremadlin (HDM201) or
    rineterkib (LTT462) and are judged by the investigator to benefit from
    continued treatment
    A.4.1Sponsor's protocol code numberCINC424A2X01B
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02386800
    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 Services AG
    B.5.2Functional name of contact pointPunktinformacyjnybadaniaklinicznego
    B.5.3 Address:
    B.5.3.1Street AddressForum 1, Novartis Campus / Lichtstrasse 35
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4056
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+41 61 3241 111
    B.5.5Fax number+41 61 3248 001
    B.5.6E-mailclinicaltrial.enquiries@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 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 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
    This roll-over study is designed to accept patients with varied disease origins. Please refer to the parent protocol for the disease background information and rationale for use of ruxolitinib in their individual indications.
    E.1.1.1Medical condition in easily understood language
    This study is designed to accept patients with varied disease origins. Please refer to the parent protocol.
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10074689
    E.1.2Term Post polycythemia vera myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10074690
    E.1.2Term Post essential thrombocythemia myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10074691
    E.1.2Term Post polycythaemia vera myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10074692
    E.1.2Term Post essential thrombocythaemia myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10036061
    E.1.2Term Polycythemia vera
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10054658
    E.1.2Term Thalassemia
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 27.0
    E.1.2Level PT
    E.1.2Classification code 10066264
    E.1.2Term Acute graft versus host disease in intestine
    E.1.2System Organ Class 10021428 - Immune system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 27.0
    E.1.2Level PT
    E.1.2Classification code 10066262
    E.1.2Term Acute graft versus host disease in skin
    E.1.2System Organ Class 10021428 - Immune system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10066263
    E.1.2Term Acute graft versus host disease in liver
    E.1.2System Organ Class 10021428 - Immune system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 27.0
    E.1.2Level PT
    E.1.2Classification code 10066260
    E.1.2Term Acute graft versus host disease
    E.1.2System Organ Class 10021428 - Immune system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 27.0
    E.1.2Level PT
    E.1.2Classification code 10066261
    E.1.2Term Chronic graft versus host disease
    E.1.2System Organ Class 10021428 - Immune system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10072160
    E.1.2Term Chronic graft versus host disease in liver
    E.1.2System Organ Class 10021428 - Immune system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10072158
    E.1.2Term Chronic graft versus host disease in intestine
    E.1.2System Organ Class 10021428 - Immune system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10072159
    E.1.2Term Chronic graft versus host disease in skin
    E.1.2System Organ Class 10021428 - Immune system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate long term safety data i.e. SAEs and AEs.
    E.2.2Secondary objectives of the trial
    To evaluate clinical benefit as assessed by the investigator

    Other secondary:
    - To evaluate long term safety data by ruxolitinib in monotherapy or in
    combination with
    panobinostat or siremadlin (HDM201) or rineterkib (LTT462)
    - To evaluate clinical benefit by ruxolitinib in monotherapy or in
    combination with panobinostat or siremadlin (HDM201) or rineterkib
    (LTT462)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patient is currently enrolled in a Novartis GDD or GMA-sponsored or
    Incyte-sponsored clinical study (where Incyte can delegate the
    sponsorship to a preferred CRO, if applicable) that is approved to enroll
    into this rollover study, and are receiving either ruxolitinib or
    combination of ruxolitinib and panobinostat, or combinations of
    ruxolitinib and siremadlin (HDM201), or ruxolitinib and rineterkib
    (LTT462), and fulfilled all of the requirements of the parent protocol.
    Please refer to the list of parent studies in Appendix 2.
    - Patient is currently benefiting from the treatment with ruxolitinib
    monotherapy or combination of ruxolitinib and panobinostat, or
    combinations of ruxolitinib and siremadlin (HDM201), or ruxolitinib and
    rineterkib (LTT462), as determined by the investigator
    - Patient has demonstrated compliance, as assessed by the investigator,
    with the parent study protocol requirements
    - Willingness and ability to comply with scheduled visits, treatment plans
    and any other study procedures
    - Patient currently has no evidence of progressive disease, as
    determined by the investigator, following previous treatment with
    ruxolitinib or combination of ruxolitinib and panobinostat, or
    combinations of ruxolitinib and siremadlin (HDM201), or ruxolitinib and
    rineterkib (LTT462)
    - Written informed consent obtained prior to enrolling in roll-over study and receiving study medication. If consent cannot be expressed in writing, it must be formally documented and witnessed, ideally via an independent trusted witness.
    Note 1: If the patient is a minor, the parent who signs the informed consent for the minor
    must be a legally recognized parent or guardian. Where deemed appropriate by the clinician, and the child’s parent or guardian, the child will also be included in the alldiscussions about the trials and the minor aged 12 and above assent will be obtained. The parent or guardian will sign on the designated line on the ICF attesting to the fact that the child had given consent.
    Note 2: if the minor is an adolescent female, she will be informed during the assent process that for safety purpose, a pregnancy test is required. She will also be told that if it is positive, she will be counseled and will be assisted in telling her parents. If the minor does not want to proceed, she will be advised not to sign consent and her enrollment in this protocol will end.
    E.4Principal exclusion criteria
    - Patient has been permanently discontinued from study treatment in the parent study due to any reason.
    - Patient’s indication is currently approved and reimbursed in the local
    corresponding country for ruxolitinib monotherapy or combination of
    ruxolitinib and panobinostat (if the patient is receiving combination
    treatment in the parent study)
    - Patient has participated in a combination trial other than the panobinostat and ruxolitinib
    combination trial (CLBH589X2106), where ruxolitinib was dispensed in combination with another study medication and the patient is still receiving combination therapy.
    - Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test.
    - Female patients of childbearing potential (e.g. are menstruating) who
    do not agree to abstinence or, if sexually active, do not agree to the use
    of highly effective contraception as defined below, throughout the study
    and for up to 30 days after stopping study treatment. If local regulations
    are more stringent than the contraception methods listed in the protocol
    and in Section 7.1.7 to prevent pregnancy, the local regulations will
    apply as described in the ICF.
    Highly effective contraception methods include: see protocol
    - Patient has participated in a combination study other than the
    CINC424H12201 platform study providing combinations of either
    ruxolitinib and siremadlin or ruxolitinib and rineterkib, where ruxolitinib
    was dispensed in combination with another study medication
    - Sexually active males, unless they use a condom during intercourse
    while taking siremadlin or rineterkib and for 2 weeks after siremadlin or
    rineterkib discontinuation, and thus do not attempt to father a child in
    this period. A condom is required to be used also by vasectomized men
    in order to prevent delivery of the drug via seminal fluid.
    - Had history of documented severe hypersensitivity
    reactions/immunogenicity to a prior biologic product in any treatment
    arm OR received a monoclonal antibody or immunoglobulin-based agent:
    • For treatment arms with rineterkib or siremadlin arms within ≤4
    weeks of screening or ≤5 half-lives whichever is shorter for rineterkib or
    siremadlin arms
    E.5 End points
    E.5.1Primary end point(s)
    Frequency and severity of SAEs/AEs
    E.5.1.1Timepoint(s) of evaluation of this end point
    through study completion estimated to be approximately 10 years
    E.5.2Secondary end point(s)
    Proportion of patients with clinical benefit as assessed by the investigator at scheduled visits

    Other secondary:
    - frequency and severity of AEs/SAEs
    - Proportion of patients with clinical benefit as assessed by the investigator at scheduled visits
    E.5.2.1Timepoint(s) of evaluation of this end point
    at every visit that occur every 12 weeks until EOT

    other secondary:
    - at every visit that occur every 12 weeks until End of Trial
    - at every visit that occur every 12 weeks until End of Trial
    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 No
    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
    To better characterize the long term safety of ruxolitinib in patients from existing global Novartis or Incyte sponsored studies who are benefiting from treatment after conclusion of the parent study
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    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) No
    E.8.2.2Placebo No
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA83
    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
    Argentina
    Chile
    Switzerland
    Taiwan
    Australia
    Brazil
    Canada
    China
    India
    Israel
    Japan
    Jordan
    Korea, Republic of
    Lebanon
    Mexico
    Russian Federation
    Saudi Arabia
    South Africa
    Thailand
    Belgium
    Bulgaria
    Czechia
    Denmark
    France
    Germany
    Greece
    Hungary
    Italy
    Lithuania
    Norway
    Poland
    Portugal
    Romania
    Slovakia
    Slovenia
    Spain
    Sweden
    Türkiye
    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
    End of the study will occur after all patients in the study have completed their last assessment per protocol.Patient may continue on study treatment until one of the following criteria is met, whichever come first
    End of Treatment
    At least one of the premature patient withdrawal criteria are met
    12 years after the first patient’s first visit into this clinical study
    Current treatment becomes commercially available and reimbursed in that indication
    PSDS becomes available in other programs
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days13
    E.8.9.2In all countries concerned by the trial years12
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days12
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 38
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 28
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 196
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 132
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 245
    F.4.2.2In the whole clinical trial 366
    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)
    Should ruxolitinib (rux) or combination of rux and panobinostat or
    combinations of rux and siremadlin or rux and rineterkib not be
    commercially available to
    patients at end of study due to local regulations, Novartis will make
    every effort to ensure that patients benefiting from treatment continue
    to have access to rux or combination of
    rux and panobinostat or combinations of rux and siremadlin or rux and
    rineterkib without interruption of treatment in accordance with local
    regulation
    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 Decision2022-01-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-11-18
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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    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
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