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    Summary
    EudraCT Number:2015-003775-30
    Sponsor's Protocol Code Number:CICL670ABE04
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2015-11-17
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2015-003775-30
    A.3Full title of the trial
    A phase II pilot study to assess the presence of molecular factors predictive for hematologic response in myelodysplastic syndrome patients receiving deferasirox therapy in hematological centers in Belgium using gene expressing profiling from baseline bone marrow.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase II study in patients with low and intermediate -1 risk myelodysplastic syndrome while on therapy with deferasirox to investigate the presence of molecular factors in the baseline bone marrow which could predict an hematologic improvement due to therapy with deferasirox
    A.4.1Sponsor's protocol code numberCICL670ABE04
    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 NV
    B.1.3.4CountryBelgium
    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 NV
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Pharma nv
    B.5.2Functional name of contact point
    B.5.3 Address:
    B.5.3.1Street AddressMedialaan 40 bus 1
    B.5.3.2Town/ cityVilvoorde
    B.5.3.3Post code1800
    B.5.3.4CountryBelgium
    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 Exjade
    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 namedeferasirox
    D.3.2Product code ICL670
    D.3.4Pharmaceutical form Dispersible 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 INNDEFERASIROX
    D.3.9.1CAS number 201530-41-8
    D.3.9.2Current sponsor codeICL670
    D.3.9.4EV Substance CodeSUB21981
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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
    The study will include two groups of patients with low and intermediate 1 risk myelodysplastic syndrome. One group consists of patients who experience an hematological response while on deferasirox therapy while the other group consists of patients who are non-responders
    E.1.1.1Medical condition in easily understood language
    treatment of iron overload
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To identify differentially expressed genes in baseline bone marrow samples of low and intermediate-1 risk MDS patients with a hematologic response vs non-responder patients based on NGS of the whole transcriptome to search for a predictive gene signature.
    E.2.2Secondary objectives of the trial
    Objective 1: To assess the mean time between the initiation of treatment of deferasirox and the emergence of hematological response in patients with low and intermediate-1 risk MDS in the responder group
    Objective 2: To evaluate treatment related changes of iron parameters (serum ferritin, transferrin, transferrin saturation) in responders versus non-responders
    Objective 3: To evaluate the effect of the differential deferasirox dosing on iron and hematological parameters in responders versus non-responders
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Written informed consent obtained prior to any other study procedure,
    2. Males or females ≥ 18 years of age,
    3. MDS according to WHO criteria lasting ≥ 14 weeks at the time of screening,
    4. IPSS score <1.5 (low and intermediate-1 risk patients) at the time of screening using the IPSS score of 1997
    5. Treatment with deferasirox:
    - Only for the responder group: Treatment with deferasirox for prevention or treatment of IOL for at least 14 weeks before screening.
    - Only for the non-responder group: Treatment with deferasirox for at least 9 months for prevention or treatment of IOL before screening to exclude patients with a late hematological response.
    6. Only for responder-group: Patient with hematological response defined according to the IWG criteria of 2006 which must last at least 8 weeks and confirmed by the scientific advisory committee.
    7. Only for non-responder group: confirmation by the scientific advisory committee that patient is eligible based on matched-pairing and confirmation of no hematological response. Minimal requirements for matched pairing include age, sex, IPSS score, hemoglobin level and transfusion need at baseline, treatment duration with deferasirox and time since MDS diagnosis. Pairing can be extended according to level of leukopenia, thrombocytopenia, serum ferritin level at baseline, comorbidities and transfusion history.
    8.Bone marrow aspirate taken at the time of MDS diagnosis (at baseline) retrievable from patient’s hospital and viably frozen. This should be checked by the treating hematologist/oncologist before referring the patient for potential inclusion to the study.
    E.4Principal exclusion criteria
    1. Known concomitant presence of anemia due to iron, B12 or folate deficiencies, auto-immune or hereditary hemolysis, gastro-intestinal bleeding, or medication induced anemia at the time of screening,
    2. Known infection with viral hepatitis B (HBV) or hepatitis C (HCV) defined as the presence in blood of HBV antigens in absence of HB antibodies, or presence of HCV antibodies at the time of screening,
    3. Known positivity to human immunodeficiency virus (HIV) measured by enzyme-linked immunosorbent assay (ELISA) or western blot at the time of screening,
    4. Patient participating in another clinical trial or receiving an investigational drug, within 1 month prior to study inclusion
    5. History of other malignancy within the last five years, with the exception of basal skin carcinoma or cervical carcinoma in situ or completely resected colonic polyps carcinoma in situ.
    6. Concomitant treatment with other drugs known or suspected to elicit hematological response (azacitidine, erythroid stimulating agents, granulocyte colony stimulating factors, lenalidomide, thalidomide, valproate, ATG, cyclosporine, arsenic trioxide). When patients are still receiving red blood cell transfusions, patients are still eligible for study inclusion as long as they meet the IWG criteria of 2006
    7. Female patients who are pregnant or breast feeding.
    E.5 End points
    E.5.1Primary end point(s)
    Descriptive list of differentially expressed genes from responders versus non-responders.
    E.5.1.1Timepoint(s) of evaluation of this end point
    after at least 3 months of treatment with deferasirox in patients experiencing a hematological response
    after at least 9 months of treatment with deferasirox in patients not experiencing an hematological response in order to exclude late responders (most responses arise between 3 and 9 months after treatment start with deferasirix)
    E.5.2Secondary end point(s)
    Endpoint 1: The time to response is defined as the time (in months) between the date of deferasirox initiation and the date of the first documented hematological response only in the responder group.
    Endpoint 2: Changes in serum ferritin levels, serum transferrin levels, transferrin saturation levels from baseline to time of response (responder group) or time to last follow up (non-responders)
    Endpoint 3: Deferasirox dose is defined as the average daily dose (mg/kg/d) given to the patient from treatment initiation to the emergence of hematological response in the responder group or the time of enrollment in the study in the non-responder group.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Endpoint 1: at least after 3 months after treatment initiation with deferasirox
    Endpoint 2:
    * for responder patients: at least after 3 months after treatment initiation with deferasirox
    * for non-responder patients: at least after 9 months after treatment initiation with deferasirox
    Endpoint 3:
    * for responder patients: at least after 3 months after treatment initiation with deferasirox
    * for non-responder patients: at least after 9 months after treatment initiation with
    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 No
    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
    The scope of the trail is to see whether we can find predictive molecular factors in the bone marrow of MDS patients which can predict the emergence of an hematological response while on deferasirox therapy
    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 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 concerned13
    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 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 be defined as 30 days after bone marrow aspirate taken at visit 1 for the last patient ~ May 2017
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    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: 20
    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 No
    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 state20
    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 are only 1 day on Exjade provided by the sponsor, before and after this day the patient is taken commercially available medication.
    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 Decision2015-12-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-01-18
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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