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    Summary
    EudraCT Number:2011-004559-38
    Sponsor's Protocol Code Number:RG_12-101
    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:2012-06-07
    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 ConcernedUK - MHRA
    A.2EudraCT number2011-004559-38
    A.3Full title of the trial
    A phase 2 study of the efficacy and safety of Deferasirox administered at early iron loading in patients with transfusion-dependent Myelodysplastic Syndromes.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of the benefit and safety of using the drug Deferasirox in patients with increased iron levels who have Myelodysplastic Syndromes
    A.3.2Name or abbreviated title of the trial where available
    Deferasirox for early iron loading in transfusion-dependant MDS
    A.4.1Sponsor's protocol code numberRG_12-101
    A.5.4Other Identifiers
    Name:Sponsors SAF number Number:ERN_11-0870
    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 SponsorUniversity of Birmingham
    B.1.3.4CountryUnited Kingdom
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCRCTU, University of Birmingham
    B.5.2Functional name of contact pointTrial Office
    B.5.3 Address:
    B.5.3.1Street AddressSchool of Cancer Sciences
    B.5.3.2Town/ cityBirmingham
    B.5.3.3Post codeB15 2TT
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number01214147673
    B.5.5Fax number01214143700
    B.5.6E-mailDe-Iron@trials.bham.ac.uk
    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 AuthorisationUnited Kingdom
    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.3.4Pharmaceutical 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.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number125 to 500
    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
    Iron overload in patients being treated for Myelodysplastic syndroms (MDS) by regular blood transfusions
    E.1.1.1Medical condition in easily understood language
    Iron accumulation in patients being treated by regular blood transfusions for a range of conditions in which the bone marrow produces abnormal cells that function poorly(Myelodysplastic syndromes).
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level LLT
    E.1.2Classification code 10068361
    E.1.2Term MDS
    E.1.2System Organ Class 100000004864
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level LLT
    E.1.2Classification code 10040310
    E.1.2Term Serum iron increased
    E.1.2System Organ Class 100000004848
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary aim of this study is to assess the activity of the study drug by measuring how long it takes for the levels of iron in the blood to rise to over 1500μg/l (a level associated with a poor prognosis and organ damage) when treated with Deferasirox.
    E.2.2Secondary objectives of the trial
    The secondary aims are to assess the proportion of patients who reach the the 1500μg/l during the 12 months of treatment, the Safety and tolerability of the drug in this population of patients, by collecting data on side effects, as well as measuring the proportion of patients maintaining a serum ferritin level in their blood of less than 1500 μg/l. We will also be measuring the cardiac and hepatic iron loading by MRI scan, in patients willing to undergo imaging, to see if the heart and liver stores of iron have increased during the study as wells as measuring the patients endocrine function to see if this has been altered during treatment.
    All patients will also be asked to provide blood samples for Biochemical iron parameter tests to look and different iron related proteins in the blood and how they change during treatment. We will be collecting data to measure the proportion of patient achieving a Haematological response, the time to this response and the duration of this respo
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    MRI T* scans will be optional and will therefore be classed as a sub-study. We will be measuring the cardiac and hepatic iron loading by MRI scan, in patients willing to undergo imaging, to see if the heart and liver stores of iron have increased during the study as wells as measuring the patients endocrine function to see if this has been altered during treatment.
    E.3Principal inclusion criteria
    • At least 18 years old.
    • Written informed consent.
    • MDS with:
    o Baseline haemoglobin concentration < 11 g/dl and clinically requiring red cell transfusion with a frequency of at least 2 units every 6 weeks for the receding 12 week period.
    o Serum ferritin >300μg/l but <1000 μg/l in absence of ongoing inflammation (CRP < 3 x ULN)
    o Serum creatinine <1.2 x ULN and/or creatinine clearance > 40 mls/min
    o ALT or AST < 2.5 ULN
    o IPSS Low/INT-1 previously untreated or having failed a therapeutic trial of erythropoetic stimulating agents (ESA) or other active MDS drug therapy, or alternatively lost their response to such therapy
    o IPSS INT-2 with < 10% blasts and lacking a complex karyotype or monosomy 7 (and with stable blood counts from diagnosis to study entry).
    E.4Principal exclusion criteria
    • Active treatment for MDS, including erythropoetic stimulating agents (ESA), 5-azacitidine, antilymphocyte globulin and low dose chemotherapy such as cytarabine during the trial and within the last 8 weeks
    • Life expectancy of less than 1 year
    • Known HIV positive
    • Active infection
    • Use of prior investigational agents within 6 weeks
    • Pregnancy or lactation
    •Other severe, concurrent medical illness that may affect the patients participation in the study, or psychiatric disorders
    • Concurrent active or previous malignancy, within the last 3 years, – except controlled, localised prostate cancer on hormone therapy or basal cell carcinoma or cervical carcinoma in situ or completely resected colonic polyps carcinoma in situ
    • Ongoing inflammation as measured by CRP > 3 x ULN
    • Serum creatinine >1.2 x ULN and/or creatinine clearance < 40 mls/min
    • ALT or AST >2.5 ULN
    • History of drug/alcohol abuse or non-compliance
    E.5 End points
    E.5.1Primary end point(s)
    The Primary outcome is Time to mean serum Ferritin > 1500 μg/l, as measured from the time of initiation using the mean serum Ferritin value of 2 consecutive measurements of Ferritin, where the first level is >1500 μg/l and CRP is < 3 times baseline measurement.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The primary activity outcome measures will be assessed in the full analysis set which includes all patients who have taken at least 1 dose of study drug and have at least one assessment of ferritin level. Analysis will occur when all patients have completed 12months of treatment or discontinued. Patients not reaching mean ferritin level greater than 1500 μg/l at the time of analysis will be censored at the last response assessment. In cases where the second serum ferritin level is not available and the first level is > 1500 μg/l, the first level is used and an event is assumed to have occurred.
    E.5.2Secondary end point(s)
    - Proportion of patients with mean serum Ferritin, of 2 consecutive measurements of Ferritin, >1500 μg/l within 12 months, where the first level is >1500 μg/l and CRP is < 3 times baseline measurement.
    - Safety and tolerability is defined in terms of the following events deemed to be related to Deferasirox and graded according to the CTCAE Criteria, version 4: a) Grade 3/4 non-haematological toxicity that does not resolve to ≤ Grade 2 within 4 weeks of deferasirox withdrawal, b) unexpected Grade 3/4 non-haematological toxicity, c) ≥Grade 2 auditory or ocular abnormalities associated with chelation therapy.
    - Proportion of patients maintaining serum ferritin <1500 μg/l at 12 months
    - Cardiac and hepatic iron loading quantified by MRI R2 and T2* is defined as the mean change in iron concentration pre-treatment and at 12 months or when serum Ferritin levels exceed 2000 μg/l. This will be available for patients treated within a centre offering iron quantitation by MRI, or willing to travel.
    - Endocrine function is defined as the mean change in the following parameters pre-treatment and at 12 months; diabetes (HbA1c), thyroid (as measured by serum thyroid hormone concentration), sex hormones (measured by serum FSH, LH, testosterone) and adrenal (measured by cortisol).
    - Proportion of patients achieving hematologic response per IWG2006 criteria
    - Time to haematological response per IWG2006 criteria
    - Duration of haematological response per IWG2006 criteria
    - Biochemical iron parameters including transferrin saturation, hepcidin, non-transferrin bound iron and GDF15 as measure by the central laboratory at UCL.
    E.5.2.1Timepoint(s) of evaluation of this end point
    The safety outcome measures will be assessed using the safety analysis set. Toxicity will be assessed continuously throughout the study according to CTC version 4. In addition, a three stage design (each of 18 patients) has been planned which incorporates formal early stopping guidelines that meet the error rate criterion of alpha of 0.1 and beta of 0.2 to assess tolerability of treatment (Section 12.4. All other analyses will occur when all patients have completed 12months of treatment or discontinued.
    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 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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 concerned16
    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
    The end of trial will be 6 months after the last data capture. This will allow sufficient time for the
    completion of protocol procedures, data collection and data input. The Trials Office will notify the MHRA and main REC that the trial has ended and will provide them with a summary of the clinical trial report within 12 months of the end of trial.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 years3
    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: 25
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 29
    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 state54
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 54
    F.4.2.2In the whole clinical trial 54
    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)
    After 12 months patients will have completed the desired treatment and the study drug will be stopped. However, patients will be closely monitored by their doctors after this period and it may be necessary to start another drug to control their iron levels in the blood. Patients will not be able to continue the trial drug beyond 12 months.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2012-08-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-07-26
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-05-01
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