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    Summary
    EudraCT Number:2010-023217-61
    Sponsor's Protocol Code Number:10/0174
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2012-01-05
    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 number2010-023217-61
    A.3Full title of the trial
    A phase IV, open-label, partial cross-over partial parallel, randomized, multi-centre study to compare the gastrointestinal tolerability of once daily oral deferasirox, when administered before or after food in patients with transfusional haemosiderosis.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    DEFERASIROX GASTROINTESTINAL TOLERABILITY STUDY
    A.3.2Name or abbreviated title of the trial where available
    DEFERASIROX GASTROINTESTINAL TOLERABILITY STUDY
    A.4.1Sponsor's protocol code number10/0174
    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 College London
    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 organisation
    B.5.2Functional name of contact point
    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 Deferasirox (Exjade®) dispersible tablets 125 mg
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/02/092
    D.3 Description of the IMP
    D.3.1Product nameExjade 125 mg
    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 code10/0174
    D.3.9.4EV Substance CodeAS3
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    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.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 Deferasirox (Exjade) dispersible tablets 250 mg
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/02/092
    D.3 Description of the IMP
    D.3.1Product nameExjade 250 mg
    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 code10/0174
    D.3.9.4EV Substance CodeAS4
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    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.IMP: 3
    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 Deferasirox (Exjade) dispersible tablets 500mg
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/02/092
    D.3 Description of the IMP
    D.3.1Product nameExjade 500mg
    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 code10/0174
    D.3.9.4EV Substance CodeAS5
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    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
    transfusional haemosiderosis
    E.1.1.1Medical condition in easily understood language
    iron overload due to multiple blood transfusions
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10019024
    E.1.2Term Haemosiderosis
    E.1.2System Organ Class 10027433 - Metabolism and nutrition 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 compare the gastrointestinal (GI) tolerability of deferasirox administered before and after meals in patients with iron overload due to multiple blood transfusions, on established deferasirox therapy. The primary endpoint is the difference in mean individual Gastrointestinal Quality of Life Index (GIQLI)score change from baseline to end of study in the before-meals arm versus after-meals arm.
    E.2.2Secondary objectives of the trial
    1) To describe the gastrointestinal tolerability of deferasirox administered before and after meals in patients with transfusional iron overload naive to deferasirox therapy. The secondary endpoint is the difference in mean individual GIQLI score change from baseline to 1 month and 2 months of before meal versus after meal deferasirox administration. 2) To compare steady state pharmacokinetics profile characteristics (AUCss, Cmax ss, Cmin ss) following 1 month of deferasirox administered either before or after meals in a crossover design in patients with established deferasirox therapy. where AUCss is Area Under the Curve at steady state (a measure of bioavailability of drug and drug clearance) Cmax ss is the maximum concentration of drug at steady state Cmin ss is the minimum concentration of drug at steady state Exploratory: 1) To investigate the relationship between plasma level of the iron-drug complex and plasma iron parameters.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Adults 18 years and over Ability to provide informed consent Ability to meet all study requirements Adequate renal function (e.g. creatinine clearance≥ 60ml/min), Wllingness to stop other iron chelation therapy, No known allergies to the drug Not pregnant or breast feeding and willing to use effective contraception For non-naive cohort: male or female participants with transfusional iron overload as defined by a minimum of 20 lifetime transfusion episodes and transfusional iron loading with ferritin levels >500 mcg/L and/or liver iron concentration >3 mg of iron/g dry weight (as previously demonstrated by liver biopsy or MRI prior to the study); established on deferasirox therapy for at least 1 year and suffering GI side effects believed to be related to their therapy as suggested by at least one of the following: - The temporal relationship of gastrointestinal side effects occurrence with the administration of deferasirox ….
    E.4Principal exclusion criteria
    Patients with gastrointestinal symptoms assumed or known NOT to be caused by deferasirox. Patients who are treated for any condition with aspirin daily, patients under chronic steroid therapy and patients on anticoagulant therapy that could all lead to potential gastrointestinal symptoms/bleeding. Presence of gastrointestinal disease that may significantly alter the absorption of deferasirox (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhoea, malabsorption syndrome, gastrointestinal or rectal bleeding). History of gastrointestinal surgery (except appendicitis and cholecystectomy) e.g. stomach/bowel surgery or awaiting elective surgery in the next 2 months. Undergoing acute medical intervention or hospitalization. Psychiatric illness (schizophrenia, major depression) which may interfere with study requirements Any other medical condition that, in the opinion of the site investigator would interfere with completing the study (visual problems or cognitive impairment. Platelet count less than 100 000 Currently on treatment for Hepatitis C Patients with severe iron loading in the heart (T2* less than 10 ms) and/or patients with severe total body iron load (liver iron concentration over 30 mg/g dry weight) Patients who have historical evidence of severe iron loading in the heart Women of child-bearing potential who are planning a pregnancy, pregnant, lactating and unwilling to use effective means of contraception. Inadequate renal function (e.g. Creatinine clearance < 60ml/min) Patients unwilling to stop using other iron chelation therapy for the trial duration Known allergy to the drug Patients on aluminium containing antacid preparations Patients who are on Vitamin C at doses higher than 200mg/day Patients receiving or having received any investigational drug within 30 days prior to study enrolment Patients unable to understand oral or written English
    E.5 End points
    E.5.1Primary end point(s)
    Primary endpoint is the between-treatment arm-difference of the mean individual change of Gastrointestinal Quality of Life Index(GIQLI) (follow-up minus baseline) in the non-naive cohort (patients on established deferasirox therapy).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Four weeks after treatment 1 in the non-naive cohort Four weeks after crossover day 1 in the non-naive cohort
    E.5.2Secondary end point(s)
    1) Difference between mean follow-up ( 1 month and 2 months) vs baseline changes of GIQLI per arm in naive cohort 2) Difference of the mean GSRS score changes in both arms of the established cohort and the naive cohort 3) Difference of the mean SF-36 score changes in both arms of the established cohort and the naive cohort 4) Arm difference of geometric mean steady state Cmin at visit 3 and visit 4 in the cross-over group 5) Arm difference of geometric mean AUC and Cmax at Visit 3 and visit 4 in cross-over group 6) Arm difference of geometric mean steady-state Cmin at Visit 3 for all patients (after 1 month of treatment) 7)Effect of food on systemic exposure of deferasirox as assessed by Pharmacokinetics approach. Pooled PK data from all arms will be analysed with a population PK model and appropriate covariates will be examined for clinical and statistical relevance.
    E.5.2.1Timepoint(s) of evaluation of this end point
    4 weeks and 8 weeks after treatment day 1
    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 No
    E.6.5Efficacy No
    E.6.6Pharmacokinetic Yes
    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) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    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 Yes
    E.8.1.7.1Other trial design description
    Partial parallel and Partial Cross over
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    pre-prandial and post-prandial IMP administration
    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 concerned2
    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 trial will end 4 weeks after the last visit of the last patient enrolled in the study. This is because patients will be followed up at 28 days from the last visit which was also the day of the last dose of IMP administered on study.
    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 months0
    E.8.9.1In the Member State concerned days29
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days29
    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: 64
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 0
    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 state64
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 64
    F.4.2.2In the whole clinical trial 64
    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)
    Established deferasirox patients (cohort I) will continue treatment after the study as by the arrangements that were in place prior to study entry. Patients naïve to deferasirox will continue the treatment post-trial as part of standard management in transfusional haemosiderosis if they are willing to do so and tolerate the treatment well. There are no provisional arrangements on the part of the IMP provider (Novartis) to secure …..
    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 Decision2012-01-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-01-25
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2012-07-16
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