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    Summary
    EudraCT Number:2017-003777-34
    Sponsor's Protocol Code Number:NL63198.041.17
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-02-22
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2017-003777-34
    A.3Full title of the trial
    Proton pump inhibition for secondary hemochromatosis in hereditary anemia, a phase III placebo controlled randomized cross-over clinical trial.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    PPI in secondary hemochromatosis.
    A.3.2Name or abbreviated title of the trial where available
    PPI Shine Again
    A.4.1Sponsor's protocol code numberNL63198.041.17
    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 SponsorUMC Utrecht
    B.1.3.4CountryNetherlands
    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 supportZonMW
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUMC Utrecht
    B.5.2Functional name of contact pointVan Creveldkliniek
    B.5.3 Address:
    B.5.3.1Street AddressHeidelberglaan 100
    B.5.3.2Town/ cityUtrecht
    B.5.3.3Post code3584CX
    B.5.3.4CountryNetherlands
    B.5.4Telephone number0031088-7558450
    B.5.6E-mailvck-research@umcutrecht.nl
    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 Esomeprazole; manufacturer Sandoz RVG 107193-4.
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Consumer Healthcare Ltd
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 nameEsomeprazole
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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
    D.8.3Pharmaceutical form of the placeboCapsule
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Iron overload (secondary hemochromatosis) in non-transfusion dependent forms of hereditary anemias. Iron overload and hereditary anemia are a growing, underestimated emerging health care problem.
    E.1.1.1Medical condition in easily understood language
    Iron overload in patients with a form of hereditary anemia not requiring 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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10002272
    E.1.2Term Anemia
    E.1.2System Organ Class 100000004851
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10057875
    E.1.2Term Secondary haemochromatosis
    E.1.2System Organ Class 100000004861
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level HLT
    E.1.2Classification code 10022979
    E.1.2Term Iron excess
    E.1.2System Organ Class 100000004861
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10055610
    E.1.2Term Hemoglobinopathy
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10074356
    E.1.2Term Non-transfusion dependent thalassemia
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To show that PPIs compared to placebo are an effective treatment of secondary hemochromatosis in a relative large number of patients with hereditary anemia and mild iron overload.
    E.2.2Secondary objectives of the trial
    To assess the safety and side effects of treatment with esomeprazole.
    To assess quality of life during treatment with esomeprazole compared with placebo.
    To evaluate cost-effectiveness of esomeprazole in treatment of iron overload in hereditary anemia.
    To assess the changes in ‘iron markers’ during treatment with esomeprazole compared with placebo.
    To assess the need for chelation therapy after one year of treatment with esomeprazole compared with placebo.
    To assess the adherence to therapy in a real life setting.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    o Diagnosis of hereditary anemia: hemoglobinopathy (including all sickle cell syndromes and beta-thalassemia), sideroblastic anemia, congenital dyserythropoietic anemia or an erythrocyte enzyme deficiency.
    o Hemoglobin before study inclusion <7.0 mmol/L
    o Clinically stable and relevant iron overload defined as either one of:
    - a baseline LIC measurement by MRI between 3 and 15 mg Fe/g without having received iron chelation 2 months prior to entering the study.
    - OR a baseline LIC measurement by MRI between 3 and 15 mg Fe/g on stable chelation therapy (deferasirox, deferoxamine or deferiprone), with documented stable dosage the preceding 2 months and no expected dose reductions or increases the next two years.
    o Aged more than 18 years and able to sign informed consent.
    o Serum transferrin saturation higher than 0.40 once during the preceding 24 months.
    o Received less than 10 units of blood during the preceding 12 months.
    o Is expected to receive less than 4 units fo blood during the following 12 months
    o Is not splenectomized during the preceding 24 months.
    E.4Principal exclusion criteria
    o Pregnancy.
    o Liver cirrhosis.
    o Heart failure.
    o Severe cardiac iron overload defined as MRI T2* < 20 ms.
    o Severe liver iron overload defined as MRI LIC > 15 mg Fe/g dw.
    o Expected poor compliance.
    o Currently taking PPI and not able to stop for personal or medical reasons.
    o Patients that are being phlebotomized as treatment for iron overload.
    o Current peptic ulcer disease, gastro-intestinal bleeding or other causes of blood loss.
    o Contra-indication for esomeprazole use.
    o Concomitant use of clopidogrel.
    o Contra-indication for MRI.
    o Received more than 4 units blood during one of the treatment periods of 12 months.
    E.5 End points
    E.5.1Primary end point(s)
    The main endpoint of this study, is the change in LIC from baseline (start of treatment) to 12 months measured by MRI of the liver. This endpoint will be used in the primary analysis to compare treatment with esomeprazole to treatment with placebo. The LIC will be expressed in mg Fe/g dw after data analysis of the T2* and T1 images of the MRI.
    E.5.1.1Timepoint(s) of evaluation of this end point
    MRI 1: baseline. The maximum time interval between start of study medication and the baseline MRI will be 14 days.
    MRI 2: after the first treatment year (T=12 months). The maximum time interval between the cross-over point and the MRI will be 7 days.
    MRI 3: after the second treatment year (T=24 months). The maximum time interval between the end of study treatment and the MRI will be 7 days.
    E.5.2Secondary end point(s)
    1. Tolerability of esomeprazole: the incidence of side effect / adverse events will be monitored every 3 months during study visits. Measurement of vitamin B12, zinc and magnesium, T0, T12 and T24. Report of airway infections.
    2. Quality of life: this will be assessed with EQ5D-forms, with time intervals of 3 months.
    3. Cost-effectiveness analysis of esomeprazole in treatment of iron overload in hereditary anemia. This will be assessed by a prospective cost-effectiveness analysis. IMCQ and iPCQ questionnaires will be filled in with time intervals of 3 months.
    4. Related changes in markers of iron metabolism:
    a. Plasma hepcidin T0.
    b. Serum ferritin T0, T12, T24.
    5. Compliance to study drug
    a. Plasma gastrin T0, T6, T12, T18, T24.
    b. Counting of the capsules.
    6. Need for chelation therapy
    E.5.2.1Timepoint(s) of evaluation of this end point
    Timepoints outlined in E.5.5. T0 = start of stdy. T6 = 6 months after start first treatment period. T12 = 12 months after start first treatment period.
    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 Yes
    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) 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 No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    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) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    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 concerned3
    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
    Last visit of the last subject undergoing the trial. The last visit is planned after the second treatment period of 12 months.
    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 months
    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: 35
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 Information not present in EudraCT
    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 state40
    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)
    None. In case of proven efficacy of esomeprazole in iron overload, effort will be made to implement this drug in current treatment protocols.
    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 Decision2018-02-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-02-08
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2021-04-12
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