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    Summary
    EudraCT Number:2018-004016-22
    Sponsor's Protocol Code Number:LA61-0218
    National Competent Authority:Greece - EOF
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-12-05
    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 ConcernedGreece - EOF
    A.2EudraCT number2018-004016-22
    A.3Full title of the trial
    Safety and acceptability of deferiprone delayed release tablets in patients with systemic iron overload
    Ασφάλεια και επίπεδο αποδοχής των δισκίων δεφεριπρόνης καθυστερημένης αποδέσμευσης από ασθενείς με συστημική υπερφόρτωση σιδήρου
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study looking at the safety and tolerability of a new formulation of deferiprone inpatients with excess iron due to frequent blood transfusions
    Μελέτη αξιολόγησης της ασφάλειας και της αποδοχής της νέας μορφής δεφεριπρόνης σε ασθενείς με περίσσεια σιδήρου λόγω συχνών μεταγγίσεων αίματος.
    A.3.2Name or abbreviated title of the trial where available
    LA61-0218
    LA61-0218
    A.4.1Sponsor's protocol code numberLA61-0218
    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 SponsorApoPharma Inc.
    B.1.3.4CountryCanada
    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 supportApoPharma Inc.
    B.4.2CountryCanada
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationApoPharma Inc.
    B.5.2Functional name of contact pointJohn Connelly
    B.5.3 Address:
    B.5.3.1Street Address200 Barmac Drive
    B.5.3.2Town/ cityToronto, Ontario
    B.5.3.3Post codeM9L 2Z7
    B.5.3.4CountryCanada
    B.5.4Telephone number1416401-7296
    B.5.5Fax number14164013869
    B.5.6E-mailjconnell@apopharma.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 No
    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 nameDeferiprone delayed release 1000 mg tablets
    D.3.4Pharmaceutical form Modified-release 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 INNDEFERIPRONE
    D.3.9.1CAS number 30652-11-0
    D.3.9.2Current sponsor codeAPO-066
    D.3.9.4EV Substance CodeSUB06941MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000
    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
    Systemic iron overload
    Συστημική υπερφόρτωση σιδήρου
    E.1.1.1Medical condition in easily understood language
    excess iron in body
    περίσσεια σιδήρου σώματος
    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 PT
    E.1.2Classification code 10065973
    E.1.2Term Iron overload
    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 evaluate the safety of deferiprone delayed release (DR) tablets in two different dosage groups in patients with systemic iron overload.
    Η αξιολόγηση της ασφάλειας των δισκίων δεφεριπρόνης καθυστερημένης αποδέσμευσης (DR) σε δύο διαφορετικές ομάδες δοσολογίας σε ασθενείς με συστημική υπερφόρτωση σιδήρου.
    E.2.2Secondary objectives of the trial
    •To evaluate the tolerability of deferiprone DR tablets in two different dosage groups in patients with systemic iron overload
    •To evaluate the acceptability to patients of deferiprone DR
    •Η αξιολόγηση της ανεκτικότητας των δισκίων δεφεριπρόνης DR σε δύο διαφορετικές ομάδες δοσολογίας σε ασθενείς με συστημική υπερφόρτωση σιδήρου
    •Η αξιολόγηση του επιπέδου αποδοχής της δεφεριπρόνης DR από τους ασθενείς
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    •Male or female aged ≥ 18 years
    •Diagnosis of thalassemia syndrome, sickle cell disease, or other disorder requiring a regular regimen of red blood cell transfusions
    •On a stable regimen (≥3 months) of Ferriprox tablets for the treatment of systemic iron overload
    •A record of at least the last 12 measured ALT and AST levels prior to baseline
    •Άνδρας ή γυναίκα, ηλικίας ≥ 18 ετών
    •Διάγνωση συνδρόμου μεσογειακής αναιμίας, δρεπανοκυτταρικής νόσου ή άλλης διαταραχής που απαιτεί ένα κανονικό σχήμα μεταγγίσεων ερυθρών αιμοσφαιρίων
    •Ένα σταθερό σχήμα δοσολογίας (≥3 μήνες) με δισκία Ferriprox για τη θεραπεία της συστημικής υπερφόρτωσης σιδήρου
    •Καταχώριση τουλάχιστον των τελευταίων 12 μετρούμενων επιπέδων ALT και AST πριν από την επίσκεψη αναφοράς
    E.4Principal exclusion criteria
    •ALT and/or AST value > 5 x ULN at screening
    •Active case of hepatitis B or C at screening
    •Τιμή ALT ή/και AST > 5 x ULN στη διαλογή
    •Ενεργό περιστατικό ηπατίτιδας Β ή C στη διαλογή
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the incidence of any post-dose occurrence of increases in ALT and/or ALT levels that meet one of the criteria for being considered a safety concern. The safety criteria are:

    •For a patient whose baseline level is within the normal range, the criterion will be reaching a value of 5 times the ULN during the study.
    •For a patient whose baseline level is above the ULN, the criterion will be reaching a value of 5 times the baseline value during the study or >10 x ULN.
    Το πρωτεύον τελικό σημείο είναι η επίπτωση οποιασδήποτε εμφάνισης αυξήσεων μετά τη δόση στα επίπεδα ALT ή/και ALT οι οποίες ικανοποιούν ένα από τα κριτήρια, για να θεωρηθούν ζήτημα ασφάλειας. Τα κριτήρια ασφάλειας είναι τα εξής:
    •Για έναν ασθενή του οποίου το επίπεδο αναφοράς εμπίπτει στο φυσιολογικό εύρος, το κριτήριο θα είναι μια τιμή 5πλάσια του ULN κατά τη διάρκεια της μελέτης.
    •Για έναν ασθενή το επίπεδο αναφοράς του οποίου είναι άνω του ULN, το κριτήριο θα είναι μια τιμή 5πλάσια της τιμής αναφοράς κατά τη διάρκεια της μελέτης ή >10 x ULN.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Adverse events will be collected throughout the study; laboratory safety assessments will be performed at screening, baseline, and Days 3, 7, 14, 21, and 28
    Συλλογή ανεπιθύμητων ενεργειών κατά τη διάρκεια της μελέτης, πραγματοποίηση εργαστηριακών αναλύσεων ασφάλειας κατά τη διαλογή, βάση και 3η, 7η,14η,21η και 28η ημέρα
    E.5.2Secondary end point(s)
    Secondary endpoints are as follows:
    •The assessment of tolerability will be based on the incidence of gastrointestinal (GI) distress reported by patients during the study.

    •The assessment of acceptability will be based on the number of patients who indicate that they prefer the DR formulation over Ferriprox IR after considering their preference with respect to scheduling, ease of administration, and tolerability.
    Τα δευτερεύοντα τελικά σημεία είναι τα εξής:
    •Η αξιολόγηση της ανεκτικότητας θα βασίζεται στην επίπτωση γαστρεντερικής (GI) δυσφορίας που αναφέρεται από τους ασθενείς κατά τη διάρκεια της μελέτης.
    •Η αξιολόγηση του επιπέδου αποδοχής θα βασίζεται στον αριθμό ασθενών οι οποίοι υποδεικνύουν ότι προτιμούν το σκεύασμα DR σε σχέση με το Ferriprox IR αφού κατόπιν εξέτασης της προτίμησής τους όσον αφορά τον προγραμματισμό, την ευκολία χορήγησης και την ανεκτικότητα.
    E.5.2.1Timepoint(s) of evaluation of this end point
    •A questionnaire asking patients for their views on the acceptability of the delayed release formulation vs. that of Ferriprox will be administered on Day28.

    •Adverse events related to gastrointestinal tolerability will be collected throughout the study as per the timepoints.
    • Την 28η ημέρα οι ασθενείς θα ερωτηθούν μέσω ερωτηματολογίου για τις απόψεις τους σχετικά με την ανεκτικότητα της μορφής βραδείας αποδέσμευσης σε σχέση με το Ferriprox
    • Ανεπιθύμητες ενέργειες σχετιζόμενες με γαστρεντερολογική δυσανεξία θα συλλεχθούν κατά τη διάρκεια της μελέτης ως τα καθορισμένα χρονικά σημεία .
    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
    Acceptability
    Ανεκτικότητα
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA2
    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
    Canada
    United States
    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
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    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 years0
    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.1.1.1In 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: 30
    F.1.3Elderly (>=65 years) No
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 30
    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. Upon completion of the study, patients will return to their regular chelationregimen.
    Κανένα. Μετά το πέρας της μελέτης οι ασθενείς θα επιστρέψουν στην συστηματική τους αποσιδήρωση.
    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 Decision2018-12-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-12-14
    P. End of Trial
    P.End of Trial StatusCompleted
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