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    Summary
    EudraCT Number:2014-001427-79
    Sponsor's Protocol Code Number:TIRCON2012V1-EXT
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-06-04
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2014-001427-79
    A.3Full title of the trial
    Long-term Safety and Efficacy Study of Deferiprone in Patients with Pantothenate Kinase-Associated Neurodegeneration (PKAN)
    Long-term Safety and Efficacy Study of Deferiprone in Patients with Pantothenate Kinase-Associated Neurodegeneration (PKAN)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An 18-month extension of an international trial of deferiprone in patients with iron storage brain disorders
    An 18-month extension of an international trial of deferiprone in patients with iron storage brain disorders
    A.3.2Name or abbreviated title of the trial where available
    Long-term safety and efficacy study of deferiprone in patients with pantothenate kinase-associated n
    Studio a lungo termine sulla sicurezza ed efficacia di deferiprone in pazienti con neurodegenerazion
    A.4.1Sponsor's protocol code numberTIRCON2012V1-EXT
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02174848
    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 SponsorAPOTEX EUROPE B.V.
    B.1.3.4CountryNetherlands
    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
    B.5.3.3Post codeM9L 2Z7
    B.5.3.4CountryCanada
    B.5.4Telephone number14164017296
    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 80 mg/mL oral solution
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Yes
    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.3.9.3Other descriptive nameDEFERIPRONE
    D.3.9.4EV Substance CodeSUB06941MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number80
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 Yes
    D.3.11.13.1Other medicinal product typeIron chelating Agent
    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
    Pantothenate Kinase-Associated Neurodegeneration (PKAN)
    Pazienti con neurodegenerazione associata a pantotenato chinasi (PKAN)
    E.1.1.1Medical condition in easily understood language
    Pantothenate Kinase-Associated Neurodegeneration (PKAN). Patients with PKAN have high levels of iron in certain brain regions, and this extra iron acts to damage brain cells and may be the cause of th
    Pazienti con neurodegenerazione associata a pantotenato chinasi (PKAN). I pazienti con PKAN presentano elevati livelli di ferro in determinate regioni cerebrali, e questo accumulo di ferro danneggia l
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10053643
    E.1.2Term Neurodegenerative disorder
    E.1.2System Organ Class 10029205 - Nervous system 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 long-term safety and tolerability of deferiprone in patients with PKAN.
    Valutare la sicurezza e la tollerabilità a lungo termine di deferiprone in pazienti con PKAN.
    E.2.2Secondary objectives of the trial
    • To evaluate the change in severity of dystonia over time in patients with PKAN treated with deferiprone.
    • To evaluate global improvement over time in patients with PKAN treated with deferiprone.
    • Valutare la variazione nella gravità della distonia nel tempo in pazienti con PKAN trattati con deferiprone.
    • Valutare il miglioramento globale nel tempo in in pazienti con PKAN trattati con deferiprone.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Completed study TIRCON2012V1

    2. Sexually active females of childbearing potential, including those who are peri-menopausal (defined as less than 2 years since last menstrual period) must have a negative pregnancy test result at Visit 1 (if applicable; in cases where the investigator determines there is no reasonable risk of pregnancy because of significant incapacity, pregnancy testing will not be performed). In addition, if applicable, they must meet at least one of the following criteria: Use an effective method of contraception during the study and within 30 days following their last dose of study medication, OR; Participate in a non-heterosexual lifestyle, OR; Have a male sexual partner who has been sterilized (supporting evidence required).

    Approved methods of contraception will consist of the following or must follow local requirements: Oral contraceptive used in conjunction with condom, diaphragm, or spermicide; Hormonal implant used in conjunction with condom, diaphragm, or spermicide; Injectable contraceptive used in conjunction with condom, diaphragm, or spermicide; Diaphragm or condom used with spermicide.

    If a hormonal contraception is used, it should have a Pearl index <1%.

    Female patients who meet any of the following criteria are not of childbearing potential and therefore do not need to practice contraception: Post-menopausal (last menstrual period > 2 years ago); Had a tubal ligation (supporting evidence required); Had a hysterectomy or oophorectomy (supporting evidence required).

    3. Fertile heterosexual males and/or their partners must agree to use an effective method of contraception during the study and for 30 days following the last dose of study medication.

    4. Patients and/or their authorized legal representatives must provide signed and dated written informed consent prior to the first study intervention, and patients must be able to adhere to study restrictions, appointments, and evaluation schedules. Patients who are minors must sign an assent form as per local regulatory requirements.
    1. Completed study TIRCON2012V1

    2. Sexually active females of childbearing potential, including those who are peri-menopausal (defined as less than 2 years since last menstrual period) must have a negative pregnancy test result at Visit 1 (if applicable; in cases where the investigator determines there is no reasonable risk of pregnancy because of significant incapacity, pregnancy testing will not be performed). In addition, if applicable, they must meet at least one of the following criteria: Use an effective method of contraception during the study and within 30 days following their last dose of study medication, OR; Participate in a non-heterosexual lifestyle, OR; Have a male sexual partner who has been sterilized (supporting evidence required).

    Approved methods of contraception will consist of the following or must follow local requirements: Oral contraceptive used in conjunction with condom, diaphragm, or spermicide; Hormonal implant used in conjunction with condom, diaphragm, or spermicide; Injectable contraceptive used in conjunction with condom, diaphragm, or spermicide; Diaphragm or condom used with spermicide.

    If a hormonal contraception is used, it should have a Pearl index <1%.

    Female patients who meet any of the following criteria are not of childbearing potential and therefore do not need to practice contraception: Post-menopausal (last menstrual period > 2 years ago); Had a tubal ligation (supporting evidence required); Had a hysterectomy or oophorectomy (supporting evidence required).

    3. Fertile heterosexual males and/or their partners must agree to use an effective method of contraception during the study and for 30 days following the last dose of study medication.

    4. Patients and/or their authorized legal representatives must provide signed and dated written informed consent prior to the first study intervention, and patients must be able to adhere to study restrictions, appointments, and evaluation schedules. Patients who are minors must sign an assent form as per local regulatory requirements.
    E.4Principal exclusion criteria
    1. Withdrew from the study TIRCON2012V1 for reasons of safety.

    2. Plan to participate in another clinical trial at any time from the day of enrolment until 30 days post-treatment in the current study.

    3. Presence of any medical, psychological, or psychiatric condition which in the opinion of the investigator would cause participation in the study to be unwise.

    4. Pregnant, breastfeeding, or planning to become pregnant during the study period.
    1. Withdrew from the study TIRCON2012V1 for reasons of safety.

    2. Plan to participate in another clinical trial at any time from the day of enrolment until 30 days post-treatment in the current study.

    3. Presence of any medical, psychological, or psychiatric condition which in the opinion of the investigator would cause participation in the study to be unwise.

    4. Pregnant, breastfeeding, or planning to become pregnant during the study period.
    E.5 End points
    E.5.1Primary end point(s)
    • Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product
    • Serious adverse events (SAEs): Frequency, severity, time to onset, duration, and relatedness to study product
    • Number of discontinuations due to AEs
    • Eventi avversi (AE): Frequenza, gravità, tempo all'insorgenza, durata e correlazione al prodotto in studio
    • Eventi avversi seri (SAE): Frequenza, gravità, tempo all'insorgenza, durata e correlazione al prodotto in studio
    • Numero di sospensioni a causa degli AE
    E.5.1.1Timepoint(s) of evaluation of this end point
    18 months
    18 mesi
    E.5.2Secondary end point(s)
    • Change in the Barry-Albright Dystonia Scale (BAD) total score from baseline (defined as prior to the start of deferiprone therapy) to Visit 4, as assessed by central evaluation of videotapes; Proportion of patients with improved or unchanged BAD scale total score between baseline and Visit 4 (responder analysis); • Change from baseline to Visit 4 in BAD scale score per body region (eyes, mouth, neck, trunk, and each upper and lower extremity), as assessed by central evaluation of videotapes; Change in score on the Patient Global Impression of Improvement (PGI-I) from Visit 1 to Visit 4; Proportion of patients showing an improvement on PGI-I at Visit 4 (responder analysis)
    • Variazione nel punteggio totale della scala Barry-Albright Dystonia Scale (BAD) dal basale (definito come valore anteriore all'avvio della terapia con deferiprone) alla Visita 4, come valutata mediante valutazione centrale di videocassette; Percentuale di pazienti con punteggio totale della scala BAD migliorato o invariato tra il basale e la Visita 4 (analisi dei responder); • Variazione dal basale alla Visita 4 nel punteggio della scala BAD per regione del corpo (occhi, bocca, collo, tronco e ciascun arto superiore e inferiore), come valutata mediante valutazione centrale di videocassette; Variazione nel punteggio della scala Patient Global Impression of Improvement (PGI-I) dalla Visita 1 alla Visita 4; Percentuale di pazienti che mostrano un miglioramento del punteggio della scala PGI-I alla Visita 4 (analisi dei responder)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Visits 1, 2, 3, 4; Visits 1, 2, 3, 4; Visits 1, 2, 3, 4; Visits 1, 2, 3, 4; Visit 4
    Visita 1, 2, 3, 4; Visita 1, 2, 3, 4; Visita 1, 2, 3, 4; Visita 1, 2, 3, 4; Visita 4
    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 Yes
    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) Yes
    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) 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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United States
    Germany
    Italy
    United Kingdom
    E.8.7Trial has a data monitoring committee Yes
    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 last patient last visit is estimated for April 2018. This will be followed by analysis and interpretation of the results.
    The last patient last visit is estimated for April 2018. This will be followed by analysis and interpretation of the results.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months4
    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 months10
    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 Yes
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 21
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 16
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 52
    F.1.3Elderly (>=65 years) No
    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 Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children > 4 years
    Adults with legal representative
    Children > 4 years
    Adults with legal representative
    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 51
    F.4.2.2In the whole clinical trial 89
    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)
    If the preliminary evidence indicates that deferiprone is safe and effective for this indication, the drug will be made available as compassionate use until we get approval for the drug. The investigators will have to request for the compassionate use. If the approval is not granted, the investigators will cancel the compassionate use.
    If the preliminary evidence indicates that deferiprone is safe and effective for this indication, the drug will be made available as compassionate use until we get approval for the drug. The investigators will have to request for the compassionate use. If the approval is not granted, the investigators will cancel the compassionate use.
    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 Decision2015-07-31
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
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