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    Summary
    EudraCT Number:2015-002432-40
    Sponsor's Protocol Code Number:042/15
    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:2021-05-27
    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 number2015-002432-40
    A.3Full title of the trial
    Safety and Efficacy of yIFN treatment in Friedreich ataxia
    Sicurezza ed efficacia del trattamento con Interferone gamma (¿IFN) nell'Atassia di Friedreich
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and Efficacy of yIFN treatment in Friedreich ataxia
    Sicurezza ed efficacia del trattamento con Interferone gamma (¿IFN) nell'Atassia di Friedreich
    A.3.2Name or abbreviated title of the trial where available
    ¿IFN in Friedreich Ataxia
    ¿IFN nella Atassia di Friedreich
    A.4.1Sponsor's protocol code number042/15
    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 SponsorASSOCIAZIONE "LA NOSTRA FAMIGLIA" - SEZIONE SCIENTIFICA I.R.C.C.S. "E.MEDEA"
    B.1.3.4CountryItaly
    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 supportAssociazione di pazienti
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationLa Nostra Famiglia - IRCCS E.Medea
    B.5.2Functional name of contact pointNucleo operativo ricerca ¿IFN
    B.5.3 Address:
    B.5.3.1Street AddressVia Don Luigi Monza 20
    B.5.3.2Town/ cityBosisio Parini (LC)
    B.5.3.3Post code23842
    B.5.3.4CountryItaly
    B.5.4Telephone number031877111
    B.5.5Fax number031877499
    B.5.6E-mailatassia.ifng.medea@gmail.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 Yes
    D.2.1.1.1Trade name IMUKIN - 6 FLACONI 0.5 ML 100 MCG
    D.2.1.1.2Name of the Marketing Authorisation holderBOEHRINGER INGELHEIM ITALIA S.P.A.
    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 nameImukin
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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
    FRDA is a progressive neurodegenerative disease inherited as recessive trait. It manifests itself usually in adolescence and affects various systems including the central and peripheral nervous system (balance, coordination, speech and sensitivity), the heart with hypertrophic cardiomyopathy, the bones with skeletal deformities (scoliosis, claw foot) and in some cases diabetes mellitus. The disease causes severe and progressive disability, significantly reducing the life expectancy of patients.
    FRDA ¿ una malattia neurodegenerativa a carattere progressivo trasmessa in forma autosomica recessiva. Esordisce in particolare in et¿ adolescenziale e colpisce vari sistemi tra cui il nervoso centrale e periferico (disturbi dell¿equilibrio, coordinazione, disturbi del linguaggio e della sensibilit¿), il cuore con cardiomiopatia ipertrofica, le ossa con deformit¿ scheletriche (scoliosi, piede cavo) ed in alcuni casi diabete mellito. La malattia causa disabilit¿ severa e progressiva, riduce in mo
    E.1.1.1Medical condition in easily understood language
    FRDA is a progressive neurodegenerative disease inherited as recessive trait. The disease manifests itself usually in adolescence
    FRDA ¿ una malattia neurodegenerativa a carattere progressivo che viene trasmessa in forma autosomica recessiva. La malattia esordisce in particolare in et¿ adolescenziale.
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10017374
    E.1.2Term Friedreich's ataxia
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10017374
    E.1.2Term Friedreich's ataxia
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic 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 test the safety of treatment with ¿IFN in FRDA patients, administered for 6 months at the initial dose of 100 ucg three times per week (first 2 weeks of treatment) and at the final dose of 200 ucg three times per week (for the remaining 22 weeks of treatment).
    Testare la sicurezza del trattamento con ¿IFN in pazienti con FRDA, somministrato per 6 mesi alla dose iniziale di 100 ucg per tre volte alla settimana (prime 2 settimane di trattamento) e alla dose finale di 200 ucg per tre volte alla settimana (per le rimanenti 22 settimane di trattamento).
    E.2.2Secondary objectives of the trial
    Test the efficacy of the treatment with ¿IFN in patients with FRDA by means of fMRI changes detected by a ¿finger tapping¿ motor task.
    Test the effect of the treatment by means of a number of secondary MRI indexes, laboratory and clinical end-points: DTI changes, brain iron content as measured by SWI, ventricular wall thickness as measured by 'Echocardiogram (ECHOCG), visual contrast tests, functional performance as measured by: Friedreich Ataxia Rating Scale (FARS), Scale for the Assessment and Rating of Ataxia (SARA), 9-Hole Peg test (9-HPT), 6-minute walk test (if applicable), levels of frataxin in cell lysates prepared from peripheral blood mononuclear cells (PBMC), changes in the quality of life and the impact of disability measures.
    Testare l'effetto del trattamento con ¿IFN sui cambiamenti riscontrabili dalla fMRI del disturbo specifico in pazienti con FRDA che svolgono il protocollo con il ¿finger tapping¿ task.
    Testare l'effetto di questo trattamento su una serie di indici secondari di MRI, end-point di laboratorio e clinici: cambiamenti DTI, contenuto cerebrale di ferro come misurato da SWI, spessore della parete ventricolare come misurato dall¿ Ecocardiogramma (EcoCG), test visivo con contrasto, le prestazioni funzionali come misurato da: Friedreich Ataxia Rating Scale (FARS), Scale for the Assessment and Rating of Ataxia (SARA), 9-Hole Peg test di (9-HPT), test del cammino di 6 minuti (se applicabile), livelli di fratassina in lisati cellulari preparati da cellule mononucleate del sangue periferico (PBMC), cambiamenti nella qualit¿ della vita e misura dell'impatto della disabilit¿.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Defined molecular diagnosis of Friedreich's ataxia

    Aged between 10 and 40 years

    Having completed the clinical-radiological study protocol within 12 months before the start of the study.

    Willingness to participate in the study.

    For women of childbearing age, availability to the use of safe contraceptive method for the entire duration of the study and for the next 60 days after the follow-up.
    Diagnosi molecolare definita di Atassia di Friedreich.

    Età tra i 10 e i 40 anni.

    Avere completato il protocollo di studio clinico-radiologico entro i 12 mesi prima dell'inizio del presente studio.

    Disponibilità a partecipare allo studio.

    Per le donne in età fertile, disponibilità all’uso di metodo contracettivo sicuro per tutta la durata dello studio e per i successivi 60 gg al follow up.
    E.4Principal exclusion criteria
    Presence of contraindications to perform the magnetic resonance.

    Clinical conditions unstable to the kidney, liver, heart, bone marrow suppression, blood dyscrasias or other medical condition unstable.

    Known allergy to latex.

    Known hypersensitivity to IFN gamma.

    Previous exposure to eritropoietina recombinant in the last 3 years.

    Simultaneus use of other drugs used to modify the disease in question (eg idebenone, deferiprone, Vit B ...)

    Women of childbearing age who are planning a pregnancy, or existing pregnancy or lactation in place.
    Presenza di controindicazioni ad eseguire la risonanza magnetica

    Condizioni cliniche instabili a carico del rene, fegato, cuore, mielosopressione, discrasia ematica o altre condizioni mediche instabili.

    Allergia conosciuta al lattice.

    Riconosciuta ipersensibilità all’IFN gamma.

    Precedente esposizione all’eritropoietina ricombinante negli ultimi 3 anni.

    Concomitante uso di altri farmaci usati per modificare la malattia in oggetto (e.s. idebenone, deferiprone, Vit B…)

    Donne in età fertile che hanno in programma una gravidanza, o gravidanza in atto oppure allattamento in atto.
    E.5 End points
    E.5.1Primary end point(s)
    Valutare la sicurezza del farmaco alla dose iniziale di 100 ucg per tre volte alla settimana (prime 2 settimane di trattamento) e di 200 ucg per tre volte alla settimana (per le rimanenti 22 settimane di trattamento) nei pazienti affetti da FRDA, tramite l'analisi del numero e della tipologia degli eventi avversi registrati.
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 mesi per paziente.
    E.5.2Secondary end point(s)
    Valutare l¿efficacia del trattamento del farmaco nei pazienti affetti da FRDA tramite la valutazione delle seguenti modifiche: ¿ di neuroimaging a carico del segnale BOLD ottenuto durante un compito motorio selettivo (finger tapping), dei parametri di DTI (FA, MD) nella sostanza bianca cerebellare, nei tratti lunghi e commisurali ed il contenuto di ferro cerebrale (SWI) confrontando i dati ottenuti alla baseline dello studio (T0), alla fine del trattamento (T6) e trascorso il periodo di wash-out (T+6) pesati con i dati ottenuti almeno 6 mesi prima dell¿inizio del trattamento; ¿ dello spessore della parete ventricolare (Eco CG) misurate a T0, T3, T6 e T+6; ¿ del test della vista con contrasto misurate a T0, T3, T6 e T+6; ¿ del contenuto di fratassina nelle PBMC misurate a T0, T3, T6 e T+6; ¿ del punteggio delle scale cliniche/funzionali: SARA, FARS, 9 HPT, misure di deambulazione misurate a T0, T3, T6 e T+6; ¿ dei cambiamenti della Qualit¿ di vita (QoL) e misurati tramite: Short Form 36 (SF-36) e WHO-DAS 2.0. misurate a T0, T3, T6 e T+6.
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 mesi per paziente
    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 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.8.2.4Number of treatment arms in the trial1
    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 No
    E.8.5.1Number of sites anticipated in the EEA1
    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 Information not present in EudraCT
    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 study will be completed two months after the last visit of the last person involved in the trial (months 19 and 20) with the analysis and interpretation of data and preparation of reports.
    Lo studio verr¿ completato 2 mesi dopo l¿ultima visita dell¿ultimo soggetto coinvolto nella sperimentazione (mesi 19 e 20) con l¿analisi e l'interpretazione dei dati e la preparazione del report.
    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 months20
    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 months20
    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: 1
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 5
    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 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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    F.4.2.2In the whole clinical trial 12
    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)
    Patients in the study will be followed with regular clinical follow-up after the conclusion of the study. In the case of positive results on the secondary end-points it is likely that a larger randomized controlled trial (RCT), double-blind will be proposed for funding. To persons who have participated in this study we will be offered participation in the study RCT.
    I pazienti partecipanti allo studio verranno seguiti con regolari follow-up clinici successivamente alla conclusione dello studio. In caso di risultati positivi sugli end-points secondari ¿ verosimile che una pi¿ ampia sperimentazione controllata randomizzata (RCT) e in doppio cieco verr¿ proposta per finanziamento. Ai soggetti che hanno partecipato a questo studio verr¿ offerta la partecipazione allo studio RCT.
    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 Decision2016-03-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-12-15
    P. End of Trial
    P.End of Trial StatusCompleted
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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