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    Summary
    EudraCT Number:2012-001881-14
    Sponsor's Protocol Code Number:GIFT/1
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2013-01-11
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2012-001881-14
    A.3Full title of the trial
    A phase II clinical trial to evaluate the safety and efficacy of interferon gamma in elevating frataxin levels in Friedreich ataxia patients
    Studio clinico di fase 2 per valutare la sicurezza e l'efficacia dell'interferone gamma nell'aumentare l'espressione di frataxina in pazienti con atassia di Friedreich
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase II clinical trial to evaluate the safety and efficacy of interferon gamma in elevating frataxin levels in Friedreich ataxia patients
    Studio clinico di fase 2 per valutare la sicurezza e l'efficacia dell'interferone gamma nell'aumentare l'espressione di frataxina in pazienti con atassia di Friedreich
    A.3.2Name or abbreviated title of the trial where available
    GIFT/1
    GIFT/1
    A.4.1Sponsor's protocol code numberGIFT/1
    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 SponsorAZIENDA UNIVERSITARIA POLICLINICO UMBERTO I DI ROMA
    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 supportATAXIA UK
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBarbara Tomassini
    B.5.2Functional name of contact pointServizio Info Sperimentazione
    B.5.3 Address:
    B.5.3.1Street AddressVia Padre Filippini 129
    B.5.3.2Town/ cityRoma
    B.5.3.3Post code00144
    B.5.3.4CountryItaly
    B.5.4Telephone number+39-3387504282
    B.5.5Fax number06-5290046
    B.5.6E-mailb.tomassini@alice.it
    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*SC 6F 0,5ML 100MCG
    D.2.1.1.2Name of the Marketing Authorisation holderBOEHRINGER INGELHEIM IT.SpA
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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/3/11/935
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Concentrate for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNINTERFERON GAMMA-1B
    D.3.9.1CAS number 98059-61-1
    D.3.9.4EV Substance CodeSUB12076MIG
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 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 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
    Friedreich's Ataxia
    Atassia di Friedreich
    E.1.1.1Medical condition in easily understood language
    Friedreich's Ataxia
    Atassia di Friedreich
    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 14.1
    E.1.2Level SOC
    E.1.2Classification code 10029205
    E.1.2Term Nervous system disorders
    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
    The primary objective of this study is to investigate whether the treatment with IFN gamma can induce significant accumulation of frataxin in FRDA patients, a possibility suggested by pre-clinical evidence in an animal model of the disease.
    L’obiettivo primario di questo studio è di stabilire se un trattamento con IFN gamma possa indurre un accumulo significativo di frataxina in pazienti FRDA, possibilità suggerita dalle evidenze pre-cliniche in un modello animale della malattia.
    E.2.2Secondary objectives of the trial
    Secondary objectives include the assessment of the safety and tolerability of IFN gamma in FRDA patients during the treatment period.
    Gli obiettivi secondari comprendono la valutazione della sicurezza e tollerabilità dell’IFN gamma in pazienti FRDA durante il periodo di trattamento.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Genetically confirmed diagnosis of Friederich’s Ataxia 2. Evidence of a personally signed and dated informed consent document indicating that the subject (or a legally acceptable representative) has been informed of all pertinent aspects of the study. 3. Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures. 4. Male and/or female subjects >/=18 .
    1. Diagnosi di atassia di Friedreich geneticamente confermata. 2. Evidenza di un documento di consenso informato personalmente firmato e datato che indichi che il soggetto (o un rappresentante legale accettabile) è stato informato su tutti gli aspetti pertinenti dello studio. 3. Soggetti che siano consenzienti e capaci di completare le visite programmate, il piano di trattamento, i test di laboratorio e le altre procedure. 4. Soggetti di sesso maschile o femminile di età &gt;/=18.
    E.4Principal exclusion criteria
    Subjects presenting with any of the following will not be included in the study: Medical History 1. Pregnant or breastfeeding women. 2. Significant concurrent medical conditions at the time of screening or baseline visit, including, but not limited to, the following: • Any major illness/condition or evidence of an unstable clinical condition (eg, renal, hepatic, hematologic, GI, endocrine, pulmonary, immunologic, or local active infection/infectious illness) that, in the investigator’s judgment, will substantially increase the risk to the subject if he or she participates in the study. • Class III or IV congestive heart failure as defined by the New York Heart Association. • Acute coronary syndrome (eg, myocardial infarction, unstable angina pectoris) and any history of significant cerebrovascular disease within 24 weeks before screening. 3. Presence of a transplanted organ. 4. Previous assumption of IFN gamma 1b.
    1. Donne in stato di gravidanza o allattamento al seno. 2. Condizioni mediche concomitanti al momento dello screening o della visita basale, comprese, ma non solo: • Qualsiasi condizione maggiore o malattia o evidenza di una condizione clinica instabile (p.es renale, epatica, ematologica, gastrointestinale, endocrina, polmonare, immunologica, o malattia infettiva/infezione locale che, a giudizio dell’investigatore, possa sostanzialmente aumentare il rischio del soggetto alla partecipazione allo studio). • Insufficienza cardiaca congestizia di Classe III o IV secondo la New York Heart Association. • Sindrome coronarica acuta (p.es, infarto miocardico, angina pectoris instabile) o storia di malattia cerebrovascolare significativa entro 24 settimane dallo screening. 3. Presenza di organi trapiantati. 4. Precedente assunzione di IFN gamma 1b.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the increase of cellular frataxin after treatment with IFN gamma.
    L’endpoint primario è l’aumento della frataxina cellulare dopo trattamento con IFN gamma.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Blood sample for the quantitation of frataxin will include in total 9 blood samples. Blood samples will be taken at the beginning of the treatment, 0 timepoint, and at days 1-7-14-15-21-28-29-35.
    I pazienti saranno soggetti ad un totale di 9 prelievi di sangue. Tali analisi verranno eseguite all’inizio del trattamento, tempo 0, e nei giorni 1-7-14-15-21-28-29-35
    E.5.2Secondary end point(s)
    Secondary endpoints are the safety and tolerability of IFN gamma in FRDA patients. The on treatment adverse events and withdrawals due to adverse effects will be reported. Any subject who receives at least 1 dose of investigational product will be included in the evaluation for safety.
    Gli obiettivi secondari sono la sicurezza e tollerabilità di IFN gamma in pazienti FRDA. Saranno riportati gli eventi avversi durante il trattamento e i ritiri dovuti a effetti avversi. Ogni individuo che riceva almeno 1 dose del prodotto studiato sarà incluso nella valutazione della sicurezza.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Patients will be evaluated at the inclusion and after 14, 28 and 58 days.
    I pazienti verranno valutati all'inclusione e dopo 14, 28 e 58 giorni.
    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 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 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 Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial1
    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 study protocol will end when at least 10 patients or all the enrolled patients have ended the clinical protocol.
    Lo studio si può considerare concluso quando almeno 10 pazienti o tutti i pazienti arruolati hanno concluso lo studio.
    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 months10
    E.8.9.1In the Member State concerned days0
    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.1.1.2Preterm 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: 15
    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 Yes
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women Yes
    F.3.3.4Nursing women Yes
    F.3.3.5Emergency situation Yes
    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 state15
    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 will continue their rehabilitative standard of care treatment.
    I pazienti proseguiranno il trattamento standard riabilitativo.
    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 Decision2013-03-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-02-28
    P. End of Trial
    P.End of Trial StatusOngoing
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