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    Summary
    EudraCT Number:2012-002566-12
    Sponsor's Protocol Code Number:DSC/11/2357/43
    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:2012-10-17
    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 number2012-002566-12
    A.3Full title of the trial
    A Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children with Duchenne Muscular Dystrophy
    Studio in due parti per valutare la sicurezza e la tollerabilita , la farmacocinetica, e gli effetti sulla istologia e i diversi parametri clinici di Givinostat in bambini deambulanti affetti da distrofia muscolare di Duchenne
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children with Duchenne Muscular Dystrophy
    Studio diviso in due parti per valutare la sicurezza e la tollerabilita’, la farmacocinetica, e gli effetti sull’istologia e i diversi paramentri clinici di Givinostat in bambini deambulanti affetti da distrofia muscolare di Duchenne
    A.3.2Name or abbreviated title of the trial where available
    DSC
    DSC
    A.4.1Sponsor's protocol code numberDSC/11/2357/43
    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 SponsorITALFARMACO
    B.1.3.4CountryItaly
    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 supportITALFARMACO
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationItalfarmaco
    B.5.2Functional name of contact pointClinical R&D Dept.
    B.5.3 Address:
    B.5.3.1Street AddressVia dei Lavoratori, 54
    B.5.3.2Town/ cityCinisello Balsamo (MI)
    B.5.3.3Post code20092
    B.5.3.4CountryItaly
    B.5.4Telephone number02 6443 2512
    B.5.5Fax number02 6443 3554
    B.5.6E-mailb.gatti@italfarmaco.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/12/1009
    D.3 Description of the IMP
    D.3.1Product nameGivinostat
    D.3.2Product code ITF2357
    D.3.4Pharmaceutical form Oral suspension
    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 INNGivinostat
    D.3.9.1CAS number 732302-99-7
    D.3.9.2Current sponsor codeITF2357
    D.3.9.3Other descriptive namegivinostat
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number100
    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 Yes
    D.3.11.13.1Other medicinal product typecomposto chimico
    D.IMP: 2
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/12/1009
    D.3 Description of the IMP
    D.3.1Product nameGivinostat
    D.3.2Product code ITF2357
    D.3.4Pharmaceutical form Capsule, hard
    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 INNGivinostat
    D.3.9.1CAS number 732302-99-7
    D.3.9.2Current sponsor codeITF2357
    D.3.9.3Other descriptive nameGivinostat
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number100
    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 Yes
    D.3.11.13.1Other medicinal product typechimico
    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
    Duchenne Muscular Dystrophy
    Distrofia Muscolare di Duchenne
    E.1.1.1Medical condition in easily understood language
    Ambulant Children with Duchenne Muscular Dystrophy
    Bambini deambulanti affetti da distrofia muscolare di Duchenne
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level SOC
    E.1.2Classification code 10028395
    E.1.2Term Musculoskeletal and connective tissue disorders
    E.1.2System Organ Class 10028395 - Musculoskeletal and connective tissue 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 establish the histologic effects of Givinostat administered chronically at the selected daily dose
    Stabilire gli effetti istologici di Givinostat somministrato in modo cronico alla dose giornaliera selezionata
    E.2.2Secondary objectives of the trial
    • To establish the effects of Givinostat administered chronically at the selected daily dose on functional parameters, such as the 6-Minute Walk Test (6MWT), North Star Ambulatory Assessment (NSAA), and performance of upper limb (PUL) • To establish the safety and tolerability of Givinostat administered chronically at the selected daily dose in children with Duchenne muscular dystrophy (DMD) • To explore the effects of Givinostat administered chronically at the selected daily dose on parameters such as magnetic resonance imaging (MRI), biomarkers, and cytokines
    • Stabilire gli effetti istologici di Givinostat somministrato in modo cronico alla dose giornaliera selezionata sui parametri funzionali, quali i tests: “6-minutes walk test” (6MWT), North Star Ambulatory Assessment (NSAA), e “performance of upper limb” (PUL) • Stabilire la sicurezza e la tollerabilita’ di Givinostat somministrato in modo cronico alla dose giornaliera selezionata in bambini affetti da distrofia muscolare di Duchenne (DMD) • Esplorare gli effetti di Givinostat somministrato in modo cronico alla dose giornaliera selezionata su parametri quali la risonanza magnetica (MRI), i biomarcatori, e le citochine
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male children aged 7 to <11 years with an immunohistochemical and molecular diagnosis of DMD. 3. Able to complete the 2 screening 6MWTs with a minimal distance of at least 250 m each. In addition, the results of these tests must be within ±30 m of each other. 4. On a stable dose of systemic corticosteroids for at least 6 months. 5. At least 6 months worth of data on the 6MWT (this will be the “historical” 6MWT). From the moment of the historical 6MWT assessment(s), the child must not have received any compound that could potentially affect the 6MWT, with the exception of the stable steroid treatment.
    1. Bambini maschi di età tra 7 e &lt;11 anni con una diagnosi di DMD immunoistochimica e molecolare. 2. Bambini in grado di completare i due test di 6MWTs allo screening, con una distanza minima di almeno 250 metri ciascuno. Inoltre, i risultati di questi tests non devono differire tra di loro di ±30 m. 4. Bambini a dosaggio stabile di corticosteroidi sistemici per almeno 6 mesi. 5. Almeno 6 mesi di valore dei dati sul 6MWT (questo sarà lo storico 6MWT). Dal momento dell’effettuazione dello storico 6MWT, il bambino non dovra’ aver assunto alcun composto che possa potenzialmente inficiare il test, ad eccezione del trattamento stabile con steroidi.
    E.4Principal exclusion criteria
    1. Initiation of systemic corticosteroid therapy within 6 months prior to the start of study drug or change in systemic corticosteroid therapy (e.g., initiation, change in type of drug, dose modification not related to body weight change, schedule modification, interruption, discontinuation, or re initiation) within 6 months prior to the start of study drug.
    2. Use of any pharmacologic treatment, other than corticosteroids, that might have an effect on muscle strength since the time of the historical 6MWT and in any case within 3 months prior to the start of study treatment (e.g., growth hormone). Vitamin D, calcium, and integrators will be allowed.
    3. Surgery that might have an effect on muscle strength or function within 3 months before study entry or planned surgery at any time during the study.
    1. Inizio di una terapia con corticosteroidi entro i 6 mesi precedenti l’assunzione del farmaco in studio o cambiamenti nella terapia con corticosteroidi sistemici (es. Inizio, modifica del tipo di farmaco, modifica del dosaggio non legata a cambiamenti di peso, modifica della posologia, interruzione, o ripresa) nei 6 mesi precedenti l’inizio del farmaco in studio. 2. Uso di qualsiasi agente farmacologico oltre i corticosteroidi, che possa avere un effetto sulla forza muscolare dal momento del valore storico del 6MWT, e in ogni caso nei 3 mesi precedenti l’inizio del farmaco in studio (es. Ormone della crescita). Sono permessi Vitamina D, calcio e integratori 3. Trattamenti chirurgici che possano avere un effetto sulla forza o sulla funzionalità muscolare, effettuati nei 3 mesi precedenti l’inizio dello studio, oppure qualsiasi intervento chirurgico, già pianificato, in qualunque momento durante lo studio.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of this study is change in the value of MFA% comparing the histology biopsies before and after 12 months of treatment with Givinostat.
    L’obiettivo primario dello studio è il cambiamento del valore di % MFA (area di fibra muscolare) confrontando le biopsie istologiche prima e dopo i 12 mesi di trattamento con Givinostat.
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 months
    12 mesi
    E.5.2Secondary end point(s)
    • Change in additional histological endpoints (i.e., cross-sectional area, inflammation, necrosis, fibrosis, and muscle regeneration) after 12 months of treatment with Givinostat at the selected daily dose • Change in muscular function after 12 months of treatment with Givinostat at the selected daily dose based on the 6MWT • Change in muscular function after 12 months of treatment with Givinostat at the selected daily dose based on the NSAA • Change in muscular function after 12 months of treatment with Givinostat at the selected daily dose based the PUL
    • Cambiamento negli obbiettivi istologici aggiuntivi (es. area cross- sectional, infiammazione, necrosi, fibrosi e rigenerazione muscolare) dopo 12 mesi di trattamento con Givinostat alla dose giornaliera selezionata. • Cambiamento nella funzionalità muscolare dopo 12 mesi di trattamento con Givinostat alla dose giornaliera selezionata sulla base del 6MWT • Cambiamento nella funzionalità muscolare dopo 12 mesi di trattamento con Givinostat alla dose giornaliera selezionata sulla base del NSAA • Cambiamento nella funzionalità muscolare dopo 12 mesi di trattamento con Givinostat alla dose giornaliera selezionata sulla base del PUL
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 months
    12 mesi
    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 Yes
    E.6.7Pharmacodynamic Yes
    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) 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 concerned5
    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 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
    All subjects, regardless of whether they complete the study or terminate early in Part 1 or Part 2 should return to the study center within 4 weeks of the last dose of study drug for the follow-up visit
    tutti i soggetti termineranno lo studio con una visita di follow up effettuata 4 settimana dopo l'ultima somministrazione di farmaco.
    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 months18
    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 Yes
    F.1.1Number of subjects for this age range: 20
    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: 20
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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 state20
    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)
    according to the standard clinical practice
    secondo la pratica clinica
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Parent Project
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2013-02-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-09-20
    P. End of Trial
    P.End of Trial StatusCompleted
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