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    Summary
    EudraCT Number:2016-000601-36
    Sponsor's Protocol Code Number:GUP15004
    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:2021-06-08
    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 number2016-000601-36
    A.3Full title of the trial
    CLINICAL EFFICACY OF NIV AND MODAFINIL ON EXCESSIVE DAYTIME SLEEPINESS: A
    MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED CLINICAL TRIAL IN DM1
    EFFICACIA DELLA VENTILAZIONE NON INVASIVA E DEL MODAFINIL SULLA ECCESSIVA SONNOLENZA DIURNA: SPERIMENTAZIONE MULTICENTRICA, RANDOMIZZATA, A DOPPIO CIECO, PLACEBO-CONTROLLATA NELLA DISTROFIA MIOTONICA DI STEINERT (DM1)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical trial on the efficacy of non invasive ventilation and modafinil on excessive daytime sleepiness in Myotonic Dystrophy type 1
    Sperimentazione clinica farmacologica sull' efficacia della ventilazione notturna e del farmaco modafinil sulla eccessiva sonnolenza diurna nella distrofia miotonica di Steinert.
    A.3.2Name or abbreviated title of the trial where available
    ModaNIV
    ModaNIV
    A.4.1Sponsor's protocol code numberGUP15004
    A.5.4Other Identifiers
    Name:Progetto Telethon-UILDMNumber:GUP15004
    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 SponsorFONDAZIONE SERENA ONLUS
    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 supportTelethon
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFondazione Serena ONLUS
    B.5.2Functional name of contact pointAmministrazione Sezione Progetti
    B.5.3 Address:
    B.5.3.1Street AddressPiazza Ospedale Maggiore 3
    B.5.3.2Town/ cityMilano
    B.5.3.3Post code20162
    B.5.3.4CountryItaly
    B.5.4Telephone number0291433753
    B.5.5Fax number914337200
    B.5.6E-mailsilvia.pugliatti@centrocliniconemo.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 PROVIGIL - 100 MG COMPRESSE 30 COMPRESSE
    D.2.1.1.2Name of the Marketing Authorisation holderTEVA ITALIA S.R.L.
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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 No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Myotonic Dystrophy type 1
    Distrofia Miotonica di tipo 1
    E.1.1.1Medical condition in easily understood language
    Muscular Dystrophy
    Distrofia Muscolare
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10068871
    E.1.2Term Myotonic dystrophy
    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
    Change from baseline of EDS as assessed by mean Maintenance of Wakefulness Test (MWT)
    L¿obiettivo primario ¿ valutare l¿efficacia clinica della ventilazione non invasiva e del modafinil sulla eccessiva sonnolenza diurna, quantificando tale riduzione con il test di Maintenance of Wakefulness Test.
    E.2.2Secondary objectives of the trial
    Change from baseline of EDS as assessed by subjective sleepiness scales (Epworth Sleepiness
    Scale, ESS and Fatigue and Daytime Sleepiness Scale, FDSS)
    l Change from baseline of EDS as assessed by 1-week actigraphy
    l Change from baseline of fatigue as assessed by the Fatigue Severity Scale (FSS)
    l Change from baseline of respiratory function (assessed by ABG and PG) at 3 and at 9 months from
    treatment
    l Change from baseline of distance walked with 6MWT, of TUG and of 9-peg hole test
    l Change from baseline of cognitive tests (TEA, TMT, Stroop, Wisconsin)
    l Change from baseline of POMS and depression and anxiety scores
    l Change from baseline of SF-36 and INQoLv2 scores
    l Improved compliance (assessed by % days used and mean daily usage as indicated by the
    ventilators¿ microchip)
    Gli obiettivi secondari saranno quelli di valutare la soggettiva riduzione della EDS, il mantenimento della funzione respiratoria, il miglioramento delle performances cognitive, motorie, della percezione di fatica, della qualit¿ di vita e della aderenza alla NIV.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Molecular diagnosis of DM1, any range of CTG expansion size (= 50)
    Adult-onset DM1
    Age range 18-75
    Patients who have never tried NIV or patients who have not been using NIV for at least 3 months
    prior to enrollment
    Epworth score = 10
    Fatigue and Sleepiness (FDSS): positive answers in items 8-12 (37)
    Raw MMSE = 20
    Patients having EDS according to MSLT = 8 minutes and ESS = 10 or positive scores for EDS on
    the FDSS scale
    Diagnosi molecolare di DM1
    DM1 ad esordio adulto
    età compresa tra 18-75
    Paziente non in NIV
    Epworth score = 10
    Fatigue and Sleepiness (FDSS): risposte affermative ai punti 8-12
    MMSE = 20
    EDS con MSLT = 8 minutes e ESS = 10
    E.4Principal exclusion criteria
    (i) Hypersensibility to modafinil
    (ii) Pregnancy or women who are in child bearing age who do not adhere to the guidelines applied during clinical trials “Recomandations related to contraception and pregnancy testing in clinical trials".
    (iii) Patients who receive drugs that may interfere with modafinil (tricyclic antidepressants like
    clomipramine, desimipramine, pregabalin, carbamazepine, valproate, lithium, hydantoins, warfarin, propranol, MAO inhibitors, erythromycin, ketoconazole, phenobarbital, rifampin, diazepam,
    hydantoins)
    (iv) Cognitive impairment as assessed by interviews or MMSE = 20
    (v) Patients with uncontrolled hypertension will be excluded
    (vi) Patients with cardiac arrhythmias (tachycardia, atrial fibrillation, atrial flutter, bradycardia meaning <60 bpm), ventricular fibrillation) will be excluded
    (vii) History of seizure
    (i) Ipersensibilità al modafinil
    (ii) Donne gravide o in età fertile che non seguono metodi contraccettivi considerati efficaci e il timing previsto per i test di gravidanza allo screening e nel corso dello studio, in linea con quanto previsto dal CTFG nelle linee guida europee (Recomandations related to contraception and pregnancy testing in clinical trials)
    (iii) Pazienti in terapia con farmaci che possono interferire con il modafinil (antidepressivi triciclici come la clomipramina, desimipramina, pregabalin, carbamazepina, valproato, litio, idantoina, warfarin, propranolo, inibitori delle MAO, eritromicina, ketoconazolo, fenobarbitale, rifampicina, diazepam)
    (iv) Compromissione cognitiva tale per cui il punteggio al MMSE = 20
    (v) Pazienti con ipertensione non controllata
    (vi) Pazienti con tachicardia, fibrillazione atriale, flutter atriale, bradicardia (< 60 bpm), fibrillazione ventricolare, extrasistole
    (vii) Storia di crisi epilettiche
    E.5 End points
    E.5.1Primary end point(s)
    Improvement of Excessive Daytime Sleepiness
    Miglioramento dell'ipersonnia diurna eccessiva
    E.5.1.1Timepoint(s) of evaluation of this end point
    after 6 months treatment/intervention
    dopo 6 mesi di trattamento per ogni paziente
    E.5.2Secondary end point(s)
    Change from baseline of EDS as assessed by subjective sleepiness scales (Epworth Sleepiness
    Scale, ESS and Fatigue and Daytime Sleepiness Scale, FDSS)
    l Change from baseline of EDS as assessed by 1-week actigraphy
    l Change from baseline of fatigue as assessed by the Fatigue Severity Scale (FSS)
    l Change from baseline of respiratory function (assessed by ABG and PG) at 3 and at 9 months from
    treatment
    l Change from baseline of distance walked with 6MWT, of TUG and of 9-peg hole test
    l Change from baseline of cognitive tests (TEA, TMT, Stroop, Wisconsin)
    l Change from baseline of POMS and depression and anxiety scores
    l Change from baseline of SF-36 and INQoLv2 scores
    l Improved compliance (assessed by % days used and mean daily usage as indicated by the
    ventilators¿ microchip)
    Miglioramento soggettivo della ipersonnia diurna, della fatica e della qualit¿ di vita; miglioramento dell'insufficienza respiratoria cronica; miglioramento dell'attenzione e dell'umore; miglioramento della qualit¿ di vita
    E.5.2.1Timepoint(s) of evaluation of this end point
    6 months after treatment
    6 mesi dopo il trattamento
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Yes
    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 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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned4
    E.8.5The trial involves multiple Member States No
    E.8.5.1Number of sites anticipated in the EEA4
    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 end with LVLS
    La conclusione della sperimentazione sar¿ con LVLS
    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 months24
    E.8.9.1In the Member State concerned days73
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months24
    E.8.9.2In all countries concerned by the trial days73
    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: 35
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 state80
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 80
    F.4.2.2In the whole clinical trial 80
    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)
    The majority of patients with DM1 complain of excessive daytime sleepiness (EDS). There is no
    accepted treatment for this complaint in DM1. EDS is mostly of central origin in DM1 but part of it is
    related to chronic respiratory insufficiency. This may be corrected by regular non-invasive nocturnal
    ventilation (NIV). If NIV and modafinil alone, or combined prove clinical efficacy we will have an impact on
    the most frequent symptom in these patients (EDS) and we expect this will improve their qual
    La maggior parte dei pazienti con DM1 presenta una ipersonnia diurna. Non esiste ad oggi un trattamento riconosciuto per tale sintomo nella DM1. L'ipersonnia diurna ha origine prevalentemente centrale ma parte ¿ dovuta ad un'insufficienza respiratoria cronica. Questa pu¿ essere corretta dall'uso quotidiano e regolare della NIV. Se la NIV e il Modafinil da soli o combinati si dimostreranno efficaci, avremo un impatto positivo su uno dei sintomi pi¿ frequenti in questi pazienti, e ci aspettiamo ch
    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 Decision2016-07-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-04-22
    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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