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    Summary
    EudraCT Number:2009-011184-36
    Sponsor's Protocol Code Number:IND #77,021
    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:2011-09-05
    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 number2009-011184-36
    A.3Full title of the trial
    Phase II Therapeutic Trial of Mexiletine in Non-Dystrophic Myotonia (IND #77,021)
    IMPIEGO DELLA MEXILETINA NELLE MIOTONIE NON DISTROFICHE: SPERIMENTAZIONE CLINICA DI FASE II (IND # 77,021)
    A.3.2Name or abbreviated title of the trial where available
    ND
    ND
    A.4.1Sponsor's protocol code numberIND #77,021
    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 SponsorDept. of Neurology - Univ. of Kansas Medical Center
    B.1.3.4CountryUnited States
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 MEXITIL*20CPS 200MG
    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 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.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMexiletine
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.3Concentration number200
    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 No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    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 disorders are divided into dystrophic myotonias (DM1 and DM2) and non-dystrophic myotonias (NDM). The dystrophic myotonias are associated with significant progressive muscular weakness and other systemic organ involvement. On the other hand, NDM usually presents with muscle stiffness as the primary symptom, and severe weakness is not considered a major feature, especially in myotonia congenita.
    Miotonie non distrofiche, cioe' le canalopatie scheletriche del cloro (es. miotonia congenita di Thomsen e di Becker) e del sodio (es. paralisi periodica ipercaliemica, paramiotonia congenita, miotonia aggravata dal potassio, acetazolamide-sensibile)
    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 14.0
    E.1.2Level LLT
    E.1.2Classification code 10032487
    E.1.2Term Other specific muscle 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
    The specific aim of this proposal is to perform a randomized, double-blind, placebo-controlled crossover study to assess whether mexiletine improves both quantitative and qualitative measures of myotonia in patients with non-dystrophic myotonia.
    Lo scopo di questo studio randomizzato, in doppio cieco, cross-over, controllato verso placebo e' quello di determinare se la mexiletina e' un farmaco efficace nelle miotonie non distrofiche
    E.2.2Secondary objectives of the trial
    -
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    At least 16 years of age 2. Clinical symptoms or signs suggestive of myotonic disorders 3. Presence of myotonic potentials on electromyography (EMG) 4. Participation in the Non-Dystrophic Myotonia Natural History study or a new patient with genetic confirmed NDM
    1. &lt; 16 anni 2. sintomi o segni clinici evocativi di miotonia 3. riscontro di scariche miotoniche allo studio EMG 4. paziente con diagnosi genetica di miotonia non distrofica
    E.4Principal exclusion criteria
    Inability or unwillingness to provide informed consent. 2. Other neurological conditions that might affect the assessment of the study measurements. 3. Genetic confirmed DM1 (CTG > 50 repeats), or DM2. 4. Patients with existing cardiac conduction defects, evidenced on EKG including but not limited to the following conditions: malignant arrhythmia or cardiac conduction disturbances (such as second degree AV block, third degree AV block, or prolonged QT interval >500 ms or QRS duration > 150 msec). 5. Current use of the following antiarrhythmic medication for a cardiac disorder: flecainide acetate, encainide, disopyramide, procainamide, quinidine, propafenone or mexiletine. 6. Women who are pregnant or lactating. 7. Patients currently on medications for myotonia such as phenytoin and flecainide acetate within 5 days of enrollment, carbamazepine and mexiletine within 3 days of enrollment, or propafenone, procainamide, disopyramide, quinidine and encainide within 2 days of enrollment. 8. Patients with an existing permanent pacemaker. 9. Patients with renal or hepatic disease, heart failure, or seizure disorders. 10. Patients on medications that produce myotonia. This includes one or more of the following: a. fibrate acid derivatives, b. hydroxymethylglutaryl CoA reductase inhibitors c. chloroquine d. colchicines
    1.mancanza di consenso informato 2. altre condizioni neurologiche che potrebbero interferire con il completamento dello studio (es disordini cognitivi/comportamentali associati; deficit neuroperiferico che interferisca con l uso corretto delle mani; disordini motori associati che interferiscano con la possibilita' di recarsi alle visite di controllo, ecc.) 3. conferma genetica di distrofia miotonica di Steinert, di tipo 1 (CTG &gt; 50) o di distrofia miotonica di tipo 2 (DM2) 4. pazienti con difetti di conduzione cardiaca sulla base del dato ECG che includano tra alti difetti: aritmie maggiori, disturbi di conduzione cardiaca (blocco AV di II grado o di III grado, intervallo QTc corretto allungato &gt; 500msec o un allungamento della durata del QRS &gt; 150 msec. 5. Utilizzo regolare di terapia antiaritmica per cause cardiologiche: flecainide acetato, encainide, disopiramide, procainamide, chinidina, propafenone, mexiletina. 6. Donne gravide o in allattamento 7. Pazienti che abbiano iniziato il trattamento antimiotonico con i seguenti farmaci: fenitoina e flecainide entro 5 giorni dall arruolamento; carbamazepina e mexiletina entro 3 giorni dall arruolamento; propafenone, procainamide, disopiramide, chinidina ed encainide entro 2 giorni dall arruolamento. 8. Pazienti portatori di pacemaker permanente 9. Pazienti con insufficienza epatica, cardiaca o epilettici 10. Pazienti in terapia con farmaci che possono indurre miotonia. Questi includono: - fibrati - inibitori della idrossimetilglutaril CoA reduttasi - clorochina - colchicina
    E.5 End points
    E.5.1Primary end point(s)
    Participant-assessed stiffness as measured by IVR. Data from the NDM natural history study has shown that stiffness is the most common symptom NDM patients encountered; and there is minimal variation of the reported symptom frequency from week to week (Wang, 2007). Therefore, the primary outcome measure will be the mean daily IVR participantassessed severity of stiffness in weeks 2 & 3, and 7 & 8.
    La modifica della miotonia valutata dai pazienti utilizzando diari elettronici. L end-point primario sara' la media del punteggio relativo alla severita' della miotonia segnalata alla 2a e 3a settimana e la media del punteggio relativo segnalata alla 7a e 8a settimana.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    E.6.3Therapy No
    E.6.4Safety No
    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 Information not present in EudraCT
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Information not present in EudraCT
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over Yes
    E.8.1.7Other Information not present in EudraCT
    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 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 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
    Regole di conclusione anticipata (Early stopping rules), come indicate nel protocollo
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months0
    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 months0
    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 Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 60
    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 Decision2010-06-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2010-02-04
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2010-12-31
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