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    Summary
    EudraCT Number:2013-000717-20
    Sponsor's Protocol Code Number:SP0967
    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:2015-04-16
    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 number2013-000717-20
    A.3Full title of the trial
    A MULTICENTER, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO INVESTIGATE THE EFFICACY AND SAFETY OF LACOSAMIDE AS ADJUNCTIVE THERAPY IN SUBJECTS WITH EPILEPSY ≥1 MONTH TO <4 YEARS OF AGE WITH PARTIAL-ONSET SEIZURES
    Studio multicentrico, in doppio cieco, randomizzato, controllato con placebo, a gruppi paralleli per valutare l’efficacia e la sicurezza di lacosamide come terapia aggiuntiva in pazienti affetti da epilessia di età pari o superiore a 1 mese e inferiore a 4 anni con crisi a insorgenza parziale
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    EFFICACY AND SAFETY OF LACOSAMIDE AS ADJUNCTIVE THERAPY IN SUBJECTS ≥1 MONTH TO <4 YEARS WITH PARTIAL-ONSET SEIZURES
    Efficacia e la sicurezza di lacosamide come terapia aggiuntiva in pazienti di età pari o superiore a 1 mese e inferiore a 4 anni affetti da epilessia con crisi a insorgenza parziale
    A.4.1Sponsor's protocol code numberSP0967
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/275/2014
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorUCB Biosciences Inc.
    B.1.3.4CountryUnited States
    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 supportUCB Biosciences Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUCB Biosciences GmbH
    B.5.2Functional name of contact pointClin Trial Reg & Results Disclosure
    B.5.3 Address:
    B.5.3.1Street AddressAlfred-Nobel-Strasse 10
    B.5.3.2Town/ cityMonheim
    B.5.3.3Post codeD-40789
    B.5.3.4CountryGermany
    B.5.4Telephone number492176481515
    B.5.5Fax number492173481572
    B.5.6E-mailclinicaltrials@ucb.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 Vimpat
    D.2.1.1.2Name of the Marketing Authorisation holderUCB Pharma SA
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 namelacosamide
    D.3.4Pharmaceutical form Syrup
    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 INNLACOSAMIDE
    D.3.9.1CAS number 175481-36-4
    D.3.9.2Current sponsor codeSPM 927
    D.3.9.3Other descriptive nameLCM
    D.3.9.4EV Substance CodeSUB25407
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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 placeboSyrup
    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
    Epilepsy with partial onset seizures
    epilessia con crisi a insorgenza parziale
    E.1.1.1Medical condition in easily understood language
    Epilepsy with partial onset seizures
    epilessia con crisi a insorgenza parziale
    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 18.0
    E.1.2Level PT
    E.1.2Classification code 10015037
    E.1.2Term Epilepsy
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to evaluate the efficacy of LCM administered concomitantly with 1 to 3 AEDs in subjects ≥1 month to <4 years of age with epilepsy who currently have uncontrolled partial-onset seizures.
    L’obiettivo primario di questo studio consiste nel valutare l’efficacia di LCM somministrato in concomitanza con 1 fino a 3 AED in pazienti affetti da epilessia di età pari o superiore a 1 mese e inferiore a 4 anni che attualmente soffrono di crisi a insorgenza parziale incontrollate.
    E.2.2Secondary objectives of the trial
    The secondary objective is to evaluate the safety and tolerability of LCM in subjects ≥1 month to <4 years of age with epilepsy who currently have uncontrolled partial-onset seizures.
    L’obiettivo secondario dello studio consiste nel valutare la sicurezza e la tollerabilità di LCM in pazienti affetti da epilessia di età pari o superiore a 1 mese e inferiore a 4 anni che attualmente soffrono di crisi a insorgenza parziale incontrollate.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subject is male or female from ≥1 month (ie, 4 weeks after full term [37 weeks gestational age]) to <4 years of age.
    • Subject has a diagnosis of epilepsy with partial-onset seizures. The results of ≥1 prior EEG and ≥1 magnetic resonance imaging/computerized tomography scan should be consistent with this diagnosis.
    • Subject weighs ≥4kg to <30kg at Visit 1.
    • Subject has uncontrolled partial-onset seizures after an adequate course of treatment (in the opinion of the investigator) with ≥2 AEDs (concurrently or sequentially).
    • Subject has experienced ≥2 partial-onset seizures with or without secondary generalization during each consecutive 7-day period during the 2 weeks prior to Visit 1.
    • Subject has ≥2 partial-onset seizures with or without secondary generalization during the 72-hour Baseline video-EEG.
    • Subject is on a stable (concurrently or sequentially) dosage regimen of 1 to 3 AEDs. The dosage regimen of concomitant AED therapy must be kept constant for a period of ≥2 weeks prior to Visit 1. A stable daily dosage regimen of a concomitant benzodiazepine (BZD) will be considered as a concomitant AED.
    • Vagus nerve stimulation is allowed and will not be counted as a concomitant AED. The VNS device must have been implanted for ≥6 months prior to Visit 1; device settings must be kept stable for ≥2 weeks prior to Visit 1 and kept stable during the Baseline, Treatment, and Transition Periods. Use of the VNS device magnet is allowed.
    • Subject is an acceptable candidate for venipuncture
    Il paziente è di sesso maschile o femminile di età pari o superiore a 1 mese (vale a dire, quattro settimane dopo il parto [37 settimane di età gestazionale]) e inferiore a 4 anni.
    • Il paziente presenta una diagnosi di epilessia con crisi a insorgenza parziale. I risultati di almeno 1 precedente elettroencefalogramma (EEG) e almeno 1 risonanza magnetica per immagini/tomografia computerizzata dovranno essere coerenti con questa diagnosi
    • Il paziente ha un peso superiore a 4 kg e inferiore a 30 kg alla Visita 1
    • Il paziente ha crisi epilettiche di origine parziale non controllate dopo un adeguato corso di trattamento (a parere dello sperimentatore) con almeno 2 AED (concomitanti o sequenziali).
    • Il paziente ha manifestato almeno 2 crisi epilettiche a insorgenza parziale, con o senza generalizzazione secondaria, in ciascun periodo consecutivo di 7 giorni nel corso delle 2 settimane precedenti la Visita 1
    • Il paziente ha manifestato almeno 2 crisi epilettiche a insorgenza parziale, con o senza generalizzazione secondaria, nel corso dei video EEG di 72 ore registrati al basale
    • Il paziente è (attualmente o sequenzialmente) sottoposto a un regime di dosaggio stabile con 1 fino a 3 AED. Il regime di dosaggio della terapia con AED concomitanti deve essere mantenuto costante per un periodo pari o superiore a 2 settimane prima della Visita 1. Un regime di dosaggio giornaliero stabile con una benzodiazepina (benzodiazepine, BZD) concomitante sarà considerato un AED concomitante
    • La stimolazione del nervo vago è consentita e non sarà considerata un AED concomitante. Il dispositivo di SNV deve essere impiantato per un periodo pari o superiore a 6 mesi prima della Visita 1; le impostazioni del dispositivo devono essere mantenute stabili per almeno 2 settimane prima della Visita 1, nonché durante il periodo basale, di trattamento e di transizione. L’uso del magnete del dispositivo di SNV è consentito
    • Il paziente è un candidato idoneo per la venipuntura.
    E.4Principal exclusion criteria
    Subject has experienced febrile seizures exclusively. The occurrence of febrile seizures in addition to partial-onset seizures is not exclusionary.
    • Subject is on a ketogenic or other specialized diet for the treatment of epilepsy. If the subject was on a ketogenic or other specialized diet in the past, they must be off this diet for ≥2 months prior to Visit 1.
    • Subject has an alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin level ≥2 times the upper limit of normal (ULN), or creatinine clearance <30mL/minute.
    • Subject has a clinically relevant ECG abnormality, in the opinion of the investigator (eg, second or third degree heart block at rest or a corrected QT interval [QTc] ≥450ms).
    • Subject has a hemodynamically significant congenital heart disease.
    • Subject has an arrhythmic heart condition requiring medical therapy.
    • Subject has a known history of severe anaphylactic reaction secondary to medication intake or serious blood dyscrasias.
    • Subject has nonepileptic events that could be confused with seizures. Subjects may be included if epileptic events can be clearly distinguished and the frequency meets the study inclusion criteria.
    • Subject has a current diagnosis of Lennox-Gastaut syndrome, epilepsia partialis continua, primary generalized epilepsy, or seizures that are not of partial-onset origin.
    • Subject has a history of status epilepticus ≤2 months prior to Screening (Visit 1).
    • Subject has been treated with ethosuximide.
    • Subject is currently being treated with vigabatrin or has discontinued use <12 months prior to Visit 1. Subjects who were previously treated with vigabatrin and have discontinued use >12 months prior to Visit 1 are eligible.
    • Subject has been treated with felbamate and has experienced any serious toxicity issues (defined as liver failure, aplastic anemia) with this treatment. Subjects treated with felbamate for <12 months are excluded. Subjects treated with felbamate for ≥12 months prior to Visit 1 and who have not experienced serious toxicity issues are eligible.
    • Subject has an acute or subacutely progressive central nervous system disease. Subject has epilepsy secondary to a progressing cerebral disease or any other progressively neurodegenerative disease (malignant brain tumor or Rasmussen Syndrome).
    • Subject has a known sodium channelopathy, such as Brugada syndrome.
    • Il paziente ha manifestato solo crisi epilettiche febbrili. La manifestazione di crisi epilettiche febbrili in aggiunta a crisi epilettiche a insorgenza parziale non è preclusiva
    • Il paziente segue una dieta chetogenica o un’altra dieta specializzata per il trattamento dell’epilessia. Qualora il paziente abbia seguito in passato una dieta chetogenica o un’altra dieta specializzata, dovrà sospendere tale dieta per un periodo pari o superiore a 2 mesi precedenti la Visita 1
    • Il paziente presenta un livello di alanina aminotransferasi (alanine aminotransferase, ALT), aspartato aminotransferasi (aspartate aminotransferase, AST) o bilirubina 2 volte pari o superiore al limite superiore della norma (Upper Limit Of Normal, ULN) o clearance della creatinina inferiore a 30 ml/minuto
    • Il paziente presenta un’anomalia rilevata nell’ECG che sia ritenuta clinicamente significativa a parere dello sperimentatore (ad esempio, arresto cardiaco di secondo o terzo grado a riposo o un prolungamento del corretto intervallo QT superiore a 450 ms)
    • Il paziente presenta un’insufficienza cardiaca congenita emodinamicamente significativa
    • Il paziente presenta una condizione di aritmia cardiaca richiedente terapia medica
    • Il paziente presenta un’anamnesi nota di grave reazione anafilattica successiva all’assunzione del farmaco in studio o una grave discrasia del sangue
    • Il paziente presenta eventi non epilettici, che possono essere confusi con crisi epilettiche. I pazienti possono essere inclusi in caso di eventi epilettici chiaramente distinguibili e di una frequenza corrispondente ai criteri di inclusione dello studio
    • Il paziente presenta una diagnosi corrente di sindrome di Lennox-Gastaut, epilessia parziale continua, epilessia primaria generalizzata o crisi epilettiche non a insorgenza parziale
    • Il paziente presenta un’anamnesi di condizione epilettica in un periodo pari o inferiore a 2 mesi precedenti la Visita di Screening (Visita 1)
    • Il paziente è stato trattato con etosuccimide
    • Il paziente è attualmente in trattamento con vigabatrina o ne ha interrotto l’uso in un periodo inferiore a 12 mesi precedenti la Visita 1. I pazienti che sono stati precedentemente trattati con vigabatrina e ne hanno interrotto l’uso per un periodo superiore a 12 mesi precedenti la Visita 1 saranno ritenuti idonei
    • Il paziente è stato trattato con felbamato e ha accusato un qualsiasi problema di tossicità grave (definita come insufficienza epatica, anemia aplastica) imputabile a questo trattamento. I pazienti trattati con felbamato per un periodo inferiore a 12 mesi saranno esclusi dallo studio. I pazienti che sono trattati con felbamato per un periodo pari o superiore a 12 mesi precedenti la Visita 1 e non hanno accusato un qualsiasi problema di tossicità grave saranno ritenuti idonei
    • Il paziente è affetto da patologia acuta o subacuta progressiva del sistema nervoso centrale. Il paziente presenta un’epilessia secondaria per una malattia cerebrale progressiva o una qualsiasi altra malattia neurodegenerativa progressiva (tumore maligno al cervello o sindrome di Rasmussen)
    • Il paziente presenta una canalopatia del sodio, tra cui la sindrome di Brugada
    E.5 End points
    E.5.1Primary end point(s)
    1) The primary efficacy variable will be the proportion of responders where a responder is a subject experiencing a 50% or greater reduction in their ADF of electrographic partial-onset seizures recorded on the 72-hour End-of-Maintenance Period video-EEG compared to the 72-hour End-of-Baseline Period video-EEG.
    2) Adverse events reported spontaneously by the subject’s parent(s) and/or legal representative(s)/caregiver(s) (in accordance with local regulation) or observed by the investigator
    3) Subject withdrawals due to AEs
    1. La variabile primaria di efficacia sarà costituita dalla percentuale di pazienti che rispondono al trattamento con una riduzione pari o superiore al 50% nelle proprie ADF di crisi epilettiche elettrografiche a insorgenza parziale registrate mediante il confronto del video EEG di 72 ore alla fine del periodo di mantenimento con il video EEG di 72 ore alla fine del periodo basale.
    2. Eventi avversi segnalati spontaneamente dal/i genitore/i e/o rappresentante/i legale/i o persona che si prende cura del paziente (in conformità con le normative locali) oppure riscontrati dallo sperimentatore.
    3. Ritiro del paziente imputabile a eventi avversi (EA).
    E.5.1.1Timepoint(s) of evaluation of this end point
    1) End-of-Maintenance Period video-EEG (Visit 6) compared to the 72-hour End-of-Baseline Period video-EEG (Visits 2 and 3)
    2) From Baseline to End-of-Treatment
    3) From Baseline to End-of-Treatment
    1) Video EEG alla fine del periodo di mantenimento (visita 6) comparato al video EEG di 72 ore alla fine del periodo basale ( visite 2 e 3)
    2) Dal basale alla fine del trattamento
    3) Dal basale alla fine del trattamento
    E.5.2Secondary end point(s)
    1) Percent and absolute change in ADF of electrographic partial-onset seizures from the End-of-Baseline Period video-EEG to the End-of-Maintenance Period video-EEG
    2) Proportion of subjects who achieved “seizure-free” status (yes/no) for subjects who completed at least 48 hours of interpretable video-EEG recording during the End-of-Maintenance Period video-EEG
    3) Proportion of subjects experiencing a ≥25% to <50%, 50% to 75%, or >75% reduction in ADF of electrographic partial-onset seizures from the end-of-Baseline Period video-EEG to the End-of-Maintenance Period video-EEG
    4) Proportion of subjects experiencing no change in ADF of electrographic partial-onset seizures (between <25% reduction and <25% increase) from the End-of-Baseline Period video-EEG to the End-of-Maintenance Period video-EEG
    5) Proportion of subjects experiencing an increase in ADF of electrographic partial-onset seizures of ≥25% from the End-of-Baseline Period video-EEG to the End-of-Maintenance Period video-EEG
    1. Variazione percentuale e assoluta nell’ADF di crisi epilettiche a insorgenza parziale misurata mediante il confronto del video EEG alla fine del periodo basale con il video EEG alla fine del periodo di mantenimento.
    2. Percentuale di pazienti che abbia raggiunto una condizione di “assenza di crisi” (sì/no) tra coloro che hanno terminato almeno 48 ore di una registrazione interpretabile del video EEG durante il video EEG registrato alla fine del periodo di mantenimento.
    3. Percentuale di pazienti che presenti una riduzione pari o superiore al 25% e inferiore al 50%, dal 50% al 75% o superiore al 75% nell’ADF di crisi epilettiche elettrografiche a insorgenza parziale misurata mediante il confronto del video EEG alla fine del periodo basale con il video EEG alla fine del periodo di mantenimento.
    4. Percentuale di pazienti che presenti un’assenza di variazioni nell’ADF di crisi epilettiche elettrografiche a insorgenza parziale (compresa tra una riduzione inferiore al 25% e un aumento inferiore al 25%) misurata mediante il confronto del video EEG alla fine del periodo basale con il video EEG alla fine del periodo di mantenimento.
    5. Percentuale di pazienti che presenti un aumento nell’ADF di crisi epilettiche elettrografiche a insorgenza parziale pari o superiore al 25%, misurata mediante il confronto del video EEG registrato alla fine del periodo basale con il video EEG registrato alla fine del periodo di mantenimento.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1) From the end-of-Baseline Period video-EEG (Visits 2 and 3) to the End-of-Maintenance Period video-EEG (Visit 6)
    2) During the the end-of-Baseline Period video-EEG (Visit 6)
    3) From the end-of-Baseline Period video-EEG (Visits 2 and 3) to the End-of-Maintenance Period video-EEG (Visit 6)
    4) From the end-of-Baseline Period video-EEG (Visits 2 and 3) to the End-of-Maintenance Period video-EEG (Visit 6)
    5) From the end-of-Baseline Period video-EEG (Visits 2 and 3) to the End-of-Maintenance Period video-EEG (Visit 6)
    1) dal Video EEG alla fine del periodo basale (visita 2 e 3) al EEG della fine del periodo di mantenimento ( visita 6)
    2) Durante il video EEG alla fine del periodo basale (visita 6)
    3) dal Video EEG alla fine del periodo basale (visita 2 e 3) al EEG della fine del periodo di mantenimento ( visita 6)
    4) dal Video EEG alla fine del periodo basale (visita 2 e 3) al EEG della fine del periodo di mantenimento ( visita 6)
    5) dal Video EEG alla fine del periodo basale (visita 2 e 3) al EEG della fine del periodo di mantenimento ( visita 6)
    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 Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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 Yes
    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 concerned18
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA87
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Argentina
    Australia
    Brazil
    Bulgaria
    Canada
    Chile
    Colombia
    Croatia
    Czech Republic
    France
    Georgia
    Germany
    Greece
    Hungary
    Israel
    Italy
    Korea, Republic of
    Mexico
    Moldova, Republic of
    Poland
    Romania
    Serbia
    Spain
    Taiwan
    Thailand
    Ukraine
    United Kingdom
    United States
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months9
    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 months9
    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.1.1Number of subjects for this age range: 244
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 122
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 122
    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 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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children from age of 1 month to less than 4 years
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 106
    F.4.2.2In the whole clinical trial 244
    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)
    Subjects who complete the Maintenance Period will be offered the opportunity to enroll in an open-label extension study (EP0034). Refer to section 5.1 of the protocol, Figure 5-1.
    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 Decision2015-05-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-05-20
    P. End of Trial
    P.End of Trial StatusOngoing
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