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    Summary
    EudraCT Number:2013-000531-27
    Sponsor's Protocol Code Number:NL43510
    National Competent Authority:Denmark - DHMA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2015-11-13
    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 ConcernedDenmark - DHMA
    A.2EudraCT number2013-000531-27
    A.3Full title of the trial
    Corticosteroids or clobazam for ESES syndrome: a European, multicenter, randomized, controlled clinical trial
    Kortikosteroider eller clobazam til ESES syndrom : et europæisk , multicenter, randomiseret, kontrolleret klinisk forsøg
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Treatment of ESES syndrome with corticosteroids or clobazam: a European study
    Behandling af ESES syndrom med kortikosteroider eller clobazam : en europæisk undersøgelse
    A.3.2Name or abbreviated title of the trial where available
    RESCUE ESES: Randomized European trial of Steroids vs Clobazam Usage for Encephalopathy with ESES
    RESCUE ESES:Randomiseret europæisk forsøg med steroider vs Clobazam til encephalopati med ESES
    A.4.1Sponsor's protocol code numberNL43510
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN42686094
    A.5.4Other Identifiers
    Name:Dutch CCMO ABR numberNumber:43510
    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 SponsorUniversity Medical Center Utrecht
    B.1.3.4CountryNetherlands
    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 supportDutch national epilepsy fund (NEF)
    B.4.2CountryNetherlands
    B.4.1Name of organisation providing supportWilhelmina Kinderziekenhuis onderzoeksfonds (WKZ-fund)
    B.4.2CountryNetherlands
    B.4.1Name of organisation providing supportEuropean Clinical Research Infrastructures Network (ECRIN)
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportMartindale Pharma
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversity Medical Center Utrecht
    B.5.2Functional name of contact pointCoordinating investigator
    B.5.3 Address:
    B.5.3.1Street AddressLundlaan 6
    B.5.3.2Town/ cityUtrecht
    B.5.3.3Post code3584 EA
    B.5.3.4CountryNetherlands
    B.5.4Telephone number0031887555555
    B.5.5Fax number0031887555350
    B.5.6E-mailf.e.jansen@umcutrecht.nl
    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 clobazam (Frisium®)
    D.2.1.1.2Name of the Marketing Authorisation holderSanofi-Aventis
    D.2.1.2Country which granted the Marketing AuthorisationDenmark
    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 INNCLOBAZAM
    D.3.9.1CAS number 22316-47-8
    D.3.9.4EV Substance CodeSUB06673MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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.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 Yes
    D.2.1.1.1Trade name prednisolon DAK
    D.2.1.1.2Name of the Marketing Authorisation holderTakeda Pharma
    D.2.1.2Country which granted the Marketing AuthorisationDenmark
    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 INNprednisolone
    D.3.9.3Other descriptive namePREDNISOLONE
    D.3.9.4EV Substance CodeSUB10018MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number2.5 to 25
    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.IMP: 3
    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 methylprednisolone
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer BV
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMethylprednisolone
    D.3.9.3Other descriptive nameMETHYLPREDNISOLONE
    D.3.9.4EV Substance CodeSUB08872MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number40 to 1000
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Encephalopathy with electrical status epilepticus in sleep, also called ESES syndrome
    Encephalopati med elektrisk status epilepticus under søvn, også kaldet ESES syndrom
    E.1.1.1Medical condition in easily understood language
    Sleep epilepsy
    Søvn epilepsi
    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 18.1
    E.1.2Level LLT
    E.1.2Classification code 10032061
    E.1.2Term Other forms of epilepsy
    E.1.2System Organ Class 100000004852
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To compare the effects on cognition of treatment with either corticosteroids or clobazam in children with ESES syndrome
    At sammenligne effekten på kognition af behandling med enten kortikosteroider eller clobazam hos børn med ESES syndrom
    E.2.2Secondary objectives of the trial
    - To compare the effects of treatment with corticosteroids or clobazam on sleep induced spike-wave index in children with ESES syndrome.
    - To compare the effects of treatment with corticosteroids or clobazam on the frequency of seizures in children with ESES syndrome.
    - To compare the side-effects and tolerability of treatment with corticosteroids or clobazam in children with ESES syndrome.
    - To compare the effects of treatment with corticosteroids or clobazam in children with ESES syndrome as measured with a VAS score.
    - To assess demographic and disease-related predictive factors, including immunological factors, of succes of treatment with corticosteroids or clobazam in children with ESES syndrome.
    - To identify a biomarker for disease activity and treatment response.
    - At sammenligne effekten af behandling med kortikosteroider eller clobazam på søvninduceret spike-wave indeks hos børn med ESES syndrom.
    - At sammenligne effekten af behandling med kortikosteroider eller clobazam på hyppigheden af anfald hos børn med ESES syndrom.
    - At sammenligne bivirkninger og tolerabilitet af behandling med kortikosteroider eller clobazam hos børn med ESES syndrom.
    - At sammenligne effekten af behandling med kortikosteroider eller clobazam hos børn med ESES syndrom målt med en VAS score.
    - At vurdere demografiske og sygdomsrelaterede prædiktive faktorer, herunder immunologiske faktorer, ved succes med behandling med kortikosteroider eller clobazam hos børn med ESES syndrom.
    - At identificere en biomarkør for sygdomsaktivitet og behandlingsrespons.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    •Age 2 to 12 years
    •A diagnosis within six months prior to study inclusion (preferentially as soon as possible) of either typical or atypical ESES syndrome (as defined in study protocol)
    •No previous treatment with either clobazam or steroids
    •No current treatment with carbamazepine, oxcarbazepine, vigabatrin, tiagabine, gabapentin and pregabalin and no treatment with any of these drugs in the previous three months;
    •Written informed consent by parents/tutors or legal representatives
    • Alder 2 til 12 år
    • En diagnose inden for seks måneder før studieinklusion påbegyndes (fortrinsvis så hurtigt som muligt) med enten typisk eller atypisk ESES syndrom (som defineret i forsøgsprotokollen)
    • Ingen tidligere behandling med enten clobazam eller steroider
    • Ingen nuværende behandling med carbamazepin, oxcarbazepin, vigabatrin, tiagabin, gabapentin og pregabalin, og ingen behandling med nogen af disse lægemidler i de foregående tre måneder;
    • skriftligt informeret samtykke fra forældremyndighedens indehaver eller værge
    E.4Principal exclusion criteria
    •Patients with a spike wave index during wakefulness of > 50%
    •Any condition that, in the investigator’s judgement, contraindicates the use of clobazam or steroids.
    • Patienter med en spike bølge indeks under vågenhed på > 50%
    • Enhver tilstand, der, i følge investigators vurdering, kontraindikerer brug en af clobazam eller steroider.
    E.5 End points
    E.5.1Primary end point(s)
    - Intelligence quotient, or developmental quotient
    - Cognitive sumscore

    Improvement is defined as significant when improved by at least 75% of the standard deviation.
    - Intelligenskvotient, eller udviklingsmæssig kvotient
    - Kognitiv sumscore

    Forbedring er defineret som signifikant, når der forbedres med mindst 75% af standardafvigelsen.
    E.5.1.1Timepoint(s) of evaluation of this end point
    after six months
    efter seks måneder
    E.5.2Secondary end point(s)
    - Individual absolute test results, and IQ scores;
    - Spike wave index during non-REM sleep. Improvement is defined as a rdecrease to less than
    25%;
    - Seizure frequency. Improvement is defined as a reduction of 50% or more as compared with
    baseline;
    - Global improvement of functioning assessed with a VAS score (-5 to 5)
    - Safety and tolerability, as assessed by the occurrence of serious
    adverse events;
    - Differences in pro-inflammatory cytokine levels in patients with ESES
    who respond to either treatment strategies compared to nonresponders.
    - Individuelle absolutte testresultater og IQ score;
    - Spike bølge indeks under ikke-REM søvn. Forbedring er defineret som en reduktion til mindre end
    25%;
    - Frekvens af anfald. Forbedring er defineret som en reduktion på 50% eller mere i forhold til
    baseline;
    - Samlede forbedring af funktionsmåde vurderet med en VAS score (-5 til 5)
    - Sikkerhed og tolerabilitet, som vurderet ud fra forekomsten af alvorlige
    bivirkninger;
    - Forskelle i pro-inflammatoriske cytokinniveauer hos patienter med ESES
    der reagerer på en af behandlingsstrategierne sammenlignet med patienter der ikke reagerer.
    E.5.2.1Timepoint(s) of evaluation of this end point
    after six and 18 months
    efter seks og 18 måneder
    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 No
    E.6.7Pharmacodynamic No
    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) 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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Blændet resultatvurdering af klinisk neurofysiolog og neuropsykolog
    Blinded outcome assessment by clinical neurophysiologist and neuropsychologist
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo No
    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.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    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
    LVLS
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    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: 130
    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: 130
    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
    Børn
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 130
    F.4.2.2In the whole clinical trial 130
    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)
    After six months of treatment, the continued care is given according to judgement of the treating physician.
    Efter seks måneders behandling, er den fortsatte pleje givet efter bedømmelse af den behandlende læge.
    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 European Clinical Research Infrastructures Network (ECRIN)
    G.4.3.4Network Country Netherlands
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-12-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-01-25
    P. End of Trial
    P.End of Trial StatusOngoing
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