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    Summary
    EudraCT Number:2013-000531-27
    Sponsor's Protocol Code Number:NL43510
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2014-07-03
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2013-000531-27
    A.3Full title of the trial
    Corticosteroids or clobazam for ESES syndrome: a European, multicenter, randomized, controlled clinical trial
    Corticoides o clobazam para el síndrome ESES: un ensayo clínico aleatorizado y controlado multicéntrico europeo.
    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
    Tratamiento para el síndrome ESES con corticoides: un Estudio Europeo.
    A.3.2Name or abbreviated title of the trial where available
    RESCUE ESES
    RESCUE ESES
    A.4.1Sponsor's protocol code numberNL43510
    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 supportFundación para la Investigación hospital Clínico San Carlos
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFundación para la Investigación hospital Clínico San Carlos
    B.5.2Functional name of contact pointUICEC
    B.5.3 Address:
    B.5.3.1Street Addressprofesor martin lagos s/n
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28040
    B.5.3.4CountrySpain
    B.5.4Telephone number00349133030002774
    B.5.5Fax number00349133035153515
    B.5.6E-mailfibucicec.hcsc@salud.madrid.org
    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.2.1.2Country which granted the Marketing AuthorisationSpain
    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/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.5
    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.2.1.2Country which granted the Marketing AuthorisationSpain
    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 Oral drops
    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 STEAGLATE
    D.3.9.1CAS number 5060-55-9
    D.3.9.4EV Substance CodeSUB10019MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number2 to 60
    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.2.1.2Country which granted the Marketing AuthorisationSpain
    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 IMPParenteral use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMETHYLPREDNISOLONE
    D.3.9.1CAS number 2375-03-3
    D.3.9.3Other descriptive nameMETHYLPREDNISOLONE SODIUM HEMISUCCINATE
    D.3.9.4EV Substance CodeSUB26423
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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
    Encefalopatía con estatus epiléptico eléctrico del sueño, también llamado sindrome ESES
    E.1.1.1Medical condition in easily understood language
    Sleep epilepsy
    Epilepsia del sueño
    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 17.0
    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
    comparar los efectos sobre la cognición del tratamiento con corticoides y del tratamiento con clobazam en niños con ESES
    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.
    - comparar los efectos de ambos tratamientos sobre el índice de puntas y ondas inducidas por el sueño en el EEG.
    - comparar entre ambos grupos la incidencia de crisis.
    - comparar la seguridad de ambos tratamientos, con el fin de evaluar qué pacientes son los que más se benefician de las terapias.
    - estudiar potenciales biomarcadores relacionados con la activación del sistema inmune.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    · Age 2 up to 12 years (not including children of 12 years old);
    · A diagnosis within six months prior to study inclusion (preferably as close to inclusion as possible) of either:
    -Bilateral sleep-induced epileptiform activity with SWI in > 85% of nonREM sleep and developmental delay, arrest, or regression (?typical ESES syndrome?)
    -Arrest or regression of development and bilateral sleep-induced epileptiform activity with a SWI in > 50%, or unilateral sleep induced epileptiform activity with SWI in > 85% of nonREM sleep (?atypical ESES syndrome?)
    -Regression of development and unilateral epileptiform activity with a SWI of > 50% of nonREM sleep (?atypical ESES syndrome?);
    · No previous treatment with either corticosteroids or clobazam
    · No current treatment nor in the previous three months with carbamazepine, oxcarbazepine, vigabatrin, tiagabine, gabapentin and pregabalin; These drugs are known to possibly worsen the outcome in children with ESES syndrome and may therefore influence treatment result. Furthermore, these drugs can increase the SWI during sleep and may cause an electrographic pattern fulfilling the criteria for ESES. Inclusion of such cases with possible ?treatment induced ESES? is not desirable.
    · Written informed consent by parents / legal representatives
    · Edad 2 - 12 años ( sin incluir los niños de 12 años) ;
    · Un diagnóstico dentro de los seis meses anteriores a la inclusión de un estudio (preferentemente lo más cerca posible inclusión como
    posible ) de cualquiera de :
    - actividad epileptiforme inducida Bilateral sueño con patrón EEG caracterizado por ondas y puntas durante más del 85% del sueño no REM y retraso en el desarrollo , la detención o regresión ( "síndrome típico ESES ")
    - detención o regresión del desarrollo y de la actividad epileptiforme inducida por el sueño - bilateral con patrón EEG caracterizado por ondas y puntas durante más del 50 % , o unilateral inducida actividad epileptiforme sueño con patrón EEG caracterizado por ondas y puntas durante más del 85% del sueño no REM ( "síndrome ESES atípica ")
    - regresión del desarrollo y de la actividad epileptiforme unilateral con patrón EEG caracterizado por ondas y puntas durante más del 50%de sueño no REM ( "síndrome ESES atípica ")
    · No tratamiento previo con corticosteroides o clobazam
    · No tratamiento actual ni en los tres meses anteriores con carbamazepina , oxcarbazepina, vigabatrina , tiagabina , gabapentina y pregabalina ; Estos medicamentos se sabe que posiblemente empeorará
    los resultados en los niños con síndrome de ESES y puede , por tanto, influir en resultado del tratamiento. Además , estos medicamentos pueden aumentar el con patrón EEG caracterizado por ondas y puntas durante el sueño y puede causar un EEG con patrón de cumplimiento de los criterios de ESES . La inclusión de este tipo de casos con tratamiento que posiblemente induzca ESES no es deseable.
    · Consentimiento informado por los padres / representantes legales por escrito
    E.4Principal exclusion criteria
    . Patients with a SWI during wakefulness of > 50%
    · Acute or chronic infectious disease (e.g. TB, HIV)
    . Immunodeficiency
    · Severe osteopenia/osteoporosis
    · Diabetes
    · Cushing syndrome
    · Severe respiratory insufficiency
    · Severe liver failure
    · Severe ulcera
    · Any other condition that, in the investigator?s judgement, contra-indicates the use of corticosteroids or clobazam.
    . Los pacientes con EEG con patrón puntas y ondas durante la vigilia de> 50%
    · Enfermedad infecciosa crónica o aguda(por ejemplo, la tuberculosis, el VIH)
    . Inmunodeficiencia
    · Severa osteopenia / osteoporosis
    · Diabetes
    · Síndrome de Cushing
    · Insuficiencia respiratoria grave
    · Insuficiencia hepática grave
    · Úlcera severa
    · Cualquier otra condición que, a juicio del investigador, contra-indica el uso de corticosteroides o clobazam.
    E.5 End points
    E.5.1Primary end point(s)
    Cognitive function at 6 months after starting treatment
    funcionamiento cognitivo a los 6 meses del inicio del tratamiento
    E.5.1.1Timepoint(s) of evaluation of this end point
    after six months
    a los 6 meses
    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.
    - funcionamiento cognitivo a los 18 meses tras el inicio del tratamiento
    - índice de puntas y ondas inducidas por el sueño
    - frecuencia de crisis
    - seguridad
    - tolerabilidad
    E.5.2.1Timepoint(s) of evaluation of this end point
    after six and 18 months
    a los 18 meses
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    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
    grupos paralelos con control activo y abierto, con evaluador ciego
    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 EEA20
    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
    ultima visita del último paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months6
    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
    niños
    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 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.
    Tras 6 meses de tratamiento se continuará administrando el cuidado que el médico responsable consideré adecuado
    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 ECRIN
    G.4.3.4Network Country Spain
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2014-09-19
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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