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    Summary
    EudraCT Number:2013-005528-40
    Sponsor's Protocol Code Number:EPISTOP
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2014-07-14
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2013-005528-40
    A.3Full title of the trial
    Long-term, prospective study evaluating clinical and molecular biomarkers of epileptogenesis in a genetic model of epilepsy – Tuberous Sclerosis Complex.
    Een lange-termijn, prospectieve studie om klinische en moleculaire biomarkers van epileptogenese in kaart te brengen in een genetisch model voor epilepsie - Tubereuze Sclerosis Complex.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Long-term, prospective study evaluating clinical and molecular biomarkers of epileptogenesis in a genetic model of epilepsy – Tuberous Sclerosis Complex.
    Een lange-termijn, prospectieve studie om klinische en moleculaire biomarkers van epileptogenese in kaart te brengen bij patiënten met Tubereuze Sclerosis Complex.
    A.3.2Name or abbreviated title of the trial where available
    EPISTOP
    EPISTOP
    A.4.1Sponsor's protocol code numberEPISTOP
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02098759
    A.5.4Other Identifiers
    Name:Dutch ABR formNumber:48101
    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 SponsorThe Children's Memorial Health Institute
    B.1.3.4CountryPoland
    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 supportEuropean Commission
    B.4.2CountryEuropean Union
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationThe Children's Memorial Health Institute
    B.5.2Functional name of contact pointEPISTOP Project Office
    B.5.3 Address:
    B.5.3.1Street AddressAl. Dzieci Polskich 20
    B.5.3.2Town/ cityWarsaw
    B.5.3.3Post code04-730
    B.5.3.4CountryPoland
    B.5.4Telephone number0048228157854
    B.5.6E-mailepistop@ipczd.pl
    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 Vigabatrin (Sabril)
    D.2.1.1.2Name of the Marketing Authorisation holderSanofi-Aventis Netherlands B.V.
    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.3.1Product namevigabatrin
    D.3.4Pharmaceutical form Granules in sachet
    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 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
    Epilepsy in tuberous sclerosis complex (TSC)
    Epilepsie bij Tubereuze Sclerosis Complex (TSC)
    E.1.1.1Medical condition in easily understood language
    Epilepsy in tuberous sclerosis complex (TSC)
    Epilepsie bij Tubereuze Sclerosis Complex
    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
    The primary objective of clinical part of EPISTOP project is to identify the clinical and molecular biomarkers of epileptogenesis in a prospective clinical study of patients with TSC.
    Het primaire doel van de EPISTOP trial is om klinische en moleculaire biomarkers van epileptogenese te identificeren, in een prospectieve klinische studie bij zuigelingen met TSC.
    E.2.2Secondary objectives of the trial
    Secondary objective of the clinical part of EPISTOP is to compare the effects of standard antiepileptic treatment in patients diagnosed as having epilepsy after clinical seizures vs after electroencephalographic epileptiform discharges, in a randomized trial in TSC patients.
    Het secundaire doel van de EPISTOP trial is om het effect van vroege behandeling - na het ontstaan van epileptiforme EEG afwijkingen - te vergelijken met het effect van standaard behandeling - na het ontstaan van klinische aanvallen -, in een randomized controlled trial.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male or female infants with a definite diagnosis of TSC (Roach criteria; Roach 1998 or DNA confirmed),
    - Age up to 4 months at the moment of enrolment,
    - No clinical seizures seen by caregivers or on baseline videoEEG recording,
    - Written informed consent of caregivers. It is possible to give consent for the observational part of the study only. In this case, the child will not enter the randomized part of the study.
    - Mannelijke of vrouwelijke kinderen met een bevestigde diagnose van TSC (Roach criteria; Roach 1998, of DNA bevestigd);
    - Niet ouder dan 4 maanden op het moment van inclusie;
    - Geen klinische aanvallen gezien door ouders/verzorgers of op het baseline videoEEG;
    - Schriftelijke toestemming van de ouders. Het is mogelijk om alleen toestemming te geven voor het observationele deel van de studie. In dat geval zal het kind niet deelnemen aan de RCT.
    E.4Principal exclusion criteria
    - Any type of seizures observed till baseline visit,
    - Antiepileptic treatment at or prior to study entry,
    - Contraindications to MRI,
    - Any severe and/or uncontrolled medical condition that is considered by the investigator as possibly affecting the EPISTOP analyses or procedures.
    - Elke type epileptische aanvallen, geobserveerd tot aan inclusie;
    - Behandeling met anti-epileptica voor of ten tijde van inclusie;
    - Contra-indicaties voor MRI-onderzoek;
    - Elke ernstige medische aandoening die volgens de onderzoeker mogelijk de procedures en/of analyses van EPISTOP kan beinvloeden.
    E.5 End points
    E.5.1Primary end point(s)
    Primary endpoint of the study is the collection of a set of molecular and clinical biomarkers in full analysis set of patients, including all TSC infants participating in the study and the control subjects.
    De primaire uitkomstparameter van de studie is de collectie van een set van moleculaire en klinische biomarkers, bij alle deelnemers aan het onderzoek, zowel kinderen met TSC, als controles.
    E.5.1.1Timepoint(s) of evaluation of this end point
    vEEG will be performed every 4 weeks (± 1 week) in the children under the age of 6 months, then every 6 weeks (± 1 week) in children under 12 months, and every 8 weeks (± 1 week) thereafter.
    MRI will be performed at baseline (unless patient had an MRI performed within 1 month before baseline visit and an additional scan cannot be performed without general anaesthesia) and then as clinically indicated, but not later than at the age of 2 years.
    Neuropsychological assessment using a specially designed targeted battery of test will be performed every 6 months.
    Blood samples will be collected at study entry, at the onset of epileptiform discharges on vEEG or at the age of 6 months, whichever is applicable, at the onset of clinical seizures and at the end of follow-up (age 2 years)
    Er zal elke 4 weken (± 1 week) een videovEEG gemaakt worden bij kinderen jonger dan 6 maanden; daarna elke 6 weken (± 1 week) bij kinderen jonger dan 12 maanden en daarna elke 8 weken tot het einde van de follow-up.
    Er zal bij inclusie een MRI-hersenen gemaakt worden (tenzij de patient binnen een maand voor de baseline visit een MRI heeft ondergaan en een extra scan niet kan worden uitgevoerd zonder algehele anesthesie) en daarna wanneer klinisch geïndiceerd, maar niet later dan op de leeftijd van 2 jaar.
    Neuropsychologisch onderzoek zal elke 6 maanden plaatsvinden.
    Bloedafname zal plaatsvinden bij inclusie, bij het ontstaan van epileptiforme afwijkingen op het EEG of op de leeftijd van 6 maanden, bij de start van klinische aanvallen en aan het einde van de follow-up (2 jaar).
    E.5.2Secondary end point(s)
    Key secondary endpoint is better efficacy of antiepileptic treatment in patients diagnosed as epileptic after electroencephalographic epileptiform discharges in comparison to patients diagnosed as having epilepsy after the onset of clinical seizures.
    De belangrijkste secundaire uitkomstparameter is het vaststellen van het meest effectieve moment van behandelen: na het ontstaan van epileptiforme EEG afwijkingen of na het ontstaan van klinische aanvallen.
    E.5.2.1Timepoint(s) of evaluation of this end point
    The parameters of efficacy assessment include: the distribution of seizure free patients, proportion of patients with drug resistant seizures, proportion of patients with normalized EEG, the neuropsychological outcome recognized as the results in a battery of tests performed at the age of 24 months.
    Parameters van effectiviteit omvatten de volgende: de verdeling van aanvalsvrije patienten, de proportie patienten met refractaire epilepsie, de proportie patienten met een genormaliseerd EEG en de uitkomsten van neuropsychologisch onderzoek op de leeftijd van 24 maanden.
    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 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) 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 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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Het meest optimale moment van behandelen.
    Different time points for treatment of epilepsy.
    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 EEA9
    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
    Last visit of the last subject
    Laatste bezoek van de laatste patient.
    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 days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months6
    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: 100
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 40
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 60
    F.1.1.5Children (2-11years) No
    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
    Infants up to 4th month of life can join the study after their caregivers/parents give consent
    Zuigelingen tot de leeftijd van 4 maanden kunnen deelnemen aan het onderzoek als hun ouders/verzorgers toestemming geven.
    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 100
    F.4.2.2In the whole clinical trial 100
    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)
    none
    geen
    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 Decision2014-07-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-12-05
    P. End of Trial
    P.End of Trial StatusOngoing
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