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    The EU Clinical Trials Register currently displays   43800   clinical trials with a EudraCT protocol, of which   7272   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2010-019519-39
    Sponsor's Protocol Code Number:RAPIT
    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:2011-12-01
    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 number2010-019519-39
    A.3Full title of the trial
    Efficacy of RAD001/everolimus in Autism and NeuroPsychological deficits in children with TSC (RAPIT-trial)
    Onderzoek naar behandeling van leer- en gedragsproblemen bij kinderen met Tubereuze Sclerose Complex met RAD001
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy of RAD001/everolimus in Autism and NeuroPsychological deficits in children with tuberous sclerosis complex (RAPIT-trial)
    Onderzoek naar behandeling van leer- en gedragsproblemen bij kinderen met Tubereuze Sclerose Complex met RAD001
    A.3.2Name or abbreviated title of the trial where available
    RAPIT
    A.4.1Sponsor's protocol code numberRAPIT
    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 SponsorErasmus MC - Department of Neurology
    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 supportSophia Stichting Wetenschappelijk Onderzoek
    B.4.2CountryNetherlands
    B.4.1Name of organisation providing supportHersenstichting
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationErasmus MC
    B.5.2Functional name of contact pointClinical trials information
    B.5.3 Address:
    B.5.3.1Street AddressDr. Molewaterplein 60
    B.5.3.2Town/ cityRotterdam
    B.5.3.3Post code3015 GE
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31107036595
    B.5.5Fax number+31107036345
    B.5.6E-mailm.c.y.dewit@erasmusmc.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 Votubia
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Ltd
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/10/764
    D.3 Description of the IMP
    D.3.1Product nameEverolimus
    D.3.2Product code L04AA18
    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 INNVotubia
    D.3.9.1CAS number 159351-69-6
    D.3.9.3Other descriptive nameEVEROLIMUS
    D.3.9.4EV Substance CodeSUB02065MIG
    D.3.10 Strength
    D.3.10.1Concentration unit ng nanogram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number5 to 15
    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
    Tuberous Sclerosis Complex
    E.1.1.1Medical condition in easily understood language
    TSC is a genetic disease that leads to intellectual disability in over 50% of patients, and to learning problems, behavioral problems, autism and epilepsy in up to 90% of patients.
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    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 is to determine the effect of Everolimus on the cognition of children with TSC, measured by
    IQ.
    E.2.2Secondary objectives of the trial
    We will also evaluate the effect on symptoms of autism spectrum disorder, other (neuro) psychological test parameters, seizure frequency, EEG-abnormalities and specific symptoms of learning disability. Furthermore, we will observe the tolerability of Everolimus in children with TSC.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Title: RAPIT-MRI
    Date: 16-08-2012
    Version: 1
    Objective: Magnetic resonance imaging (MRI) of the brain will be made for each participant of the RAPIT trial before and after trial participation. This way, the effect of the investigational drug on the structural abnormalities in the brain can be assessed, and related to cognitive performance.
    Primary outcome will be white matter integrity (DWI/DTI), volume of (sub)cortical tubers, subependymal nodules (SEN) and subependymal giant-cell astrocytomas (SEGA).
    Secondary outcome will be the correlation between structural brain changes and changes in neuropsychological functioning.
    This study is a substudy of the RAPIT trial. Participants of the RAPIT trial will get the choice to have the MRI before and after trial participation. If participant and/or his/her parents do not want to have the MRI, the participant can still enter the RAPIT trial.
    E.3Principal inclusion criteria
    • Children with a definite diagnosis of TSC between 3 and 18 years.
    • With an IQ estimated <80 and/or special schooling and/or autism spectrum disorder and/or learning disability
    requiring remedial teaching.
    • Written informed consent by parents/care-takers, and the patient if he or she is 12 years or older and cognitively
    able to consent.
    • In girls after menarche, appropriate contraception must be used or abstinence practiced.
    E.4Principal exclusion criteria
    • Renal or liver dysfunction
    • Surgery <6wk before entering the study
    • Current infection at time of inclusion
    • Additional diseases or disorders that may influence the endpoints
    • Allergy for any of the components of the study medication
    E.5 End points
    E.5.1Primary end point(s)
    Cognitive functioning: Mullen scales (IQ)
    E.5.1.1Timepoint(s) of evaluation of this end point
    IQ measurements will be obtained at inclusion and at the last study visit.
    E.5.2Secondary end point(s)
    We will also evaluate the effect on symptoms of autism spectrum disorder, other (neuro) psychological test parameters, seizure frequency, EEG-abnormalities and specific symptoms of learning disability. Furthermore, we will observe the tolerability of Everolimus in children with TSC.
    E.5.2.1Timepoint(s) of evaluation of this end point
    All tests will be executed at inclusion and at the last study visit.
    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 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) 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 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    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
    The trial ends when the last patient has completed 12 months of treatment and all tests of the last visit are done.
    For details see protocol.
    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 months0
    E.8.9.1In the Member State concerned 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: 60
    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: 45
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 15
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2011-12-01. Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children aged >12 years that might have developmental delay.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state60
    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 the trial, patients will continue their normal treatment as they did during the trial, because the study medication will be given on top of the standard care. If it turns out everolimus has multiple benefits for the patients, a follow-up trial will be started to follow patients that want to start everolimus again.
    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 Decision2011-12-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-03-14
    P. End of Trial
    P.End of Trial StatusOngoing
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