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    Summary
    EudraCT Number:2011-003784-30
    Sponsor's Protocol Code Number:CHUBX2011/20
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2011-12-13
    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 ConcernedFrance - ANSM
    A.2EudraCT number2011-003784-30
    A.3Full title of the trial
    Rubinstein-Taybi syndrome: approach to functional imaging and therapeutic trial
    SYNDROME DE RUBINSTEIN-TAYBI : APPROCHE EN IMAGERIE FONCTIONNELLE ET ESSAI THERAPEUTIQUE
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Rubinstein-Taybi syndrome: approach to functional imaging and therapeutic trial
    Rubinstein-Taybi syndrome: approach to functional imaging and therapeutic trial
    A.3.2Name or abbreviated title of the trial where available
    RUBIVAL
    A.4.1Sponsor's protocol code numberCHUBX2011/20
    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 SponsorCHU de bordeaux
    B.1.3.4CountryFrance
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 Depakine
    D.2.1.1.2Name of the Marketing Authorisation holderSanofi-Aventis
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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 liquid
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPBuccal use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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 placeboOral liquid
    D.8.4Route of administration of the placeboBuccal use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Rubinstein-Taybi Syndrome
    Syndrome de Rubinstein-Taybi
    E.1.1.1Medical condition in easily understood language
    Rubinstein-Taybi Syndrome
    Syndrome de Rubinstein-Taybi
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10039281
    E.1.2Term Rubinstein-Taybi syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Evaluate long term memory with subtest “point location” – CMS and “image recognition” RBMT
    A patient is said to be responder if after one year his or her test result increase for one point at least one of the two tests : a increase of one point to “point location” or “image recognition”.
    Estimer l’efficacité clinique, après un an de traitement par le valproate de sodium (30mg/kg/j), sur l’évolution de la mémoire à long terme chez des enfants atteint de SRT.
    E.2.2Secondary objectives of the trial
    Evaluate the impact after one year of treatment with sodium valproate through:
    - a special brain imaging profile and motor skills (posturology and motor coordination in a visuo-manual pointing task)
    - cognitive and developmental profile
    - histone acetylation profile
    Estimer l’effet après un an de traitement par le valproate de sodium, par:
    - Un profil particulier en imagerie cérébrale et la motricité (posturologie, et coordination motrice dans une tache de pointage visio-manuel, et une tâche d’interception d’un mobile)
    - le profil cognitif et développemental
    - le profil d’acétylation des histones
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Children over 6 and under 21
    - RTS confirmed by a genetic study with a CBP gene or EP300 gene mutation
    - Sufficient cognitive capacities for neuropsychological evaluation
    - Free and informed consent of the parents or guardians
    - Children affiliated to or benefiting of the French social welfare system
    - Enfant de plus de 6 ans et de moins de 21 ans
    - SRT confirmé par étude génétique avec mutation du gène CREBBP ou du gène EP300
    - Capacités cognitives permettant l’évaluation neuropsychologique
    - Consentement libre et éclairé des titulaires de l’autorité parentale
    - Enfant affilié ou ayant droit au régime de sécurité sociale
    E.4Principal exclusion criteria
    - Contraindication to sodium valproate
    - Women of reproductive age without contraception means
    - Case history of sodium valproate treatment
    - Monotherapy treatment for epilepsy with Lamictal with a dosage superior to 5 mg/kg/j
    - Pregnancy
    - breastfeeding
    - renal failure
    - enzymatic deficit of urea cycle
    - Contre-indication au valproate de sodium
    - Femme en âge de procréer sans moyen de contraception efficace (oestroprogestatif, dispositif intrautérin)
    - ATCD de traitement par valproate de sodium
    - Epilepsie traitée par Lamictal en monothérapie à une posologie supérieure à 5 mg/kg/j
    - Antécédent familial d’hépatite sévère notamment médicamenteuse
    - Hépatite aiguë ou chronique.
    - Femme enceinte.
    - Allaitement
    - Insuffisance rénale
    - Déficit enzymatique du cycle de l’urée
    E.5 End points
    E.5.1Primary end point(s)
    - Memory tests (assessing memory learning)
    A patient is said to be responder if after one year his or her test result increase for one point at least one of the two tests : a increase of one point to “point location” or “image recognition”.
    Evaluation de la mémoire à long terme par le subtest « localisation des points » du CMS et « reconnaissance d’images » du RBMT.
    Le subtest localisation de point est noté sur 6. Le subtest « reconnaissance d’images » du RBMT est noté sur 10.
    Les patients répondeurs sont définis comme ceux montrant une amélioration de la mémoire à long terme cliniquement considérée comme pertinente, c'est-à-dire se traduisant par une amélioration de 1 point au moins à l’un des 2 tests : épreuves de Localisation des points – CMS ou de Reconnaissance d’images – RBMT (1 point si + 1 image quand < 5 image reconnu à V0 ; + 20% quand >5 images reconnus à V0).
    Les critères d’efficacité retenus sont ceux évalués à 12 mois de traitement
    E.5.1.1Timepoint(s) of evaluation of this end point
    one year
    un an
    E.5.2Secondary end point(s)
    - Special brain imaging profile and motor skills (posturology and motor coordination in a visuo-manual pointing task)
    - Cognitive and developmental profile
    - Histone acetylation profile
    - Profil particulier en imagerie cérébrale et motricité (posturologie, et coordination motrice dans une tache de pointage visio-manuel)
    - Profil cognitif et développemental
    - Profil d’acétylation des histones
    E.5.2.1Timepoint(s) of evaluation of this end point
    one year
    un an
    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 No
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months30
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months30
    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.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) Yes
    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
    minors and adults under guardianship
    mineur et majeurs sous tutelle
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state60
    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 Decision2012-02-10
    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 Status
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