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    Summary
    EudraCT Number:2011-004349-42
    Sponsor's Protocol Code Number:NP27936
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-05-21
    Trial results Removed from public view
    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 number2011-004349-42
    A.3Full title of the trial
    A randomized, double-blind, 12- week, parallel group, placebo-controlled study of the efficacy and safety of RO4917523 in patients with Fragile X Syndrome.
    Un estudio aleatorizado, doble ciego, con grupos paralelos y controlado por placebo de 12 semanas de duración para evaluar la eficacia y la seguridad de RO4917523 en pacientes con Síndrome de X Frágil
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of the efficacy and safety of RO4917523 in patients with Fragile X Syndrome
    Estudio de eficacia y seguridad de RO4917523 en pacientes con Síndrome de X Frágil
    A.4.1Sponsor's protocol code numberNP27936
    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 SponsorF. Hoffmann-La Roche Ltd.
    B.1.3.4CountrySwitzerland
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportF. Hoffmann-La Roche Ltd.
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationF.Hoffmann-La Roche Ltd.
    B.5.2Functional name of contact pointTrial Information Support Line-TISL
    B.5.3 Address:
    B.5.3.1Street AddressGrenzacherstrasse 124
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4070
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+4161688 1111
    B.5.5Fax number+4161691 9319
    B.5.6E-mailglobal.rochegenentechtrials@roche.com
    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 No
    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 namemGlu5 antagonist
    D.3.2Product code Ro 491-7523/F18
    D.3.4Pharmaceutical form Capsule
    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.3.9.2Current sponsor codeRO4917523
    D.3.9.3Other descriptive namemGlu5 antagonist
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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 No
    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 namemGlu5 antagonist
    D.3.2Product code Ro 491-7523/F19
    D.3.4Pharmaceutical form Capsule
    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.3.9.2Current sponsor codeRO4917523
    D.3.9.3Other descriptive namemGlu5 antagonist
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1.0
    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 placeboCapsule
    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
    Fragile X Syndrome (FXS)
    Síndrome de X Frágil
    E.1.1.1Medical condition in easily understood language
    FXS is a genetic condition caused by a change in the FMR1 gene. A defect in this gene causes your body to produce too little or none of a protein that is needed for normal brain development.
    Síndrome X Frágil:condición genética por cambio en gen FMR1. Un defecto en este gen provoca que el cuerpo produzca muy poca/ninguna cantidad de proteina necesaria para dllo. normal cerebro.
    E.1.1.2Therapeutic area Psychiatry and Psychology [F] - Mental Disorders [F03]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level LLT
    E.1.2Classification code 10025463
    E.1.2Term Major depressive disorder, single episode
    E.1.2System Organ Class 10037175 - Psychiatric disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    ? To evaluate the efficacy of 12-week treatment with RO4917523 in patients with Fragile X Syndrome (FXS) as measured by the ADAMS social avoidance factor.
    ? To evaluate the safety and tolerability of 12-week treatment with RO4917523 in patients with FXS
    Evaluar la eficacia de un tratamiento de 12 semanas de duración con RO4917523 en pacientes con SXF medida según el factor de
    evitación social de la Escala de Ansiedad, Depresión y Estado de Ánimo (ADAMS, por sus siglas en ingles: Anxiety Depression and
    Mood Scale).
    ? Evaluar la seguridad y la tolerabilidad de un tratamiento de 12 semanas de duración con RO4917523 en pacientes con SXF.
    E.2.2Secondary objectives of the trial
    ? Change from baseline in symptoms as measured by the: Aberrant Behaviour Checklist (ABC) total, ABC factor scores, Anxiety Depression and Mood Scale (ADAMS) total, ADAMS factor scores, and Social Responsiveness Scale (SRS)
    ? Change from baseline in cognitive function as measured by the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS)
    ? Clinical Global Impression-Improvement (CGI-I) and change from baseline in the Clinical Global Impression-Severity (CGI-S)
    ? Clinical response (at least 25% improvement in the ABC total score and a CGI-I score of 1 or 2)
    ? Change in the caregiver-identified most troubling symptom as measured by the Visual Analogue Scale (VAS)
    ? Change from baseline in adaptive behaviour skills as measured by the Vineland Adaptive Behavior Scale (VABS-II)
    ? Cambio respecto a síntomas iniciales medidos según: puntuación total según la Lista de Comprobación de Comportamientos Aberrantes, puntuaciones de factores ABC, puntuación total en la escala ADAMS, puntuaciones de factores de escala ADAMS y la Escala de Respuesta Social (SRS)
    ? Cambio respecto a valores iniciales en función cognitiva, medida según Batería Repetible para la Evaluación del
    Estado Neuropsicológico (RBANS)
    ? Impresión Clínica Global-Mejoría (CGI-I) y cambio respecto de los valores iniciales de Impresión Clínica Global Gravedad de la Enfermedad (CGI-S)
    ? Respuesta clínica (mejoría de al menos un 25 % en la puntuación total ABC y puntuación de 1 o 2 en CGI-I)
    ? Cambio en síntomas más problemáticos identificados por el
    cuidador, medidos según Escala Visual Analógica (EVA)
    ? Cambio en las habilidades de comportamiento adaptativo respecto a valores iniciales, medidas según la Escala de Conducta Adaptativa de Vineland-II (VABS-II)
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Sampling Substudy for the Roche clinical Repository:to identify
    biomarkers
    NP27936D - 16 April 2012. Same protocol as main study
    Subestudio de Muestras para el banco de muestras clínicas de Roche: para identificar biomarcadores
    NP27936D - 16 Abril 2012
    Mismo protocolo que el estudio principal
    E.3Principal inclusion criteria
    ? Adults and adolescents (16 to 50)
    ? CGI-S of 3 (mildly ill) or more
    ? ABC total score of 20 or more
    ? Diagnosis of FXS with a confirmed FMR1 full mutation
    ? Adultos y adolescentes (de 16 a 50)
    ? CGI-S de 3 (moderadamente enfermos) o más
    ? Puntuación total de ABC de 20 o más
    ? Diagnóstico de SXF con mutación completa del FMR1 confirmada
    E.4Principal exclusion criteria
    ? Have previously received treatment with another mGlu5 receptor antagonist within 18 months or RO4917523
    ? Enrollment/participation in any interventional study (clinical trial) involving an investigational drug (unapproved) or non-drug treatment within the prior 3 months or 5 times the half-life (whichever is longer)
    ? Any uncontrolled, unstable clinically significant psychiatric condition other than FXS that may interfere with interpretation of safety and efficacy evaluations (e.g. Attention Deficit Hyperactivity Disorder (ADHD))
    ? Current symptoms or presumption of psychosis or euphoria, history of catatonia, hallucinations or delusional thoughts
    ? History of suicidal behaviour or otherwise considered a high suicidal risk by the investigator
    ? Ha recibido un tratamiento previo con otro antagonista de receptor del mGlu5 hace menos de 18 meses, o con RO4917523
    ? Inclusión/participación en cualquier estudio de intervención (ensayo clínico) sobre un fármaco experimental (no aprobado) o un tratamiento sin fármacos durante los 3 meses anteriores o 5 veces la semivida (el período de mayor duración)
    ? Cualquier trastorno psiquiátrico no controlado o inestable y clínicamente significativo, distinto del SXF, que pudiera interferir con la interpretación de las evaluaciones de la seguridad y la eficacia (Trastorno de Deficit de Atención e Hiperactividad)
    ? Síntomas manifiestos o presunción de psicosis o euforia, historial de catatonia, alucinaciones o pensamientos delirantes
    ? Historial de conducta suicida o cualquier otro indicio que el investigador considere con alto riesgo de suicidio
    E.5 End points
    E.5.1Primary end point(s)
    Change in the ADAMS social avoidance factor from baseline to end of treatment
    Cambio en el factor de evitación social de la Escala de Ansiedad,
    Depresión y Estado de Ánimo (ADAMS) desde el inicio hasta el final del tratamiento
    E.5.1.1Timepoint(s) of evaluation of this end point
    Change from baseline to end of treatment (12 weeks)
    Cambio desde el inicio hasta el final del tratamiento (12 semanas)
    E.5.2Secondary end point(s)
    ? Change from baseline in symptoms as measured by the ABC and ADAMS (total and factor scores), and the SRS
    ? Change from baseline in cognitive function as measured by the RBANS
    ? CGI-I and change from baseline in the CGI-S
    ? Clinical response (at least 25% improvement in the ABC total score and a CGI-I score of 1 or 2)
    ? Change in the caregiver-identified most troubling symptom as measured by the VAS
    ? Change from baseline in adaptive behavior skills as measured by the VABS-II
    ? To investigate the pharmacokinetics and exposure response relationship of RO4917523 in patients with FXS using a population analysis approach.
    ? Cambio respecto a síntomas iniciales medidos por ABC y ADAMS (puntuaciones de factores y total) y la Escala (SRS)
    ? Cambio respecto a valores iniciales en función cognitiva, medida según RBANS
    ? CGI-I y cambio respecto de los valores iniciales de CGI-S
    ? Respuesta clínica (mejoría de al menos un 25 % en la puntuación total ABC y puntuación de 1 o 2 en CGI-I)
    ? Cambio en síntomas más problemáticos identificados por el
    cuidador, medidos según EVA
    ? Cambio en las habilidades de comportamiento adaptativo respecto a valores iniciales, medidas según VABS-II
    ? Investigar la farmacocinética y la relación exposición-respuesta del fármaco RO4917523 en pacientes con SXF utilizando
    un método de análisis de población
    E.5.2.1Timepoint(s) of evaluation of this end point
    Change from baseline to end of treatment (12 weeks)
    Cambio desde el inicio hasta el final del tratamiento (12 semanas)
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Patient Reported Outcomes, Psychometric testing of assessment scales for use in FXS
    Resultados comunicados del Paciente, ensayos psicométricos de las escalas de evaluación para su uso en Síndrome de X frágil
    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 trial3
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Argentina
    Canada
    Chile
    France
    Mexico
    Peru
    Spain
    Sweden
    United Kingdom
    United States
    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
    LVLS
    última visita del último paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months5
    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: 49
    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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 49
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 131
    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 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
    Patient?s care-giver will provide consent on the patient?s behalf.
    El cuidador del paciente facilitará el consentimiento en nombre del paciente
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state60
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 50
    F.4.2.2In the whole clinical trial 180
    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)
    Ro4917523 will not be provided to patients after they complete the study. Patients will continue to receive the same level of care as the received prior to enrolment into the study.
    Ro4917523 no se facilitará a los pacientes tras la finalización del estudio. Los pacientes continuarán recibiendo el mismo nivel de cuidados que recibían antes de su participación en el estudio
    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 Decision2012-08-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-07-11
    P. End of Trial
    P.End of Trial StatusCompleted
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