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    Summary
    EudraCT Number:2010-022638-96
    Sponsor's Protocol Code Number:CAFQ056B2214
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2011-04-12
    Trial results View 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 number2010-022638-96
    A.3Full title of the trial
    A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety of AFQ056 in adolescent patients with Fragile X Syndrome
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate the efficacy of AFQ056 in treating abnormal behaviors showed by adolescents (12-17 year-old) with Fragile X Syndrome
    A.4.1Sponsor's protocol code numberCAFQ056B2214
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/152/2011
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorNovartis Pharma Services AG
    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 supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Pharma S.A.S.
    B.5.2Functional name of contact pointInformation et Communication médica
    B.5.3 Address:
    B.5.3.1Street Address2 et 4 rue Lionel Terray
    B.5.3.2Town/ cityRueil Malmaison
    B.5.3.3Post code92500
    B.5.3.4CountryFrance
    B.5.4Telephone number+33155.47.66.00
    B.5.5Fax number+33155.47.60.50
    B.5.6E-mailicm.phfr@novartis.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 nameAFQ056
    D.3.2Product code AFQ056
    D.3.4Pharmaceutical form Capsule, hard
    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 codeAFQ056
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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 nameAFQ056
    D.3.2Product code AFQ056
    D.3.4Pharmaceutical form Capsule, hard
    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 codeAFQ056
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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, hard
    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
    E.1.1.1Medical condition in easily understood language
    Fragile X Syndrome
    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 13.1
    E.1.2Level PT
    E.1.2Classification code 10017324
    E.1.2Term Fragile X 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
    • To assess the efficacy of three doses of AFQ056 versus placebo in reducing the ABC-C Total score after 12 weeks of treatment in FXS patients with fully-methylated FMR1 gene.
    E.2.2Secondary objectives of the trial
    Key secondary objective:
    • To assess the efficacy of three doses of AFQ056 versus placebo in reducing the ABC-C Total score after 12 weeks of treatment in FXS patients with partially-methylated FMR1 gene.

    Please refer to study protocol for other secondary and exploratory objectives

    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients eligible for inclusion in this study have to fulfill all of the following criteria at Screening (V1), unless another time point prior to randomization is specified:

    1. the caregiver/legal guardian must be able to communicate well with the investigator and must understand and support the study requirements by providing written informed consent. Where possible the patient should also provide his or her written assent (in accordance with local ethical/regulatory requirements);

    2. male or female, between 12 and 17 years of age, inclusive;

    3. have a previous diagnosis of FXS based upon documented genetic testing results (full mutation >200 CGG repeats). The diagnosis will need to be confirmed by genetic testing prior to the patient entering the Placebo Run-in Period;

    4. have a Clinical Global Impression Severity Score (CGI-S) of ≥ 4 (moderately ill);

    5. have a score of > 20 in the ABC-C total scale;

    6. have an IQ < 70 as measured by the Leiter International Performance Scale – Revised (Leiter-R);

    7. have a caregiver who spends, on average, at least six hours per day with the patient, who is willing and capable of supervising treatment, providing input into efficacy and safety assessments, and accompanying the patient to all study visits;

    8. have a caregiver who, in the opinion of the investigator, is a reliable source of information regarding the patient and the severity of his/her disease.
    E.4Principal exclusion criteria
    Patients fulfilling any of the following criteria prior to randomization are not eligible
    for inclusion in this study :

    1. female patients who are sexually active at any time during the study. Sexual activity should be carefully evaluated by the Investigator and if sexual activity is
    confirmed or suspected, the female participant should not be enrolled into the study.
    Furthermore, the Investigator should continue to evaluate the possibility of sexual activity throughout the study and the caregiver/legal guardian should be instructed to immediately inform the Investigator if the female participant becomes or is suspected to have become sexually active during the study. In these cases, appropriate actions should be taken by the Investigator and the patient must be discontinued from the study;

    2. any advanced, severe or unstable disease that may interfere with the primary or secondary study outcome evaluations or put the patient at special risk;

    3. past medical history of clinically significant ECG abnormalities or QTcF > 450 msec for males and > 470 msec for females at screening or baseline;

    4. lab screening values that include AST, ALT, GGT, total bilirubin or creatinine ≥ 1.5 X
    ULN (upper limit of normal) for the central laboratory; total or unconjugated (indirect)
    bilirubin levels up to 3 X ULN for the central laboratory are allowed if the patient has a diagnosis of Gilbert’s syndrome;

    5. have history of major surgery or major medical event that require hospitalization or major surgery within the past 6 months, unless they recovered fully or are considered clinically stable;

    6. donated or lost more than 2.4 mL of blood per kg of body weight within (4) weeks prior to Screening (V1) or longer if required by local regulation;

    7. history and/or presence of schizophrenia, bipolar disease, psychosis, confusional states and/or repeated hallucinations as per Diagnostic and Statistical Manual of Mental Disorders (DSM-IV) criteria;

    8. history of suicidal behavior or considered a high suicidal risk;

    9. history of severe self-injurious behavior;

    10. history of uncontrolled seizure disorder or resistant to therapy within the past 2 years (Patients who are clinically stable under anti-convulsant therapy for the past 2 years are allowed);

    11. history of clinically significant allergies requiring hospitalization or non-inhaled
    corticosteroid therapy (asthma, anaphylaxis, etc.);

    12. history of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether or not there is evidence of local recurrence or metastases;

    13. pregnant or nursing (lactating) females, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG serum pregnancy test;

    14. patients on any treatment regimen, including psychotropic and/or current anticonvulsant therapy that has not been stable for ≥ 6 weeks prior to randomization;

    15. current treatment with more than two psychoactive medications, excluding anti-epileptics;

    16. patients who are using (or used within 6 weeks before randomization) concomitant medications that are potent inhibitors or inducers of CYP3A4 (e.g., ketoconazole, ritonavir, carbamazepine, etc.) (Refer to Appendix 2 for a list of medications);

    17. patients who are using (or used within 6 weeks before randomization) digoxin or warfarin;

    18. patients using glutamatergic agents (riluzole, memantine, etc.) or lithium within 6 weeks of randomization (Refer to Appendix 2 for a list of medications);

    19. patients planning to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study;

    20. use of investigational drugs at the time of enrollment, or within 6 weeks or 5 half-lives of randomization, whichever is longer;

    21. participation in any clinical investigation within 6 weeks prior to randomization or longer if required by local regulation;

    22. patients who, in the opinion of the investigator, are unsuitable in any other way to participate in this study, including being unable to comply with the requirements of the study or displaying abnormalities in safety assessments at baseline;

    23. unable to swallow study medication as demonstrated by a swallowing test using placebo capsules at the Screening visit;

    24. patients who weigh less than 32 kg (= 10th percentile of body weight for a 12-year old child).

    No additional exclusions may be applied by the investigator, in order to ensure that the study population will be representative of all eligible patients.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy analyses will be based on the Aberrant Behavour Checklist - Community Edition (ABC-C)
    Total score.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At multiple visits:

    -7, -4, -2 weeks before Baseline

    Visit at baseline

    2, 4, 8, 12 weeks after baseline
    E.5.2Secondary end point(s)
    - Aberrant Behavour Checklist - Community Edition (ABC-C): Total score and subscales

    - Clinical Global Impression - Improvement (CGI)

    - Repetitive Behavior Scale - Revised (RBS-R)
    E.5.2.1Timepoint(s) of evaluation of this end point
    ABC-C: -7, -4, -2 weeks before Baseline; Visit at baseline; 2, 4, 8, 12
    weeks after baseline

    CGI: Visits at 2, 4, 8 12 weeks after Baseline

    RBS-R: Visit at -3 weeks before Baseline; Visit at Baseline; Visits at 4 and 12 weeks after Baseline
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    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
    Tolerability
    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 trial4
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    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
    Australia
    Denmark
    France
    Germany
    Italy
    Sweden
    Switzerland
    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
    last visit of last subject
    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 months5
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days2
    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: 180
    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: 180
    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 Yes
    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
    Fragile X syndrome is a form of mental retardation. The parents or legal guardian must sign Informed Consent.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state16
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 96
    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)
    Expected normal treatment
    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 Decision2011-05-25
    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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