A randomized, double-blind, placebo-controlled, parallel group study to evaluate AFQ056 in adult patients with Fragile X Syndrome - ND

• Conditions: Fragile X Syndrome; MedDRA version: 9.1Level: PTClassification code 10017324

• Registration Number: EUCTR2009-013667-19-IT
• Lead Sponsor: OVARTIS FARMA

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-12-24
• Last Update Posted: 30 September 2013

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 160

Inclusion Criteria

Inclusion criteria • 18 and 45 years of age, inclusive • males and females; females must be following an acceptable method of birth control according to the protocol • 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 • have a Clinical Global Impression Severity Score (CGI-S) of = 4 (moderately ill) • have a score of > 20 in the ABC-C total scale • have a documented Intelligence Quotient (IQ) score lower than two standard deviations below the IQ test median • have a caregiver who spends, on average, at least six hours per day with the patient, who is willing and capable of supervising treatment, provInclusion criteria • 18 and 45 years of age, inclusive • males and females; females must be following an acceptable method of birth control according to the protocol • 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 • have a Clinical Global Impression Severity Score (CGI-S) of = 4 (moderately ill) • have a score of > 20 in the ABC-C total scale • have a documented Intelligence Quotient (IQ) score lower than two standard deviations below the IQ test median • 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 visitsiding input into efficacy and safety assessments, and accompanying the patient to all study visits
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Exclusion criteria • any advanced, severe or unstable disease • history and/or presence of schizophrenia, bipolar disease, psychosis, confusional states and/or repeated hallucinations as per DSM-IV (Diagnostic and Statistical Manual of Mental Disorders) criteria • history of suicidal behavior or considered a high suicidal risk • history of severe self-injurious behavior • 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 not excluded) • history of clinically significant allergies requiring hospitalization or non-inhaled corticosteroid therapy (asthma, anaphylaxis, etc.) • 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 • any treatment regimen, including psychotropic and/or anticonvulsant therapy that has not been stable for = 6 weeks prior to randomization • current treatment with more than two psychoactive medications, excluding medication used specifically for seizure control • using (or used within 6 weeks before randomization) concomitant medications that are potent inhibitors or inducers of CYP3A4 • using glutamatergic agents (riluzole, memantine, etc.) or lithium within 6 weeks of randomization • planning to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Primary: To assess the efficacy of three doses of AFQ056 versus placebo in reducing the Aberrant Behavior Checklist – Community edition (ABC-C) Total score after 12 weeks of treatment in FXS patients with fully-methylated (FM) FMR1 gene.;Secondary Objective: Secondary: 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 (PM) FMR1 gene.;Primary end point(s): Primary: To assess the efficacy of three doses of AFQ056 versus placebo in reducing the Aberrant Behavior Checklist – Community edition (ABC-C) Total score after 12 weeks of treatment in FXS patients with fully-methylated (FM) FMR1 gene.