A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety of AFQ056 in adolescent patients with Fragile X Syndrome

ovartis Pharma Services AG0 sites180 target enrollmentApril 12, 2011

Overview

No summary available.

Registry

who.int

Start Date

April 12, 2011

End Date

TBD

Last Updated

8 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

•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.
•Are the trial subjects under 18? yes

•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;