A Study of RO4917523 in Patients With Fragile X Syndrome

Study Overview
=================
Brief Summary
-----------------
This multi-center, randomized, double-blind, placebo-controlled, parallel-group study will investigate the efficacy and safety of RO4917523 in adolescent and adult patients with fragile X syndrome. Patients will be randomized to receive oral doses of 0.5 mg or 1.5 mg of RO4917523, or matching placebo once daily. The anticipated time on study treatment is 12 weeks.

Official Title
-----------------
A Randomized, Double-blind, 12-week, Parallel Group, Placebo-controlled Study of Efficacy and Safety of RO4917523 in Patients With Fragile X Syndrome.

Conditions
-----------------
Fragile X Syndrome

Intervention / Treatment
-----------------
* Drug: Placebo
* Drug: RO4917523 0.5 mg
* Drug: RO4917523 1.5 mg


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Adult and adolescent patients, 14-50 years of age Diagnosis of fragile X syndrome with a confirmed fragile X mental retardation 1 (FMR1) full mutation and qualifying scores on the Aberrant Behavior Checklist (ABC) and CGI-S Patients must agree to either remain completely abstinent or to use two effective contraceptive methods during and 3 weeks after the study Exclusion Criteria: Previous treatment with another metabotropic glutamate (mGLU) receptor antagonist within 18 months or with RO4917523 Participation in a clinical trial involving an investigational (unapproved) drug within 3 months or 5 times the half-life (whichever is longer) before start of this study Any uncontrolled, unstable clinically significant psychiatric condition other than fragile X syndrome History of suicidal behavior

Ages Eligible for Study
-----------------
Minimum Age: 14 Years
Maximum Age: 50 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: RO4917523 0.5 mg<br> | Drug: RO4917523 0.5 mg<br>* 0.5 mg orally once a day for 12 weeks<br>|
| Experimental: RO4917523 1.5 mg<br> | Drug: RO4917523 1.5 mg<br>* 1.5 mg orally once a day for 12 weeks<br>|
| Placebo Comparator: Placebo<br> | Drug: Placebo<br>* placebo to RO4917523 orally once a day for 12 weeks<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Anxiety Depression and Mood Scale (ADAMS) total score |  | 12 weeks | 
| Safety (incidence of adverse events) |  | 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Social Responsiveness Scale (SRS) |  | 12 weeks | 
| Change in Clinical Global Impressions Scale - Improvement (CGI-I) |  | 12 weeks | 
| Change in Clinical Global Impressions Scale - Severity of Illness (CGI-S) |  | 12 weeks | 
| Change in Aberrant Behavior Checklist total score |  | 12 weeks | 
| Change in Aberrant Behavior Checklist factor scores |  | 12 weeks | 
| Change in Anxiety Depression and Mood Scale (ADAMS) factor scores |  | 12 weeks |