A Study of RO4917523 in Patients With Fragile X Syndrome

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

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01517698

Enrollment

185

Registered

2012-01-25

Start date

2012-05-31

Completion date

2014-04-30

Last updated

2016-07-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Fragile X Syndrome

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.

Interventions

DRUGPlacebo

placebo to RO4917523 orally once a day for 12 weeks

DRUGRO4917523 0.5 mg

0.5 mg orally once a day for 12 weeks

DRUGRO4917523 1.5 mg

1.5 mg orally once a day for 12 weeks

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

DOUBLE (Subject, Investigator)

Eligibility

Sex/Gender

ALL

Age

14 Years to 50 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
Change in Anxiety Depression and Mood Scale (ADAMS) total score	12 weeks
Safety (incidence of adverse events)	12 weeks

Secondary

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

Countries

Argentina, Canada, Chile, France, Mexico, Peru, Spain, Sweden, United Kingdom, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026