A Study of RO4917523 in Patients With Fragile X Syndrome

Phase 2

Completed

• Conditions: Fragile X Syndrome
• Interventions: Drug: RO4917523 0.5 mg; Drug: RO4917523 1.5 mg; Drug: Placebo

• Registration Number: NCT01517698
• Lead Sponsor: Hoffmann-La Roche

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2012-01-20
• Study First Submitted QC: 2012-01-24
• Study First Posted: 2012-01-25
• Study Start: 2012-05
• Primary Completion: 2014-04
• Study Completion: 2014-04
• Status Verified: 2016-07
• Disposition First Submitted: 2016-07-06
• Disposition First Submitted QC: 2016-07-06
• Last Update Submitted: 2016-07-06
• Disposition First Posted: 2016-07-11
• Last Update Posted: 2016-07-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 185

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
RO4917523 0.5 mg	RO4917523 0.5 mg	-
RO4917523 1.5 mg	RO4917523 1.5 mg	-
Placebo	Placebo	-

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
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