A Study of RO4917523 in Patients With Fragile X Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 21 July 2016
• Main ID: NCT01517698
• Date of registration: 20/01/2012
• Prospective Registration: Yes
• Primary sponsor: Hoffmann-La Roche
• Public title: A Study of RO4917523 in Patients With Fragile X Syndrome
• Scientific title: A Randomized, Double-blind, 12-week, Parallel Group, Placebo-controlled Study of Efficacy and Safety of RO4917523 in Patients With Fragile X Syndrome.
• Date of first enrolment: May 2012
• Target sample size: 185
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01517698
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

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

Contacts

• Name: Clinical Trials
• Affiliation: Hoffmann-La Roche

Key inclusion & exclusion 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



Age minimum: 14 Years
Age maximum: 50 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Fragile X Syndrome

Intervention(s)

Drug: RO4917523 1.5 mg

Drug: RO4917523 0.5 mg

Drug: Placebo

Primary Outcome(s)

Safety (incidence of adverse events) [Time Frame: 12 weeks]

Change in Anxiety Depression and Mood Scale (ADAMS) total score [Time Frame: 12 weeks]

Secondary Outcome(s)

Change in Aberrant Behavior Checklist factor scores [Time Frame: 12 weeks]

Change in Aberrant Behavior Checklist total score [Time Frame: 12 weeks]

Change in Anxiety Depression and Mood Scale (ADAMS) factor scores [Time Frame: 12 weeks]

Change in Social Responsiveness Scale (SRS) [Time Frame: 12 weeks]

Change in Clinical Global Impressions Scale - Improvement (CGI-I) [Time Frame: 12 weeks]

Change in Clinical Global Impressions Scale - Severity of Illness (CGI-S) [Time Frame: 12 weeks]

Secondary ID(s)

NP27936

Source(s) of Monetary Support

Please refer to primary and secondary sponsors