A Study of RO4917523 in Patients With Fragile X Syndrome

Fragile X Syndrome Clinical Trial

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01517698
• Other study ID #: NP27936
• Status: Completed
• Phase: Phase 2
• First received: January 20, 2012
• Last updated: July 6, 2016
• Start date: May 2012
• Est. completion date: April 2014

Study information

• Verified date: July 2016
• Source: Hoffmann-La Roche
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 185
• Est. completion date: April 2014
• Est. primary completion date: April 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 14 Years to 50 Years

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Fragile X Syndrome
• Syndrome

Intervention

Drug:
• Placebo
placebo to RO4917523 orally once a day for 12 weeks
• RO4917523 0.5 mg
0.5 mg orally once a day for 12 weeks
• RO4917523 1.5 mg
1.5 mg orally once a day for 12 weeks

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Hoffmann-La Roche

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Anxiety Depression and Mood Scale (ADAMS) total score		12 weeks	No
Primary	Safety (incidence of adverse events)		12 weeks	No
Secondary	Change in Social Responsiveness Scale (SRS)		12 weeks	No
Secondary	Change in Clinical Global Impressions Scale - Improvement (CGI-I)		12 weeks	No
Secondary	Change in Clinical Global Impressions Scale - Severity of Illness (CGI-S)		12 weeks	No
Secondary	Change in Aberrant Behavior Checklist total score		12 weeks	No
Secondary	Change in Aberrant Behavior Checklist factor scores		12 weeks	No
Secondary	Change in Anxiety Depression and Mood Scale (ADAMS) factor scores		12 weeks	No