Fragile X Syndrome (FXS) MedDRA version: 14.1 Level: LLT Classification code 10025463 Term: Major depressive disorder, single episode System Organ Class: 100000004873
Conditions
Interventions
Product Name: mGlu5 antagonist
Product Code: Ro 491-7523/F18
Pharmaceutical Form: Capsule
Current Sponsor code: RO4917523
Other descriptive name: mGlu5 antagonist
Concentration unit: mg milligram(s)
C
Sponsors
F. Hoffmann-La Roche Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Adults and adolescents (14 to 50) • CGI-S of 3 (mildly ill) or more • ABC total score of 20 or more • Diagnosis of FXS with a confirmed FMR1 full mutation Are the trial subjects under 18? yes Number of subjects for this age range: 60 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 120 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Have previously received treatment with another mGlu5 receptor antagonist within 18 months or RO4917523 • Enrollment/participation in any interventional study (clinical trial) involving an investigational drug (unapproved) or non-drug treatment within the prior 3 months or 5 times the half-life (whichever is longer) • Any uncontrolled, unstable clinically significant psychiatric condition other than FXS that may interfere with interpretation of safety and efficacy evaluations (e.g. Attention Deficit Hyperactivity Disorder (ADHD)) • Current symptoms or presumption of psychosis or euphoria, history of catatonia, hallucinations or delusional thoughts • History of suicidal behaviour or otherwise considered a high suicidal risk by the investigator
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To evaluate the efficacy of 12-week treatment with RO4917523 in patients with Fragile X Syndrome (FXS) as measured by the ADAMS total score • To evaluate the safety and tolerability of 12-week treatment with RO4917523 in patients with FXS ;Secondary Objective: • Change from baseline in symptoms as measured by the: Aberrant Behaviour Checklist (ABC) total, ABC factor scores, Anxiety Depression and Mood Scale (ADAMS) total, ADAMS factor scores, and Social Responsiveness Scale (SRS) • Change from baseline in cognitive function as measured by the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) • Clinical Global Impression-Improvement (CGI-I) and change from baseline in the Clinical Global Impression-Severity (CGI-S) • Clinical response (at least 25% improvement in the ABC total score and a CGI-I score of 1 or 2) • Change in the caregiver-identified most troubling symptom as measured by the Visual Analogue Scale (VAS) • Change from baseline in adaptive behaviour skills as measured by the Vineland Adaptive Behavior Scale (VABS-II) ;Primary end point(s): Change in the ADAMS total score from baseline to end of treatment;Timepoint(s) of evaluation of this end point: Change from baseline to end of treatment (12 weeks) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Change from baseline in symptoms as measured by the ABC and ADAMS (total and factor scores), and the SRS • Change from baseline in cognitive function as measured by the RBANS • CGI-I and change from baseline in the CGI-S • Clinical response (at least 25% improvement in the ABC total score and a CGI-I score of 1 or 2) • Change in the caregiver-identified most troubling symptom as measured by the VAS • Change from baseline in adaptive behavior skills as measured by the VABS-II • To investigate the pharmacokinetics and exposure response relationship of RO4917523 in patients with FXS using a population analysis approach. ;Timepoint(s) of evaluation of this end point: Change from baseline to end of treatment (12 weeks) | — |
Countries
Argentina, Canada, Chile, France, Mexico, Peru, Spain, Sweden, United Kingdom, United States
Contacts
Public ContactTrial Information Support Line-TISL
F.Hoffmann-La Roche Ltd.
Outcome results
None listed