A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome
- Registration Number
- NCT01750957
- Lead Sponsor
- Hoffmann-La Roche
- Brief Summary
This randomized, double-blind, placebo-controlled, parallel-arm study will evaluate the safety and exploratory efficacy and pharmacokinetics of RO4917523 in pediatric patients with fragile X syndrome. Patients will be randomized to receive one of 2 dose levels of RO4917523 or placebo orally daily for 12 weeks.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 47
Inclusion Criteria
- Children and adolescents, 5 to 13 years of age
- Diagnosis of fragile X syndrome based on prior DNA testing confirming Fragile X Mental Retardation 1 (FMR1) full mutation and qualifying scores on the ABC and CGI-S
Exclusion Criteria
- Previous treatment with another mGlu5 receptor antagonist within the prior 3 months
- Participation in a clinical trial involving an investigational drug (unapproved) or non-drug treatment within the prior 6 weeks or 5 times the half-life (whichever is longer) before the start of this study
- Any uncontrolled, unstable clinically significant psychiatric condition other than fragile X syndrome
- History of suicidal behavior
- Other protocol defined inclusion/exclusion criteria may apply
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo - RO4917523 Dose A RO4917523 - RO4917523 Dose B RO4917523 -
- Primary Outcome Measures
Name Time Method Safety: Incidence of adverse events 15 weeks
- Secondary Outcome Measures
Name Time Method Efficacy: Neuropsychological/behavioral assessment scales (ADAMS/Clinical Global Impressions CGI-S, CGI-I/ GBAS/Aberrant Behavior Checklist ABC/Repeatable Battery for the Assessment of Neuropsychological Status RBANS/VAS behavior) 15 weeks Pharmacokinetics: Clearance (CL/F) up to Week 12 Pharmacokinetics: Volume of distribution at steady-state (Vss/F) up to Week 12