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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
47
Unnamed facility
Long Beach, California, United States
Unnamed facility
Sacramento, California, United States
Safety: Incidence of adverse events
Time frame: 15 weeks
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)
Time frame: 15 weeks
Pharmacokinetics: Clearance (CL/F)
Time frame: up to Week 12
Pharmacokinetics: Volume of distribution at steady-state (Vss/F)
Time frame: up to Week 12
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Unnamed facility
San Diego, California, United States
Unnamed facility
Aurora, Colorado, United States
Unnamed facility
Washington D.C., District of Columbia, United States
Unnamed facility
Decatur, Georgia, United States
Unnamed facility
Chicago, Illinois, United States
Unnamed facility
Iowa City, Iowa, United States
Unnamed facility
Louisville, Kentucky, United States
Unnamed facility
Baltimore, Maryland, United States
...and 10 more locations