Efficacy and Safety of RO4917523
Research type
Research Study
Full title
A randomized, double-blind, 12- week, parallel group, placebo-controlled study of the efficacy and safety of RO4917523 in patients with Fragile X Syndrome.
IRAS ID
101886
Contact name
Andrew Stanfield
Sponsor organisation
F. Hoffmann-La Roche Ltd.
Eudract number
2011-004349-42
Clinicaltrials.gov Identifier
Research summary
Fragile X syndrome (FXS) is the most commonly inherited cause of mental retardation, and is more frequent in males than females. Symptoms of FXS include attention deficit and hyperactivity, anxiety with mood lability and obsessive-compulsive or autistic behaviors.There is a high unmet need for effective therapies in the treatment of FXS, with current medications aimed at treating symptoms of the disease rather than focused at the underlying mechanism. Metabotropic glutamate receptors play a variety of roles in neuronal functions. Increased signaling in some of these receptors resulting from the absence of Fragile X Mental Retardation Protein (FMRP) may account for the impairment in mental processes and other clinical symptoms associated with FXS. This phase II, randomised, double-blind, parallel-group study aims to evaluate the efficacy and safety of two fixed doses of RO4917523 compared to placebo in male and female patients with FXS, ages 16 to 50 years. Patients will be in the study for approximately 18 weeks: up to 3 weeks in screening followed by a 12-week treatment period and then a 3-week follow-up period. Approximately 180 patients (60 per treatment arm - RO4917523 0.5 mg, RO4917523 1.5 mg or placebo) will be enrolled into this study from approximately 50 study centres worldwide. Eligible patients will have their diagnosis of FXS confirmed by a mandatory DNA sample taken at screening and must have a reliable caregiver that is able and willing to provide information regarding the patient??s behaviour and symptoms and able to administer treatment. The primary objective of this current study is to evaluate the efficacy of 12-week treatment with RO4917523 in patients with FXS as measured by the Anxiety Depression and Mood Scale social avoidance factor and to evaluate the safety and tolerability of RO4917523 in patients with FXS.
REC name
Scotland A REC
REC reference
12/SS/0106
Date of REC Opinion
16 Jul 2012
REC opinion
Further Information Favourable Opinion