A Randomized, Double-Blind, 12- Week Parallel Group, Placebo-Controlled Study of the Efficacy and Safety of RO4917523 in Patients with Fragile X Syndrome (NP27936)

Service: Trial Number:
018-11
Principal Investigator: Conducted at:
Miller Children‘s Hospital Long Beach
Currently enrolling additional patients:
Yes
Age Range:
7 years & up

Fragile X syndrome is the most common inherited form of autism and mental disability. The mutation is located on the X chromosome and results in abnormal brain development and function including inattention, hyperactivity, social withdrawal, anxiety and impaired cognitive function and language development. The purpose of this study is to evaluate the study drug (RO4917523), an oral medication to reduce anxiety and social withdrawal in Fragile X patients. Study participants will be asked to come to Dr. Feldman’s clinic for a total of 7 or 8 outpatient visits over approximately 18 weeks. The visits include the following: (1) the parent/guardian/caregiver will be asked to complete behavior forms, review medical and physical history, and review all prescribed medicines, and (2) participants will undergo a complete physical exam including vital signs, an electrocardio­gram, a blood sample, and a urine sample. Study participants will take oral capsules daily of either active drug or placebo. There are 2 study drug doses (.5 mg and 1.5mg) which were selected from a dose ranging phase II study where these 2 doses had the most positive results for improved behavior. The study is blinded and prospective to evaluate study drug safety, effectiveness and pharmacokinetics (how well it is being absorbed).

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