Study authors included Christina Henderson, Aileen M. Healy, Matthew Shumway, Rebecca S. Hammond, Friso R. Postima, Christopher Brynczka, Roger Rush and Randall L. Carpenter of Seaside Therapeutics; Lasani Wijetunge and Peter C. Kind of the University of Edinburgh; Mika Nakamoto Kinoshita and Stephen T. Warren of Emory University; Alicia Thomas and Richard Paylor of Baylor College of Medicine; Peter W. Vanderklish of The Scripps Research Institute; and Mark F. Bear of the Massachusetts Institute of Technology.
The accompanying paper, "Effects of STX209 (arbaclofen) on neurobehavioral function in children and adults with fragile X syndrome: a randomized, controlled, Phase 2 trial," detailed the results previously announced from the Company's Phase 2 trial. The randomized, double-blind, placebo-controlled Phase 2 study was designed to evaluate the clinical effects of STX209 in 63 subjects, age 6-40 years, with a full mutation of the FMR1 gene. As previously reported, STX209 was well-tolerated and no metabolic side effects were observed. The most common adverse events in subjects receiving STX209 were upper respiratory infections (13%), sedation (8%) and headache (8%), compared to 10%, 2% and 2%, respectively, while receiving placebo. In per-protocol analyses, improvement was seen on the visual analog scale (VAS) ratings of parent-nominated problem behaviors and positive trends were observed on multiple global measures. While improvement was noted on the Irritability subscale of the Aberrant Behavior Checklist (ABC-I), the study's primary endpoint, the magnitude was comparable to that observed on placebo and did not achieve statistical significance.
Social avoidance is a core symptom
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