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OV101 (gaboxadol) as therapy of Angelman syndrome

AAN 2019
Phase 2, STARS
The randomised phase 2 trial STARS showed clinical benefit of OV101 (gaboxadol) in patients with Angelman syndrome versus placebo [1]. Improvements were seen in sleep, motor, and behavioural function, using the Clinical Global Impressions - Improvement scale (CGI-I). The authors think CGI-I may be particularly well suited for assessing clinical improvements in a highly heterogeneous disorder like Angelman syndrome. Angelman syndrome is a rare, genetic, neurodevelopmental disorder, characterised by intellectual disability, seizures, and severe impairments in speech, behaviour, motor skills, and sleep. In the STARS trial, OV101 once-daily (15 mg) and twice-daily (10 mg, 15 mg) were tested versus placebo. The primary objective was safety and tolerability over 12 weeks. In the 78 patients who completed the study, most adverse events were mild, with similar frequencies in all 3 groups. Global improvement was observed after 12 weeks with OV101 once-daily ...

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