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A pilot study of golexanolone, a new GABA-A receptor-modulating steroid antagonist, in patients with covert hepatic encephalopathy

Journal of Hepatology May 16, 2021

Montagnese S, Lauridsen M, Vilstrup H, et al. - Researchers conducted the study for reporting data on the safety, pharmacokinetics and effectiveness of golexanolone, a GABA-A receptor-modulating steroid antagonist, in adult patients with cirrhosis. Adults with Child-Pugh A/B cirrhosis and abnormal continuous reaction time on screening were randomized to 3 weeks of dosing with golexanolone (10, 40, or 80 mg BID) or placebo after single/multiple ascending dose studies. Golexanolone was well tolerated and linked to better cognitive performance. Such findings support the therapeutic potential of golexanolone by implicating GABA-A receptor-modulating neurosteroids in the pathogenesis of HE.

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