A randomised, double-blind, placebo-controlled, two-part study to evaluate the pharmacokinetics, safety and tolerability, and preliminary efficacy of two dose levels of golexanolone in subjects with primary biliary cholangitis, fatigue, and cognitive dysfunction.

Primary biliary cholangitis (PBC)

Part A: Frequency, intensity, and seriousness of adverse events (AEs), changes from baseline to Day 5 in laboratory parameters and clinical safety parameters., Part B: Frequency, intensity, and seriousness of AEs, changes from baseline to Day 28 in laboratory parameters and clinical safety parameters.

Part A: PK parameters: after the first dose; after the last dose and accumulation ratio between first and last dose., Part A: Metabolite profile in human plasma and urine (to be reported separately)., Part B : Change from baseline to Day 28 in the following HRQoL measures: − PBC-40 scores for each of the domains (cognition, itch, fatigue, social, emotional, and general symptoms). − EQ-5D-3L tool, Part B: Change from baseline to Day 28 in daytime sleepiness related symptoms using the Epworth Sleepiness Scale (ESS)., Part B: Change from baseline to Day 28 in a battery of cognitive tests, including: − Portosystemic Hepatic Encephalopathy Score (PHES) total score − Rey Auditory Verbal Learning test (RAVLT) − Delis and Kaplan Executive Function System (D-KEFS) Letter and Category fluency subtests, Part B: Clinical Global Impression of change, PBC version (CGI-C-PBC)., Part B: Lowest plasma concentration before the next dose (Ctrough) will be assessed pre-dose on Days 1, 7, 14 and 28.

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