Evaluating Two Doses of Golexanolone for Safety, Tolerability, and Effects on Fatigue and Cognition in Adults With Primary Biliary Cholangitis

What this Trial Is About

Researchers are evaluating the safety, tolerability, and initial effects of two dosages of golexanolone compared to placebo in adults with Primary Biliary Cholangitis PBC who experience significant fatigue and cognitive symptoms. This phase 1b2 randomized, double-blind, placebo-controlled study focuses on patients with non-cirrhotic or mild cirrhotic PBC Child-Pugh class A who are on stable standard care medication. The study aims to understand how golexanolone affects fatigue, daytime sleepiness, cognitive function, and overall quality of life. The study has two parts Part A assesses safety, tolerability, and pharmacokinetics of golexanolone 40 mg taken twice daily for 5 days. Part B evaluates safety, tolerability, and effects of two dose levels 40 mg and 80 mg, twice daily compared to placebo over 28 days. Participants receive soft gelatin capsules orally twice daily during these periods. The study uses a parallel design with randomized allocation and quadruple masking to compare golexanolone doses and placebo. Participants will be monitored from enrollment through 5 days in Part A and 28 days in Part B, with assessments including adverse event tracking, quality of life questionnaires PBC-40, EQ-5D-3L, sleepiness scales, cognitive tests, and investigator impressions of treatment effect. Pharmacokinetic sampling occurs on days 1, 14, and 28 in Part B. Safety and tolerability are closely observed, with the total participation lasting up to 28 days depending on the part enrolled.

A Two-part Study to Investigate the Effects in Adults of Two Doses of Golexanolone in Patients With Primary Biliary Cholangitis (PBC) With Fatigue and Cognitive Dysfunction