NCT07304843

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

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 (PBC), Fatigue, and Cognitive Dysfunction

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 84 (estimated)
• Sponsor: Umecrine Cognition AB · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The present phase 1b/2 randomised, double-blind, placebo-controlled, two-part study is designed to evaluate the safety, tolerability, pharmacokinetic characteristics and preliminary efficacy of two dose levels of golexanolone compared with placebo among subjects with a history of non-cirrhotic or Child-Pugh class A cirrhotic Primary Biliary Cholangitis (PBC) with clinically significant fatigue and cognitive symptoms on stable background standard of care (SoC) PBC medication. The objectives of this research study are to assess the safety and tolerability as well the pharmacokinetic (PK) characteristics of golexanolone administered 40 mg BID for 5 days in the target population (part A) and to assess the safety and tolerability, the effects of golexanolone on health-related quality of life (HRQoL), including fatigue, day-time sleepiness and cognitive function as well as Investigator's overall impression of treatment effect of 28 days twice per day (BID) treatment with two dose levels of golexanolone versus placebo (part B).

Conditions

• Primary Biliary Cholangitis (PBC)

Interventions

Type	Name	Description
DRUG	golexanolone	soft gelatin capsules, oral dosage twice per day for up to 28 days
DRUG	golexanolone	soft gelatin capsules, oral dosage twice a day for up to 28 days
DRUG	Placebo	soft gelatin capsules, oral dosage twice a day for up to 28 days
DRUG	golexanolone	soft gelatin capsules, oral dosage twice per day for 5 days
DRUG	Placebo	soft gelatin capsules, oral dosage twice per day for 5 days

Timeline

• Start date: 2023-04-14
• Primary completion: 2026-06-30
• Completion: 2026-06-30
• First posted: 2025-12-26
• Last updated: 2026-03-09

Locations

36 sites across 8 countries: Germany, Greece, Hungary, Italy, Serbia, Spain, Turkey (Türkiye), United Kingdom