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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

NCT07304843 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 84

Last updated 2026-05-14

No results posted yet for this study

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

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

Sponsors & Collaborators

  • Umecrine Cognition AB

    lead INDUSTRY

Principal Investigators

  • Pernilla Sandwall · Umecrine Cognition

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-04-14
Primary Completion
2026-08-31
Completion
2026-08-31

Countries

  • Germany
  • Greece
  • Hungary
  • Italy
  • Serbia
  • Spain
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07304843 on ClinicalTrials.gov