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Two-part test of two doses of golexanolone for adults with PBC who have fatigue and thinking problems.

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

Phase1; Phase2 Interventional Umecrine Cognition AB · NCT07304843

This trial will test whether two doses of golexanolone can help reduce fatigue and thinking problems in adults with primary biliary cholangitis who are on stable treatment.

Quick facts

PhasePhase1; Phase2
Study typeInterventional
Enrollment84 (estimated)
Ages18 Years and up
SexAll
SponsorUmecrine Cognition AB Industry-sponsored
Locations36 sites (Düsseldorf and 35 other locations)
Trial IDNCT07304843 on ClinicalTrials.gov

What this trial studies

This randomized, double-blind, placebo-controlled two-part protocol enrolls adults with non-cirrhotic or Child-Pugh A primary biliary cholangitis who have clinically significant fatigue and cognitive symptoms while on stable standard therapy. Part A is a phase 1b pharmacokinetic and safety evaluation of golexanolone 40 mg twice daily for 5 days, and Part B is a phase 2, 28-day randomized comparison of two dose levels given twice daily versus placebo. The study captures safety, tolerability, pharmacokinetics, health-related quality of life outcomes (including PBC-40 fatigue and cognitive domains), daytime sleepiness, and investigator global impression of effect. Participants are randomized and blinded to treatment to provide controlled preliminary efficacy and safety data.

Who should consider this trial

Good fit: Adults (≥18 years) with a confirmed diagnosis of PBC (non-cirrhotic or Child-Pugh A), a PBC-40 fatigue score ≥29 and cognitive score ≥16 at screening, on stable PBC standard-of-care therapy for at least 3 months, and meeting reproductive safety requirements are the intended participants.

Not a fit: People with more advanced liver disease beyond Child-Pugh A, those without clinically significant fatigue or cognitive symptoms, or those with unstable PBC treatment or contraindications to the investigational drug are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, golexanolone could offer a new oral option to reduce fatigue and cognitive symptoms and improve quality of life for people with PBC.

How similar studies have performed: This approach is relatively novel for PBC fatigue and cognitive symptoms and there is limited prior clinical data in this exact indication, though related neurosteroid-targeting agents have had early-phase testing in liver-related cognitive disorders.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Male and female subjects age ≥ 18 years
* Diagnosis of PBC based on the presence of ≥2 of 3 key disease characteristics
* Clinically significant fatigue defined for the purposes of this study as a PBC-40 fatigue domain score of ≥29 at screening
* Clinically significant cognitive symptoms, defined for the purposes of this study as a PBC-40 cognitive domain ≥16 at screening
* Stable PBC SoC therapy (if any),for at least 3 months prior to randomisation
* For all women of childbearing potential (WOCBP) a negative pregnancy test at screening and a negative urine dip-stick pregnancy test at baseline, prior to first dose of IMP
* WOCBP must be willing to use a contraceptive method with a failure rate of \< 1% and agree to continue use of this method for the duration of the study and thereafter for 1 month after the last dosing of the IMP
* Females of non-childbearing potential must have documented tubal ligation or hysterectomy; or be post-menopausal
* Fertile male subjects must be willing to use condom and assure that their female partner will use contraceptive methods with a failure rate of \< 1%
* Willing and able to give informed consent
* The subject should be judged by the Investigator to be lucid and oriented to person, place, time, and situation when giving the informed consent

Exclusion Criteria:

* Child-Pugh class B or C cirrhosis
* Clinical evidence of hepatic decompensation (e.g. current or prior HE, ascites, or variceal bleeding)
* History of hepatocellular carcinoma
* Bilirubin \>1.5 x ULN
* Glomerular filtration rate (GFR) \<35 mL/min/1.73m2
* Low Haemoglobin (HB), i.e. subjects with moderate/severe anaemia
* Low S-B12 or low P-folate
* Evidence of biliary obstruction
* Any positive result on screening for human immunodeficiency virus (HIV), or hepatitis B (serum hepatitis B surface antigen positive)
* Prolonged QTcF (\>500 ms), or any clinically significant abnormality in the resting ECG, as judged by the Investigator (at screening)
* Concomitant disease characterised by chronic fatigue and/or cognitive impairment
* Clinically significant bowel disease, including obstruction, active inflammatory bowel disease, or malabsorption
* Clinically significant sleep apnoea
* An uncontrolled thyroid disorder
* Subjects with a history of or currently active immune disorders (i.e. uncontrolled) other that PBC (including autoimmune disease) and/or diseases requiring immunosuppressive drugs
* Clinical diagnosis of autoimmune hepatitis overlap
* The presence, as judged by the Investigator, of clinically significant concomitant illness which would jeopardise safe participation in the study and /or the interpretation of study findings
* Regular use of prescribed or over the counter (OTC) medications known to cause fatigue or cognitive dysfunction
* Use of prohibited medications within 14 days prior to randomisation
* Anticipated change in PBC medication and/or significant medical or surgical intervention within the duration of the study
* Regular (more than 1 week per month) alcohol consumption in excess of 14 units per week
* Administration of another new chemical entity or has participated in any other clinical study that included drug treatment with the last administration within 3 months prior to administration of IMP in this study
* Females who are pregnant, nursing or actively trying to conceive a child
* Expected inability to swallow the required number of IMP capsules at the applicable dose level
* History of severe allergy/hypersensitivity or on-going allergy/hypersensitivity, as judged by the Investigator

Where this trial is running

Düsseldorf and 35 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Primary Biliary CholangitisPBCCognitionFatigue
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.