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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 | Clinical Trials | Clareo Health

RECRUITINGPHASE1/PHASE2

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

NCT07304843•Umecrine Cognition AB

View on ClinicalTrials.gov

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).

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

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
+1 more criteria

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)
+15 more criteria

Locations (36)

University Hospital Düsseldorf

Düsseldorf, Germany

University of Leipzig

Leipzig, Germany

Hippokration General Hospital of Athens

Athens, Greece

University Hospital of Patras

Pátrai, Greece

Bekes County Central Hospital

Gyula, Hungary

Facility of CRU Hungary Ltd.

Kistarcsa, Hungary

Università di Milano-Bicocca, S.C. ASST Grande Ospedale Metropolitano Niguarda, Dipartimento di Medicina e Chirurgia, Epatologia e Gastroenterologia

Milan, Italy

Fondazione IRCCS San Gerardo dei Tintori, Autoimmune Liver Disease Centre, ERN-Rare Liver, Department of Medicine and Surgery, Division of Gastroenterology

Monza, Italy

University of Padova, Department of Surgery, Oncology and Gastroenterology

Padua, Italy

University Hospital Paolo Giaccone, University of Palermo

Palermo, Italy

Key Dates

Start Date

April 14, 2023

Primary Completion Date

June 30, 2026

Completion Date

June 30, 2026

Last Updated

March 9, 2026

Enrollment

ESTIMATED participants

Conditions

Primary Biliary Cholangitis (PBC)

Interventions

golexanolone

DRUG

golexanolone

DRUG

Placebo

DRUG

golexanolone

DRUG

Placebo

DRUG

Contacts

Pernilla Sandwall

CONTACT

+46 70 630 65 19 pernilla.sandwall@umecrine.se

Biochemical Response and Clinical Outcomes in Patients With PBC

NCT07449793

Primary Biliary Cholangitis (PBC)Primary Biliary Cholangitis

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RECRUITINGPHASE3

Fenofibrate in Combination With Ursodeoxycholic Acid in Primary Biliary Cholangitis: a Real World Study

NCT06755541

Primary Biliary Cholangitis (PBC)

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 9, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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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

Inclusion Criteria

Exclusion Criteria

Biochemical Response and Clinical Outcomes in Patients With PBC

Fenofibrate in Combination With Ursodeoxycholic Acid in Primary Biliary Cholangitis: a Real World Study

FIREFLY Trial: Fenofibrate Intervention---Randomized Evaluation in First-Line PBC Therapy

EMPower Mechanisms

A Multicenter, Randomized, Controlled Trial of Prednisone Combined With Ursodeoxycholic Acid in the Treatment of Primary Biliary Cholangitis With Moderate to Severe Interface Hepatitis Characteristics

PET/CT Scans Using the Tracer 11C-Csar, a Bile Acid Analog, to Depict and Visualize Changes in the Hepatobiliary System in Patients With Primary Biliary Cholangitis Before and After Treatment.