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Clinical Trials in the UK / NCT07304843
Active, not recruiting Phase 1/2

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 · tracked via the Priya Life Science UK tracker
Sponsor
Umecrine Cognition AB
Phase
Phase 1/2
Started
2023-04-14
Last updated
2026-08-28

Condition(s) studied

Primary Biliary Cholangitis (PBC)

Investigational drug(s) / intervention(s)

golexanolonegolexanolonePlacebogolexanolonePlacebo

golexanolone: soft gelatin capsules, oral dosage twice per day for up to 28 days

golexanolone: soft gelatin capsules, oral dosage twice a day for up to 28 days

Placebo: soft gelatin capsules, oral dosage twice a day for up to 28 days

golexanolone: soft gelatin capsules, oral dosage twice per day for 5 days

Placebo: soft gelatin capsules, oral dosage twice per day for 5 days

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).

Eligibility

Sex
ALL
Min age
18 Years
Max age
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (38)

FacilityCityRegionStatus
University Hospital Düsseldorf Düsseldorf Germany
University of Leipzig Leipzig Germany
Hippokration General Hospital of Athens Athens Greece
University Hospital of Patras Pátrai Greece
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
University of Padova, Department of Surgery, Oncology and Gastroenterology Padova Italy
University Hospital Paolo Giaccone, University of Palermo Palermo Italy
A. Gemelli Polyclinic, Sacro Cuore Catholic University Roma Italy
Humanitas University Rozzano Italy
University of Udine Udine Italy
University Medical Center "Zvezdara" Belgrade Serbia
Hospital Universitario Parc Taulí Barcelona Spain
Hospital Clinic Barcelona Barcelona Spain
Hospital General Universitario Gregorio Marañón Madrid Spain
Hospital 12th October, Madrid Madrid Spain
Hospital Universitario Virgen De La Victoria Málaga Spain
University Hospital Complex of Pontevedra & IIS Galicia South, Pontevedra Pontevedra Spain
University Hospital Marquez de Valdecilla, Santander Santander Spain
Virgen del Rocio University Hospital Seville Spain
Hospital Universitario y Politécnico La Fe Valencia Spain
Hacettepe University, Fakulty of Medicine, Department of Gastroenterology and Hepatology Ankara Turkey (Türkiye)
9 Eylul University Research and Application Hospital Department of Gastroenterology Balçova Turkey (Türkiye)
Dicle University Faculty of Medicine Department of Gastroenterology Diyarbakır Turkey (Türkiye)
Ege University Faculty of Medicine, Department of Gastroenterology Izmir Turkey (Türkiye)
Kocaeli University Faculty of Medicine Gastroenterology and Hepatology Department Kocaeli Turkey (Türkiye)
Recep Tayyip Erdoğan University Training and Research Hospital, Department of Gastroenterology Rize Turkey (Türkiye)
Harran Üniversitesi Osmanbey Campus Department of Gastroenterology Sanliurfa Turkey (Türkiye)
Karadeniz Technical University, Farabi Hospital, Department of Gastroenterology Trabzon Turkey (Türkiye)
NIHR Birmingham BRC Birmingham United Kingdom
Glasgow Royal Infirmary Glasgow United Kingdom
Royal Free London NHS Foundation Trust London United Kingdom
Guy's and St Thomas' Hospital, London London United Kingdom
Freeman Hospital Newcastle United Kingdom
Nottingham Digestive Diseases Centre and Biomedical Research Centre Nottingham University Hospitals NHS Trust, Queen's Medical Centre Nottingham United Kingdom
Oxford University Hospitals NHS Foundation Trust Oxford United Kingdom
Dept of Gastroenterology & Hepatology Portsmouth Hospitals University NHS Trust Queen Alexandra Hospital Portsmouth United Kingdom
Royal Wolverhampton NHS Trust, New Cross Hospital Wolverhampton United Kingdom
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07304843 on ClinicalTrials.gov ↗ ← All trials in the UK