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
NCT ID: NCT07304843
Last Updated: 2025-12-26
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
84 participants
INTERVENTIONAL
2023-04-14
2026-06-30
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Part B: Treatment arm 1 (golexanolone 40 mg)
40 mg golexanolone BID
golexanolone
soft gelatin capsules, oral dosage twice per day for up to 28 days
Part B: Treatment arm 2 (golexanolone 80 mg)
80 mg golexanolone BID
golexanolone
soft gelatin capsules, oral dosage twice a day for up to 28 days
Placebo
Part B: Placebo BID
Placebo
soft gelatin capsules, oral dosage twice a day for up to 28 days
Part A: Golexanolone
40 mg golexanolone BID
golexanolone
soft gelatin capsules, oral dosage twice per day for 5 days
Part A: Placebo
Placebo BID
Placebo
soft gelatin capsules, oral dosage twice per day for 5 days
Interventions
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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
Eligibility Criteria
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Inclusion Criteria
* 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
* 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
18 Years
ALL
No
Sponsors
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Umecrine Cognition AB
INDUSTRY
Responsible Party
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Principal Investigators
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Pernilla Sandwall
Role: STUDY_DIRECTOR
Umecrine Cognition
Locations
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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
University of Padova, Department of Surgery, Oncology and Gastroenterology
Padua, , 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 12th October, Madrid
Madrid, , 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
Hacettepe University, Fakulty of Medicine, Department of Gastroenterology and Hepatology
Ankara, , 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)
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
Countries
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Central Contacts
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Facility Contacts
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Hans Bock
Role: primary
Johannes Wiegand
Role: primary
Spilios Manolakopoulos
Role: primary
Christos Triantos
Role: primary
Tibor Martyin
Role: primary
Geza Lakner
Role: primary
Nora Cazzagon
Role: primary
Vicenza Calvaruso
Role: primary
Luca Miele
Role: primary
Ana Lleo De Nalda
Role: primary
Pierluigi Toniutto
Role: primary
Petar Svorcan
Role: primary
Mercedes Vergara
Role: primary
Maria Londono
Role: primary
Elena Gomez-Dominguez
Role: primary
Indhira Perez Medrano
Role: primary
Alvaro Diaz Gonzalez
Role: primary
Javier Ampuero
Role: primary
Yasemin Balaban
Role: primary
Kendal Yalcin
Role: primary
Elvan Isik
Role: primary
Hasan Yilmaz
Role: primary
Palak Trivedi
Role: primary
Stephen Barclay
Role: primary
Douglas Thorburn
Role: primary
Sreelakshmi Kotha
Role: primary
David Jones
Role: primary
Stephen Ryder
Role: primary
Emma Culver
Role: primary
Richard Aspinall
Role: primary
Sarah Townsend
Role: primary
Provided Documents
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Document Type: Study Protocol
Other Identifiers
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2024-515907-20-00
Identifier Type: CTIS
Identifier Source: secondary_id
IRAS ID 1003871
Identifier Type: OTHER
Identifier Source: secondary_id
515-04-27580-22-1
Identifier Type: OTHER
Identifier Source: secondary_id
E-66175679-514.02.02-1264666
Identifier Type: OTHER
Identifier Source: secondary_id
UCAB-CT-05
Identifier Type: -
Identifier Source: org_study_id