Actively Recruiting
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
Led by Umecrine Cognition AB · Updated on 2026-05-14
84
Participants Needed
40
Research Sites
176 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male and female subjects age 63 18 years
- Diagnosis of PBC based on the presence of 652 of 3 key disease characteristics
- Clinically significant fatigue defined as a PBC-40 fatigue domain score of 63 29 at screening
- Clinically significant cognitive symptoms defined as a PBC-40 cognitive domain 63 16 at screening
- Stable PBC standard of care therapy for at least 3 months prior to randomisation
- Negative pregnancy test at screening and baseline for women of childbearing potential
- Women of childbearing potential willing to use highly effective contraception during and 1 month after the study
- Females of non-childbearing potential must have tubal ligation, hysterectomy, or be post-menopausal
- Fertile male subjects willing to use condoms and ensure female partner uses effective contraception
- Willing and able to give informed consent
- Judged lucid and oriented when giving informed consent
You will not qualify if you...
- Child-Pugh class B or C cirrhosis
- Clinical evidence of hepatic decompensation such as hepatic encephalopathy, ascites, or variceal bleeding
- History of hepatocellular carcinoma
- Bilirubin greater than 1.5 times the upper limit of normal
- Glomerular filtration rate below 35 mL/min/1.73m2
- Moderate or severe anemia
- Low vitamin B12 or folate levels
- Evidence of biliary obstruction
- Positive screening for HIV or hepatitis B
- Prolonged QTcF over 500 ms or significant ECG abnormalities
- Chronic fatigue or cognitive impairment caused by other diseases
- Significant bowel disease including obstruction or inflammatory bowel disease
- Clinically significant sleep apnea
- Uncontrolled thyroid disorder
- Active or uncontrolled immune disorders other than PBC
- Diagnosis of autoimmune hepatitis overlap
- Other serious illnesses that may affect safety or study results
- Regular use of medications causing fatigue or cognitive dysfunction
- Use of prohibited medications within 14 days prior to randomisation
- Anticipated changes in PBC medication or major medical/surgical interventions during the study
- Regular alcohol consumption exceeding 14 units per week
- Participation in another drug study within 3 months prior
- Pregnant, nursing, or actively trying to conceive females
- Inability to swallow required capsules
- History of severe or ongoing allergies as judged by the Investigator
AI-Screening
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Trial Site Locations
Total: 40 locations
1
University Hospital Düsseldorf
Düsseldorf, Germany, 40225
Actively Recruiting
2
University of Leipzig
Leipzig, Germany, 04103
Actively Recruiting
3
Hippokration General Hospital of Athens
Athens, Greece, 11527
Actively Recruiting
4
University Hospital of Patras
Pátrai, Greece, 26504
Actively Recruiting
5
Bekes County Central Hospital
Gyula, Hungary, 5700
Withdrawn
6
Facility of CRU Hungary Ltd.
Kistarcsa, Hungary, 2143
Completed
7
Università di Milano-Bicocca, S.C. ASST Grande Ospedale Metropolitano Niguarda, Dipartimento di Medicina e Chirurgia, Epatologia e Gastroenterologia
Milan, Italy, 20162
Actively Recruiting
8
Fondazione IRCCS San Gerardo dei Tintori, Autoimmune Liver Disease Centre, ERN-Rare Liver, Department of Medicine and Surgery, Division of Gastroenterology
Monza, Italy, 20900
Not Yet Recruiting
9
University of Padova, Department of Surgery, Oncology and Gastroenterology
Padova, Italy, 35128
Actively Recruiting
10
University Hospital Paolo Giaccone, University of Palermo
Palermo, Italy, 90127
Actively Recruiting
11
A. Gemelli Polyclinic, Sacro Cuore Catholic University
Roma, Italy, 168
Actively Recruiting
12
Humanitas University
Rozzano, Italy, 20089
Actively Recruiting
13
University of Udine
Udine, Italy, 33100
Actively Recruiting
14
University Medical Center "Zvezdara"
Belgrade, Serbia, 11000
Actively Recruiting
15
Hospital Universitario Parc Taulí
Barcelona, Spain, 08208
Actively Recruiting
16
Hospital Clinic Barcelona
Barcelona, Spain, 8036
Actively Recruiting
17
Hospital General Universitario Gregorio Marañón
Madrid, Spain, 28007
Actively Recruiting
18
Hospital 12th October, Madrid
Madrid, Spain, 28041
Actively Recruiting
19
Hospital Universitario Virgen De La Victoria
Málaga, Spain, 29010
Actively Recruiting
20
University Hospital Complex of Pontevedra & IIS Galicia South, Pontevedra
Pontevedra, Spain, 36071
Actively Recruiting
21
University Hospital Marquez de Valdecilla, Santander
Santander, Spain, 39008
Actively Recruiting
22
Virgen del Rocio University Hospital
Seville, Spain, 41013
Actively Recruiting
23
Hospital Universitario y Politécnico La Fe
Valencia, Spain, 46026
Actively Recruiting
24
Hacettepe University, Fakulty of Medicine, Department of Gastroenterology and Hepatology
Ankara, Turkey (Türkiye), 06230
Actively Recruiting
25
9 Eylul University Research and Application Hospital Department of Gastroenterology
Balçova, Turkey (Türkiye), 35340
Not Yet Recruiting
26
Dicle University Faculty of Medicine Department of Gastroenterology
Diyarbakır, Turkey (Türkiye), 21280
Actively Recruiting
27
Ege University Faculty of Medicine, Department of Gastroenterology
Izmir, Turkey (Türkiye), 35100
Actively Recruiting
28
Kocaeli University Faculty of Medicine Gastroenterology and Hepatology Department
Kocaeli, Turkey (Türkiye), 41100
Actively Recruiting
29
Recep Tayyip Erdoğan University Training and Research Hospital, Department of Gastroenterology
Rize, Turkey (Türkiye), 53020
Not Yet Recruiting
30
Harran Üniversitesi Osmanbey Campus Department of Gastroenterology
Sanliurfa, Turkey (Türkiye), 63300
Not Yet Recruiting
31
Karadeniz Technical University, Farabi Hospital, Department of Gastroenterology
Trabzon, Turkey (Türkiye), 61080
Not Yet Recruiting
32
NIHR Birmingham BRC
Birmingham, United Kingdom, B15 2TT
Actively Recruiting
33
Glasgow Royal Infirmary
Glasgow, United Kingdom, G4 0SF
Actively Recruiting
34
Royal Free London NHS Foundation Trust
London, United Kingdom, NW3 2QG
Actively Recruiting
35
Guy's and St Thomas' Hospital, London
London, United Kingdom, SE1 7EH
Actively Recruiting
36
Freeman Hospital
Newcastle, United Kingdom, NE7 7DN
Actively Recruiting
37
Nottingham Digestive Diseases Centre and Biomedical Research Centre Nottingham University Hospitals NHS Trust, Queen's Medical Centre
Nottingham, United Kingdom, NG7 2UH
Actively Recruiting
38
Oxford University Hospitals NHS Foundation Trust
Oxford, United Kingdom
Actively Recruiting
39
Dept of Gastroenterology & Hepatology Portsmouth Hospitals University NHS Trust Queen Alexandra Hospital
Portsmouth, United Kingdom, PO6 3LY
Actively Recruiting
40
Royal Wolverhampton NHS Trust, New Cross Hospital
Wolverhampton, United Kingdom, WV10 0QP
Actively Recruiting
Research Team
P
Pernilla Sandwall
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
5
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