RecruitingPhase I/IIInterventional

A two-part study to investigate the effects in adults of two doses of golexanolone in patients with primary biliary cholangitis with fatigue and cognitive dysfunction

This research study is investigating a new medication called golexanolone. It's for adults who have a liver disease called Primary Biliary Cholangitis (PBC) and are experiencing significant tiredness (fatigue) and difficulty with memory or concentration (cognitive dysfunction). The study aims to find out if golexanolone is safe, how well the body handles it, and if it can improve these common and challenging symptoms of PBC. Participants receive either golexanolone or a dummy pill (placebo). The study involves regular clinic visits, blood tests, and questionnaires to track health and symptoms. It hopes to improve understanding and treatment options for people living with PBC.

At a glance

Status

Recruiting

Phase

Phase I/II

Sponsor

Umecrine Cognition AB

Enrolment target

100

Start

14 Apr 2023

Estimated completion

30 Jun 2026

Patients with primary biliary cholangitis with fatigue and cognitive dysfunction

What is this study about?

Imagine feeling constantly drained and finding it hard to focus or remember things, all while dealing with a liver condition. This is the reality for many people with Primary Biliary Cholangitis (PBC), a long-term liver disease that affects how well your liver works. This study focuses on two common and difficult symptoms of PBC: extreme tiredness, called fatigue, and problems with thinking, learning, and remembering things clearly, known as cognitive dysfunction.

Researchers are testing a new drug called golexanolone. This medicine was developed because scientists believe it might help with thinking problems related to long-term liver conditions. Based on earlier research in animals and in people with a different liver condition called cirrhosis, there's hope that golexanolone could help improve both the thinking difficulties and the fatigue experienced by people with PBC. The main goals of this study are to see how safe golexanolone is, how it's processed by the body, and most importantly, if it can make a difference to these challenging symptoms compared to a dummy pill.

By taking part, you would be helping doctors and scientists learn more about PBC and whether this new treatment could help people in the future. Even if it doesn't directly help you, your participation is very valuable to medical science.

Key takeaways

Tests a new medicine, golexanolone, for PBC.
Aims to help with fatigue and thinking problems.
Compares golexanolone to a dummy pill (placebo).
Involves clinic visits, tests, and questionnaires.
Participation helps future PBC treatments.
Your doctor will decide if you can join.

Who may be eligible?

This study is looking for adults aged 18 or over who have been diagnosed with Primary Biliary Cholangitis (PBC). You'll also need to be experiencing significant tiredness and have problems with your thinking or memory, as assessed by a doctor. Your current PBC treatment should also be stable for at least three months before joining.

There are certain health conditions or medicines that might mean you can't join the study. For example, if your liver disease is very severe, or you have certain heart conditions, or if you are pregnant or planning to become pregnant, you might not be able to take part. Women who could become pregnant must agree to use reliable birth control during the study.

Ultimately, a study doctor will carefully review your health information to ensure your safety and that the study is a good fit for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have a diagnosis of Primary Biliary Cholangitis (PBC)?
Do you experience significant tiredness (fatigue)?
Do you sometimes have trouble with your memory or thinking clearly?
Has your current PBC treatment been stable for at least 3 months?
Are you generally in good health otherwise?

Answer every question to see your result.

What does participation involve?

If you join the study, it will last for up to 8 weeks. You'll have 5 visits to a clinic and receive 2 phone calls during this time. Throughout the study, you'll take either golexanolone (at one of two doses) or a dummy pill (placebo) by mouth twice a day for 28 days.

During your clinic visits, you can expect several checks and tests. These include providing your basic health information, having a physical examination, and giving blood and urine samples. You'll also be asked questions about your general health and fill out questionnaires related to your fatigue, anxiety, mental well-being, and sleep. You'll have a longer interview (around 30-45 minutes) with a health professional not involved in treating you directly, which will be video-recorded, to talk about your condition. You will also do some tests to check your memory and thinking skills, like recalling words. You'll also be asked to keep a diary of when you take your study medicine and any health changes you notice.

Potential risks and benefits

Taking part in this study may or may not directly benefit your health. While early research suggests golexanolone might help with tiredness and thinking problems in PBC, there's no guarantee the study drug will improve your condition. If you receive the dummy pill (placebo), there will be no direct medical benefit. However, your participation will provide important information that could help doctors understand and treat PBC better in the future, benefiting others. All medical studies carry some risks, such as side effects from the medication or discomfort from tests; these will be fully explained to you before you decide to join. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (38)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

The Newcastle upon Tyne Hospitals NHS Foundation Trust
City only
Newcastle upon Tyne, England
Nottingham University Hospitals NHS Trust - Queen's Medical Centre Campus
City only
Nottingham, England
Portsmouth Hospitals University NHS Trust
City only
Portsmouth, England
Royal Free London NHS Foundation Trust
City only
London, England
The Royal Wolverhampton NHS Trust
City only
Wolverhampton, England
NIHR Birmingham BRC
Unverified
Birmingham, England
Glasgow Royal Infirmary
Unverified
Glasgow, Scotland
Guy's and St Thomas' Hospital, London
Unverified
London, England
Oxford University Hospitals NHS Foundation Trust - Octave Duo Covid19 Trials
Unverified
Oxford, England
University Hospital Düsseldorf
Unverified
Düsseldorf, Germany
University of Leipzig
Unverified
Leipzig, Germany
University Hospital of Patras
Unverified
Patras, Greece

Common questions

What is Primary Biliary Cholangitis (PBC)?

PBC is a long-term liver disease that slowly damages the bile ducts in the liver, which can affect how well your liver works.

What is 'cognitive dysfunction'?

This term means having difficulties with thinking, focusing, remembering, or learning new things clearly.

What is a 'placebo'?

A placebo is a dummy pill that looks exactly like the study drug but contains no active medicine. It helps researchers see if the real drug is making a difference.

Will I know if I'm getting the real drug or the placebo?

No, this is a 'blinded' study, meaning neither you nor your study doctor will know whether you are receiving golexanolone or the placebo. This helps ensure unbiased results.

How long will I be in the study?

Your involvement in the study will last for a maximum of 8 weeks, including clinic visits and phone calls.

How to find out more

Pernilla Sandwall

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

Always speak to your GP or specialist before deciding to take part in a study.