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, fatigue, and cognitive dysfunction.
Primary biliary cholangitis (PBC) is a long-term liver condition. Even when liver treatments work well, many people still live with disabling fatigue, daytime sleepiness, and difficulties with concentration or memory, often referred to as 'brain fog'. This clinical trial tests an experimental capsule called golexanolone to find out if it can safely improve these daily symptoms. The trial is split into two parts. In the first part, doctors check how safe the medicine is over five days and how the body handles it. In the second part, participants take the capsules for 28 days. Because this is a placebo-controlled trial, some people will take the real medicine while others take a dummy capsule that looks identical. Researchers will monitor safety, run blood tests, and measure changes in memory, energy levels, and overall quality of life.
At a glance
What is this study about?
Primary biliary cholangitis, or PBC, is an autoimmune condition where the body mistakenly attacks the tiny bile ducts inside the liver. Over time, this causes bile to build up, which can damage liver tissue. While current treatments can slow liver damage, they rarely fix other exhausting symptoms. Many people with PBC struggle daily with extreme tiredness, poor sleep, and 'brain fog'—problems with memory, focus, and quick thinking that make everyday life much harder.
This clinical trial is evaluating an experimental drug called golexanolone (also known as GR3027). Scientists believe that certain chemical signals in the brain become unbalanced in chronic liver conditions, contributing directly to mental fatigue and memory problems. Golexanolone is designed to target these brain signals to see if it can clear the mental fog and restore energy.
Taking part in this study helps researchers find out two crucial things: whether golexanolone is safe to take, and whether it genuinely improves day-to-day well-being. If successful, this research could lead to one of the first targeted treatments specifically designed to tackle the debilitating mental and physical exhaustion of PBC, offering hope for better symptom relief in the future.
Key takeaways
- Tests a new medicine, golexanolone, designed to ease fatigue and 'brain fog' in PBC.
- Open to adults aged 18 and older living with primary biliary cholangitis.
- Involves taking daily capsules for either 5 days (Part A) or 28 days (Part B).
- Uses memory tests and symptom questionnaires to see if quality of life improves.
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
This trial is open to adults aged 18 and over who have been diagnosed with primary biliary cholangitis (PBC). To be considered, you must also be experiencing significant fatigue and thinking or memory troubles related to your liver condition.
Both men and women can join. Because this is an early-stage safety trial, the study team will want to make sure your liver disease and any other health conditions are stable before you take part.
You may not be eligible if you have certain other severe medical conditions, if you are pregnant or breastfeeding, or if you are taking specific medications that could interfere with the study drug. The trial doctor will review your full medical records and run routine health checks to ensure taking part is safe 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.
- I am aged 18 or older.
- I have a confirmed medical diagnosis of primary biliary cholangitis (PBC).
- I struggle with persistent tiredness, fatigue, or daytime sleepiness.
- I experience 'brain fog', forgetfulness, or trouble concentrating.
- I am able and willing to attend clinic visits and swallow daily capsules.
What does participation involve?
If you join the trial, you will be invited to a clinic for health checks, including physical exams, blood tests, and urine tests. You will take the study capsules by mouth. The study is divided into two phases: Part A checks safety over 5 days, while Part B tests the drug over 28 days.
During your visits, doctors will measure how the drug breaks down in your body. You will also complete short memory and thinking games (such as word recall and problem-solving puzzles) and fill out questionnaires about your tiredness, daytime sleepiness, itchiness, and mood. The trial team will track your health closely throughout the study and for a short follow-up period afterwards.
Potential risks and benefits
Locations (5)
- —UnverifiedHungary
- —UnverifiedSpain
- —UnverifiedItaly
- —UnverifiedGermany
- —UnverifiedGreece
Common questions
What is a placebo?
A placebo is a harmless dummy capsule that looks exactly like the real medicine but contains no active ingredients. It helps researchers prove whether any improvements are truly caused by the study drug.
Can I continue taking my normal PBC medicines?
In most cases, yes. You can usually continue standard treatments such as ursodeoxycholic acid (UDCA), but the study doctors will check all your regular medicines beforehand to make sure they are safe to combine.
Will taking part cost me anything?
No, the study medication and all trial tests are provided free of charge. Most trials also reimburse reasonable travel expenses for hospital visits.
How long will I be in the study?
Depending on which part of the trial you join, you will take the study medication for either 5 days or 28 days, alongside some time before and after for screening and follow-up visits.
What happens if I change my mind?
You can leave the study at any time without giving a reason. Deciding to withdraw will never affect your relationship with your doctors or the quality of your normal medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.