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
This research is looking into a new medication called golexanolone for adults diagnosed with Primary Biliary Cholangitis (PBC). The main goal is to help those who experience significant tiredness and issues with memory or clear thinking due to their PBC. The study is split into two parts: first, a short-term check of safety and how the medicine moves through the body. Then, a longer part compares two different doses of golexanolone with a dummy pill (placebo) over 28 days. Researchers want to know if golexanolone is safe, well-tolerated, and can improve tiredness, daytime sleepiness, thinking abilities, and overall quality of life for people with PBC.
At a glance
What is this study about?
This study is about an illness called Primary Biliary Cholangitis, or PBC. It's a long-term liver condition. Many people with PBC feel very tired and can have trouble with their memory or concentration. This research is testing a new medicine called golexanolone to see if it can help with these specific problems.
The study has two main parts. The first part is shorter and focuses on understanding how safe the medicine is and how the body handles it over five days. The second part is longer, lasting 28 days. In this part, some people will get golexanolone, and others will get a 'dummy' pill (placebo). This helps the researchers find out if the medicine is truly making a difference. During this part, they will look at different doses of the medicine.
The main aim is to check if golexanolone is safe and if it can improve how tired people feel, reduce daytime sleepiness, help with clear thinking, and generally improve their daily life. The study design ensures that neither you nor your doctor will know if you are receiving the active drug or the placebo, which helps to get unbiased results.
Key takeaways
- Tests new medicine (golexanolone) for PBC fatigue and thinking problems.
- Compares golexanolone to a dummy pill (placebo).
- Focuses on safety and whether symptoms improve.
- Involves regular clinic visits and health checks.
- Participation is for adults with PBC and specific symptoms.
Who may be eligible?
To join this study, you need to be an adult, aged 18 or older, and have a confirmed diagnosis of PBC. A key part of joining is that you must be experiencing significant tiredness and problems with your thinking, memory, or concentration due to your PBC. If you are already taking medication for your PBC, it needs to have been a stable dose for at least three months.
There are also some reasons why you might not be able to join. For example, if your liver disease is very severe, or if you have other serious health problems like certain heart conditions, a known history of hepatitis B or HIV, or very low blood levels of certain vitamins. Certain levels of bilirubin (a substance in your blood) or kidney function issues could also mean you're not suitable.
If you are a woman who could become pregnant, you'll need to use very effective birth control during and for a month after the study. Men capable of fathering a child will also need to use condoms and ensure their partner uses birth control. This is to make sure no one becomes pregnant during the study, as the effects of the new medicine on pregnancy are unknown.
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) and problems with concentration or memory because of your PBC?
- If you take PBC medication, has your dose been stable for at least 3 months?
- Are you able to attend study appointments regularly?
What does participation involve?
If you decide to take part in this study, you will first go through a screening process to make sure you fit all the necessary criteria. Once you've been accepted, you would be randomly assigned to either receive golexanolone (at one of two possible doses) or a placebo (a dummy pill). You will take the study medication twice a day.
For the first part of the study, you'd take the medication for 5 days. For the second part, it would be for 28 days. Throughout this time, you'll have regular visits to the clinic where you'll be assessed. These assessments will involve giving blood samples, physical examinations, and completing questionnaires about your tiredness, sleepiness, how you're thinking, and your general quality of life. The doctors will also keep a close eye on your health to check for any side effects. You'll need to attend follow-up appointments after you stop taking the medication.
Potential risks and benefits
Locations (40)
- University Hospital DüsseldorfVerified postcodeDüsseldorf, Germany· Recruiting
- University of LeipzigVerified postcodeLeipzig, Germany· Recruiting
- Hippokration General Hospital of AthensVerified postcodeAthens, Greece· Recruiting
- University Hospital of PatrasVerified postcodePátrai, Greece· Recruiting
- Bekes County Central HospitalVerified postcodeGyula, Hungary· Withdrawn
- Facility of CRU Hungary Ltd.Verified postcodeKistarcsa, Hungary· Completed
- Università di Milano-Bicocca, S.C. ASST Grande Ospedale Metropolitano Niguarda, Dipartimento di Medicina e Chirurgia, Epatologia e GastroenterologiaVerified postcodeMilan, Italy· Recruiting
- Fondazione IRCCS San Gerardo dei Tintori, Autoimmune Liver Disease Centre, ERN-Rare Liver, Department of Medicine and Surgery, Division of GastroenterologyVerified postcodeMonza, Italy· Not yet recruiting
- University of Padova, Department of Surgery, Oncology and GastroenterologyVerified postcodePadova, Italy· Recruiting
- University Hospital Paolo Giaccone, University of PalermoVerified postcodePalermo, Italy· Recruiting
- A. Gemelli Polyclinic, Sacro Cuore Catholic UniversityVerified postcodeRoma, Italy· Recruiting
- Humanitas UniversityVerified postcodeRozzano, Italy· Recruiting
Common questions
What is PBC?
PBC, or Primary Biliary Cholangitis, is a long-term liver condition where the small bile ducts in the liver are slowly destroyed.
What does a 'placebo' mean?
A placebo is a 'dummy' pill that looks just like the study medicine but contains no active drug. It helps researchers compare if the real medicine is having an effect.
What are 'fatigue' and 'cognitive dysfunction'?
Fatigue means extreme tiredness that doesn't get better with rest. Cognitive dysfunction refers to problems with memory, concentration, or clear thinking.
Will I know if I'm getting the real medicine?
No, this is a 'double-blind' study, meaning neither you nor your doctor will know if you're receiving the study medicine or the placebo until after the study ends.
What does 'stable PBC medication' mean?
It means if you take medication for your PBC, you need to have been on the same dose for at least three months without any changes.
How to find out more
Pernilla Sandwall
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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