Not recruitingRare disease

Study on Golexanolone for Patients with Primary Biliary Cholangitis Experiencing Fatigue and Cognitive Dysfunction

Verified siteInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition called Primary Biliary Cholangitis (PBC), which is a disease that affects the liver. People with PBC often experience significant tiredness and problems with thinking clearly. The trial is testing a new treatment called Golexanolone, which is taken as a capsule. The study will compare the effects of Golexanolone with a placebo to understand its safety, how well it is tolerated, and its initial effectiveness in treating symptoms of PBC.

The study is divided into two parts. In the first part, participants will take a 40 mg dose of Golexanolone twice a day for five days. This part aims to see how safe the treatment is and how well participants can handle it. In the second part, participants will take two different dose levels of Golexanolone twice a day for 28 days. This part will also focus on safety and tolerance, as well as any changes in symptoms like fatigue and cognitive issues.

Throughout the study, researchers will monitor participants for any side effects and changes in their health. They will also look at how the treatment affects quality of life, including aspects like sleepiness and cognitive function. The goal is to gather information that could help improve treatment options for people with PBC in the future.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
Upon joining the study, an initial assessment will be conducted to confirm eligibility. This includes verifying age, medical history, and current health status.
A diagnosis of primary biliary cholangitis (PBC) will be confirmed through specific tests, such as antibody tests and liver biopsy results.
A pregnancy test will be required for women of childbearing potential.
Step 2
Part a treatment
In Part A, participants will receive golexanolone at a dose of 40 mg, taken orally twice a day (BID) for 5 days.
Participants will be monitored for any side effects and changes in health through regular check-ups and laboratory tests.
Step 3
Part a evaluation
After completing the 5-day treatment, an evaluation will be conducted to assess the safety and tolerability of the medication.
Participants will undergo tests to measure any changes in health and to identify any adverse events.
Step 4
Part b treatment
In Part B, participants will receive one of two dose levels of golexanolone, taken orally twice a day (BID) for 28 days.
Regular monitoring will continue to ensure safety and to track any side effects or health changes.
Step 5
Part b evaluation
At the end of the 28-day treatment, a comprehensive evaluation will be conducted.
This includes assessing changes in quality of life, cognitive function, and any adverse events experienced during the trial.

Who can join the trial?

12 criteria

Men and women aged between 18 and 75 years.
Willing and able to provide informed consent, meaning you understand the study and agree to participate.
You should be clear-minded and aware of who you are, where you are, the time, and the situation when giving consent.
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Diagnosed with Primary Biliary Cholangitis (PBC) based on at least two of the following:
- Presence of specific antibodies related to PBC.
- Higher than normal levels of a liver enzyme called alkaline phosphatase (ALP).
- A liver biopsy that matches PBC.
- Experiencing significant fatigue, measured by a specific score of 29 or higher in a fatigue questionnaire.
- Experiencing significant cognitive symptoms, measured by a specific score of 16 or higher in a cognitive questionnaire.
- If you are on standard PBC treatment, it should be stable for at least 3 months before joining the study. This treatment may include medications like UDCA, OCA, bezafibrate, and/or fenofibrate.
- Women who can become pregnant must have a negative pregnancy test before starting the study and agree to use a reliable birth control method during the study and for one month after.
- Women who cannot become pregnant must have proof of surgical sterilization, such as tubal ligation or hysterectomy, or be post-menopausal (no periods for 12 months).
- Men who can father children must agree to use condoms and ensure their female partner uses a reliable birth control method to prevent pregnancy during the study and for one month after. Men who are surgically sterile do not need to follow these birth control rules.

Who cannot join the trial?

7 criteria

Patients with a history of any other liver disease besides Primary Biliary Cholangitis (PBC) cannot participate.
Patients who have severe liver damage classified as Child-Pugh class B or C are excluded. Child-Pugh class is a system used to measure the severity of liver disease.
Patients with any other serious medical condition that might interfere with the study are not allowed to join.
Patients who are pregnant or breastfeeding cannot take part in the study.
Patients who have participated in another clinical trial within the last 30 days are excluded.
Patients with a known allergy or sensitivity to the study medication or its ingredients cannot participate.
Patients who are unable to comply with the study procedures or follow-up visits are not eligible.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
ASST Grande Ospedale Metropolitano Niguarda
Milan, Italy
Verified
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Palermo, Italy
Verified
Azienda Ospedaliera di Padova
Padua, Italy

Trial locations

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Investigated drugs

Golexanolone is a medication being studied for its potential to help people with primary biliary cholangitis, a liver disease that can cause fatigue and problems with thinking. The trial is looking at how safe and tolerable golexanolone is for patients, as well as how it might help with their symptoms. Participants in the study will take golexanolone to see if it can improve their energy levels and cognitive function, which are often affected by this condition.

Golexanolone

What is already known about the treatment

Golexanolone – Golexanolone is administered orally in the form of tablets, taken twice a day (BID). It is currently being studied in clinical trials to evaluate its safety, tolerability, and preliminary effectiveness in treating primary biliary cholangitis (PBC), particularly in patients experiencing fatigue and cognitive dysfunction. The medication works by targeting specific pathways in the brain that may be involved in these symptoms, although the exact molecular mechanism is still under investigation. Golexanolone is classified as a potential therapeutic agent for liver-related conditions, specifically focusing on improving quality of life for PBC patients.

Investigated diseases

Primary biliary cholangitis – Primary biliary cholangitis is a chronic disease that slowly destroys the bile ducts in the liver. Over time, the damage to these ducts can lead to a buildup of bile, which can harm the liver. The disease often begins with inflammation in the small bile ducts, which can progress to scarring and eventually lead to liver damage. Symptoms may include fatigue, itching, and dry eyes and mouth. As the disease progresses, it can cause more severe symptoms related to liver function. The progression of the disease can vary greatly among individuals.