Clinical trials on Primary Biliary Cirrhosis

Overview of Primary Biliary Cirrhosis

Primary Biliary Cirrhosis (PBC), now more commonly referred to as Primary Biliary Cholangitis, is a chronic liver disease characterized by the progressive destruction of the bile ducts within the liver. This autoimmune condition leads to a buildup of bile, causing liver inflammation and eventually fibrosis, which can progress to cirrhosis and liver failure if left untreated. The exact cause of PBC remains unknown, but it is believed to involve a combination of genetic predisposition and environmental factors triggering an abnormal immune response against the bile duct cells.

The diagnosis of PBC is often made through a combination of symptoms, blood tests showing elevated levels of liver enzymes and antibodies specific to the condition, and sometimes liver biopsy. Common symptoms include fatigue, itching (pruritus), dry eyes and mouth, and right upper quadrant abdominal pain. However, many patients may be asymptomatic in the early stages of the disease. Treatment focuses on slowing the disease progression, managing symptoms, and monitoring for complications. Ursodeoxycholic acid (UDCA) is the primary medication used, which can improve liver function and may delay the need for liver transplantation in advanced cases.

Living with PBC requires regular monitoring and a proactive approach to managing symptoms and preventing complications. Lifestyle changes, such as a healthy diet, regular exercise, and avoiding alcohol, are recommended to support liver health. Additionally, patients may benefit from joining support groups and educational programs to better understand their condition and connect with others facing similar challenges. With appropriate management, many individuals with PBC can lead a normal life expectancy, although close monitoring and treatment adjustments are essential to manage the disease effectively over time.

Prognosis for Primary Biliary Cirrhosis

Primary Biliary Cirrhosis (PBC) is a chronic liver disease characterized by the progressive destruction of the bile ducts within the liver. The long-term prospects for individuals with PBC can vary significantly. The disease tends to progress slowly, and it may remain asymptomatic for years, with a normal life expectancy for many. However, without intervention, PBC can eventually lead to liver scarring, known as cirrhosis, and its associated complications. The rate of disease progression is highly individual, influenced by various factors including age at diagnosis, overall health, and response to any potential treatments. In advanced stages, PBC may result in liver failure, necessitating consideration for liver transplantation. Early detection and monitoring are crucial in managing the disease’s progression and improving the long-term outlook. Regular follow-up care is important to assess liver function and manage the condition effectively.

Complications in Primary Biliary Cirrhosis

Primary Biliary Cirrhosis (PBC) may lead to several complications that significantly impact health and daily living. Common issues include:

  • Fatigue: Can render even simple tasks overwhelming.
  • Itching: Often severe and persistent, causing discomfort and sleep disturbances.
  • Jaundice: Characterized by yellowing of the skin and eyes due to liver impairment.
  • Ascites: Fluid accumulation in the abdomen, which can be uncomfortable and may lead to breathing difficulties.
  • Osteoporosis: Weakened bones, increasing the risk of fractures.
  • Digestive problems: Due to the liver’s reduced ability to process fats, leading to vitamin deficiencies and related health issues.

These complications can significantly affect the quality of life, making daily activities and overall well-being more challenging to maintain.

Treatment Methods for Primary Biliary Cirrhosis

For the management of Primary Biliary Cirrhosis, several non-clinical trial treatments are recommended:

  • Dietary adjustments: Reduction of salt and the avoidance of alcohol, incorporation of fruits, vegetables, and whole grains, along with the limitation of processed foods.
  • Regular physical activity: Tailored to the capacity of the individual, aids in maintaining a healthy weight and reducing liver strain.

Pharmacotherapy options include medications that may be prescribed to manage symptoms and slow the progression of the disease. Ursodeoxycholic acid is commonly used to improve liver function. Other medications may be recommended based on specific symptoms or complications.

Modern technology offers tools for the monitoring of health and the management of treatment plans. Mobile apps can track medication schedules, dietary intake, and exercise routines. Wearable devices can monitor vital signs and activity levels, providing data that can assist in personalizing treatment approaches.

The adoption of these lifestyle changes, the utilization of pharmacotherapy, and the leveraging of modern technology can collectively contribute to the effective management of Primary Biliary Cirrhosis.

  • CT-EU-00036665

    Evaluating itch relief in liver disease with linerixibat

    The GLISTEN study is a detailed research project exploring the effectiveness of a medication called linerixibat in treating severe itching in people with a liver condition known as Primary Biliary Cholangitis (PBC). In this study, participants are given either linerixibat or a placebo to see which one better reduces itching and improves sleep and overall quality of life. This research is important for finding new ways to help people with PBC who experience discomfort from itching.

    • Linerixibat
  • Effectiveness of setanaxib in primary biliary cholangitis

    This clinical trial aims to study the effectiveness of a new medication called setanaxib on patients suffering from primary biliary cholangitis (PBC), a type of liver disorder, and with elevated liver stiffness. The drug is being tested for its ability to improve patients’ health over a course of a year. Participants of the study, adults aged 18 years and older, are either not responding adequately or are intolerant to a common PBC treatment, Ursodeoxycholic acid (UDCA). The study employs a randomized double-blind method, meaning patients are randomly placed into groups and neither patients nor researchers know who gets the actual drug or a placebo. The trial will last 52 weeks with an extension phase that could last an additional year. 

    • Setanaxib