Reviewed by Michael Gill, B. Sc.
Recently Completed Studies with FDA Approved Treatments for Primary Biliary Cirrhosis
Treatment
Year
Sponsor
HTD1801 (BUDCA)
2021
HighTide Biopharma Pty Ltd

What Are Primary Biliary Cirrhosis Clinical Trials?

Primary Biliary Cirrhosis is now known as Primary Biliary Cholangitis (PBC). It is a chronic condition of the liver. In PBC, the small ducts of the bile in the patient's liver become inflamed. Inflammation further damages the ducts. It causes bile to build up, resulting in liver damage, such as scarring, cirrhosis, and liver failure. It is thought of as an autoimmune disease. Therefore, the immune system attacks the healthy bile duct cells.

It is a progressive liver disorder with initial symptoms like fatigue, jaundice, and pruritus. However, as the disease progresses, patients may experience issues such as:

  • Abdominal pain
  • Small white/yellow bumps on the skin and around their eyes
  • Swelling of the feet and legs
  • Internal bleeding

Primary Biliary Cirrhosis clinical trials are studies conducted to understand the condition better and explore novel treatments, their efficacy, and potential side effects.

Why Is Primary Biliary Cirrhosis Being Studied Through Clinical Trials?

The PBC condition is more common in females than males. Research shows that nearly 65 out of 100,000 women are diagnosed with PBC. Initially affecting people in their middle age, the average age of the patient at the time of diagnosis is 60. Moreover, the condition is also found in white people more than in ethnic and racial groups.

Therefore, Primary Biliary Cirrhosis clinical trials are underway to determine the best methods for early diagnosis, prevention, and cure. Moreover, these trials provide quality insights into determining the condition's root cause. Before it is widely available, new medications, medical devices, and therapies are assessed and tested through clinical trials for efficacy and adverse reactions.

What Are the Types of Treatments Available for Primary Biliary Cirrhosis?

Though there is no cure for the condition, drugs can slow the progression of primary biliary cirrhosis and control its symptoms. For example, Ursodiol is available under different brand names, which replicate the bile acid (ursodeoxycholic acid or UDCA) that occurs naturally. It helps move the bile from the liver into the intestine. This medication is effective in improving liver function.

Patients who don't completely respond to Ursodiol may be prescribed medication to suppress their immune system. However, a liver transplant may also be advised for patients with PBC when the disease can't be managed or controlled. This procedure may be required for survival.

What Are Some Recent Breakthrough Clinical Trials for Primary Biliary Cirrhosis?

2016: A Placebo-Controlled Trial of Obeticholic Acid in Primary Biliary Cholangitis- The study assessed 217 patients for 12 months in a phase three trial. These patients did not respond well to Ursodiol. Hence, they received an obeticholic acid dose of 10mg or a placebo. It was concluded that obeticholic acid as monotherapy or combined with Ursodiol decreased alkaline phosphatase and bilirubin levels.

2020: Experimental Pharmacological Agents for the Treatment of Primary Biliary Cholangitis- This clinical review provides an overview of the experimental pharmacological agents used for PBC treatment. It concluded by stating that bezafibrate, in combination with UDCA, improved the biochemistry of the patients. Hence, combination treatments with different agents seem promising in managing biochemistry and PBC disease symptoms.

Who Are Some of the Key Opinion Leaders/Researchers/Institutions Conducting Primary Biliary Cirrhosis Clinical Trial Research?

American Liver Foundation

Serving for more than 46 years, ALF is dedicated to promoting research and support services for the diagnosis, prevention, and treatment of liver diseases.

Indiana University School of Medicine

It is committed to transforming health care with medical education and ground-breaking clinical research. Physicians and scientists at the IU institute carry out research studies to develop a better understanding of PBC.

About The Author

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 17th, 2021

Last Reviewed: November 19th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.