An on-going study of an investigational drug for primary biliary cholangitis has been given the go-ahead to continue by a safety monitoring board. The planned review recommended that the Phase 2 study continues without changing the study protocol. For more detailed information about this, you can read the source news release here, at Business Wire.

About Primary Biliary Cholangitis

Primary biliary cholangitis (PBC), often called primary biliary cirrhosis, is a type of liver disease that may get worse over time, and, in some cases, can lead to liver failure. Some people with PBC don’t experience symptoms, whereas others may develop aches, fatigue, and itchiness, amongst other possible signs. PBC is caused by the body’s immune system mistakenly attacking bile ducts and damaging them, resulting in a build-up of bile in the liver. To find out more about PBC, click here.

The Study

A Phase 2 clinical trial is investigating the drug GKT831 for its potential use as a treatment for PBC. It is a 24-week double-blind study that will compare the effects of the drug to those of a placebo in approximately one hundred patients with PBC. All the patients who take part are given UDCA (ursodeoxycholic acid), which is a treatment for PBC, and then, depending on which of the three groups they are in, either a placebo, or GKT831 once per day, or GKT831 twice per day. For patients who complete the study, there are plans to begin an open-label extension study (if the safety data continues to be favourable), which would allow them to continue treatment with GKT831.

Recent Updates

Genkyotex, the company holding the study, has announced updates about the trial. According to their press release, a planned data review by an independent safety monitoring board has taken place. The board has recommended that the study continues without amending its protocol. This decision was based on data collected from 77 patients, 60 of who had undergone at least six weeks of treatment. Genkyotex also says that they expect interim data about the efficacy of the drug to be available in Fall 2018, and the final study results to be released in the first half of 2019.

Primary Biliary Cholangitis (PBC)

What is primary biliary cholangitis (PBC)?

Primary biliary cholangitis (PBC), formerly known as primary biliary cirrhosis, is a chronic disease of the liver characterized by a slow, progressive damage to the bile ducts located in the liver. Bile is a substance produced by the liver that is responsible for the digestion of fat and fat soluble vitamins in the intestine, and damage to these ducts prevents the flow of bile out of the liver and causes a buildup of bile and other substances within the liver.  The accumulation of these substances can lead to scarring (fibrosis) and advanced scarring (cirrhosis), which ultimately can lead to liver failure.

What are the symptoms of PBC?

Many people can go years without any symptoms of PBC. Early symptoms include:

  • Fatigue, often severe
  • Persistent itching of the skin, especially on the bottoms of the feet
  • Darkening of the skin
  • Small white bumps under the skin mainly around the eyes
  • Dry mouth
  • Muscle and joint pain

Advanced symptoms include:

  • Yellowing of the skin (jaundice)
  • Swelling of feet and lower legs
  • Chronic diarrhea
  • Internal bleeding of stomach and throat
  • Fragile bones
  • Underactive thyroid
  • Swollen distended abdomen due to fluid accumulation (ascites)

What causes PBC?

The exact cause of PBC is unknown. The condition is mainly thought to be due to problems with the immune system that cause the body to attack the bile ducts of the liver, and it is thought that an environmental cause may trigger this autoimmune response.  Examples of these triggers include exposure to toxins or infection. Though not known to be an inherited disease, PBC is more common in individuals with a family history. PBC is not related to alcohol consumption.

Are there treatment options available for PBC?

There is no known cure for PBC. Current available treatments can dramatically slow disease progression, help with symptoms, and help avoid complications, so early detection is key. Treatments include:

  • Medications to help with the elimination and proper traveling of bile through the liver. These medications include Ursodiol (brand name, Actigall) and Ubeticholic acid (brand name, Ocaliva)
  • Medication to help with itching
  • Vitamin supplements to help with jaundice
  • Avoidance of alcohol
  • Liver transplant for advanced cases

Where can I find more information about PBC?

PBC Clinical Practice Guidelines

Primary Biliary Cholangitis (PBC) Articles