FOR US RESIDENTS ONLY

If you are eligible for our studies, you have the option of participating at a clinic or from the comfort of your home.
If you travel, related expenses may be reimbursed as permitted by study site policies.

What is PBC?1

Primary biliary cholangitis (PBC) is a progressive inflammatory liver disease that causes the small bile ducts in the liver to become injured and inflamed—and eventually destroyed. When there are no bile ducts, bile builds up and causes liver damage, ultimately requiring a liver transplant for many patients.

While PBC affects mostly women, men may also develop this condition. The exact cause is unknown, but it may be linked with issues in the immune system. People with a family member who has PBC may also have a higher chance of developing it.


How is PBC diagnosed?2

Doctors may diagnose PBC based on your medical and family history, a physical exam, and the results of medical tests. Medical tests used to diagnose PBC may include blood tests, imaging tests, and liver biopsy.


Common symptoms of PBC1

Itch
Fatigue
Pain
Yellowing of the skin and eyes (jaundice)
Weak and brittle bones (osteoporosis)
Depression
Weight loss
Pruritus, or itching, is one of the most common symptoms reported by 80-100%
of patients with cholestatic liver disease, which includes PBC.3
Patients with PBC who itch don't have a rash. They often scratch all over their body to relieve their itch. The itch often affects their quality of life, leading to lack of sleep, depression, social isolation, and more.3
Treatments that may be recommended to try to control itch related to PBC.
These may not be effective for everyone.3
  • Antihistamines
  • Cholestyramine
  • Rifampin
  • Opioid antagonists
  • Sertraline

Enroll in our clinical study

In our clinical study, an investigational medicine is tested on volunteers to verify its safety and efficacy in itch associated with PBC compared to a placebo with no active ingredients.

Learn about the VANTAGE Study

What is a clinical study?4

A clinical study, also called a clinical trial, is a research test to see if an investigational medicine is safe and effective in human volunteers. It compares the investigational medicine to a placebo (a treatment with no active ingredients). These medicines have not been approved by the FDA (Food and Drug Administration) or other health organizations. However, they can still be given to people as part of the study. Participants play a vital role in advancing medicine for current and future generations.

Additional resources to help you learn more about PBC


Mirum Pharmaceuticals, Inc. is a biopharmaceutical company conducting clinical studies to investigate a potential treatment for itch associated with PSC and PBC in pediatric and adult individuals.

For more information about Mirum, please visit MirumPharma.com.

References

1. Mayo Clinic. Primary biliary cholangitis. Accessed February 10, 2025. https://www.mayoclinic.org/diseases-conditions/primary-biliary-cholangitis/symptoms-causes/syc-20376874. 2. National Institute of Diabetes and Digestive and Kidney Diseases. Primary biliary cholangitis. National Institutes of Health. Accessed February 12, 2025. https://www.niddk.nih.gov/health-information/liver-disease/primary-biliary-cholangitis. 3. Patel SP, Vasavda C, Ho B, Meixiong J, Dong X, Kwatra SG. Cholestatic pruritus: Emerging mechanisms and therapeutics. J Am Acad Dermatol. 2019;81(6):1371-1378. doi:10.1016/j.jaad.2019.04.035. 4. World Health Organization. Clinical trials. WHO. Accessed February 12, 2025. https://www.who.int/health-topics/clinical-trials#tab=tab_1.