Study to Evaluate the Efficacy and Safety of K-808 (Pemafibrate) in Participants With Primary Biliary Cholangitis (PBC) With Inadequate Response to Ursodeoxycholic Acid (UDCA) and/or Obeticholic Acid (OCA) Treatment.
This research study is looking at a new medicine called K-808 for a long-term liver illness known as Primary Biliary Cholangitis, or PBC. PBC causes damage to the small bile ducts in your liver. We are recruiting people who already take standard PBC medicines like ursodeoxycholic acid (UDCA) or obeticholic acid (OCA), but whose condition hasn't fully improved. The study aims to find out if two different doses of K-808 are effective and safe in helping to manage PBC. Some participants will receive one of the K-808 doses, while others will receive a placebo (a dummy drug with no active medicine) to compare the results. This is a Phase 2 study, meaning it's an earlier stage of testing to see if the drug works and is safe enough for further research.
At a glance
What is this study about?
This study is about a condition called Primary Biliary Cholangitis, often shortened to PBC. PBC is a long-term liver disease where the tiny bile ducts inside your liver slowly get damaged. These ducts usually help carry a fluid called bile that aids digestion. When they're damaged, bile can build up and harm the liver over time. Medicines like Ursodeoxycholic Acid (UDCA) and Obeticholic Acid (OCA) are commonly used to treat PBC.
However, for some people, these treatments don't fully control the condition. This study is testing a new medicine, K-808, to see if it can help these individuals. We want to find out if K-808 can improve how people with PBC are doing and if it is safe to use. We'll be looking at two different amounts (doses) of K-808.
Since this is a 'Phase 2' study, it means we are in the earlier stages of testing this new medicine. We're trying to understand if it works, how much of it might be needed, and to check its safety before potentially moving on to larger studies. Your participation could help us learn more about treating PBC in the future.
Key takeaways
- This study is for adults with Primary Biliary Cholangitis (PBC).
- It's testing a new medicine called K-808 for those not fully responding to current treatments.
- Participants will receive K-808 or a placebo, alongside their usual PBC medication.
- Regular clinic visits and blood tests will be required to monitor health.
- It's a Phase 2 study, focused on safety and how well the new medicine works.
- Participation is voluntary, and you can leave the study at any time.
Who may be eligible?
To join this study, you must be 18 years old or older. You'll need to have a confirmed diagnosis of Primary Biliary Cholangitis (PBC). This diagnosis is usually made by a doctor based on your medical history, specific blood tests (like having positive antibodies often linked to PBC, or high levels of alkaline phosphatase – ALP), or a liver biopsy.
For this study, your blood test results for ALP, a liver enzyme, must be at least one and a half times higher than the normal range, even if you're already on standard PBC medication. This indicates your PBC isn't fully controlled by your current treatment.
There are also some reasons why you might not be able to join. For example, if your liver tests are extremely high (very high ALP, ALT, or AST levels), or if you have other serious liver conditions like alcoholic liver disease, active Hepatitis B or C, or another condition causing bile duct blockages. We also can't include people with certain other genetic liver conditions or autoimmune diseases that might make the study results unclear or put your health at risk. The study team will review all your medical information carefully to make sure it's safe and appropriate for you to participate.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 18 years old or older?
- Do you have a diagnosis of Primary Biliary Cholangitis (PBC)?
- Are your liver enzyme (ALP) levels still high despite current PBC treatment?
- Do you have any other significant liver conditions or very high liver test results?
- Do you have a history of certain other medical conditions (e.g., severe heart disease, uncontrolled diabetes, active hepatitis)?
What does participation involve?
If you decide to join this study, you would first have a screening visit to check if you meet all the requirements. This will involve blood tests and a review of your medical history. Once confirmed, you would start taking either one of the two doses of the new medicine (K-808) or a placebo (a dummy pill that looks like the study drug but contains no active medicine). You would continue taking your regular PBC medications (like UDCA or OCA) as prescribed by your doctor during the study.
The study involves regular visits to the clinic for check-ups, more blood tests to monitor your liver function and overall health, and to make sure the medicine is safe. The frequency and total number of visits would be explained in detail by the study team. You would also be asked to report any new symptoms or side effects you experience. The total duration of your participation in the study, including all follow-up, will be clearly explained by the research team.
Potential risks and benefits
Locations (38)
- UA Thomas D. Boyer Liver InstituteVerified postcodeTucson, United States
- Southern California Research Center - CoronadoVerified postcodeCoronado, United States
- Cedars-Sinai Medical CenterVerified postcodeLos Angeles, United States
- Velocity Clinical ResearchVerified postcodeSanta Ana, United States
- Peak Gastroenterology Associates Colorado SpringsVerified postcodeColorado Springs, United States
- University of Florida Hepatology Research at CTRBVerified postcodeGainesville, United States
- Florida Research InstituteVerified postcodeLakewood Rch, United States
- University of Miami Leonard M. Miller School of MedicineVerified postcodeMiami, United States
- Springfield ClinicVerified postcodeSpringfield, United States
- Mercy Medical Center - Mcauley PlazaVerified postcodeBaltimore, United States
- CommonSpirit Health Research InstituteVerified postcodeOmaha, United States
- New York University Hepatology AssociatesVerified postcodeNew York, United States
Common questions
What is Primary Biliary Cholangitis (PBC)?
PBC is a long-lasting liver disease where the tiny tubes in your liver that carry bile get damaged. This can affect how your liver works over time.
What is K-808?
K-808 is a new medicine being tested. We hope it might help people with PBC whose current treatments aren't fully managing their condition.
What is a placebo?
A placebo is a 'dummy' pill that looks just like the study medicine but doesn't contain any active drug. It helps us compare the new medicine's effects accurately.
Will I have to stop my current PBC medicine?
No, you will continue taking your current PBC medicines, like Ursodeoxycholic Acid (UDCA) or Obeticholic Acid (OCA), as normally prescribed by your doctor.
What is a Phase 2 study?
A Phase 2 study is an early stage of testing a new medicine. It helps us see if the drug works, what dose might be best, and to check for any safety concerns.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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