Primary Biliary Cholangitis?
A clinical study is evaluating an investigational product as a potential additional treatment for primary biliary cholangitis (PBC).
The purpose of this clinical research study is to evaluate the effects of an investigational product (setanaxib) as a potential additional treatment for PBC.
There will be about 318 participants enrolled in the study worldwide. During the Main Treatment Period of the study, participants will be randomised into one of three oral treatment groups to receive one of the following:
- Setanaxib 1200 mg/day
- Setanaxib 1600 mg/day
- Placebo (a pill that contains no active drug)
This part of the study is double-blind. This means that neither you nor the study doctor will know which treatment you are taking. Studies are blinded to help prevent any bias in the results.
After the Main Treatment Period of the study, there will be an Extension Phase Treatment Period. During this phase all participants will receive setanaxib in a dose of either 1200 mg/day or 1600 mg/day.
You may be eligible to participate in the TRANSFORM Study if you:
- Are age 18 or older.
- Have liver disease and liver stiffness.
- Have taken ursodeoxycholic acid (UDCA) to treat PBC for the past 6 months (and have been on a stable dose for 3 months) or cannot take UDCA and your last dose of UDCA was at least 3 months ago.
Note: If you have taken obeticholic acid (OCA), fenofibrate or bezafibrate to treat PBC, you must have been taking these agents for at least 6 months and on a stable dose for the past 3 months.
Participation in the study will last about 120 weeks, or about 2 years and 4 months. The study is divided into the following periods:
Screening Period (4 weeks):
- The study doctor will do some tests to see if you are eligible for the study.
Main Treatment Period (52 weeks)
- You will be randomly assigned to one of three oral treatment groups. Neither you nor the study staff will know which treatment you will receive. Depending on your treatment group, you will take:
- Setanaxib 1200 mg/day
- Setanaxib 1600 mg/day
- Placebo (a pill that contains no investigational product)
- During this period of the study, you will have up to 8 visits with the study doctor. You will also have 7 phone call visits with the study doctor.
- If you complete the 52-week Main Treatment Period, you may enter the Extension Phase Treatment Period.
Extension Phase Treatment Period (52 weeks)
- In the Extension Phase Treatment Period, all participants will receive setanaxib. Your dose of setanaxib will be either 1200 mg/day or 1600 mg/day.
- During the Extension Phase Treatment Period, you will have a phone call with the study staff 2 weeks after you start the Extension Phase Treatment Period, and you will have follow-up visits with the study staff every 3 months.
Follow-Up Period (12 Weeks)
- Once you have completed the Main Treatment Period and the Extension Phase Treatment Period, or if you decide to drop out of the study, you will have a Safety Follow-up Visit 30 days and 12 weeks after your last treatment dose.
- If you decline the Extension Phase Treatment Period, you will have an End-of-Treatment Visit and Follow-up Visit 30 days and 12 weeks after you complete the Main Treatment Period.
All eligible study participants will receive the following at no cost:
- The study investigational product (setanaxib) or placebo.
- Study-related visits, assessments and tests, such as urine and blood tests, an electrocardiogram (ECG), magnetic resonance elastography (MRE) and transient elastography (FibroScan).
- You may also be reimbursed for reasonable expenses such as travel costs for attending the study visits.
Some medications may interact with the investigational product and are not allowed during the study. Please ask the study doctor before taking any new medication.
Before a new therapy can be made available to the general public, multiple studies involving healthy volunteers and patient volunteers must be conducted to evaluate the safety and effectiveness of the therapy. These studies, referred to as clinical trials, are regulated by the Food and Drug Administration (FDA) in the United States and by similar regulatory agencies worldwide.
Your participation in the TRANSFORM Study will help us understand whether the active investigational product, setanaxib, is effective in treating adults with primary biliary cholangitis. A prior clinical study with setanaxib was conducted in PBC patients that included 111 participants.
Participation in this clinical research study is completely voluntary. Your decision to participate or not participate will have no effect on the medical care you receive now or in the future. You may leave the study at any time and for any reason.