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.
Frequently asked questions
Adults ages 18 or older who have been diagnosed with primary biliary cholangitis (PBC).
The Main Treatment Period of this study is double-blind. This means that during this part of the study neither you nor the study doctor will know if you are taking the investigational product or a placebo. During the Extension Phase Treatment Period, all participants will take the investigational product.
It is up to you to decide if you would like to participate in this phase or not. If you do not want to participate in the Extension Phase Treatment Period, your treatment will end and the study doctor will continue to monitor you for 12 weeks.
During a clinical study, you’ll receive the medication that is being studied. This will be done at scheduled times and may occur only once or multiple times throughout the study, depending on the medication. If you participate, you may also have the following procedures performed to monitor your health: vital signs, blood work, physical exams or other types of assessments, such as an electrocardiogram (ECG), magnetic resonance elastography (MRE) or a transient elastography (FibroScan), that are necessary to gather the required information about the study medication.
In the TRANSFORM Study, you may not receive the study medication, but instead receive a placebo. You will have a 2:1 chance of receiving the setanaxib or placebo in this study. All study volunteers are always closely monitored by clinic staff and the medical team to ensure their safety.
Placebo: A placebo looks like the investigational product being tested, but it doesn’t have any active ingredients. In placebo-controlled research, one group is given the inactive treatment, while another group is given the active treatment. This is the best way to find out if the treatment being tested works better than receiving no treatment at all.
Participation in any clinical study is completely voluntary. Your decision to participate – or not participate — in this clinical study will have no effect on the medical care that you receive now or in the future. If you are eligible and choose to participate in the study, you may leave the clinical study at any time, and for any reason.
Genkyotex may reimburse study participants for study-related travel and expenses. For more information, please speak with a member of the study staff during your study visits.
There are many reasons to participate in clinical research. For starters, it allows participants to play a more proactive role in their own health by receiving a new treatment for an ongoing disease or condition. Participants also play an important part in bringing much needed drugs to market, benefiting thousands of people who also have the disease or condition. In many cases, this can be life changing. In addition to benefiting those with the disease, participants may be paid for their time and travel expenses.
Every clinical trial must be reviewed, and is continually monitored, by a regulatory review committee to ensure the risks are as low as possible and are worth any potential benefits to the study participant. As a volunteer, you have the right to discontinue your participation and leave the study at any time and for any reason, with no penalty or loss of benefits to which you are otherwise entitled.
Genkyotex is responsible for conducting this clinical study.