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Primary biliary cholangitis (PBC, previously known as primary biliary cirrhosis) is a chronic, serious, and potentially life-threatening liver disease. It is estimated that 65 of every 100,000 women and 12 of every 100,000 men in the United States have PBC.¹
The most common early symptoms of PBC are itching (pruritus) and fatigue,[¹] which can be very debilitating for some people.² Approximately 60% of people with PBC do not have symptoms at the time they are diagnosed with the disease. Progression of PBC is associated with an increased risk of liver cancer and liver-related mortality.3,4,5
Currently, there are only two treatment options, ursodeoxycholic acid (UDCA, or Ursodiol) and obeticholic acid (Ocaliva®), that have received approval from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency for the treatment of PBC.
Despite these available treatments for PBC, people living with this disease still have an unmet need for additional therapies. Approximately 40% of people with PBC treated with UDCA have an inadequate response to the medication and approximately 50% of people with PBC taking Ocaliva® do not respond well to therapy and continue to have disease symptoms, even though they are taking medication.6
RESPONSE (study number CB8025-32048) is a Phase 3 clinical research study being conducted to learn more about the effectiveness and safety of seladelpar, a new investigational drug for the potential treatment of patients with PBC.
The RESPONSE study is enrolling people who have been using UDCA but have not achieved the recommended treatment goal, or who develop troublesome side effects from UDCA that prevent them from continuing to take this treatment. People who tried and cannot take Ocaliva® may also be eligible. In this study, we want to find out if seladelpar can improve PBC-related blood laboratory tests and can improve symptoms of PBC, such as itching.
To qualify for this study, you must:
- Be 18 to 75 years old
- Have been on a stable and recommended dose of UDCA for the past 12 months or have developed troublesome side effects from UDCA that required you to stop taking the medication
- Be diagnosed with PBC
- Have blood laboratory test results in the following range:
- Alkaline phosphatase (ALP) at least 1.67 times the upper limit of normal (ULN)
- Bilirubin test levels less than two times the ULN
- Doctors will check other requirements to confirm that you qualify for this study.
What Happens if I Qualify?
After you decide to volunteer, the study doctor and staff will provide you with an informed consent document. If you consent to participate in the study, the study staff will give you some tests to make sure you qualify. This qualification is called the “screening period” and getting all the results can last approximately four weeks.
If you qualify during the screening period and you want to participate, you will receive the study drug or placebo to take orally (by mouth) one time each day for 52 weeks. In this study, there is a 2 in 3 chance that you will receive seladelpar and a 1 in 3 chance you will receive placebo in this study. Placebo is an inactive substance that looks like seladelpar. During the study, neither you nor your doctor will know if you are receiving placebo or seladelpar.
During the study treatment period (a total of about 60 weeks), you will have approximately six visits to the study doctor’s office. You may also have a follow-up visit after you leave the study. You will be reimbursed for study-related travel expenses you incur and a travel concierge service may help you plan your travel. For some visits you may opt in to have a nurse come to your home instead of visiting the study site.
After completion of this study, you may be offered the opportunity to participate in the ASSURE long-term study of seladelpar. Everyone in the ASSURE long-term study will receive seladelpar (5 or 10 mg) at no cost for up to five years or until seladelpar is available for sale after its first commercial approval, or the study is discontinued, whichever occurs first.
All study medication and study-related care will be at no cost to you. If you are interested in getting screened to participate, talk to your doctor.
Seladelpar is a first-in-class oral, selective PPARδ agonist in development for the treatment of people with PBC and other liver diseases.
Seladelpar has received Breakthrough Therapy Designation (FDA) and PRIME status (EMEA) and Orphan Drug Designation in the U.S. and Europe. It is backed by an extensive body of clinical evidence and global patient experience through its Phase 2 and Phase 3 (ENHANCE) studies of seladelpar in PBC.
Currently, over 700 individuals, including healthy volunteers and people with PBC and other liver diseases have received seladelpar in clinical trials. Data from these clinical trials have demonstrated that seladelpar may improve PBC-related blood laboratory tests and symptoms of PBC, thus suggesting that it may benefit PBC patients. The results provided the foundation for RESPONSE.
To learn more about the RESPONSE study, visit: www.pbcstudies.com