A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC) (VISTAS)
General Information:
The purpose of this clinical research study is to learn more about the use of the study medicine, volixibat, for the treatment of pruritus (itching) associated with Primary Sclerosing Cholangitis (PSC), and to assess the possible impact on the disease progression of PSC.
Eligibility:
Inclusion Criteria:
- Provide freely signed informed consent and be willing to comply with all study visits and requirements through end of study, including the follow-up period.
- Subjects aged ≥12 years for eligible regions; otherwise ≥18 years
- Confirmed diagnosis of large duct or small duct PSC based on American Association for the Study of Liver Disease (AASLD) guidelines.
- Qualified pruritus associated with PSC as assessed by Adult ItchRO.
- Ursodeoxycholic acid (UDCA) and anti-pruritic medication use will be allowed if meeting additional criteria.
- Concomitant Inflammatory Bowel Disease (IBD) is allowed if meeting additional criteria.
Exclusion Criteria:
- Pruritus associated with an etiology other than PSC
- Evidence or clinical suspicion of decompensated cirrhosis, or a history of decompensation events
- History of ileostomy or small bowel surgery/resection or other surgeries that may have disrupted the enterohepatic circulation
- Evidence, history, or suspicion of other liver diseases; PSC patients with AIH are not excluded.
- Bile duct stent or percutaneous bile duct drain placement, or balloon dilatation procedure of a stricture within 12 weeks of screening
- Exceeding pre-defined biochemical values for alanine aminotransferase/aspartate aminotransferase (ALT/AST), estimated glomerular filtration rate (eGFR), serum creatinine (sCr), platelet count, international normalized ratio (INR) and total bilirubin
- History of liver transplantation
Study Details:
The VISTAS clinical trial is evaluating an investigational medication called volixibat in patients as a potential treatment option for itch due to PSC.
Study participation will last 7~8 months with the option to continue in the long-term extension phase, where eligible participants will receive the investigational medication, volixibat for an indefinite amount of time. During this time, you will take study treatment as instructed and will have appointments where the study team will perform study-related tests and procedures and ask about your health. During the study treatment period, you will need to complete an eDiary once each day.
The study also now includes a “decentralized or virtual site” option (available only in the USA) where all participants can enroll in the trial from the comfort of their home: all study activities will be performed at home by health professionals and supervised by a study physician using virtual visits. Participants who prefer to participate via the traditional hospital- or clinic-based approach may continue to do so.
Description of treatment or intervention:
Volixibat blocks the main transporter protein (IBAT) that is normally responsible for bringing bile acids back into the body from the gut. As a result, volixibat treatment is expected to lower bile acid levels in the blood which in turn may help ease itch experienced by people with PSC.
Patient Participation Requirements:
To determine potential study eligibility, please fill out the prescreening form at: https://vistaspscstudy.com/screener/
Possible risks and side effects:
Volixibat is still being studied, so not all of its side effects are known. The most commonly known side effects of volixibat treatment can include gastrointestinal side effects such as diarrhea, abdominal pain, nausea and vomiting. If you qualify for the study you will be given more detailed information about the possible risks and side effects of volixibat by a study doctor or member of their staff.
Progress to date:
In the June 2024 interim analysis, safety and effectiveness of volixibat met thresholds to continue the VISTAS study.