The VISTAS Study is seeking adults with PSC to learn if the study drug, volixibat, might be an effective and safe treatment option for intense itching due to PSC compared to placebo (drug with no active ingredient).
Take an active role in your treatment while helping to contribute to medical advancements for present and future generations. The VISTAS Study is designed to give medical professionals the information they need to potentially help you and other PSC patients suffering from cholestatic itch.
Receive standard medical care for PSC from an experienced medical team.
PSC is a rare liver disease that causes damage to the bile ducts inside and outside the liver. It is frequently associated with chronic infections and inflammatory bowel disease (IBD).
A common complication from PSC is intense itching which can lead to fatigue, social isolation, and an overall lower quality of life. Currently, there are no approved or effective medications to treat PSC or PSC-associated itch.
The VISTAS Study will evaluate the safety and effectiveness of an investigational medication named volixibat. Information about volixibat’s effects on PSC-associated itch and on markers associated with PSC disease progression will be obtained in the VISTAS Study.
First, you must be evaluated to determine if you meet the criteria for entry into the study. During the screening period of 4 weeks, study staff will assess your itch and your health and determine if you are eligible for the VISTAS Study. Upon entering the screening period, you will need to complete an eDiary once each day.
If you qualify for the study, you will enter the study treatment period of 7 months. 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 questions about your health. During the study treatment period, you will need to complete an eDiary once each day.
Whether you are treated with the investigational medicine will be determined randomly. You will receive standard medical care for PSC from an experienced medical team, plus oral, twice-daily dosing with the investigational medicine (volixibat) or placebo.
After successful completion of the study treatment period, you will be able to continue participation, if eligible, in the Long-Term Extension (LTE) period, where you will receive volixibat and continue to be closely monitored by the study doctor for up to 2 years.
At the end of your study participation, you will return for a safety follow-up visit to close out your participation in the VISTAS Study.
Additional study requirements and exclusions apply. A study representative will discuss these with you during the screening period.
Through clinical research, scientists and doctors determine whether a new medical strategy, drug, or device is safe and effective for people. New treatments are only approved through clinical research studies, which rely on potential study participants, like you, to evaluate their safety and efficacy. Participation is voluntary and study participants may withdraw from the study at any time.
Investigational medications are not yet approved by any government health authority such as the FDA. A clinical research study is used to test the safety and efficacy (or effectiveness) of an investigational medication.
Clinical trials are important to help determine if new medications are both safe and effective. Typically, investigational medications must pass through three phases of development in clinical trials, called Phase 1, Phase 2, and Phase 3 for approval. Results from each phase must be properly assessed before medications can be approved for use in the public.
Volixibat has already been studied in more than 400 patients in earlier Phase 1 and Phase 2 studies in patients with conditions other than PSC. The VISTAS Study is a Phase 2 study.
The VISTAS Study is seeking adults with PSC to learn if the study drug, volixibat, might be an effective and safe treatment option for intense itching due to PSC compared to placebo (drug with no active ingredient).
Qualified participants may receive compensation for time and travel related expenses (including long distance travel). Study-related medications, procedures and examinations are provided at no cost to study participants.
Patient participation in the VISTAS Study is expected to last 8 months. There will be 10 planned study visits that occur over this time. All study participants who successfully complete the study treatment period and meet eligibility criteria may participate in the Long Term Extension period.
This initial appointment is an opportunity for you to:
Mirum Pharmaceuticals, Inc., based in Foster City, California, is a biopharmaceutical company focused on the development and commercialization of therapies to treat debilitating liver diseases. Mirum is developing two ileal bile acid transporter (IBAT) inhibitors for the treatment of cholestatic liver diseases, including maralixibat for pediatric liver diseases, and volixibat for adult liver diseases.
In addition to PSC, volixibat is being evaluated in intrahepatic cholestasis of pregnancy (ICP) and primary biliary cholangitis (PBC).
Mirum works closely with advocates and the patient communities to understand and address the most critical aspects affecting patients and families living with rare liver diseases.
For more information about Mirum, please visit MirumPharma.com.
To learn more, please talk to your doctor for more information. You can also visit www.clinicaltrials.gov using the study ID: VLX-301.