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Study on Volixibat for Treating Itching in Patients with Primary Sclerosing Cholangitis

Fast replyInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition called Primary Sclerosing Cholangitis (PSC), which is a disease that affects the bile ducts in the liver and can cause itching, known as pruritus. The study is testing a new treatment called volixibat, which is taken as a hard capsule by mouth. Volixibat is an investigational drug, meaning it is still being studied and is not yet approved for general use. It works as an ileal bile acid transporter inhibitor, which means it helps to reduce the amount of bile acid in the body, potentially easing the itching associated with PSC.

The purpose of the study is to evaluate how effective and safe volixibat is compared to a placebo in treating the itching caused by PSC. Participants in the study will be randomly assigned to receive either volixibat or a placebo, and neither the participants nor the researchers will know who is receiving which treatment. This is known as a double-blind study. The study will last for a period of time, during which participants will take the medication and attend regular study visits to monitor their condition and any changes in their symptoms.

Throughout the study, the main focus will be on observing changes in the severity of itching using a tool called the Adult ItchRO. This tool helps to measure how the itching affects daily life. The study aims to provide valuable information on whether volixibat can effectively reduce itching in people with PSC, potentially leading to a new treatment option for this challenging condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, you will be required to provide freely signed informed consent. This means you agree to participate after understanding the study's purpose and requirements.

    You must be willing to comply with all study visits and requirements through the end of the study, including the follow-up period.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to confirm your eligibility. This includes verifying your age, which should be at least 12 years in eligible regions or 18 years otherwise.

    A confirmed diagnosis of primary sclerosing cholangitis (PSC) is required, consistent with established guidelines.

    Your average daily Adult ItchRO score must be at least 1 during the two consecutive weeks leading to the second visit.

  3. Step 3

    Medication administration

    You will be randomly assigned to receive either the investigational drug volixibat or a placebo. Both are administered as hard capsules for oral use.

    The dosage, frequency, and duration of administration will be explained to you by the study team.

  4. Step 4

    Ongoing assessments

    Throughout the study, regular assessments will be conducted to monitor your condition and the effects of the medication.

    These assessments will include measuring changes in your itch severity using the Adult ItchRO tool.

  5. Step 5

    End of study

    At the end of the study, a final assessment will be conducted to evaluate the overall efficacy and safety of the treatment.

    You will be required to attend a follow-up period as part of the study's completion process.

Who can join the trial?

6 criteria

  • Provide freely signed informed consent, which means you agree to participate after understanding the study, and be willing to attend all study visits and follow all study requirements until the end, including the follow-up period.
  • Be at least 12 years old in eligible regions; otherwise, you must be at least 18 years old.
  • Have a confirmed diagnosis of Primary Sclerosing Cholangitis (PSC) before the second visit, according to specific medical guidelines.
  • Have an average daily score of 1 or higher on the Adult ItchRO tool, which measures itching, during the two weeks leading up to the second visit.
  • Use of Ursodeoxycholic acid (UDCA) and anti-itch medication is allowed if you meet additional criteria.
  • Having Inflammatory Bowel Disease (IBD) at the same time is allowed if you meet additional criteria.

Who cannot join the trial?

4 criteria

  • Patients who do not have **pruritus** (itching) associated with **Primary Sclerosing Cholangitis (PSC)** cannot participate.
  • Patients who are not within the specified age range for the study cannot participate.
  • Patients who belong to a group that is not included in the study cannot participate.
  • Patients who are part of a vulnerable population that is not selected for the study cannot participate.
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Investigated drugs

Volixibat is a medication being studied for its potential to help reduce itching in people with a liver condition called Primary Sclerosing Cholangitis (PSC). This condition can cause a lot of discomfort due to itching, and Volixibat is being tested to see if it can make this symptom better. The study aims to find out if Volixibat can effectively decrease the sensation of itching, improving the quality of life for those affected by PSC.

What is already known about the treatment

Volixibat – Volixibat is administered orally in the form of a pill and is currently being studied in clinical trials for its effectiveness and safety in treating itching (pruritus) associated with a liver condition called Primary Sclerosing Cholangitis (PSC). This medication is not yet widely used in medicine, as it is still under investigation. The main therapeutic indication for Volixibat is to relieve itching in patients with PSC. It works by inhibiting a protein in the liver that affects bile acid transport, which can help reduce the buildup of substances that cause itching. Volixibat is classified as a bile acid transporter inhibitor.

Investigated diseases

Primary Sclerosing Cholangitis (PSC) – Primary Sclerosing Cholangitis is a chronic liver disease characterized by inflammation and scarring of the bile ducts. Over time, this scarring can lead to blockages, causing bile to accumulate in the liver. This accumulation can result in liver damage and eventually liver failure. The disease often progresses slowly, with symptoms such as fatigue, jaundice, and abdominal pain. Pruritus, or severe itching, is a common symptom associated with PSC, caused by the buildup of bile acids in the bloodstream. The exact cause of PSC is unknown, but it is often associated with inflammatory bowel diseases.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-505764-11-00Protocol codeVLX-301Estimated enrolment387 patientsSponsorMirum Pharmaceuticals Inc.

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

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