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NCT Number: NCT04663308

A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Primary Sclerosing Cholangitis Bile Duct Diseases Biliary Tract Diseases Cholangitis Cholangitis, Sclerosing Cholestasis Digestive System Diseases Pathological Conditions, Signs and Symptoms Pruritus Signs and Symptoms Skin Diseases Skin Manifestations Skin and Connective Tissue Diseases

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Hospital Italiano de Buenos Aires, Buenos Aires, Argentina

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Provide freely signed informed consent and assent (as applicable) 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.
  • 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 disease; 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

Treatment and study plan

Volixibat

Drug

Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.

Other names: SHP626

Placebo

Drug

Capsules matched to study drug minus active substance

Primary outcomes

  1. Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire

    Time frame: Baseline through to Week 28

    The Adult ItchRO is an 11-point NRS measurement of itch severity ranging from 0=no itch to 10=worst possible itch.

Secondary outcomes

  1. Proportion of participants with itch response using the Adult ItchRO

    Time frame: Baseline through to Week 28

  2. The incidence of adverse events

    Time frame: Baseline through to Week 28

  3. Changes in serum bile acid levels

    Time frame: Baseline through to Week 28

  4. Changes in alkaline phosphatase

    Time frame: Baseline through to Week 28

  5. Changes in total bilirubin levels

    Time frame: Baseline through to Week 28

  6. Change in Primary Sclerosing Cholangitis-Specific Patient-Reported Outcome

    Time frame: Baseline through to Week 28

  7. Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire

    Time frame: Baseline through to Week 28

    The PROMIS® Fatigue questionnaire is a 5-point response scale. Respondents endorse each item using a 5-point response scale: Never, Rarely, Sometimes, Often, and Always.

  8. Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire

    Time frame: Baseline through to Week 28

    The PROMIS® Sleep Disturbance in an 8-item measure that assesses quality of sleep, sleep depth, and restoration associated with sleep. It is a 5-point response scale, the specific response options vary by items. The first 4 items use response options that range from Not at all to Very much. The next 3 items use response options that range from Never to Always. The final item assesses sleep quality ranging from Very poor to Very good.

Sponsors and collaborators

Lead sponsor

Mirum Pharmaceuticals, Inc.

Industry

Registry information

Official study title

A Randomized Double-Blind Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in the Treatment of Cholestatic Pruritus in Patients With Primary Sclerosing Cholangitis

Acronym: VISTAS

Important dates

Study start
2020
Primary completion
2026
Study completion
2027
First posted
Dec 11, 2020
Registry last updated
Feb 4, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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