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NCT Number: NCT05050136

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

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 Biliary Cholangitis (PBC), and to assess the possible impact on the disease progression of PBC.

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Key information

Conditions

Primary Biliary Cholangitis Bile Duct Diseases Biliary Tract Diseases Cholangitis Cholestasis Cholestasis, Intrahepatic Digestive System Diseases Fibrosis Liver Cirrhosis Liver Cirrhosis, Biliary Liver Diseases PBC Pathologic Processes Pathological Conditions, Signs and Symptoms Pruritus Signs and Symptoms Skin Diseases Skin Manifestations Skin and Connective Tissue Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Hospital de Alta Complejidad En Red El Cruce, San Juan Bautista, Buenos Aires, Argentina

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Provide signed informed consent at the screening visit as well as comply with all study visits and requirements through the end of the study
  • Male or female, age ≥18 years at the screening visit
  • Confirmed diagnosis of PBC in line with the AASLD guidelines
  • UDCA, anti-pruritic medication and PPAR agonist use will be allowed if meeting additional criteria
  • Qualified pruritus associated with PBC as assessed by Adult ItchRO

Exclusion criteria

  • Pruritus associated with an etiology other than PBC
  • Evidence or clinical suspicion of decompensated cirrhosis or a history of decompensation events
  • Current symptomatic cholelithiasis or inflammatory gallbladder disease
  • History of small bowel surgery/resection impacting the terminal ileum that may disrupt the enterohepatic circulation.
  • Evidence, history, or suspicion of other liver diseases; PBC patients with AIH are not excluded
  • 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 without the active pharmaceutical ingredient

Primary outcomes

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

    Time frame: Baseline 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 to week 28

  2. Incidence of adverse events

    Time frame: Baseline to week 28

  3. Changes in alkaline phosphatase

    Time frame: Baseline to week 28

  4. Changes in total bilirubin

    Time frame: Baseline to week 28

  5. Changes in serum bile acid levels

    Time frame: Baseline to week 28

  6. Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40)

    Time frame: Baseline to week 28

    PBC-40 questionnaire is scored on a scale of 40 to 200, with higher scores indicating poorer quality of life

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

    Time frame: Baseline 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 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 Biliary Cholangitis

Acronym: VANTAGE

Important dates

Study start
2021
Primary completion
2027
Study completion
2027
First posted
Sep 20, 2021
Registry last updated
May 14, 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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