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

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ClinicalTrials.gov Identifier: NCT05050136
Recruitment Status : Recruiting
First Posted : September 20, 2021
Last Update Posted : December 2, 2022
Sponsor:
Information provided by (Responsible Party):
Mirum Pharmaceuticals, Inc.

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Primary Biliary Cholangitis PBC Drug: Volixibat Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 260 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : September 22, 2021
Estimated Primary Completion Date : December 2023
Estimated Study Completion Date : December 2025


Arm Intervention/treatment
Experimental: Volixibat 20mg
Participants randomized to this arm will receive volixibat 20mg twice daily.
Drug: Volixibat
Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
Other Name: SHP626

Experimental: Volixibat 80mg
Participants randomized to this arm will receive volixibat 80mg twice daily.
Drug: Volixibat
Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
Other Name: SHP626

Placebo Comparator: Placebo
Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Drug: Placebo
Capsules matched to study drug without the active pharmaceutical ingredient




Primary Outcome Measures :
  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 Outcome Measures :
  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 aspartate aminotransferase [ Time Frame: Baseline to week 28 ]
  4. Changes in alanine aminotransferase [ Time Frame: Baseline to week 28 ]
  5. Changes in alkaline phosphatase [ Time Frame: Baseline to week 28 ]
  6. Changes in total bilirubin [ Time Frame: Baseline to week 28 ]
  7. Changes in direct bilirubin [ Time Frame: Baseline to week 28 ]
  8. Changes in fasting serum bile acid levels [ Time Frame: Baseline to week 28 ]
  9. 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

  10. 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.

  11. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 and anti-pruritic medication 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05050136


Locations
Show Show 37 study locations
Sponsors and Collaborators
Mirum Pharmaceuticals, Inc.
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Responsible Party: Mirum Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT05050136    
Other Study ID Numbers: VLX-601
First Posted: September 20, 2021    Key Record Dates
Last Update Posted: December 2, 2022
Last Verified: November 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Mirum Pharmaceuticals, Inc.:
Pruritus
PBC
Itching
Cholangitis
Primary Biliary Cholangitis
Hepatobiliary disorders
Additional relevant MeSH terms:
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Cholangitis
Liver Cirrhosis, Biliary
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Cholestasis, Intrahepatic
Cholestasis
Liver Diseases
Liver Cirrhosis
Fibrosis
Pathologic Processes
Volixibat
Lipid Regulating Agents