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

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ClinicalTrials.gov Identifier: NCT04663308
Recruitment Status : Recruiting
First Posted : December 11, 2020
Last Update Posted : May 22, 2023
Sponsor:
Information provided by (Responsible Party):
Mirum Pharmaceuticals, Inc.

Study Description
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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 Sclerosing Cholangitis (PSC), and to assess the possible impact on the disease progression of PSC.

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

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 200 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 Sclerosing Cholangitis
Actual Study Start Date : December 18, 2020
Estimated Primary Completion Date : September 2024
Estimated Study Completion Date : December 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Itching

Arms and Interventions
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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 minus active substance


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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 fasting serum bile acid levels [ Time Frame: Baseline through to Week 28 ]
  4. Changes in alanine transaminase [ Time Frame: Baseline through to Week 28 ]
  5. Changes in aspartate transaminase [ Time Frame: Baseline through to Week 28 ]
  6. Changes in alkaline phosphatase [ Time Frame: Baseline through to Week 28 ]
  7. Changes in bilirubin levels [ Time Frame: Baseline through to Week 28 ]
  8. Change in Primary Sclerosing Cholangitis-Specific Patient-Reported Outcome [ Time Frame: Baseline through to Week 28 ]
  9. 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.

  10. 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.


Eligibility Criteria
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Ages Eligible for Study:   16 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. 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.
  2. Subjects aged ≥16 years for eligible regions
  3. Confirmed diagnosis of large duct or small duct PSC based on American Association for the Study of Liver Disease (AASLD) guidelines.
  4. Qualified pruritus associated with PSC as assessed by Adult ItchRO.
  5. Ursodeoxycholic acid (UDCA) and anti-pruritic medication use will be allowed if meeting additional criteria.
  6. Concomitant Inflammatory Bowel Disease (IBD) is allowed if meeting additional criteria.

Exclusion Criteria:

  1. Pruritus associated with an etiology other than PSC
  2. Evidence or clinical suspicion of decompensated cirrhosis, or a history of decompensation events
  3. History of ileostomy or small bowel surgery/resection or other surgeries that may have disrupted the enterohepatic circulation
  4. Evidence, history, or suspicion of other liver diseases
  5. Bile duct stent or percutaneous bile duct drain placement, or balloon dilatation procedure of a stricture within 12 weeks of Screening
  6. 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
  7. History of liver transplantation
Contacts and Locations
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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): NCT04663308


Contacts
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Contact: Clinical Trials Mirum +16506674085 clinicaltrials@mirumpharma.com
Contact: Medinfo Mirum medinfo@mirumpharma.com

Locations
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Sponsors and Collaborators
Mirum Pharmaceuticals, Inc.
More Information
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Responsible Party: Mirum Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04663308    
Other Study ID Numbers: VLX-301
2020-003027-41 ( EudraCT Number )
First Posted: December 11, 2020    Key Record Dates
Last Update Posted: May 22, 2023
Last Verified: May 2023

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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
PSC
Itch
Itching
Cholestasis
Additional relevant MeSH terms:
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Cholangitis
Cholangitis, Sclerosing
Bile Duct Diseases
Biliary Tract Diseases
 Digestive System Diseases
Volixibat
Lipid Regulating Agents