Trial Review

Please note the ANZCTR will be unattended from Friday 20 December 2024 for the holidays. The Registry will re-open on Tuesday 7 January 2025. Submissions and updates will not be processed during that time.

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04663308




Registration number
NCT04663308
Ethics application status
Date submitted
17/11/2020
Date registered
11/12/2020
Date last updated
16/12/2024

Titles & IDs
Public title
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)
Scientific 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
Secondary ID [1] 0 0
2020-003027-41
Secondary ID [2] 0 0
VLX-301
Universal Trial Number (UTN)
Trial acronym
VISTAS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Sclerosing Cholangitis 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Infection 0 0 0 0
Other infectious diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Volixibat
Treatment: Drugs - Placebo

Experimental: Part 1 Arm 1: Volixibat 20mg - Participants randomized to this arm will receive volixibat 20mg twice daily.

Experimental: Part 1 Arm 2: Volixibat 80mg - Participants randomized to this arm will receive volixibat 80mg twice daily.

Placebo comparator: Part 1 Arm 3: Placebo - Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.

Experimental: Part 2 Arm 1: Volixibat Selected Dose 20mg - Participants randomized to this arm will receive volixibat 20mg twice daily.

Placebo comparator: Part 2 Arm 2: Placebo - Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.


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

Treatment: Drugs: Placebo
Capsules matched to study drug minus active substance
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire
Timepoint [1] 0 0
Baseline through to Week 28
Secondary outcome [1] 0 0
Proportion of participants with itch response using the Adult ItchRO
Timepoint [1] 0 0
Baseline through to Week 28
Secondary outcome [2] 0 0
The incidence of adverse events
Timepoint [2] 0 0
Baseline through to Week 28
Secondary outcome [3] 0 0
Changes in serum bile acid levels
Timepoint [3] 0 0
Baseline through to Week 28
Secondary outcome [4] 0 0
Changes in alkaline phosphatase
Timepoint [4] 0 0
Baseline through to Week 28
Secondary outcome [5] 0 0
Changes in total bilirubin levels
Timepoint [5] 0 0
Baseline through to Week 28
Secondary outcome [6] 0 0
Change in Primary Sclerosing Cholangitis-Specific Patient-Reported Outcome
Timepoint [6] 0 0
Baseline through to Week 28
Secondary outcome [7] 0 0
Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire
Timepoint [7] 0 0
Baseline through to Week 28
Secondary outcome [8] 0 0
Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire
Timepoint [8] 0 0
Baseline through to Week 28

Eligibility
Key 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 =12 years for eligible regions; otherwise =18 years
3. Confirmed diagnosis of large duct or small duct PSC based on American Association for the Study of Liver Disease (AASLD) guidelines.
4. 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.
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 disease; PSC patients with AIH are not excluded.
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
18/12/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2025
Actual
Sample size
Target
200
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
University of the Sunshine Coast - Sippy Downs
Recruitment postcode(s) [1] 0 0
4556 - Sippy Downs
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
District of Columbia
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
Mississippi
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Pennsylvania
Country [13] 0 0
United States of America
State/province [13] 0 0
Tennessee
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
United States of America
State/province [15] 0 0
Utah
Country [16] 0 0
United States of America
State/province [16] 0 0
Virginia
Country [17] 0 0
United States of America
State/province [17] 0 0
Washington
Country [18] 0 0
Argentina
State/province [18] 0 0
Buenos Aires
Country [19] 0 0
Argentina
State/province [19] 0 0
Ciudad Autónoma Buenos Aires
Country [20] 0 0
Belgium
State/province [20] 0 0
Brussels
Country [21] 0 0
Belgium
State/province [21] 0 0
Gent
Country [22] 0 0
Canada
State/province [22] 0 0
Alberta
Country [23] 0 0
Canada
State/province [23] 0 0
Ontario
Country [24] 0 0
Canada
State/province [24] 0 0
Calgary
Country [25] 0 0
Canada
State/province [25] 0 0
Montréal
Country [26] 0 0
France
State/province [26] 0 0
Grenoble
Country [27] 0 0
France
State/province [27] 0 0
Lille Cedex
Country [28] 0 0
France
State/province [28] 0 0
Paris Cedex 12
Country [29] 0 0
Germany
State/province [29] 0 0
Lower Saxony
Country [30] 0 0
Germany
State/province [30] 0 0
Nordrhine-Westphalia
Country [31] 0 0
Germany
State/province [31] 0 0
Berlin
Country [32] 0 0
Germany
State/province [32] 0 0
Erlangen
Country [33] 0 0
Germany
State/province [33] 0 0
Kiel
Country [34] 0 0
Germany
State/province [34] 0 0
Leipzig
Country [35] 0 0
Germany
State/province [35] 0 0
Magdeburg
Country [36] 0 0
Germany
State/province [36] 0 0
Tübingen
Country [37] 0 0
Germany
State/province [37] 0 0
Wiesbaden
Country [38] 0 0
Israel
State/province [38] 0 0
Hadera
Country [39] 0 0
Israel
State/province [39] 0 0
Haifa
Country [40] 0 0
Israel
State/province [40] 0 0
Jerusalem
Country [41] 0 0
Israel
State/province [41] 0 0
Nahariyya
Country [42] 0 0
Israel
State/province [42] 0 0
Petach Tikva
Country [43] 0 0
Israel
State/province [43] 0 0
Ramat Gan
Country [44] 0 0
Israel
State/province [44] 0 0
Tel Aviv
Country [45] 0 0
Italy
State/province [45] 0 0
Bologna
Country [46] 0 0
Italy
State/province [46] 0 0
Milan
Country [47] 0 0
Italy
State/province [47] 0 0
Monza
Country [48] 0 0
Italy
State/province [48] 0 0
Napoli
Country [49] 0 0
Italy
State/province [49] 0 0
Padova
Country [50] 0 0
Netherlands
State/province [50] 0 0
Amsterdam
Country [51] 0 0
Netherlands
State/province [51] 0 0
Groningen
Country [52] 0 0
Netherlands
State/province [52] 0 0
Nijmegen
Country [53] 0 0
Netherlands
State/province [53] 0 0
Rotterdam
Country [54] 0 0
Spain
State/province [54] 0 0
Barcelona
Country [55] 0 0
Spain
State/province [55] 0 0
Madrid
Country [56] 0 0
Switzerland
State/province [56] 0 0
Zürich
Country [57] 0 0
United Kingdom
State/province [57] 0 0
Hampstead
Country [58] 0 0
United Kingdom
State/province [58] 0 0
Headington
Country [59] 0 0
United Kingdom
State/province [59] 0 0
Belfast
Country [60] 0 0
United Kingdom
State/province [60] 0 0
Birmingham
Country [61] 0 0
United Kingdom
State/province [61] 0 0
Cambridge
Country [62] 0 0
United Kingdom
State/province [62] 0 0
Edinburgh
Country [63] 0 0
United Kingdom
State/province [63] 0 0
Leeds
Country [64] 0 0
United Kingdom
State/province [64] 0 0
London
Country [65] 0 0
United Kingdom
State/province [65] 0 0
Newcastle Upon Tyne
Country [66] 0 0
United Kingdom
State/province [66] 0 0
Nottingham
Country [67] 0 0
United Kingdom
State/province [67] 0 0
Southampton

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Mirum Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Clinical Trials Mirum
Address 0 0
Country 0 0
Phone 0 0
+16506674085
Fax 0 0
Email 0 0
Clinicaltrials@mirumpharma.com
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT04663308