A study to evaluate efficacy and safety of an investigational drug named volixibat in patients with itching caused by primary sclerosing cholangitis

Phase 1

Conditions: MedDRA version: 20.1Level: LLTClassification code 10036732Term: Primary sclerosing cholangitisSystem Organ Class: 100000004871
Pruritus associated with Primary Sclerosing Cholangitis (PSC)
Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

Registration Number: EUCTR2020-003027-41-DE

Lead Sponsor: Mirum Pharmaceuticals, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 300

Inclusion Criteria

For screening:
1. Provide freely signed informed consent and be willing to comply with all study visits and requirements through the end of the long-term extension period.
2. Age =16 years for eligible regions; otherwise =18 years
3. Confirmed diagnosis of large duct or small duct PSC based on AASLD guidelines.
4. Qualified pruritus associated with PSC as assessed by Adult ItchRO
5. Completion of =80% of daily Adult ItchRO assessments during the screening period
6. UDCA and anti-pruritic medication use will be allowed if meeting additional criteria. Concomitant IBD is allowed if meeting additional criteria
7. Participants with AIH who are on stable treatment with medications

For randomization
1. Completion of =80% of Adult ItchRO assessments during the single-blind, placebo run- in period
2. Study drug compliance of =80% during the single-blind, placebo run-in period

Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 170
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 30

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. Presumptive or diagnosed ascending cholangitis within 12 weeks of screening through Day 1
4. Placement of a percutaneous drain or biliary stent within 12 weeks of screening through Day 1
5. Balloon dilatation procedure of a stricture within 12 weeks of screening through Day 1
6. History of ileostomy or small bowel surgery/resection or other surgeries that may have disrupted the enterohepatic circulation
7. ECG with clinically significant abnormalities as determined by the investigator
8. Evidence, history, or suspicion of other liver and GI-related diseases and malignancies
9. Positive for HIV antibody
10. Any active infection that, in the opinion of the investigator or medical monitor, would preclude successful participation in the study
11. Unstable and/or serious medical disease that is likely to impair the participant’s ability to participate in all aspects of the study, confound efficacy and/or safety assessments, or result in substantially shortened life expectancy
12. Clinically relevant alcohol use disorder or drug abuse within 24 weeks of screening

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the efficacy of volixibat versus placebo for the treatment of pruritus as measured by the change in the Adult ItchRO tool in participants with PSC.;Secondary Objective: To evaluate the efficacy of volixibat versus placebo for the treatment of pruritus in participants with PSC using additional measures of pruritus efficacy.<br><br>To evaluate safety and tolerability of volixibat versus placebo in participants with PSC-associated pruritus.<br><br>To assess volixibat pharmacodynamics.<br><br>To evaluate quality of life in participants with PSC-associated pruritus treated with volixibat versus placebo.;Primary end point(s): Mean change in the Adult ItchRO comparing baseline with the average of the weekly averaged worst daily itch scores from Week 12 through Week 24 ;Timepoint(s) of evaluation of this end point: Baseline from Week 12 through Week 24

Secondary Outcome Measures

Name	Time	Method

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