A study to evaluate efficacy and safety of an investigational drug namedvolixibat in patients with itching caused by primary biliary cholangitis

Phase 1

Conditions: Cholestatic Pruritus in Patients with Primary Biliary Cholangitis (PBC)
MedDRA version: 21.0Level: PTClassification code 10080429Term: Primary biliary cholangitisSystem Organ Class: 10019805 - Hepatobiliary disorders
Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

Registration Number: EUCTR2021-001389-39-DE

Lead Sponsor: Mirum Pharmaceuticals, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 260

Inclusion Criteria

1. Provide signed informed consent at the screening visit as well as comply with all study visits and requirements through the end of the study.
2. Male or female, age =18 years at the screening visit.
3. Confirmed diagnosis of PBC in line with the AASLD guidelines.
4. UDCA and anti-pruritic medication use will be allowed if meeting additional criteria.
5. Qualified pruritus associated with PBC as assessed by Adult ItchRO.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 180
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 80

Exclusion Criteria

1. Pruritus associated with an etiology other than PBC.
2. Evidence or clinical suspicion of decompensated cirrhosis or a history of decompensation events.
3. Current symptomatic cholelithiasis or inflammatory gallbladder disease.
4. History of small bowel surgery/resection impacting the terminal ileum that may disrupt the enterohepatic circulation.
5. Evidence, history, or suspicion of other liver diseases.

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 in participants with PBC as measured by the change in the Adult ItchRO tool in participants with PBC;Secondary Objective: - To evaluate the efficacy of volixibat versus placebo for the treatment of pruritus in participants with PBC using additional measures of pruritus efficacy.<br>- To evaluate safety and tolerability of volixibat versus placebo in participants with PBC-associated pruritus.<br>- To assess volixibat pharmacodynamics.<br>- To evaluate Quality of Life in participants with PBC-associated pruritus treated with volixibat versus placebo.;Primary end point(s): Mean change in Adult ItchRO score comparing baseline with the average of the weekly averaged daily itch scores from Week 16 to end of double-blind study treatment;Timepoint(s) of evaluation of this end point: Baseline and Week 16 to end of double-blind study treatment

Secondary Outcome Measures