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

Phase 2

Recruiting

• Conditions: Primary Biliary Cholangitis; PBC

• Registration Number: NCT05050136
• Lead Sponsor: Mirum Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-9-9
• Last Update Posted: 2 September 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 180

Inclusion Criteria

Inclusion Criteria:<br><br> - Provide signed informed consent at the screening visit as well as comply with all<br> study visits and requirements through the end of the study<br><br> - Male or female, age =18 years at the screening visit<br><br> - Confirmed diagnosis of PBC in line with the AASLD guidelines<br><br> - UDCA and anti-pruritic medication use will be allowed if meeting additional criteria<br><br> - Qualified pruritus associated with PBC as assessed by Adult ItchRO<br><br>Exclusion Criteria:<br><br> - Pruritus associated with an etiology other than PBC<br><br> - Evidence or clinical suspicion of decompensated cirrhosis or a history of<br> decompensation events<br><br> - Current symptomatic cholelithiasis or inflammatory gallbladder disease<br><br> - History of small bowel surgery/resection impacting the terminal ileum that may<br> disrupt the enterohepatic circulation.<br><br> - Evidence, history, or suspicion of other liver diseases; PBC patients with AIH are<br> not excluded<br><br> - History of Liver transplantation

Exclusion Criteria

Not provided

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire

Secondary Outcome Measures

Name	Time	Method
Proportion of participants with itch response using the Adult ItchRO;Incidence of adverse events;Changes in alkaline phosphatase;Changes in total bilirubin;Changes in serum bile acid levels;Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40);Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire;Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire