A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis
- Registration Number
- NCT05050136
- Lead Sponsor
- Mirum Pharmaceuticals, Inc.
- 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 Biliary Cholangitis (PBC), and to assess the possible impact on the disease progression of PBC.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 180
- Provide signed informed consent at the screening visit as well as comply with all study visits and requirements through the end of the study
- Male or female, age ≥18 years at the screening visit
- Confirmed diagnosis of PBC in line with the AASLD guidelines
- UDCA and anti-pruritic medication use will be allowed if meeting additional criteria
- Qualified pruritus associated with PBC as assessed by Adult ItchRO
- Pruritus associated with an etiology other than PBC
- Evidence or clinical suspicion of decompensated cirrhosis or a history of decompensation events
- Current symptomatic cholelithiasis or inflammatory gallbladder disease
- History of small bowel surgery/resection impacting the terminal ileum that may disrupt the enterohepatic circulation.
- Evidence, history, or suspicion of other liver diseases; PBC patients with AIH are not excluded
- History of Liver transplantation
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Part 1 Arm 1: Volixibat 20mg Volixibat Participants randomized to this arm will receive volixibat 20mg twice daily. Part 1 Arm 2: Volixibat 80mg Volixibat Participants randomized to this arm will receive volixibat 80mg twice daily. Part 1 Arm 3: Placebo Placebo Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. Part 2 Arm 1: Volixibat Selected Dose 20mg Volixibat Participants randomized to this arm will receive volixibat 20mg twice daily. Part 2 Arm 2: Placebo Placebo Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
- Primary Outcome Measures
Name Time Method Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire Baseline 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
Name Time Method Incidence of adverse events Baseline to week 28 Changes in alkaline phosphatase Baseline to week 28 Changes in total bilirubin Baseline to week 28 Proportion of participants with itch response using the Adult ItchRO Baseline to week 28 Changes in serum bile acid levels Baseline to week 28 Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40) Baseline to week 28 PBC-40 questionnaire is scored on a scale of 40 to 200, with higher scores indicating poorer quality of life
Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire Baseline 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.
Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire Baseline 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.
Trial Locations
- Locations (96)
Southern California Research Center
🇺🇸Coronado, California, United States
Cedars Sinai Medical Center
🇺🇸Los Angeles, California, United States
University of Colorado
🇺🇸Aurora, Colorado, United States
Covenant Metabolic Specialists
🇺🇸Fort Myers, Florida, United States
UF Hepatology Research at CTRB
🇺🇸Gainesville, Florida, United States
UF Health Gastroenterology- Emerson
🇺🇸Jacksonville, Florida, United States
IHS Health
🇺🇸Kissimmee, Florida, United States
Schiff Center for Liver Diseases
🇺🇸Miami, Florida, United States
Advanced Research Institute, Inc
🇺🇸New Port Richey, Florida, United States
Covenant Research and Clinics, LLC
🇺🇸Sarasota, Florida, United States
Scroll for more (86 remaining)Southern California Research Center🇺🇸Coronado, California, United States