Primary Biliary Cholangitis, PBC
Conditions
Keywords
Pruritus, PBC, Itching, Cholangitis, Primary Biliary Cholangitis, Hepatobiliary disorders
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.
Interventions
DRUGVolixibat
Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
DRUGPlacebo
Capsules matched to study drug without the active pharmaceutical ingredient
Sponsors
Mirum Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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, anti-pruritic medication and PPAR agonist use will be allowed if meeting additional criteria * Qualified pruritus associated with PBC as assessed by Adult ItchRO
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| Proportion of participants with itch response using the Adult ItchRO | Baseline to week 28 | — |
| Incidence of adverse events | Baseline to week 28 | — |
| Changes in alkaline phosphatase | Baseline to week 28 | — |
| Changes in total bilirubin | 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. |
Countries
Argentina, Belgium, Brazil, Canada, China, France, Germany, Israel, Italy, Japan, Netherlands, Spain, Switzerland, United Kingdom, United States
Outcome results
None listed