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.
Brief Title
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis
Categories
Completion Date
Completion Date Type
Estimated
Conditions
Primary Biliary Cholangitis
PBC
Eligibility Criteria
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
* 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
Inclusion Criteria
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
* 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
Gender
All
Gender Based
false
Keywords
Pruritus
PBC
Itching
Cholangitis
Primary Biliary Cholangitis
Hepatobiliary disorders
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Minimum Age
18 Years
NCT Id
NCT05050136
Org Class
Industry
Org Full Name
Mirum Pharmaceuticals, Inc.
Org Study Id
VLX-601
Overall Status
Recruiting
Phases
Phase 2
Primary Completion Date
Primary Completion Date Type
Estimated
Official Title
A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in the Treatment of Cholestatic Pruritus in Patients With Primary Biliary Cholangitis
Primary Outcomes
Outcome Description
The Adult ItchRO is an 11-point NRS measurement of itch severity ranging from 0=no itch to 10=worst possible itch.
Outcome Measure
Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire
Outcome Time Frame
Baseline to week 28
Secondary Outcomes
Outcome Time Frame
Baseline to week 28
Outcome Measure
Proportion of participants with itch response using the Adult ItchRO
Outcome Time Frame
Baseline to week 28
Outcome Measure
Incidence of adverse events
Outcome Time Frame
Baseline to week 28
Outcome Measure
Changes in alkaline phosphatase
Outcome Time Frame
Baseline to week 28
Outcome Measure
Changes in total bilirubin
Outcome Time Frame
Baseline to week 28
Outcome Measure
Changes in serum bile acid levels
Outcome Description
PBC-40 questionnaire is scored on a scale of 40 to 200, with higher scores indicating poorer quality of life
Outcome Time Frame
Baseline to week 28
Outcome Measure
Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40)
Outcome Description
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.
Outcome Time Frame
Baseline to week 28
Outcome Measure
Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire
Outcome Description
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.
Outcome Time Frame
Baseline to week 28
Outcome Measure
Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
18
Investigators
Investigator Type
Principal Investigator
Investigator Name
Kristina Chacko
Investigator Email
krchacko@montefiore.org
Investigator Department
Medicine
Investigator Division
Hepatology
Investigator Sponsor Organization
Montefiore
Study Department
Medicine
Study Division
Hepatology
Categories Mesh Debug
Digestive System --- DIGESTIVE SYSTEM DISEASES
Liver --- DIGESTIVE SYSTEM DISEASES
Digestive System --- CHOLESTASIS, INTRAHEPATIC
Liver --- CHOLESTASIS, INTRAHEPATIC
Digestive System --- CHOLESTASIS
Liver --- CHOLESTASIS
Digestive System --- BILE DUCT DISEASES
Liver --- BILE DUCT DISEASES
Digestive System --- BILIARY TRACT DISEASES
Liver --- BILIARY TRACT DISEASES
Digestive System --- LIVER DISEASES
Liver --- LIVER DISEASES
Liver --- LIVER CIRRHOSIS
MeSH Terms
LIVER CIRRHOSIS, BILIARY
PRURITUS
CHOLANGITIS
DIGESTIVE SYSTEM DISEASES
CHOLESTASIS, INTRAHEPATIC
CHOLESTASIS
BILE DUCT DISEASES
BILIARY TRACT DISEASES
LIVER DISEASES
LIVER CIRRHOSIS
FIBROSIS
PATHOLOGIC PROCESSES
PATHOLOGICAL CONDITIONS, SIGNS AND SYMPTOMS
SKIN DISEASES
SKIN AND CONNECTIVE TISSUE DISEASES
SKIN MANIFESTATIONS
SIGNS AND SYMPTOMS
VOLIXIBAT