NCT05050136
Recruiting
Phase 2
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
Overview
- Phase
- Phase 2
- Intervention
- Volixibat
- Conditions
- Primary Biliary Cholangitis
- Sponsor
- Mirum Pharmaceuticals, Inc.
- Enrollment
- 260
- Locations
- 167
- Primary Endpoint
- Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire
- Status
- Recruiting
- Last Updated
- last month
Overview
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.
Investigators
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
Arms & Interventions
Part 1 Arm 1: Volixibat 20mg
Participants randomized to this arm will receive volixibat 20mg twice daily.
Intervention: Volixibat
Part 1 Arm 2: Volixibat 80mg
Participants randomized to this arm will receive volixibat 80mg twice daily.
Intervention: Volixibat
Part 1 Arm 3: Placebo
Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Intervention: Placebo
Part 2 Arm 1: Volixibat Selected Dose 20mg
Participants randomized to this arm will receive volixibat 20mg twice daily.
Intervention: Volixibat
Part 2 Arm 2: Placebo
Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Intervention: Placebo
Outcomes
Primary Outcomes
Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire
Time Frame: 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 Outcomes
- 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)
- Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire(Baseline to week 28)
- Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire(Baseline to week 28)
Study Sites (167)
Loading locations...
Similar Trials
Active, not recruiting
Phase 2
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)Primary Sclerosing CholangitisNCT04663308Mirum Pharmaceuticals, Inc.182
Completed
Phase 2
Study Evaluating ERB-041 in Endometriosis in Reproductive-Age WomenEndometriosisNCT00110487Wyeth is now a wholly owned subsidiary of Pfizer200
Withdrawn
Phase 3
Study of a Drug [DCVax (tm)-Prostate] to Treat Prostate Cancer When Hormone Therapy is no Longer Effective.Prostate CancerNCT00043212Northwest Biotherapeutics
Unknown
Phase 2
Dapsone for Acute Ischemia Stroke StudyCerebral StrokeCerebrovascular Accident, AcuteCerebrovascular StrokeStroke, AcuteStrokeNCT01144650Cidat, S.A. de C.V.300
Completed
Phase 2
Efficacy and Safety of Avanafil in the Patients With Erectile DysfunctionErectile DysfunctionNCT02477436Pusan National University Hospital159
Related News
Phase 3 Trial Evaluating Maralixibat for Cholestasis-Related Pruritus- A Phase 3 clinical trial (EXPAND) is underway to assess maralixibat's efficacy in treating pruritus associated with rare cholestatic conditions, excluding Alagille syndrome and PFIC.
- The EXPAND trial aims to enroll 90 participants aged 6 months and older across multiple sites in the U.S., South America, Europe, and the Middle East, with enrollment expected to conclude in 2026.
- Participants will receive either maralixibat or a placebo, with the primary goal of assessing changes in the caregiver-reported ItchRO(Obs) severity score.
- Mirum Pharmaceuticals is also investigating volixibat for pruritus in primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), with Phase 2 trial data expected in 2026.Volixibat Receives FDA Breakthrough Therapy Designation for Cholestatic Pruritus in Primary Biliary Cholangitis- The FDA granted Breakthrough Therapy Designation to volixibat for treating cholestatic pruritus in patients with primary biliary cholangitis (PBC).
- This designation is based on positive interim results from the Phase 2b VANTAGE study, showcasing statistically significant pruritus improvement versus placebo.
- Volixibat, an oral ileal bile acid transporter (IBAT) inhibitor, could expedite development and review for this burdensome symptom of PBC.
- Enrollment in the confirmatory portion of the VANTAGE study is ongoing, with completion expected in 2026, marking a significant step forward.