A study to evaluate efficacy and safety of an investigational drug named volixibat in patients with itching caused by primary biliary cholangitis

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 (VANTAGE) - VANTAGE

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2021-001389-39-DE

Enrollment

260

Registered

2021-10-01

Start date

2022-04-19

Completion date

Unknown

Last updated

2024-07-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cholestatic Pruritus in Patients with Primary Biliary Cholangitis (PBC) MedDRA version: 21.0 Level: PT Classification code 10080429 Term: Primary biliary cholangitis System Organ Class: 10019805 - Hepatobiliary disorders

Interventions

Product Name: volixibat Pharmaceutical Form: Capsule, hard INN or Proposed INN: volixibat potassium CAS Number: 1431935-92-0 Current Sponsor code: VLX, V-0027 Other descriptive name: volixibat Concent

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Provide signed informed consent at the screening visit as well as comply with all study visits and requirements through the end of the study. 2. Male or female, age =18 years at the screening visit. 3. Confirmed diagnosis of PBC in line with the AASLD guidelines. 4. UDCA and anti-pruritic medication use will be allowed if meeting additional criteria. 5. Qualified pruritus associated with PBC as assessed by Adult ItchRO. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 180 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 80

Exclusion criteria

Exclusion criteria: 1. Pruritus associated with an etiology other than PBC. 2. Evidence or clinical suspicion of decompensated cirrhosis or a history of decompensation events. 3. Current symptomatic cholelithiasis or inflammatory gallbladder disease. 4. History of small bowel surgery/resection impacting the terminal ileum that may disrupt the enterohepatic circulation. 5. Evidence, history, or suspicion of other liver diseases.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the efficacy of volixibat versus placebo for the treatment of pruritus in participants with PBC as measured by the change in the Adult ItchRO tool in participants with PBC;Secondary Objective: - To evaluate the efficacy of volixibat versus placebo for the treatment of pruritus in participants with PBC using additional measures of pruritus efficacy. - To evaluate safety and tolerability of volixibat versus placebo in participants with PBC-associated pruritus. - To assess volixibat pharmacodynamics. - To evaluate Quality of Life in participants with PBC-associated pruritus treated with volixibat versus placebo.;Primary end point(s): Mean change in Adult ItchRO score comparing baseline with the average of the weekly averaged daily itch scores from Week 16 to end of double-blind study treatment;Timepoint(s) of evaluation of this end point: Baseline and Week 16 to end of double-blind study treatment	—

Measure

Time frame

Main Objective: To evaluate the efficacy of volixibat versus placebo for the treatment of pruritus in participants with PBC as measured by the change in the Adult ItchRO tool in participants with PBC;Secondary Objective: - To evaluate the efficacy of volixibat versus placebo for the treatment of pruritus in participants with PBC using additional measures of pruritus efficacy. - To evaluate safety and tolerability of volixibat versus placebo in participants with PBC-associated pruritus. - To assess volixibat pharmacodynamics. - To evaluate Quality of Life in participants with PBC-associated pruritus treated with volixibat versus placebo.;Primary end point(s): Mean change in Adult ItchRO score comparing baseline with the average of the weekly averaged daily itch scores from Week 16 to end of double-blind study treatment;Timepoint(s) of evaluation of this end point: Baseline and Week 16 to end of double-blind study treatment

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Secondary

Measure	Time frame
Secondary end point(s): - Proportion of participants achieving a reduction of at least 2 points in the weekly averaged daily Adult ItchRO score from baseline to the end of double-blind study treatment - Proportion of participants with relative reductions from baseline in the weekly averaged daily Adult ItchRO score at end of double-blind study treatment of: - =30% - =50% - Mean number of days for each participant with daily Adult ItchRO score at least 2 points lower than the baseline score from baseline to end of double-blind study treatment - Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), events of clinical interest (ECIs), and AEs that lead to discontinuation of study drug - Incidence of clinically relevant laboratory abnormalities - Changes in liver enzymes (ALT, AST, ALP, total bilirubin, and direct bilirubin) - Changes in fasting sBA - Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40) from baseline to end of double-blind study treatment - Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue score from baseline to end of double-blind study treatment - Change in PROMIS sleep score from baseline to end of double-blind study treatment ;Timepoint(s) of evaluation of this end point: Baseline and end of double-blind study treatment	—

Measure

Time frame

Secondary end point(s): - Proportion of participants achieving a reduction of at least 2 points in the weekly averaged daily Adult ItchRO score from baseline to the end of double-blind study treatment - Proportion of participants with relative reductions from baseline in the weekly averaged daily Adult ItchRO score at end of double-blind study treatment of: - =30% - =50% - Mean number of days for each participant with daily Adult ItchRO score at least 2 points lower than the baseline score from baseline to end of double-blind study treatment - Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), events of clinical interest (ECIs), and AEs that lead to discontinuation of study drug - Incidence of clinically relevant laboratory abnormalities - Changes in liver enzymes (ALT, AST, ALP, total bilirubin, and direct bilirubin) - Changes in fasting sBA - Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40) from baseline to end of double-blind study treatment - Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue score from baseline to end of double-blind study treatment - Change in PROMIS sleep score from baseline to end of double-blind study treatment ;Timepoint(s) of evaluation of this end point: Baseline and end of double-blind study treatment

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Countries

Canada, France, Germany, Israel, Italy, United Kingdom, United States

Contacts

Public ContactMirum Clinical Lead

Mirum Pharmaceuticals, Inc.

clinicaltrials@mirumpharma.com+16506674085

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026