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A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis

ConditionsPrimary Biliary CholangitisPBC
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorMirum Pharmaceuticals, Inc.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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.

Trial design

Design model

Parallel

Treatments tested in this trial

  • Volixibat

    Drug

    Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.

  • Placebo

    Drug

    Capsules matched to study drug without the active pharmaceutical ingredient

Treatment groups

260 Participants
are divided into 5 treatment groups
Group A: Part 1 Arm 1: Volixibat 20mgExperimental treatment 1 intervention
Group B: Part 1 Arm 2: Volixibat 80mgExperimental treatment 1 intervention
Group C: Part 1 Arm 3: PlaceboPlacebo comparator 1 intervention
Group D: Part 2 Arm 1: Volixibat Selected Dose 20mgExperimental treatment 1 intervention
Group E: Part 2 Arm 2: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire

The Adult ItchRO is an 11-point NRS measurement of itch severity ranging from 0=no itch to 10=worst possible itch.

Time frame
Baseline to week 28

Secondary outcomes

1

Proportion of participants with itch response using the Adult ItchRO

Time frame
Baseline to week 28
2

Incidence of adverse events

Time frame
Baseline to week 28
3

Changes in alkaline phosphatase

Time frame
Baseline to week 28
4

Changes in total bilirubin

Time frame
Baseline to week 28

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Mirum Pharmaceuticals, Inc.

Lead sponsor

Source ClinicalTrials.gov
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