VANTAGE
Research type
Research Study
Full 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 (VANTAGE)
IRAS ID
297743
Contact name
Lara Longpre
Contact email
Sponsor organisation
Mirum Pharmaceutical Inc.
Eudract number
2021-001389-39
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
152328, IND Number
Duration of Study in the UK
4 years, 6 months, 2 days
Research summary
Primary biliary cholangitis (PBC) is a, rare, liver disease in which the bile ducts of the liver are slowly destroyed by the body’s own immune system. PBC is usually diagnosed in patients between 40 and 60 years of age, however patients diagnosed at an earlier age tend to have a worse prognosis.
Severe cholestatic pruritus (itching) is a distressing symptom of PBC that has been reported in up to 70% of patients in prior studies. It can impact the quality of life of patients and their daily life activities, including sleep, resulting in fatigue and depression. Currently there are no drugs approved to treat these symptoms and most of the drugs being used provide relief only in a minority of patients.
Volixibat is a drug being developed by Mirum pharmaceuticals to reduce bile acids within the liver and therefore reduce pruritus associated with PBC.
The study consists of 4 periods:
Screening ( Up to 4 weeks): To assess eligibility for the study.
Study Treatment (28 weeks): This consists of 8 In-clinic visits and 2 or more telephone visits. Eligible patients will be given either placebo (no drug), or Volixibat at 20mg dose twice daily or 80mg dose twice daily. This will be decided randomly.
Open Label Extension (Up to 2 years): After completing the Study Treatment Period, patients who remains eligible and choose to continue in the study will be given volixibat twice a day for up to 2 years. This will consist of up to 10 In-clinic visits and all patients will receive Volixibat during this phase of the study.
Safety Follow-Up period: 1 final In-Clinic visit approximately 30 days following study treatment.
Approximately 260 people will be enrolled in this study worldwide.REC name
South Central - Hampshire B Research Ethics Committee
REC reference
21/SC/0347
Date of REC Opinion
17 Dec 2021
REC opinion
Further Information Favourable Opinion