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

JRCT ID: jRCT2041240104

Registered date:09/10/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Cholestatic Pruritus in Patients with Primary Biliary Cholangitis
• Date of first enrollment: 02/12/2024
• Target sample size: 10
• Countries of recruitment: US,Japan,France,Japan,Germany,Japan,Italy,Japan,Spain,Japan,China,Japan,Argentina,Japan,Brazil,Japan,Canada,Japan,Israel,Japan,UK,Japan
• Study type: Interventional
• Intervention(s): Volixibat (20 mg or 80 mg) or placebo is administered orally twice daily.

Outcome(s)

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

Secondary Outcome

- Proportion of participants with itch response using the Adult ItchRO
- Incidence of adverse events
- Changes in alkaline phosphatase
- Changes in total bilirubin
- Changes in serum bile acid levels
- Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40)
- Change in Patient-Reported Outcomes Measurement Information System (PROMIS) fatigue questionnaire
- Change in Patient-Reported Outcomes Measurement Information System (PROMIS) sleep disturbance questionnaire

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include 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 and anti-pruritic medication use will be allowed if meeting additional criteria
- Qualified pruritus associated with PBC as assessed by Adult ItchRO

Exclude 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

Related Information

• Primary Sponsor: Misaki Masako
• Secondary ID(s): NCT05050136