NCT05050136进行中（未招募）2 期
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
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 333
- 试验地点
- 293
- 主要终点
- Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
Part 1 Arm 1: Volixibat 20mg
Experimental
Participants randomized to this arm will receive volixibat 20mg twice daily.
干预措施: Volixibat (Drug)
Part 1 Arm 2: Volixibat 80mg
Experimental
Participants randomized to this arm will receive volixibat 80mg twice daily.
干预措施: Volixibat (Drug)
Part 1 Arm 3: Placebo
Placebo Comparator
Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
干预措施: Placebo (Drug)
Part 2 Arm 2: Placebo
Placebo Comparator
Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
干预措施: Placebo (Drug)
Part 2 Arm 1: Volixibat Selected Dose 20mg
Experimental
Participants randomized to this arm will receive volixibat 20mg twice daily.
干预措施: Volixibat (Drug)
结局指标
主要结局
Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire
时间窗: 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.
次要结局
- 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)
研究者
研究点 (293)
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相关资讯
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.last yearVolixibat 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.last year