Abatacept For The Treatment Of Primary Biliary Cirrhosis With An Incomplete Biochemical Response To Ursodeoxycholic Acid
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 16
- 试验地点
- 2
- 主要终点
- Biochemical Response
研究概览
简要总结
The purpose of this study is to determine if abatacept (Orencia) is effective in patients with primary biliary cirrhosis who do not respond adequately to standard treatment with ursodeoxycholic acid (UDCA, Urso, Ursodiol, Actigall).
详细描述
This is an open label, active treatment trial to assess the efficacy and safety of abatacept in subject with PBC who have had an incomplete biochemical response to UDCA. In this trial, 20 subjects with PBC who have had an incomplete biochemical response to UDCA will be assigned to treatment with weekly subcutaneous injections of 125 mg of abatacept. The treatment phase of the study will last 24 weeks with an off-treatment follow up at Week 36.
Inclusion criteria include:
- Confirmed diagnosis of PBC
- Alkaline phosphatase > 1.67 times the upper limit of normal after 6 months of treatment with UDCA
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Confirmed PBC diagnosis based upon at least 2 of 3 criteria
- •Anti-mitochondrial antibody (AMA) titer > 1:40
- •Alkaline phosphatase > 1.5 times the upper limit of normal for at least 6 months
- •Liver biopsy findings consistent with PBC
- •Incomplete response to UDCA defined by an alkaline phosphatase > 1.67 X the upper limit of normal after 6 months of UDCA at a minimum dose of 13 mg/kg/d
- •Taking a stable dose of UDCA for at least 3 months prior to Day 0
- •aspartate aminotransferase (AST) and alanine aminotransferase ALT < 5 times the upper limit of normal
排除标准
- •Presence of concomitant liver diseases including viral hepatitis, primary sclerosing cholangitis, alcoholic liver disease, Wilson's disease, hemochromatosis, or Gilbert's syndrome.
- •Prior liver transplantation
- •Decompensated liver disease
- •Use of immunosuppressants within 6 months of Day 0
- •Use of biologic agents within 12 months of Day 0
结局指标
主要结局
Biochemical Response
时间窗: Week 24
Number of Participants with a decrease of alkaline phosphatase by \> 40%of the Day 0 level at 24 weeks of treatment.
次要结局
- Absolute Change in Alkaline Phosphatase(Week 24)
- Primary Billiary Cholangitis Quality of Life(Week 24)
- Percent Change in Alkaline Phosphatase(Week 24)
- Drug Safety(Weeks 2, 4, 12, 24, and 36)
- Liver Stiffness Measured by Magnetic Resonance Elastography(Week 24)
- Absolute Change in Alanine Transferase (ALT)(Week 24)
- Percent Change in Alanine Transferase (ALT)(Week 24)
研究者
Christopher Bowlus, MD
Professor, Gastroenterology and Hepatology, Internal Medicine
University of California, Davis