NCT02955602已完成2 期
An 8-week, Dose Ranging, Open Label, Randomized, Phase 2 Study With a 44-week Extension, to Evaluate the Safety and Efficacy of MBX-8025 in Subjects With Primary Biliary Cholangitis (PBC) and an Inadequate Response to or Intolerance to Ursodeoxycholic Acid (UDCA)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 119
- 试验地点
- 38
- 主要终点
- Relative Change From Baseline in Serum Alkaline Phosphatase (ALP) at Week 8
研究概览
简要总结
An 8-week, dose ranging, open label, randomized, Phase 2 study with a 44-week extension, to evaluate the safety and efficacy of MBX-8025 in subjects with Primary Biliary Cholangitis (PBC) and an inadequate response to or intolerance to ursodeoxycholic acid (UDCA)
详细描述
Primary:
To evaluate the safety and efficacy of MBX-8025 2 mg, 5 mg, and 10 mg over 8 weeks of treatment
Secondary:
To evaluate the safety and efficacy of MBX-8025 2 mg, 5 mg, and 10 mg over 12 and 26 weeks of treatment
To evaluate the safety and efficacy of MBX-8025 2 mg, 5 mg, and 10 mg over 52 weeks of treatment
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
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must have given written informed consent (signed and dated) and any authorizations required by local law
- •18 to 75 years old (inclusive)
- •Male or female with a diagnosis of PBC, by at least two of the following criteria:
- •History of AP above ULN for at least six months
- •Positive AMA titers (>1/40 on immunofluorescence or M2 positive by enzyme linked immunosorbent assay (ELISA) or positive PBC-specific antinuclear antibodies
- •Documented liver biopsy result consistent with PBC
- •On a stable and recommended dose of UDCA for the past twelve months or intolerant to UDCA
- •AP ≥ 1.67 × ULN
- •Females of reproductive potential must use at least one barrier contraceptive and a second effective birth control method during the study and for at least 90 days after the last dose. Male subjects who are sexually active with female partners of reproductive potential must use barrier contraception and their female partners must use a second effective birth control method during the study and for at least 90 days after the last dose
排除标准
- •A medical condition, other than PBC, that in the investigator's opinion would preclude full participation in the study or confound its results (e.g., cancer on active treatment)
- •AST or ALT > 3 × ULN
- •Total bilirubin > 2.0 mg/dL
- •Total bilirubin > ULN AND albumin < LLN with the exception to subjects with Gilbert's Syndrome. Subjects with Gilbert's syndrome are excluded if Direct Bilirubin > ULN.
- •Auto-immune hepatitis
- •Primary sclerosing cholangitis
- •Known history of alpha-1-Antitrypsin deficiency
- •Known history of chronic viral hepatitis
- •Creatine kinase above ULN
- •Serum creatinine above ULN
- •For females, pregnancy or breast-feeding
- •Use of colchicine, methotrexate, azathioprine, or systemic steroids in the two months preceding screening
- •Current use of fibrates or simvastatin
- •Current use of obeticholic acid
- •Use of an experimental or unapproved treatment for PBC
- •Use of experimental or unapproved immunosuppressant
- •Adverse event leading to MBX-8025 discontinuation from CymaBay's phase 2 PBC study (CB8025-21528)
- •Any other condition(s) that would compromise the safety of the subject or compromise the quality of the clinical study, as judged by the Investigator
研究组 & 干预措施
MBX-8025 (2 mg)
Experimental
MBX-8025 2 mg capsule once daily
干预措施: MBX-8025 2 mg Capsule (Drug)
MBX-8025 (5 mg)
Experimental
MBX-8025 5 mg capsule once daily
干预措施: MBX-8025 5 mg Capsule (Drug)
MBX-8025 (10 mg)
Experimental
MBX-8025 10 mg capsule once daily
干预措施: MBX-8025 10 mg Capsule (Drug)
结局指标
主要结局
Relative Change From Baseline in Serum Alkaline Phosphatase (ALP) at Week 8
时间窗: 8 weeks
Relative change from baseline in serum ALP levels at Week 8 (endpoint). The modified Intent-to-Treat (mITT) analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. n, denotes number of subjects evaluable for the respective timepoints
次要结局
- Percentage of Participants Meet Published PBC Response Criteria - Paris I(12 weeks and 52 weeks)
- Percentage of Participants Meet Composite Endpoint of AP and Total Bilirubin Criteria at Week 12 and Week 52(12 weeks and 52 weeks)
- Percent Change in Serum Alkaline Phosphatase (ALP)(12 weeks and 52 weeks)
- Change in Bilirubin - Total Bilirubin (TB) From Baseline to 12 Weeks and 52 Weeks(12 weeks and 52 weeks)
- Percentage of Participants Meet Published PBC Response Criteria - Paris II(12 weeks and 52 weeks)
- Percentage of Participants Meet Published PBC Response Criteria - Toronto I(12 weeks and 52 weeks)
- Change in Aspartate Aminotransferase (AST) From Baseline to 12 Weeks and 52 Weeks(12 weeks and 52 weeks)
- Percentage of Participants Meet Composite Endpoint Criteria of ALP and Total Bilirubin(12 Weeks and 52 Weeks)
- UK-PBC Risk Score Value(12 weeks and 52 weeks)
- Absolute Change From Baseline in Serum Alkaline Phosphatase (ALP) at Week 12 and Week 52(12 weeks and 52 weeks)
- Change in Alanine Aminotransferase (ALT) From Baseline to 12 Weeks and 52 Weeks(12 weeks and 52 weeks)
- Change in Gamma-glutamyl Transferase (GGT) From Baseline to 12 Weeks and 52 Weeks(12 weeks and 52 weeks)
- Change From Baseline in PBC-40 Quality of Life (QoL) at Week 12 and Week 52(12 weeks and 52 weeks)
- Change From Baseline in Pruritus Visual Analog Score (VAS) at Week 12 and Week 52(12 weeks and 52 weeks)
- Percentage of Participants Meet Published PBC Response Criteria - Barcelona(12 weeks and 52 weeks)
- Change in GLOBE PBC Score From Baseline to 12 Weeks and 52 Weeks(12 weeks and 52 weeks)
- Participants Meet Rotterdam Criteria(12 weeks and 52 weeks)
- Absolute Change in MELD Score From Baseline to 12 Weeks and 52 Weeks(12 weeks and 52 weeks)
研究者
研究点 (38)
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