NCT04024813终止2 期
A Phase 2, Randomized, Double Blind, Placebo Controlled, Multiple Center Study to Evaluate the Safety, Tolerability, and Efficacy of Seladelpar Administered for 24 Weeks in Adult Patients With Primary Sclerosing Cholangitis (PSC)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 1
- 试验地点
- 10
- 主要终点
- Relative Change in Baseline Serum Alkaline Phosphatase (AP) at Week 24
研究概览
简要总结
The objectives of this study are to evaluate the effect of seladelpar treatment compared to placebo on efficacy, safety, and tolerability in patients with primary sclerosing cholangitis (PSC).
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Dose masking
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 diagnosis of primary sclerosing cholangitis (PSC) based on any two of the following three criteria:
- •Historical evidence of an elevated alkaline phosphatase (AP) > upper limit of normal (ULN) from any prior laboratory result
- •Liver biopsy consistent with PSC
- •Abnormal cholangiography consistent with PSC as measured by magnetic resonance cholangiopancreatography (MRCP), endoscopic retrograde cholangiopancreatography (ERCP), or percutaneous transhepatic cholangiography (PTC)
- •Individuals must have the following specific additional laboratory parameters measured by the Central Laboratory at Screening:
- •AP ≥ 1.5 × ULN and < 8 × ULN
- •Total bilirubin ≤ 2 × ULN
- •Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 5 × ULN
- •Estimated glomerular filtration rate (eGFR) > 60 mL/min/1.73 m^2
- •Platelets ≥ 140 × 10^3/µL
- •International Normalized Ratio (INR) ≤ 1.3 (in the absence of warfarin or other anticoagulant therapy)
- •Albumin ≥ 3.5 g/dL
- •Patients taking ursodeoxycholic acid (UDCA) will be allowed to enroll if meeting the following criteria:
- •Total daily dose of ≤ 20 mg/kg/day
- •A minimum of 6 months of stable treatment
- •Minimum of 12 weeks off treatment prior to Screening if UDCA is recently discontinued
排除标准
- •Clinically significant acute or chronic liver disease of an etiology other than PSC
- •Patients with a diagnosis of overlapping autoimmune hepatitis (AIH) and PSC
- •Secondary or immunoglobulin G4 (IgG4) related sclerosing cholangitis
- •Small duct PSC
- •Presence of a cholangiocarcinoma on cholangiography or MRI at Screening as determined by the central radiologist and the principal investigator (PI) or medical monitor
- •Bile duct stent or percutaneous bile duct drain placement, or balloon dilatation procedure of a stricture within 12 weeks of Screening
- •History, evidence, or high suspicion of cholangiocarcinoma or other hepatobiliary malignancy based on imaging, screening laboratory values, and/or clinical symptoms
- •Presumptive or diagnosed acute cholangitis within 12 weeks of Screening and through Day 1
- •Evidence of compensated or decompensated cirrhosis based on histology, relevant medical complications, or laboratory parameters:
- •Historical liver biopsy demonstrating cirrhosis (eg, Ludwig Stage 4 or Ishak Stage 5)
- •Current or prior history of decompensated liver disease, including ascites, hepatic encephalopathy, variceal bleeding or other clinical conditions consistent with cirrhosis and/or portal hypertension,
- •Liver stiffness > 14.4 kPa by FibroScan, or
- •Combined low platelet count (< 140 × 10^3/µL ) with one of the following:
- •Serum albumin < 3.5 g/dL,
- •INR > 1.3 (not due to antithrombotic agent use), or
- •Total bilirubin > ULN
- •Prior or actively listed for liver transplantation
- •Prior exposure to seladelpar
- •Note: Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Placebo (N=25)
Placebo Comparator
Placebo for the remainder of the study
干预措施: Placebo to match Seladelpar (Drug)
Seladelpar 5 mg (N=25)
Experimental
5 mg seladelpar daily for the remainder of the study
干预措施: Seladelpar (Drug)
Seladelpar 10 mg (N=25)
Experimental
10 mg seladelpar for the remainder of the study
干预措施: Seladelpar (Drug)
Seladepar 25 mg (N=25)
Experimental
25 mg seladelpar for the remainder of the study
干预措施: Seladelpar (Drug)
结局指标
主要结局
Relative Change in Baseline Serum Alkaline Phosphatase (AP) at Week 24
时间窗: Baseline, Week 24
次要结局
- Number of Participants With Treatment-emergent Adverse Events (TEAEs)(Up to Day 59)
- Incidence of Primary Sclerosing Cholangitis (PSC)-Related Symptoms or Procedures(Up to 24 weeks)
- Incidence of Hepatic Disease Progression Events(Up to 24 weeks)
研究者
研究点 (10)
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