A Phase 2b/3, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Safety, and Tolerability of Aldafermin in Subjects With Primary Sclerosing Cholangitis (ALPINE-PSC).
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- 入组人数
- 300
- 主要终点
- Change From Baseline in Enhanced Liver Fibrosis Score at Week 96
研究概览
简要总结
A multi-center evaluation of aldafermin in a randomized, double-blind, placebo-controlled study in subjects with Primary Sclerosing Cholangitis.
详细描述
Evaluation of Efficacy, Safety and Tolerability of Aldafermin in a Phase 2b/3, Randomized, Double-blind, Placebo-controlled, Multi-center Study in Subjects with Primary Sclerosing Cholangitis (ALPINE-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)
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
- •Able and willing to give informed consent
- •Confirmed diagnosis of PSC based on either:
- •Abnormal cholangiography consistent with PSC as measured by MRCP with contrast
- •Liver biopsy consistent with PSC
- •LSM by VCTE, indicative of at least F2 liver fibrosis stage
- •Laboratory parameters:
- •TBL ≤2.5 mg/dL unless known for Gilbert's Syndrome
- •ALT and ALT <5x ULN
- •MELD score ≤12
排除标准
- •Clinically significant acute or chronic liver disease of an etiology other than PSC
- •Alternate causes of sclerosing cholangitis including IgG4 related sclerosing cholangitis
- •Subjects with moderate to severe hepatic impairment
- •Subjects with moderate to severe renal impairment
- •Placement of bile duct stent or percutaneous bile duct drain within 12 weeks of Screening
研究组 & 干预措施
aldafermin
Aldafermin 3mg given subcutaneously, daily, for up to 6 years
干预措施: aldafermin (Drug)
placebo
Placebo given subcutaneously, daily, for up to 6 years
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline in Enhanced Liver Fibrosis Score at Week 96
时间窗: 96 Weeks
Enhanced Liver Fibrosis (ELF) score is a non-invasive blood test derived from the measurement of hyaluronic acid (HA), amino terminal propeptide of type III procollagen (PIIINP), and tissue inhibitor of metalloprotease 1 (TIMP1) using a proprietary algorithm (Siemens). ELF score is a laboratory test, is unitless, and is used as a continuous variable. The minimal ELF score is zero, the maximal ELF score is unknown. The higher the ELF score, the worse the disease outcome. ELF is a score on a scale of severity assessment against biopsy-proven fibrosis. A score of \<7.7 is none to mild, \> 7.7-9.8 is moderate, \> 9.8 is severe.
次要结局
未报告次要终点