A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Efruxifermin in Subjects With Compensated Cirrhosis Due to NASH/MASH
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 2,150
- 试验地点
- 321
- 主要终点
- Time from randomization to first occurrence of disease progression as measured by composite of protocol-specified clinical events
研究概览
简要总结
This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study in subjects with compensated cirrhosis due to NASH/MASH.
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 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Cohort 1: Biopsy proven compensated cirrhosis (fibrosis stage 4) due to NASH/MASH and NAS score of >=3 (at least 1 in each category) or evidence of steatosis and 2 current features of metabolic comorbidities
- •Cohort 2: Biopsy proven or non-invasively diagnosed compensated cirrhosis (fibrosis stage 4) due to NASH/MASH
排除标准
- •Other causes of liver disease based on medical history and/or liver histology and/or central laboratory results
- •Type 1 diabetes or unstable Type 2 diabetes
- •Any current or prior history of decompensated liver disease
- •Other inclusion and exclusion criteria may apply.
研究组 & 干预措施
EFX 50 mg
干预措施: Efruxifermin (Drug)
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Time from randomization to first occurrence of disease progression as measured by composite of protocol-specified clinical events
时间窗: 5 years
Cohort 1 only: ≥ 1 stage improvement in fibrosis and no worsening of steatohepatitis
时间窗: 96 Weeks
Based on NASH CRN fibrosis score (scored by a fibrosis score of 0-4, where 0 = no fibrosis, 1 = centrilobular pericellular fibrosis, 2 = centrilobular and periportal fibrosis, 3 = bridging fibrosis, 4 = cirrhosis)
Time from randomization to the first significant clinical event including disease progression, liver decompensation events, etc.
时间窗: 5 years
Measured by composite of protocol-specified clinical events
Cohort 1 only: Proportion of subjects with ≥ 1 stage improvement in fibrosis and no worsening of steatohepatitis
时间窗: 96 Weeks
Based on NASH CRN fibrosis score (scored by a fibrosis score of 0-4, where 0 = no fibrosis, 1 = centrilobular pericellular fibrosis, 2 = centrilobular and periportal fibrosis, 3 = bridging fibrosis, 4 = cirrhosis)
次要结局
- To assess the safety and tolerability of EFX through the reporting of extent of exposure (weeks)(96 Weeks)
- Number of participants with abnormal laboratory tests results, abnormal ECGs, abnormal ultrasounds, abnormal vital sign assessments(96 Weeks)
- Cohort 1 only: ≥ 1 stage improvement in fibrosis(96 Weeks, 5 Years)
- Change from baseline of markers of liver injury(96 Weeks)
- Change from baseline of lipoproteins(96 Weeks)
- Change from baseline of body weight (kg)(96 Weeks)
- To assess the safety and tolerability of EFX through the reporting of adverse events (severity of events)(96 Weeks)
- Cohort 1 only: ≥ 1 stage improvement in fibrosis and no worsening of steatohepatitis(5 Years)
- Cohort 1 only: Resolution of NASH/MASH(96 Weeks, 5 Years)
- Cohort 1 only: Resolution of NASH/MASH and a ≥ 1 stage improvement in fibrosis(96 Weeks, 5 Years)
- To assess the safety and tolerability of EFX through the reporting of adverse events (frequency of events)(96 Weeks)
- Change from baseline of non-invasive markers of liver fibrosis(96 Weeks, 5 Years)
- Change from baseline of markers of liver injury(96 Weeks, 5 Years)
- Change from baseline of lipoproteins(96 Weeks, 5 Years)
- Change from baseline of markers of insulin sensitivity and glycemic control(96 Weeks, 5 Years)
- Change from baseline of body weight (kg)(96 Weeks, 5 Years)
- Safety and tolerability of EFX through the reporting of extent of exposure (weeks)(From first dose through end of study (up to 5 Years))
- Safety and tolerability of EFX through the reporting of adverse events (frequency of events)(From first dose through end of study (up to 5 Years))
- Safety and tolerability of EFX through the reporting of adverse events (severity of events)(From first dose through end of study (up to 5 Years))
- Number of participants with abnormal laboratory tests results, abnormal ECGs, abnormal ultrasounds, abnormal vital sign assessments(From first dose through end of study (up to 5 years))
- Cohort 1 only: Proportion of subjects with ≥ 1 stage improvement in fibrosis and no worsening of steatohepatitis(5 Years)
- Cohort 1 only: Proportion of subjects with ≥ 1 stage improvement in fibrosis(96 Weeks)
- Cohort 1 only: Proportion of subjects with Resolution of NASH/MASH(96 Weeks)
- Cohort 1 only: Proportion of subjects with Resolution of NASH/MASH and a ≥ 1 stage improvement in fibrosis(96 Weeks)
- Change from baseline of non-invasive markers of liver fibrosis(96 Weeks)
- Change from baseline of markers of insulin sensitivity and glycemic control(96 Weeks)