Non-alcoholic Steatohepatitis Registry Platform Study
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- AstraZeneca
- 入组人数
- 1,122
- 试验地点
- 16
- 主要终点
- 1.Patient demography
研究概览
简要总结
This is a multi-center, prospective, observational registry platform study aimed at describing the clinical characteristics and diagnosis and treatment patterns of Chinese patients with nonalcoholic steatohepatitis (NASH) with fibrosis.
详细描述
Non-alcoholic fatty liver disease (NAFLD) is a metabolic stress-induced liver injury closely related to insulin resistance and genetic susceptibility, and its disease spectrum includes non-alcoholic fatty liver (NAFL), non-alcoholic steatohepatitis (NASH), cirrhosis and hepatocellular carcinoma (HCC). NASH is a severe form of NAFLD, defined as more than 5% hepatocellular steatosis combined with intralobular inflammation and hepatocellular ballooning degeneration. By conducting research on the NASH patient registry platform and collecting real-world data on the clinical characteristics and diagnosis and treatment patterns of NASH patients, including demographics, clinical characteristics, treatment, outcomes, healthcare utilization, lifestyle, and genotype, on the one hand, it can provide evidence of scientific value for clinical practice and formulate diagnosis and treatment guidelines, and on the other hand, it can promote the clinical development of NASH drugs in China.
This is a multi-center, prospective, observational registry platform study. Patient recruitment is tentatively scheduled from December 2023 to December 2024. NASH with fibrosis that meets the inclusion criteria but does not meet the exclusion criteria will be consecutively enrolled in the selected center. The estimated number of patients is 2,500, which will be adjusted according to the progress of research. Among these subjects, information on two genes, PNPLA3 and HSD17B13, will be collected from approximately 500 NASH patients with fibrosis stage F2 or higher.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •(1) Subjects must be between 18 and 75 years old when signing the informed consent form, and must have the ability to sign the consent form independently; (2) NASH patients who meet one of the following conditions:
- •Liver biopsy was performed within 24 months before enrollment, and the pathological diagnosis was NASH and the fibrosis stage was F2 or above;
- •FibroScan-AST (FAST) score ≥ 0.30 within 24 months before enrollment;
- •There are any of the following evidences of fatty liver
- •Controlled attenuation parameter (CAP) > 285 dB/m within 24 months before enrollment;
- •Liver ultrasonography showed fatty liver within 24 months before enrollment;
- •Magnetic resonance imaging-derived proton density fat fraction (MRI-PDFF) examination within 24 months before enrollment showed liver fat fraction ≥ 5%; and any of the following evidence of liver fibrosis
- •Liver stiffness measurement (LSM) ≥ 7.5kPa by vibration-controlled transient elastography (VCTE) within 24 months before enrollment;
- •The elasticity value of magnetic resonance elastography (MRE) examination within 24 months before enrollment is ≥2.93kPa;
- •If there is no liver biopsy and no liver elastography equipment is available in the research center, FIB-4>1.3 (under 65 years old) or>2 (over 65 years old) within 3 months before enrollment.
排除标准
- •History of liver transplantation;
- •Patients with bleeding tendency or coagulation dysfunction (for example, bleeding tendency, such as hemophilia, suspected hemangioma or suspected hydatid infection);
- •Exclude liver diseases caused by the following causes: alcoholic steatohepatitis, drug-induced, viral or autoimmune hepatitis, primary hemochromatosis, α1-antitrypsin deficiency, hepatolenticular degeneration, hypothyroidism, inflammatory bowel disease, Cushing's syndrome, celiac disease, β lipoprotein deficiency, lipoatrophic diabetes mellitus, Mauriac syndrome, hypopituitarism, hypogonadism, polycystic ovarian syndrome;
- •Have a history of malignant tumors in the past 5 years, excluding malignant tumors that have been cured by the investigator's judgment;
- •Pregnant or lactating women.
结局指标
主要结局
1.Patient demography
时间窗: Baseline
Gender (male, female) ，age (years)，time since pathological diagnosis of NASH (months)， proportion of comorbidities
2.The proportion of 4 NASH treatment modalities at baseline and follow-up
时间窗: up to 3-year follow-up
NASH treatment modalities include：Drug treatment，Non-drug treatment，Combined treatment and No treatment received
3.NASH Knowledge, Belief and Practice Questionnaire responses at baseline and follow-up
时间窗: up to 3-year follow-up
Describe the responses of patients to the NASH Knowledge, Belief and Practice Questionnaire at baseline and follow-up periods. The questionnaire includes NASH patients' knowledge, attitude, and behavior towards NASH disease, NASH-related medical service needs, expectations, satisfaction, and does not involve the evaluation of scores.
次要结局
- 2.The annual frequency of liver related visits(up to 3-year follow-up)
- 3.Total liver-related cost(up to 3-year follow-up)
- 1. The correlation between non-invasive diagnostic methods and pathological results of liver biopsy in NASH patients(up to 3-year follow-up)