Clinical trial NCT06123858: Non-alcoholic Steatohepatitis Registry Platform Study
Official title: Non-alcoholic Steatohepatitis Registry Platform Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: NASH With Fibrosis
Keywords: NASH；
NAFLD；
fibrosis.
Lead sponsor: AstraZeneca
Sponsor class: INDUSTRY
First posted: 2023-11-09
Enrollment: 2500
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Subjects with NASH and fibrosis.

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

(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:

1. Liver biopsy was performed within 24 months before enrollment, and the pathological diagnosis was NASH and the fibrosis stage was F2 or above;
2. FibroScan-AST (FAST) score ≥ 0.30 within 24 months before enrollment;
3. There are any of the following evidences of fatty liver

   1. Controlled attenuation parameter (CAP) \> 285 dB/m within 24 months before enrollment;
   2. Liver ultrasonography showed fatty liver within 24 months before enrollment;
   3. 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

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   1. Liver stiffness measurement (LSM) ≥ 7.5kPa by vibration-controlled transient elastography (VCTE) within 24 months before enrollment;
   2. The elasticity value of magnetic resonance elastography (MRE) examination within 24 months before enrollment is ≥2.93kPa;
   3. 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.

Exclusion Criteria:

1. History of liver transplantation;
2. Patients with bleeding tendency or coagulation dysfunction (for example, bleeding tendency, such as hemophilia, suspected hemangioma or suspected hydatid infection);
3. 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;
4. Have a history of malignant tumors in the past 5 years, excluding malignant tumors that have been cured by the investigator's judgment;
5. Pregnant or lactating women.

Primary outcomes:
measure: 1.Patient demography
timeFrame: Baseline
measure: 2.The proportion of 4 NASH treatment modalities at baseline and follow-up
timeFrame: up to 3-year follow-up
measure: 3.NASH Knowledge, Belief and Practice Questionnaire responses at baseline and follow-up
timeFrame: up to 3-year follow-up
Secondary outcomes:
measure: 1. The correlation between non-invasive diagnostic methods and pathological results of liver biopsy in NASH patients
timeFrame: up to 3-year follow-up
measure: 2.The annual frequency of liver related visits
timeFrame: up to 3-year follow-up
measure: 3.Total liver-related cost
timeFrame: up to 3-year follow-up
Other outcomes:
measure: 1.Clinical, histological and biological factors associated NASH diagnosis will be assessed using multivariate correlation
timeFrame: up to 3-year follow-up
measure: 2.Incidence of endpoint events during follow-up period
timeFrame: up to 3-year follow-up
measure: 3.Changes in Liver enzymes levels during the follow-up period compared with the baseline period
timeFrame: up to 3-year follow-up
measure: 4.Changes in the degree of steatosis during the follow-up period compared with the baseline period
timeFrame: up to 3-year follow-up
measure: 5.Changes in fibrosis grades during the follow-up period compared with the baseline period
timeFrame: up to 3-year follow-up
Locations:
city: Beijing
country: China
facility: Research Site
city: Binzhou
country: China
facility: Research Site
city: Changchun
country: China
facility: Research Site
city: Chengdu
country: China
facility: Research Site
city: Fuzhou
country: China
facility: Research Site
city: Guangzhou
country: China
facility: Research Site
city: Hangzhou
country: China
facility: Research Site
city: Inner Mongolia
country: China
facility: Research Site
city: Nanchang
country: China
facility: Research Site
city: Qingdao
country: China
facility: Research Site
city: Shanghai
country: China
facility: Research Site
city: Shenyang
country: China
facility: Research Site
city: Taiyuan
country: China
facility: Research Site
city: Tianjin
country: China
facility: Research Site
city: Wenzhou
country: China
facility: Research Site
city: Xi'an
country: China
facility: Research Site
city: Xuzhou
country: China
facility: Research Site
city: Zhengzhou
country: China
facility: Research Site
Study officials:
affiliation: Hepatopancreatobiliary Center, Beijing Tsinghua Changgung Hospital, Tsinghua University
name: Lai Wei
role: PRINCIPAL_INVESTIGATOR
References: