Clinical trial NCT05896956: The Construction of Clinical Database and Multiomics Biobank Based on a Multicentral Prospective Cohort of Benign and Malignant Biliary Tract Diseases
Official title: The Construction of Clinical Database and Multiomics Biobank Based on a Multicentral Prospective Cohort of Benign and Malignant Biliary Tract Diseases 胆道系统良恶性肿瘤前瞻性登记队列数据库与多组学生物样本库建设
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Biliary Tract Diseases
Gallbladder Cancer
Extrahepatic Bile Duct Cancer
Intrahepatic Cholangiocarcinoma
Biliary Tract Neoplasms
Gall Stone
Keywords: biliary tract diseases
gallbladder cancer
biliary tract neoplasms
biliary tract cancer
cohort study
Lead sponsor: RenJi Hospital
Sponsor class: OTHER
First posted: 2023-06-09
Enrollment: 500
Sex: ALL
Minimum age (years): 18
Maximum age (years): 74
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients with benign biliary tumors and biliary malignant tumors diagnosed by pathology, and combined with clinical manifestations and imaging results, the first diagnosis and discharge were diagnosed as patients with advanced biliary malignant tumors.

Brief summary: The aim of the study is to establishing a standardized biobank and a clinical information database for patients with benign and malignant tumors of the biliary system. With follow-up plans and advanced multiomics technology, a multiomics database for patients with benign and malignant tumors of the biliary tract will be further established. Based on the above work, real-world clinical research on the diagnosis and treatment of biliary tract tumors is about to be carried out, and a high-standard cohort research foundation is laid for precision therapy based on multiomics characteristics and molecular typing of biliary tract tumors.

Detailed description: 1. Trial population From the date of commencement of the study, patients who presented to the participating medical institutions in the study and were clinically diagnosed as benign or malignant tumors of the biliary system, and who met the inclusion criteria and did not meet any of the exclusion criteria, were enrolled in the study cohort after signing an informed consent form.

Diagnostic criteria:

Patients with benign biliary tumors and biliary malignant tumors diagnosed by pathology, and combined with clinical manifestations and imaging results, the first diagnosis and discharge were diagnosed as patients with advanced biliary malignant tumors.

Inclusion Criteria:

1. Sign informed consent, have good compliance, and be willing to accept follow-up and provide blood samples
2. Age 18-74 years old, gender is not limited
3. Clinical diagnosis of benign and malignant tumors of the biliary system, including unoperated patients preliminarily diagnosed as benign and malignant tumors of the biliary tract according to the results of imaging examinations and laboratory tests, or pathological examination of patients treated with surgery confirmed as benign and malignant tumors of the gallbladder
4. The primary tumor is located in the extrahepatic bile duct, intrahepatic bile duct, gallbladder floor, gallbladder body, gallbladder neck or gallbladder duct
5. Karnofsky performance score greater than 50

Exclusion Criteria:

1. Patients with biliary malignant tumors, biliary malignant tumor foci are not primary lesions.
2. Patients with biliary malignant tumors, combined with serious central nervous system diseases, respiratory diseases, autoimmune diseases, chronic renal insufficiency and other diseases, long-term use of immunosuppressants, combined with serious uncontrolled infections
3. Patients with biliary malignant tumors, who also have active cardiovascular and cerebrovascular diseases, have cerebrovascular accidents, myocardial infarction, unstable angina pectoris, or grade II. or above congestive heart failure according to the standards of the New York Heart Association, and require serious arrhythmias requiring drug treatment
4. Patients with biliary malignant tumors, women of childbearing age who have a positive blood pregnancy test or have not had a pregnancy test, pregnant or breastfeeding women
5. The patient is participating in other therapeutic clinical trials where treatment measures cannot be clarified or treatment information cannot be collected 2. Sample size calculation In this study, based on the retrospective cohort statistics of CRGGC study, the average number of gallbladder cancer patients admitted to the hospital in previous years, and the proportion of gallbladder cancer to biliary malignant tumors in previous epidemiological investigations, the number of potential research subjects actually diagnosed and treated by each participating unit was estimated, and the study sample size was calculated. The number of cases of benign and malignant biliary tumors included in the cohort of benign and malignant tumors of the biliary tract in this study is expected to reach 60% of the total number of hospitalized patients admitted by the participating units every year, and it is expected that no less than 250 patients with benign and malignant tumors of the biliary tract will be enrolled every year, and 500 patients are expected to be enrolled within a baseline period of 2 years from the date of the start of the study.

3. Specific research content The target population of this study is patients with benign and malignant tumors of the biliary system, and the potential sample group is patients diagnosed with benign and malignant tumors of the biliary system in the participating medical institutions since December 31, 2022 and meet the criteria for inclusion and exclusion of the study, and based on the retrospective cohort statistics of the CRGGC study, the average number of patients with gallbladder cancer admitted to the hospital in previous years, as well as the proportion of gallbladder cancer to biliary malignant tumors in previous epidemiological investigations, the number of potential research subjects actually diagnosed and treated by each participating unit was estimated. Calculate the study sample size. The number of cases of benign and malignant biliary tumors included in the cohort of benign and malignant tumors of the biliary tract in this study is expected to reach 60% of the total number of hospitalized patients admitted by the participating units every year, and it is expected that no less than 250 patients with benign and malignant tumors of the biliary tract will be enrolled every year, and 500 patients are expected to be enrolled within a baseline period of 2 years from the date of the start of the study. This study cohort is a single-disease registry cohort, which does not involve grouping at the stage of establishing the registration cohort, but in the data analysis stage, it can be divided into different subgroups such as biliary malignant tumors, benign biliary tumors, gallbladder cancer, intrahepatic cholangiocarcinoma, and extrahepatic cholangiocarcinoma according to the diagnosis of the included patients.

The research objectives of this study mainly include: (1) establishing a structured special disease dataset standard for biliary tumors based on the diagnosis and treatment norms and clinical pathways of biliary tract tumors; (2) Carry out clinical registration research on biliary tumors and build a special disease cohort; (3) Establish a biobank of biliary tumor cohort, including clinical diagnosis and treatment information such as patient diagnosis, surgery, treatment, progression, survival, and biological samples such as tumor tissue and body fluids; (4) Formulate standard operating specifications and sample quality control plans for the collection, transportation, acceptance, preparation, preservation and detection of biliary tumor biological specimen banks; (5) Formulate unified specimen bank management standards and information and data management processes, promote the intelligent management of specimen banks, and realize the linkage and integration of biobanks and specialized disease cohort databases; (6) Combine biliary tumor database, biomics technology, such as genomics, transcriptomics, proteomics, metabolomics technology, and big data acquisition, storage analysis technology to complete the molecular typing of biliary tumors.

The outcome indicators of this study included overall survival time OS, recurrence-free survival time PFS, R0 resection rate of patients treated with biliary malignancy, and objective response rate ORR. The main measurement indicators of this study include the patient's demographic information, past history, life history, admission, preoperative examination information, surgery, postoperative situation, discharge, follow-up, outcome indicators, routine examination results of biological samples and multi-omics sequencing results, the above data are derived from the original records of prospective case questionnaires, the original records of patients' electronic medical records and the examination results of patients' biological samples. The outcome of follow-up was defined as patients having been followed up for five years, or having a patient lost to follow-up or dying during follow-up.

Eligibility criteria: Inclusion Criteria:

1. Sign informed consent, have good compliance, and be willing to accept follow-up and provide blood samples.
2. Age 18-74 years old, gender is not limited.
3. Clinical diagnosis of benign and malignant tumors of the biliary system, including unoperated patients preliminarily diagnosed as benign and malignant tumors of the biliary tract according to the results of imaging examinations and laboratory tests, or pathological examination of patients treated with surgery confirmed as benign and malignant tumors of the gallbladder.
4. The primary tumor is located in the extrahepatic bile duct, intrahepatic bile duct, gallbladder floor, gallbladder body, gallbladder neck or gallbladder duct.
5. Karnofsky performance score greater than 50.

Exclusion Criteria:

1. Patients with biliary malignant tumors, biliary malignant tumor foci are not primary lesions.
2. Patients with biliary malignant tumors, combined with serious central nervous system diseases, respiratory diseases, autoimmune diseases, chronic renal insufficiency and other diseases, long-term use of immunosuppressants, combined with serious uncontrolled infections.
3. Patients with biliary malignant tumors, who also have active cardiovascular and cerebrovascular diseases, have cerebrovascular accidents, myocardial infarction, unstable angina pectoris, or grade II. or above congestive heart failure according to the standards of the New York Heart Association, and require serious arrhythmias requiring drug treatment.
4. Patients with biliary malignant tumors, women of childbearing age who have a positive blood pregnancy test or have not had a pregnancy test, pregnant or breastfeeding women.
5. The patient is participating in other therapeutic clinical trials where treatment measures cannot be clarified or treatment information cannot be collected.

Primary outcomes:
measure: overall survival
timeFrame: The last follow-up ended with the first of all patients followed up to death, loss to follow-up or withdrawal of informed consent, or follow-up for 2 years.
Secondary outcomes:
measure: progression-free survival
timeFrame: The last follow-up ended with the first of all patients followed up to death, loss to follow-up or withdrawal of informed consent, or follow-up for 2 years.
Other outcomes:
Locations:
Study officials:
affiliation: RenJi Hospital
name: Ying-Bin Liu, PhD
role: PRINCIPAL_INVESTIGATOR
References: