Ambispective Cohort Study of Precision Medicine for Primary Hepatobiliary Cancer Based on Next-generation Sequencing (CCGLC-006)

Exploring the precise medicine of patients with primary hepatobiliary cancer. And evaluate the efficacy and safety of individualized treatment regimens for primary hepatobiliary cancer based on next-generation sequencing.

Study Overview

• Status: Recruiting
• Conditions: Hepatocellular Carcinoma; Biliary Tract Cancer
• Intervention / Treatment: Genetic: precise medicine

Detailed Description

The purpose of this study is to explore the precise treatment in hepatobiliary cancer patients and evaluate drug efficacy, progression free and overall survival. This trial study is based on genetic tests, then therapeutic target drugs are administered according to the genetic test reports. Patients with genetic abnormalities (such as mutations, amplifications, or translocations) may benefit from precise treatment which targets particular genetic abnormality. The identifications of these genetic abnormalities may help treat hepatobiliary cancer patients.

The genetic tests are performed for the eligible subjects in this study, then therapeutic target drugs are administered according to the genetic test reports. While the precise treatments, follow-ups are conducted to evaluate the efficacy and safety of the target drugs for the subjects, until the overall survival.

• Study Type: Observational
• Enrollment (Estimated): 200

Contacts and Locations

• Study Contact: Name: Bixiang Zhang, PhD; Phone Number: 86-027-83665293; Email: bixiangzhang@hust.edu.cn

Study Locations

• China
  • Hubei
    • Wuhan, Hubei, China, 430030
      • Recruiting
      • Hepatic Surgery Center, Tongji Hospital, Huazhong University of Science and Technology
      • Contact: Ze-yang Ding, M.D.; Phone Number: +8613407156200; Email: dingzyang@sina.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients diagnosed primary hepatobiliary cancer in Tongji Hospital, Wuhan with results of NGS tests.

Description

Inclusion Criteria:

1. Age from 18 to 65, male or female.
2. Radiologically and pathologically confirmed as hepatobiliary cancers with stage IV.
3. Palliative care as the preferred.
4. The result of next-generation sequencing (NGS) test show the patient has gene mutation and also can be treated by the right commercial products that have been approved by the China Food and Drug Administration (CFDA) or the Food and Drug Administration (FDA).
5. ECOG performance status 0-2.
6. Life expectancy ≥3 months.
7. Agree to sign informed consent form.

Exclusion Criteria:

1. Hepatobiliary cancer patient with stageI-III, or with any of the following items will not be eligible for screening. Such as, suitable for the treatment of radical resection, radical resection but evaluation unmeasurable.
2. The result of NGS test show the patient has no gene mutation, or has gene mutation but no medicine.
3. ECOG performance status ≥ 3.
4. Female patients who are pregnant or not using a contraceptive method of birth control.
5. History or presence of serious cardiovascular or cerebrovascular abnormalities.
6. Abnormalities of the hepatic or renal functions, such as jaundice, ascites, bilirubin ≥ 1.5×ULN, alkaline phosphatase ≥ 3×ULN, persistent protein urine≥ grade 3 (according to National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTC-AE] v4.0), creatinine ratio > 3.5g/24 hours, renal failure.
7. Persistent infection > grade 2 (according to National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTC-AE] v4.0).
8. Patient has underwent a major operation 4 weeks prior to screening or has not yet recovered from the operation.
9. Patient with epilepsy, known or untreated brain metastases.
10. The presence of wounds, ulcers or fractures that can not be healed, or with a past history of transplantation.
11. The presence of bleeding events ≥grade 3 (according to National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTC-AE] v4.0) , present evidence or past history of coagulation dysfunction disorders.
12. Known human immunodeficiency virus (HIV) infection history.
13. Patient with drug abuse or unstable compliance.
14. The presence of unresolved toxicity caused by any previous treatment/operation > grade 1 (according to National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTC-AE] v4.0), except alopecia, anemia or hypothyroidism).
15. Investigator consider that the patient should not be enrolled in this study by careful assessment.
16. The subjects participate in any other clinical trial in the meantime.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Precise medicine; All patients should accept next-generation sequencing (NGS) test before treatment.	Genetic: precise medicine; During screening stage, all patients should accept next-generation sequencing (NGS) test.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Progression-free survival (PFS)	Time of progression will be obtained by telephone interview or medical treatment records.	From treatment initiation for advanced or metastatic HCC to the first date of disease progression for any cause up to 2 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Disease Control Rate (DCR)	The proportion of patients who had either stable disease (SD) for ≥ 6 months, a CR or PR after initiation of treatment for HCC	From treatment initiation to SD, CR or PR, up to 2 years
Overall Survival (OS)	Include all-cause death of patients in this study.	2 years
Objective response rates (ORR)	Percentage of patients whose tumors have a complete or partial response to treatment.	From treatment initiation to CR or PR, up to 2 years
Duration of Response (DOR)	duration from the first assessment of the tumor was CR or PR to the time that the first assessment for PD or date of death from any cause	From treatment initiation to PD, up to 2 years
Adverse Drug Reaction (ADR)/Adverse Event (AE)	Patients with treatment-related adverse events as assessed by CTCAE v4.0.	Through study completion, an average of 2 years.
Quality of Life (QoL) after treatment	The life quality of every subject will be assessed every 3 months according to the FACT-Hep questionnaire, which assesses generic HRQL concerns and disease-specific issues.	Through study completion, an average of 2 years.

Collaborators and Investigators

• Sponsor: Tongji Hospital
• Investigators: Study Chair: Wanguang Zhang, Tongji Hospital; Principal Investigator: Zeyang Ding, Tongji Hospital

Study record dates

Study Major Dates

• Study Start (Actual): May 1, 2020
• Primary Completion (Estimated): December 31, 2025
• Study Completion (Estimated): December 31, 2025

Study Registration Dates

• First Submitted: August 28, 2022
• First Submitted That Met QC Criteria: August 28, 2022
• First Posted (Actual): August 30, 2022

Study Record Updates

• Last Update Posted (Estimated): December 19, 2023
• Last Update Submitted That Met QC Criteria: December 13, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Keywords: Liver Neoplasms; NGS; Biliary Tract Neoplasms
• Additional Relevant MeSH Terms: Digestive System Diseases; Neoplasms; Neoplasms by Site; Digestive System Neoplasms; Biliary Tract Diseases; Biliary Tract Neoplasms
• Other Study ID Numbers: TJ-IRB20220129

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: The data have the gene mutations information of patients.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No