Clinical trial NCT05520788: Ambispective Cohort Study of Precision Medicine for Primary Hepatobiliary Cancer Based on Next-generation Sequencing
Official title: Ambispective Cohort Study of Precision Medicine for Primary Hepatobiliary Cancer Based on Next-generation Sequencing
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hepatocellular Carcinoma
Biliary Tract Cancer
Keywords: Liver Neoplasms
Biliary Tract Neoplasms
NGS
Lead sponsor: Tongji Hospital
Sponsor class: OTHER
First posted: 2022-08-30
Enrollment: 200
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients diagnosed primary hepatobiliary cancer in Tongji Hospital, Wuhan with results of NGS tests.

Brief summary: 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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Progression-free survival (PFS)
timeFrame: From treatment initiation for advanced or metastatic HCC to the first date of disease progression for any cause up to 2 year
Secondary outcomes:
measure: Overall Survival (OS)
timeFrame: 2 years
measure: Objective response rates (ORR)
timeFrame: From treatment initiation to CR or PR, up to 2 years
measure: Disease Control Rate (DCR)
timeFrame: From treatment initiation to SD, CR or PR, up to 2 years
measure: Duration of Response (DOR)
timeFrame: From treatment initiation to PD, up to 2 years
measure: Adverse Drug Reaction (ADR)/Adverse Event (AE)
timeFrame: Through study completion, an average of 2 years.
measure: Quality of Life (QoL) after treatment
timeFrame: Through study completion, an average of 2 years.
Other outcomes:
Locations:
city: Wuhan
country: China
facility: Hepatic Surgery Center, Tongji Hospital, Huazhong University of Science and Technology
state: Hubei
Study officials:
affiliation: Tongji Hospital
name: Wanguang Zhang
role: STUDY_CHAIR
affiliation: Tongji Hospital
name: Zeyang Ding
role: PRINCIPAL_INVESTIGATOR
References: