Precision medicine for liver and biliary cancers using genetic testing

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

This study is testing if personalized treatments based on genetic testing can help people with liver and bile duct cancers live longer and feel better.

Quick facts

• Study type: Observational
• Enrollment: 200 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Tongji Hospital (other)
• Locations: 1 site (Wuhan, Hubei)
• Trial ID: NCT05520788 on ClinicalTrials.gov

What this trial studies

This observational study aims to explore precision treatment options for patients with primary hepatobiliary cancers, specifically hepatocellular carcinoma and biliary tract cancer. It evaluates the efficacy and safety of individualized treatment regimens based on next-generation sequencing (NGS) results. Eligible patients will undergo genetic testing to identify specific mutations, amplifications, or translocations, allowing for targeted therapies that align with their genetic profiles. The study will monitor treatment outcomes, including progression-free survival and overall survival, to assess the effectiveness of these personalized approaches.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 65 with stage IV hepatobiliary cancers confirmed by radiological and pathological assessments.

Not a fit: Patients with earlier stages of hepatobiliary cancer (stage I-III) or those without identifiable genetic mutations may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to more effective and tailored treatment options for patients with hepatobiliary cancers.

How similar studies have performed: Other studies utilizing precision medicine approaches based on genetic testing have shown promising results, indicating potential for success in this novel application.

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.

Where this trial is running

Wuhan, Hubei

• Hepatic Surgery Center, Tongji Hospital, Huazhong University of Science and Technology — Wuhan, Hubei, China (RECRUITING)

Study contacts

• Principal investigator: Zeyang Ding — Tongji Hospital
• Study coordinator: Bixiang Zhang, PhD

Conditions: Hepatocellular Carcinoma, Biliary Tract Cancer, Liver Neoplasms, Biliary Tract Neoplasms, NGS