Exploratory Study on the Therapeutic Effect Prediction Model of Advanced BTC Immunotherapy

Phase 1

Active, not recruiting

• Conditions: Biliary Tract Cancer; Predictive Cancer Model; Immune Checkpoint Inhibitor
• Interventions: Drug: Immune Checkpoint Inhibitors

• Registration Number: NCT06074029
• Lead Sponsor: Peking Union Medical College Hospital

Brief Summary

1. Establish a predictive model for the efficacy of immune checkpoint inhibitors (ICI) in Chinese patients with biliary tract cancers. By analyzing the dynamic changes of circulating tumor DNA (ctDNA) and other clinical and pathological features before and after ICI treatment in a cohort of patients with biliary tract tumors, a predictive model can be established to evaluate the efficacy of ICI treatment in the early stages or even before treatment, serving as a reliable tool for selecting patients who are likely to benefit from ICI treatment.

2. Investigate the clinical features of populations that benefit from different immune combination therapies. By comparing the differences and enrichment of mutations between patients receiving different treatment regimens, and if patients have sufficient pre-treatment tissue, further comparisons of differentially expressed genes and pathways may be made.

Detailed Description

Not available

Study Timeline

• Study Start: 2021-12-01
• Study First Submitted: 2023-09-24
• Study First Submitted QC: 2023-10-05
• Study First Posted: 2023-10-10
• Primary Completion: 2023-12-30
• Status Verified: 2024-02
• Last Update Submitted: 2024-02-07
• Last Update Posted: 2024-02-08
• Study Completion: 2024-08-01

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

• The subjects voluntarily participate in the study and agree to sign the informed consent form, are compliant, and cooperate with follow-up.
• Patients with advanced biliary tract cancers (including gallbladder cancer, intrahepatic cholangiocarcinoma (ICC), hilar cholangiocarcinoma, or distal cholangiocarcinoma) with histologically, cytologically, or clinically confirmed.
• Patients who have undergone curative surgery and experienced disease recurrence after more than 6 months; or patients who have completed adjuvant therapy (chemotherapy and/or radiotherapy) and have been disease-free for more than 6 months after completing adjuvant therapy are eligible for inclusion.
• Regardless of gender, age 18-80 years old.
• ECOG PS of 0-2 with expected survival time ≥12 weeks.
• According to RECIST V1.1, the patient had at least one measurable lesion.
• The patient is suitable for treatment with immune checkpoint inhibitors (PD-1/PD-L1 inhibitor).

Exclusion Criteria

• Suffering from other malignant tumors.
• Previous systemic treatment received.
• Patients with serious organic diseases cannot receive the treatment designed in this study;
• Having a psychiatric disorder does not guarantee compliance with this study.
• Patients with symptomatic brain metastasis or complications related to brain metastasis or cognitive impairment.
• Adverse events (except hair loss of any grade) from previous antitumor therapy did not return to ≤ grade 1 or better (According to CTCAE version 5.0).
• Previously received any cell or organ transplant treatment.
• Allergy to the therapeutic drugs of this study or CT or MRI-enhanced contrast agents.
• Received local hepatobiliary treatment (including various types of ablations, percutaneous ethanol or acetic acid injection, high-intensity focused ultrasound, transarterial embolization, chemotherapy, or chemoembolization) within 14 days before the start of the study treatment.
• The physician determines that there are any contraindications to ICI treatment.
• During the second blood monitoring, the disease changes could not be judged (such as unexplained ascites, worsening symptoms); Or non-disease progression, but the next immunotherapy is not planned.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Immunotherapy cohort	Immune Checkpoint Inhibitors	Patients who were administered with immunotherapy

Primary Outcome Measures

Name	Time	Method
Progression free survival	Baseline up to approximately 12 months	It refers to the time from the first administration of the regimen to the first occurrence of disease progression or death due to any cause in the subjects.

Secondary Outcome Measures

Name	Time	Method
Overall survival	Baseline up to approximately 15 months	The time from randomization to death due to any cause in the subjects.
Objective response rate	Baseline up to approximately 6 months	The proportion of patients whose tumor volume shrinks to a predetermined value and can maintain the minimum required duration, including complete response and partial response.
Time to treatment discontinuation	Baseline up to approximately 12 months	The time from the beginning of ICI drug use to the cessation of ICI drug use for any reason

Trial Locations

Locations (1)

Chinese Academy of Medical Sciences & Peking Union Medical College Hospital (CAMS&PUMCH); 🇨🇳 Beijing, Beijing, China