A Randomized Trial of Surgical Decision-Making Guided by TDTP-RECIST

N/ANot Yet RecruitingNCT07208526

Shanghai Zhongshan Hospital270 enrolled

Overview

This project aims to conduct a prospective, multicenter, randomized controlled clinical trial. The study plans to enroll 270 patients with locally advanced iCCA who have successfully undergone GOLP conversion therapy. Participants will be randomized at a 1:2:2 ratio into three arms: Control Arm (continued medication), Direct Surgery Arm, and TDTP-RECIST Assessment Arm. The primary endpoint is Overall Survival (OS).

Intrahepatic cholangiocarcinoma (iCCA) is a highly aggressive malignancy with a poor prognosis, and a majority of patients are ineligible for curative surgery upon initial diagnosis. Although the GOLP conversion therapy regimen, pioneered by our research team, has demonstrated promising results in preliminary studies-achieving an 80% objective response rate and a 63% R0 resection rate-its clinical value requires validation through high-level evidence-based medicine. Concurrently, the conventional RECIST 1.1 criteria are inadequate for accurately assessing treatment response in iCCA, leading to challenges in surgical decision-making. To address this, we have developed the innovative TDTP-RECIST evaluation system, which preliminary data suggest can more precisely predict surgical opportunities and survival benefits. This multicenter randomized controlled trial aims to primarily validate the clinical value of the GOLP regimen as conversion therapy for locally advanced intrahepatic cholangiocarcinoma (iCCA), thereby providing high-level evidence-based medical support for its establishment as a standard treatment protocol. Furthermore, the study seeks to verify the efficacy and superiority of the TDTP-RECIST evaluation system in guiding surgical decision-making following conversion therapy for iCCA. The ultimate goals are to establish an individualized treatment pathway based on TDTP-RECIST assessments, optimize the timing of surgery, reduce unnecessary surgical interventions and healthcare resource waste, and consequently offer a reliable decision-making framework for the conversion therapy of locally advanced iCCA. This will provide a precise and powerful tool to facilitate the transition from "unresectable" to "curable" disease.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Conditions

Intrahepatic Cholangiocarcinoma (Icc)Conversion Therapy TDTP-RECIST GOLP Regimen

Interventions

GOLP regimenDRUG

PD1 antibody (Toripalimab) combined with GEMOX chemotherapy and Lenvatinib neoadjuvant treatment

SurgeryPROCEDURE

R0 resection

TDTP-RECISTDIAGNOSTIC_TEST

Treatment selection between continued GOLP therapy and surgical R0 resection based on TDTP-RECIST criteria.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 75 Years

Medical Language ↔ Plain English

Inclusion Criteria: \- Subjects eligible to participate in this study must meet all of the following criteria: 1. Male or female aged 18-75 years; 2. Patients must provide signed informed consent prior to enrollment, demonstrating the ability to understand and willingness to sign the written informed consent form; 3. Pathologically confirmed diagnosis of intrahepatic cholangiocarcinoma; 4. Locally advanced disease, failure to achieve R0 resection, and absence of distant metastasis; 5. At least one measurable lesion; 6. Eastern Cooperative Oncology Group (ECOG) performance status score of 0; 7. Child-Pugh class A liver function; Exclusion Criteria: \- Subjects who meet any of the following exclusion criteria are not permitted to enroll in this study: 1. Pathologically diagnosed hepatocellular carcinoma, combined hepatocellular-cholangiocarcinoma, or other non-cholangiocarcinoma malignant components; 2. Patients with postoperative recurrence, or those who have previously received PD-1/PD-L1 antibodies, CTLA-4 antibodies, lenvatinib, or chemotherapy; 3. History or current diagnosis of other malignancies; 4. Active tuberculosis infection; 5. Active, known, or suspected autoimmune disease; 6. History of interstitial lung disease, or non-infectious pneumonitis requiring steroid treatment; 7. Significant clinically significant bleeding symptoms within 3 months prior to enrollment, or clear bleeding tendency; 8. Suspected allergy to the investigational drug(s).

Locations (1)

Zhongshan hospital, Shanghai

Shanghai, China

Outcomes

Primary Outcomes

Overall survival

the time period from the randomization of the patient to the death event due to any reason

Time frame: 24 months

Secondary Outcomes

Event-free survival

From randomization to the occurrence of the following events: disease progression prevents radical surgery; local or distant recurrence; second primary tumor; death due to various causes.

Time frame: 24 months

Objective response rate

The proportion of patients whose tumor volume has decreased to a predetermined value

Time frame: 6 months

Pathological remission rate

the ratio of the estimated active tumor size divided by the tumor bed size

Time frame: 6 months

Recurrence-free survival

the time from surgery to disease recurrence or all-cause death

Time frame: 24 months

R0 Resection Rate

Curative Surgery Resection Rate

Time frame: 12 months

Adverse events

the severity of adverse events will be evaluated according to the NCI CTCAE 5.0 standard

Time frame: 12 months

Central Contacts

Xiao-Yong Huang, MD

CONTACT

+8615021519215 huang.xiaoyong@zs-hospital.sh.cn

Guo-Ming Shi, MD

CONTACT

+8613916969578 shi.guoming@zs-hospital.sh.cn

Data from ClinicalTrials.gov

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