Intrahepatic Cholangiocarcinoma (Icc)NCT07208526
A Randomized Trial of Surgical Decision-Making Guided by TDTP-RECIST
A study testing GOLP regimen, Surgery and TDTP-RECIST in people with Intrahepatic Cholangiocarcinoma (Icc) and Conversion Therapy, run by Shanghai Zhongshan Hospital at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Intrahepatic Cholangiocarcinoma (Icc), Conversion Therapy, TDTP-RECIST
- What is being tested?
- GOLP regimen, Surgery and TDTP-RECIST
- Type of treatment
- Pill or oral medicine, Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 270 participants
- Where
- 1 siteChina
- Study dates
- 1 Dec 2025to 31 Dec 2028
- Phase
- Interventional
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- -
- Subjects eligible to participate in this study must meet all of the following criteria:
- Male or female aged 18-75 years;
- Patients must provide signed informed consent prior to enrollment, demonstrating the ability to understand and willingness to sign the written informed consent form;
- Pathologically confirmed diagnosis of intrahepatic cholangiocarcinoma;
- Locally advanced disease, failure to achieve R0 resection, and absence of distant metastasis;
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- At least one measurable lesion;
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0;
- Child-Pugh class A liver function;
Who cannot join
- -
- Subjects who meet any of the following exclusion criteria are not permitted to enroll in this study:
- Pathologically diagnosed hepatocellular carcinoma, combined hepatocellular-cholangiocarcinoma, or other non-cholangiocarcinoma malignant components;
- Patients with postoperative recurrence, or those who have previously received PD-1/PD-L1 antibodies, CTLA-4 antibodies, lenvatinib, or chemotherapy;
- History or current diagnosis of other malignancies;
- Active tuberculosis infection;
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- Active, known, or suspected autoimmune disease;
- History of interstitial lung disease, or non-infectious pneumonitis requiring steroid treatment;
- Significant clinically significant bleeding symptoms within 3 months prior to enrollment, or clear bleeding tendency;
- Suspected allergy to the investigational drug(s).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Zhongshan hospital, Shanghai
Shanghai, 200032
Xiao-yong Huang, MD, +8615021519215, huang.xiaoyong@zs-hospital.sh.cn
Guo-Ming Shi, MD, +8613916969578, shi.guoming@zs-hospital.sh.cn
About this study
Official title: A Multicenter Randomized Controlled Study on Surgical Decision-Making Guided by the TDTP-RECIST Evaluation System for Locally Advanced Intrahepatic Cholangiocarcinoma Following GOLP Conversion Therapy
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).
Read the detailed description
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.
- Being tested
- GOLP regimen, Surgery, TDTP-RECIST
- Medical subject headings
- Cholangiocarcinoma, Cirrhosis, Familial, with Pulmonary Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GOLP regimenDrug
PD1 antibody (Toripalimab) combined with GEMOX chemotherapy and Lenvatinib neoadjuvant treatment
SurgeryProcedure or surgery
R0 resection
TDTP-RECISTDiagnostic test
Treatment selection between continued GOLP therapy and surgical R0 resection based on TDTP-RECIST criteria.
| Study group | Role | What they receive |
|---|---|---|
| Medication Group | Placebo comparator | Upon randomization, patients will continue to receive the GOLP regimen for up to 8 cycles, followed by maintenance therapy with lenvatinib plus toripalimab for one year, until disease progression, withdrawal from the study, or the occurrence of intolerable toxicity. Receives: Drug: GOLP regimen |
| Surgery Group | Experimental | Surgical resection is to be performed within 2-4 weeks after randomization, followed by adjuvant therapy with capecitabine at a dose of 1250 mg/m² twice daily, administered orally for eight cycles (each cycle consists of 14 days of medication followed by a 7-day rest period, constituting a 3-week treatment cycle). Receives: Procedure: Surgery |
| TDTP Group | Experimental | Following randomization, patients will be evaluated using the TDTP-RECIST system. Those who meet the predetermined cutoff value (63%) will proceed to surgical resection. Patients not meeting the cutoff value will continue the GOLP regimen for up to 8 cycles, during which those who subsequently meet the cutoff value will undergo surgery. Postoperative adjuvant therapy will consist of capecitabine (1250 mg/m², BID, orally) for eight cycles (each cycle: 14 days on, 7 days off, constituting a 3-week cycle). Patients who do not meet the cutoff value after 8 cycles of GOLP therapy will receive maintenance therapy with lenvatinib plus toripalimab for up to one year or until intolerable toxicity occurs. Receives: Diagnostic Test: TDTP-RECIST |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival
the time period from the randomization of the patient to the death event due to any reason
Measured: 24 months
Secondary outcomes (6)
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.
Measured: 24 months
Objective response rate
The proportion of patients whose tumor volume has decreased to a predetermined value
Measured: 6 months
Pathological remission rate
the ratio of the estimated active tumor size divided by the tumor bed size
Measured: 6 months
Recurrence-free survival
the time from surgery to disease recurrence or all-cause death
Measured: 24 months
R0 Resection Rate
Curative Surgery Resection Rate
Measured: 12 months
Adverse events
the severity of adverse events will be evaluated according to the NCI CTCAE 5.0 standard
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Oct 2025
- Study start1 Dec 2025 (estimated)
- Primary completion30 Jun 2028 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Intrahepatic Cholangiocarcinoma (Icc)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 270 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Zhongshan Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Shanghai Geriatric Medical Center, Shanghai Jiao Tong University Affiliated Sixth People's Hospital, Shanghai 10th People's Hospital, Oriental Hepatobiliary Surgery Hospital Affiliated to Naval Military Medical University
- Responsible party
- Principal investigator, Jia Fan, Shanghai Zhongshan Hospital
- Investigators
- Jia Fan, MD&PhD, Study chair, Shanghai Zhongshan Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07208526
- Sponsor's protocol ID
- ZSAB-TDTP-transGOLP
- Status last verified
- Sep 2025
- First submitted
- 28 Sep 2025
- Last updated
- 6 Oct 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Shi G, Huang X, Li X, Liang F, Gao Q, Zhang D, Lu J, Ji Y, Hu Z, Chen Y, Qiu S, Yi Y, Zhu X, Sun H, Shi Y, Peng M, Wang X, Huang C, Ding Z, He Y, Shen Y, Xu Y, Xiao Y, Hu J, Zhou J, Fan J. Conversion therapy of tislelizumab plus lenvatinib and GEMOX in unresectable locally advanced biliary tract cancer (ZSAB-TransGOLP): a multicentre, prospective, phase 2 study. Lancet Oncol. 2025 Oct;26(10):1334-1345. doi: 10.1016/S1470-2045(25)00376-6. Epub 2025 Aug 29.
PubMed 40889502Shi GM, Huang XY, Wu D, Sun HC, Liang F, Ji Y, Chen Y, Yang GH, Lu JC, Meng XL, Wang XY, Sun L, Ge NL, Huang XW, Qiu SJ, Yang XR, Gao Q, He YF, Xu Y, Sun J, Ren ZG, Fan J, Zhou J. Toripalimab combined with lenvatinib and GEMOX is a promising regimen as first-line treatment for advanced intrahepatic cholangiocarcinoma: a single-center, single-arm, phase 2 study. Signal Transduct Target Ther. 2023 Mar 17;8(1):106. doi: 10.1038/s41392-023-01317-7.
PubMed 36928584Oh DY, He AR, Bouattour M, Okusaka T, Qin S, Chen LT, Kitano M, Lee CK, Kim JW, Chen MH, Suksombooncharoen T, Ikeda M, Lee MA, Chen JS, Potemski P, Burris HA 3rd, Ostwal V, Tanasanvimon S, Morizane C, Zaucha RE, McNamara MG, Avallone A, Cundom JE, Breder V, Tan B, Shimizu S, Tougeron D, Evesque L, Petrova M, Zhen DB, Gillmore R, Gupta VG, Dayyani F, Park JO, Buchschacher GL Jr, Rey F, Kim H, Wang J, Morgan C, Rokutanda N, Zotkiewicz M, Vogel A, Valle JW. Durvalumab or placebo plus gemcitabine and cisplatin in participants with advanced biliary tract cancer (TOPAZ-1): updated overall survival from a randomised phase 3 study. Lancet Gastroenterol Hepatol. 2024 Aug;9(8):694-704. doi: 10.1016/S2468-1253(24)00095-5. Epub 2024 May 29.
PubMed 38823398Kelley RK, Ueno M, Yoo C, Finn RS, Furuse J, Ren Z, Yau T, Klumpen HJ, Chan SL, Ozaka M, Verslype C, Bouattour M, Park JO, Barajas O, Pelzer U, Valle JW, Yu L, Malhotra U, Siegel AB, Edeline J, Vogel A; KEYNOTE-966 Investigators. Pembrolizumab in combination with gemcitabine and cisplatin compared with gemcitabine and cisplatin alone for patients with advanced biliary tract cancer (KEYNOTE-966): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2023 Jun 3;401(10391):1853-1865. doi: 10.1016/S0140-6736(23)00727-4. Epub 2023 Apr 16.
PubMed 37075781Colangelo M, Di Martino M, Polidoro MA, Forti L, Tober N, Gennari A, Pagano N, Donadon M. Management of intrahepatic cholangiocarcinoma: a review for clinicians. Gastroenterol Rep (Oxf). 2025 Jan 26;13:goaf005. doi: 10.1093/gastro/goaf005. eCollection 2025.
PubMed 39867595Moris D, Palta M, Kim C, Allen PJ, Morse MA, Lidsky ME. Advances in the treatment of intrahepatic cholangiocarcinoma: An overview of the current and future therapeutic landscape for clinicians. CA Cancer J Clin. 2023 Mar;73(2):198-222. doi: 10.3322/caac.21759. Epub 2022 Oct 19.
PubMed 36260350