Combining toripalimab with GemCis for treating resectable intrahepatic cholangiocarcinoma

Toripalimab Combined With Gemcitabine and Cisplatin (GemCis) as Preoperative Neoadjuvant Therapy for Resectable Intrahepatic Cholangiocarcinoma (ICC) With High Risk of Recurrence, Phase II, Single-center, Randomized Controlled Trial

This study is testing if combining a new drug called toripalimab with standard chemotherapy can help people with resectable intrahepatic cholangiocarcinoma do better before surgery.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 70 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Tianjin Medical University Cancer Institute and Hospital (Academic / other)
• Drugs / interventions: toripalimab
• Locations: 1 site (Tianjin, Tianjin Municipality)
• Trial ID: NCT06721286 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the effectiveness and safety of toripalimab in combination with gemcitabine and cisplatin (GemCis) as a preoperative neoadjuvant therapy for patients with resectable intrahepatic cholangiocarcinoma (ICC) at high risk of recurrence. Participants will receive the treatment before surgery to assess its impact on tumor characteristics and surgical outcomes. The study aims to determine if this combination can improve patient prognosis compared to standard treatment options.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older with a confirmed diagnosis of resectable intrahepatic cholangiocarcinoma meeting specific tumor criteria.

Not a fit: Patients who have previously received systemic treatment or have severe organ dysfunction may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could enhance surgical outcomes and reduce recurrence rates for patients with intrahepatic cholangiocarcinoma.

How similar studies have performed: Other studies have shown promising results with similar neoadjuvant therapies in various cancers, suggesting potential for success in this approach.

Eligibility criteria

Inclusion Criteria:

1. Age ≥ 18, male or female;
2. Pathological diagnosis confirmed intrahepatic cholangiocarcinoma, which could be resected surgically; One of the following conditions shall be met:

   2.1 Single tumor, diameter greater than 5cm (T1b) 2.2 Single tumor with vascular invasion (T2), multiple tumors ≤ 3 2.3 Tumor penetrating visceral peritoneum (T3) or directly invading surrounding organs (T4) 2.4 Suspicious lymph node metastasis in Zone 8, 12 or 13
3. Can not received systemic treatment before participating in the study;
4. ECOG PS score 0-1;
5. The main organs function normally, and there is no serious blood, heart, lung, liver, kidney, bone marrow and other functional abnormalities and immune deficiency diseases.
6. Laboratory inspection shall meet the following requirements:

   Women of childbearing age must have a negative pregnancy test (serum or urine) within 14 days before enrollment, and voluntarily use appropriate methods of contraception during the observation period and within 8 weeks after the last administration of the study drug; For men, they should be surgically sterilized or agree to use appropriate methods of contraception during the observation period and within 8 weeks after the last administration of the study drug.
7. The patient voluntarily participated and signed the informed consent form;
8. It is expected that the compliance is good, and the efficacy and adverse reactions can be followed up according to the requirements of the scheme.

Exclusion Criteria:

1. Patients who received PD-1, PD-L1, PD-L2, CTLA-4 before enrollment, or directly received another stimulatory or co inhibitory T cell receptor (such as CTLA-4, 0X40, CD137);
2. Any other research drugs within 4 weeks before enrollment;
3. Any active autoimmune disease or history of autoimmune disease (such as interstitial pneumonia, uveitis, enteritis, hepatitis, hypophysitis, vasculitis, myocarditis, nephritis, hyperthyroidism, hypothyroidism after hormone replacement therapy);
4. Congenital or acquired immune function defects, such as human immunodeficiency virus (HIV) infection;
5. Uncontrollable clinical cardiac symptoms or diseases, such as NYHA II and above heart failure unstable angina pectoris, myocardial infarction within one year, and clinically significant supraventricular or ventricular arrhythmia requiring clinical intervention;
6. Severe infection (such as intravenous drip of antibiotics, antifungal or antiviral drugs) occurred within 4 weeks before the first drug administration, or fever of unknown cause\>38.5 ° C occurred during screening/before the first drug administration;
7. History of allogeneic organ transplantation or allogeneic hematopoietic stem cell transplantation;
8. Live attenuated vaccine shall be inoculated within 4 weeks before the first administration or planned during the study period;
9. Suffered from or accompanied by other system malignant tumors in the last 5 years (except cured skin basal cell carcinoma, cervical carcinoma in situ and ovarian cancer);
10. Allergic to any test drug;
11. Pregnant and lactating women, fertile subjects are unwilling to take effective contraceptive measures;
12. Uncontrollable psychosis;
13. Other situations that the researcher thinks are not suitable for inclusion. If the patient has central nervous system metastasis, has serious laboratory examination abnormalities, and is accompanied by family or social factors, it will affect the safety of the subject, or the collection of data and samples.

Where this trial is running

Tianjin, Tianjin Municipality

• Tianjin Medical University Cancer Institute and Hospital — Tianjin, Tianjin Municipality, China (Recruiting)

Study contacts

• Study coordinator: Qiang Wu

Conditions Intrahepatic Cholangiocarcinoma; neoadjuvant therapy; toripalimab