Actively Recruiting
A Single-Arm Study of Pembrolizumab With Gemcitabine and Cisplatin as Perioperative Therapy for Potentially Resectable Intrahepatic Cholangiocarcinoma
Led by M.D. Anderson Cancer Center · Updated on 2026-04-16
24
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying whether adding pembrolizumab to the standard chemotherapy drugs cisplatin and gemcitabine can improve long-term outcomes for patients with intrahepatic cholangiocarcinoma, a type of liver cancer, after surgery. This phase 2 single-arm study focuses on patients with high-risk but operable disease confined to the liver, bile duct, and regional lymph nodes. The research aims to evaluate recurrence-free survival and major pathological response, among other safety and efficacy markers. Participants will receive four cycles of combined chemotherapy with pembrolizumab, gemcitabine, and cisplatin, each cycle lasting 21 days, administered intravenously before and after their scheduled surgery. Clinic visits occur every three weeks during the approximately nine months of chemotherapy treatment. After this treatment phase, participants will be followed up regularly every three months for 2 to 4 years to monitor their health and disease status. Throughout the study, participants will undergo assessments including imaging scans, laboratory tests, and biomarker analyses to evaluate treatment response, immune effects, and safety. Researchers will track adverse events using standard criteria and monitor outcomes such as overall survival and progression-free survival. Additional studies will explore immune responses and microbiome changes before and after treatment. Total participation may last several years, encompassing treatment and extended follow-up.
CONDITIONS
Brief Title
A Single-Arm Study of Pembrolizumab With Gemcitabine and Cisplatin as Perioperative Therapy for Potentially Resectable Intrahepatic Cholangiocarcinoma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent before any screening procedures
- Age 18 years or older at consent
- Confirmed diagnosis of intrahepatic cholangiocarcinoma or suspected biliary adenocarcinoma measurable by RECIST 1.1
- High-risk but resectable tumors confined to liver, bile duct, and/or regional lymph nodes with specific features seen on recent imaging
- ECOG performance status 0 or 1
- Adequate blood counts and liver function within specified limits
- Adequate kidney function (creatinine clearance 50 mL/min)
- Controlled hepatitis B or C infection as specified
- Willingness and ability to participate and comply with trial requirements
- Prior or concurrent malignancies allowed if not interfering with safety or efficacy assessment
- Use of adequate contraception for women of childbearing potential and men
- Ability to understand and sign informed consent
- Includes English and non-English speaking patients
- Well-controlled HIV infection with stable antiretroviral therapy and specific immune criteria
You will not qualify if you...
- Prior therapy with anti-PD-1, anti-PD-L1, or similar immune agents
- Previous systemic therapy for intrahepatic cholangiocarcinoma
- Certain tumor types such as small cell cancer, neuroendocrine tumors, lymphoma, sarcoma, or mixed histology
- Recent systemic anti-cancer therapy or investigational agents within 4 weeks
- Uncontrolled serious illnesses including recent heart problems
- Active uncontrolled infections requiring systemic treatment
- Recent radiotherapy within 2 weeks before study start
- Recent live vaccines within 30 days before first study dose
- Participation in another investigational study within 4 weeks
- Immunodeficiency or recent immunosuppressive therapy
- Active progressing additional malignancies within 2 years
- Active central nervous system metastases or carcinomatous meningitis
- Severe hypersensitivity to pembrolizumab or its components
- Active autoimmune diseases requiring recent systemic treatment
- History or current pneumonitis/interstitial lung disease requiring steroids
- Concurrent active hepatitis B and C infections
- Conditions or therapies interfering with study participation
- Psychiatric or substance abuse disorders interfering with cooperation
- Pregnancy, breastfeeding, or planning conception during study and 120 days after
- History of allogenic tissue or organ transplant
- Cognitive impairment
- HIV-infected participants with history of Kaposi's sarcoma or Multicentric Castleman's Disease
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 9 months
Participants receive 4 cycles (21 days each) of pembrolizumab with gemcitabine and cisplatin chemotherapy before and after their scheduled surgery.
Clinic visits every 3 weeks during chemotherapy
Duration - 2 to 4 years
Participants are monitored after treatment completion to assess safety, survival, and disease recurrence.
Clinic visits every 3 months
Trial Site Locations
Total: 1 location
1
M D Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
H
Hop Tran Cao, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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