Actively Recruiting
A Clinical Trial Assessing the Safety and Efficacy of Intravenous HNF4α srRNA for the Treatment of Patients With Advanced Intrahepatic Cholangiocarcinoma
Led by Shanghai Changzheng Hospital · Updated on 2025-04-24
9
Participants Needed
1
Research Sites
8 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and efficacy of intravenous HNF4α srRNA treatment in adults with advanced intrahepatic cholangiocarcinoma (ICC), a type of liver cancer. This early phase 1, single-arm, open-label pilot study aims to assess how well patients tolerate the treatment and its effects on tumor response and survival. The study also explores the impact of the drug on tumor biomarkers and patient-reported outcomes over an average period of two years. Participants will receive HNF4α srRNA intravenously through a peripheral vein. The dosing schedule includes a second dose about 14 days after the initial treatment, followed by subsequent doses approximately every 28 days. Dose adjustments may be made based on patient tolerance and response. This dose escalation study tests increasing doses of 25, 50, and 100 micrograms. After at least four treatment cycles, some patients with stable or progressive disease may continue treatment combined with immunotherapy, targeted therapy, or chemotherapy, with doses adjusted as needed. During the study, participants will be closely monitored for safety and tolerability during a 14-day observation after the first dose. Researchers will regularly assess tumor response using RECIST v1.1 criteria and collect data on progression-free survival, overall survival, and quality of life through patient-reported outcomes. Laboratory tests, tumor biomarker analysis, and clinical evaluations will occur throughout the treatment and follow-up period lasting up to two years. The study aims to provide detailed information on treatment effects and participant experiences over time.
CONDITIONS
Brief Title
A Clinical Trial Evaluating the Safety and Efficacy of Intravenous HNF4α srRNA in Treating Advanced ICC Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Males or females aged 18 years or older
- Histologically or cytologically confirmed intrahepatic cholangiocarcinoma
- Patients not suitable for surgery, liver transplantation, or ablation therapy, or with post-surgical recurrence or metastasis
- Patients not suitable for local or systemic treatment, or who have progressed after at least one chemotherapy regimen including gemcitabine, fluoropyrimidine, or platinum
- Life expectancy of 12 weeks or more
- Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 2
- Males with fertility and females of childbearing potential willing to use effective contraception during the study and for 6 months after
- Negative serum pregnancy test for females of childbearing age within 7 days before first dose
- Willingness and ability to provide written informed consent and comply with study protocol
You will not qualify if you...
- Albumin less than 25 g/L or total bilirubin more than 5 times the upper limit of normal
- Aspartate aminotransferase (AST), alkaline phosphatase (ALP), or alanine aminotransferase (ALT) more than 10 times the upper limit of normal
- Creatinine greater than 1.5 times the upper limit of normal or creatinine clearance less than 40 mL/min
- Absolute neutrophil count less than 1.0×10⁹/L, platelets less than 30×10⁹/L, or hemoglobin less than 8.5 g/dL
- International normalized ratio (INR) greater than 2.3
- Poorly controlled hypertension, diabetes, serious heart or lung diseases, or serious dysfunction
- Receipt of local or systemic anti-tumor treatments within 4 weeks or chemotherapy, other trial drugs, or radiotherapy within 2 weeks, except as assessed for disease progression
- Incurable brain metastasis
- Toxicities from prior treatments still grade 2 or higher (except hair loss and those judged tolerable by researchers)
- Recent liver cirrhosis complications such as gastrointestinal bleeding, overt hepatic encephalopathy, or refractory ascites within 2 weeks
- Uncontrolled active infection
- History of other malignancies within 5 years except those with negligible risk
- Hepatitis B virus DNA over 500 copies/mL or hepatitis C virus RNA over 15 U/mL
- Positive for human immunodeficiency virus (HIV)
- Allergy to contrast agents
- Pregnant or lactating women, or women who may become pregnant
- Any medical condition that may prevent participation as judged by the investigator
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 - Repeated 28-day cycles, starting with a second dose 14 ± 3 days after the first, continuing based on patient response and tolerance
Participants receive intravenous HNF4α srRNA for advanced intrahepatic cholangiocarcinoma. The initial dose is followed by a second dose about 14 days later, then subsequent doses every 28 days with possible adjustments based on tolerance and response. After at least 4 cycles, participants with stable or progressive disease may continue treatment alone or combined with other therapies.
1 to 2 visits per cycle (in-person) depending on treatment schedule and assessments
Trial Site Locations
Total: 1 location
1
Shanghai Changzheng Hospital, Naval Medical University
Shanghai, Shanghai Municipality, China, 200003
Actively Recruiting
Research Team
W
Wen-Ping Xu, MD. PhD
W
Weifen Xie, MD. PhD
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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