Study of Intravenous HNF4 srRNA to Treat Adults With Advanced Intrahepatic Cholangiocarcinoma Evaluating Safety, Tolerability, and Effectiveness in a Pilot Study

What this Trial Is About

Researchers are evaluating the safety, tolerability, and efficacy of intravenous HNF4 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 HNF4 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.

A Clinical Trial Evaluating the Safety and Efficacy of Intravenous HNF4α srRNA in Treating Advanced ICC Patients