NCT07245901

Safety and Tolerability of a mRNA Vaccine Encoding Tumor-Specific Circular RNA Antigens in Combination With Anti-PD-1 Monoclonal Antibody in Patients With Advanced Solid Tumors

An Investigator-initiated, Open-Label Clinical Study of a mRNA Vaccine Encoding Tumor-Specific Circular RNA Antigens in Combination With Anti-PD-1 Monoclonal Antibody in Patients With Advanced Solid Tumors

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is an Investigator-initiated, Open-Label Clinical Study of a mRNA Vaccine Encoding Tumor-Specific Circular RNA Antigens in Combination with anti-PD-1 monoclonal antibody in Patients with Advanced Solid Tumors. Preclinical studies of this project have identified that circFAM53B, which is specifically overexpressed in tumor cells, possesses the ability to encode a cryptic peptide, FAM53B-219aa, representing a potential tumor vaccine target. In this study, the sequence encoding the cryptic antigen from circFAM53B will be linearized and formulated as an mRNA vaccine (circFAM53B mRNA injection). The vaccine will be administered via lipid nanoparticle (LNP) encapsulation, and its safety and efficacy will be evaluated in combination with anti-PD-1 monoclonal antibody therapy.

Detailed description

This investigator-initiated clinical trial is designed to evaluate the safety and preliminary efficacy of the circFAM53B mRNA vaccine in combination with anti-PD-1 monoclonal antibody therapy using a "3+3" dose-escalation design. The trial consists of two phases: a dose-escalation phase and a dose-expansion phase. In the dose-escalation phase, patients with advanced solid tumors exhibiting high circFAM53B-219aa expression who have failed prior standard therapies will be enrolled. Approximately 10-20 participants will receive the circFAM53B mRNA vaccine to determine the safety profile (DLT) and identify the maximum tolerated dose (MTD). Based on the results from the dose-escalation phase, the dose-expansion phase will be conducted at the identified MTD. This phase will enroll participants from two different tumor types, with approximately 10 patients per cohort, to receive the circFAM53B mRNA vaccine in combination with toripalimab.

Conditions

• Solid Tumor

Interventions

Type	Name	Description
DRUG	circFAM53B mRNA vaccine	IM injection on Day 1 of each 21-day cycle for up to 9 cycles
DRUG	toripalimab	Intravenous infusion once every 21 days

Timeline

• Start date: 2025-12-05
• Primary completion: 2028-12-05
• Completion: 2030-12-05
• First posted: 2025-11-24
• Last updated: 2025-11-24