Safety, Tolerability, and Preliminary Antitumor Activity of Cationic Peptide-IL22BP mRNA in Advanced Solid Tumors

A Clinical Trial of Cationic Peptide-IL22BP mRNA for Safety, Tolerability, and Preliminary Antitumor Activity in Patients With Advanced Malignant Solid Tumors

The goal of this phase 1 clinical trial is to evaluate the safety, tolerability, and preliminary antitumor activity of a peptide-delivered IL-22BP biotherapy in patients with advanced solid tumors. The main questions it aims to answer are:

Is the IL-22BP formulation safe and tolerable? Does the IL-22BP formulation show preliminary antitumor activity?

Study Overview

• Status: Not yet recruiting
• Conditions: Solid Tumor Cancer; Cancer Metastatic; mRNA Vaccine; Peptides
• Intervention / Treatment: Biological: CP-IL22BP mRNA intratumorally injection

Detailed Description

This is a phase 1, single-arm, open-label, dose-escalation clinical trial designed to evaluate a peptide-based IL-22BP mRNA gene therapy in patients with refractory advanced malignant solid tumors. The investigational product uses a peptide delivery platform composed of natural amino acids, which is intended to improve biocompatibility, reduce toxicity, simplify manufacturing, and avoid key patent limitations associated with conventional lipid nanoparticle (LNP) systems. Unlike a traditional tumor vaccine, this intervention is designed as a targeted gene therapy that promotes intratumoral expression of IL-22BP protein to inhibit tumor progression. Preclinical toxicology studies of the peptide-mRNA complex have shown an acceptable safety profile, with no notable abnormalities in hematological or biochemical parameters. The study will use a standard "3+3" dose-escalation design to assess three fixed mRNA dose levels. The primary objective is to characterize the safety and tolerability of the IL-22BP gene formulation, and the secondary objective is to evaluate its preliminary antitumor activity.

• Study Type: Interventional
• Enrollment (Estimated): 18
• Phase: Phase 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Male or female patients aged ≥18 years and ≤70 years.
• Histopathologically confirmed advanced recurrent/metastatic malignant solid tumors that are refractory to second-line treatment and have no available standard clinical therapeutic options (e.g., advanced soft tissue sarcoma, advanced head and neck squamous cell carcinoma, malignant melanoma, etc.).
• Eastern Cooperative Oncology Group (ECOG) performance status score of 0-1.
• Expected overall survival ≥3 months.
• Interval from the last chemotherapy, radiotherapy, or surgery ≥28 days.
• Interval from the last use of nitrosourea or mitomycin C ≥6 weeks.
• Adequate organ function, defined by the following laboratory parameters within 14 days prior to enrollment:
• Hemoglobin ≥90 g/L (no blood transfusion within 14 days).
• Absolute neutrophil count >1.5 × 10⁹/L.
• Platelet count ≥80 × 10⁹/L.
• Total bilirubin ≤1.5 × upper limit of normal (ULN).
• Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≤2.5 × ULN (≤5 × -ULN in case of liver metastasis).
• Creatinine clearance ≥60 mL/min (calculated by Cockcroft-Gault formula).
• Left ventricular ejection fraction (LVEF) ≥50%.
• Signed written informed consent.

Exclusion Criteria:

• Participation in another investigational drug clinical trial within 4 weeks.
• Tumor located adjacent to major blood vessels or trachea.
• Uncontrolled cardiac clinical symptoms or diseases, including New York Heart Association (NYHA) Class ≥2 heart failure, unstable angina, myocardial infarction within 1 year, or clinically significant supraventricular/ventricular arrhythmias requiring treatment or intervention.
• Female patients who are pregnant or lactating.
• Active pulmonary tuberculosis, bacterial or fungal infection (≥Grade 2 per NCI-CTCAE version 5.0), active human immunodeficiency virus (HIV) infection, active hepatitis B virus (HBV) infection, or hepatitis C virus (HCV) infection.
• History of uncontrollable psychoactive substance abuse or presence of mental disorders.
• Any active autoimmune disease or history of autoimmune disease, including but not limited to uveitis, enteritis, hypophysitis, nephritis, hyperthyroidism, and hypothyroidism.
• Subjects with vitiligo or childhood asthma in complete remission (no adult intervention required) are eligible; subjects with asthma requiring bronchodilator therapy are excluded.
• Receiving immunosuppressive therapy.
• History of drug abuse or known medical, psychological, or social conditions that interfere with study compliance (e.g., alcoholism or illicit drug addiction).
• Known hypersensitivity, allergy, or intolerance to the study drug CPIL22BP mRNA (including any excipients), or history of severe allergic reactions to any drugs, foods, or vaccines (e.g., anaphylactic shock, allergic laryngeal edema, allergic dyspnea, allergic purpura, thrombocytopenic purpura, local Arthus reaction, etc.).
• Female subjects planning pregnancy, or male subjects whose partner plans pregnancy, from screening until 12 months after the last study drug injection.
• Any concomitant disease that, in the investigator's judgment, may seriously compromise patient safety or prevent the subject from completing the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Intratumoral CP-IL22BP mRNA injection group	Biological: CP-IL22BP mRNA intratumorally injection; CP-IL22BP mRNA will be administered via intratumoral injection. A total of 5 doses will be given, with subsequent doses administered once weekly after the first injection.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants Experiencing Dose-Limiting Toxicities (DLTs)	DLT is defined as any Treatment-Related Adverse Event (TRAE) or clinically significant laboratory abnormality occurring during the DLT observation period. TRAEs include events judged by the investigator to be "definitely," "probably," or "possibly" related to the study treatment. Severity will be graded according to NCI-CTCAE v5.0.	From the first dose to the end of treatment at 9 weeks
Number of participants with treatment discontinuation due to treatment-related adverse events during the first treatment cycle	Treatment-related adverse events (TRAEs) will be assessed and graded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Treatment discontinuation is defined as permanent cessation of CP-IL22BP mRNA administration due to investigator-determined TRAEs occurring during the first treatment cycle.	From the first dose to the end of treatment at 9 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Progression-Free Survival	Progression-Free Survival (PFS) is defined as the time interval from the start of treatment until the first documented occurrence of disease progression (PD) according to RECIST v1.1, or death due to any cause, whichever occurs first.	Up to 6 months from the date of the first dose.
Overall Survival	OS is defined as the time interval from the start of treatment to death due to any cause. For participants who are still alive at the end of the study, data will be censored at the last known date of follow-up.	Up to 12 months from the date of the first dose.
Objective Response	Number of participants achieving a Best Overall Response (BOR) of either Complete Response (CR) or Partial Response (PR) according to RECIST v1.1. Due to the small sample size, data will be reported as absolute counts.	Time Frame: Up to 6 months from the date of the first dose.

Collaborators and Investigators

• Sponsor: West China Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): May 30, 2026
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): December 30, 2027

Study Registration Dates

• First Submitted: April 30, 2026
• First Submitted That Met QC Criteria: May 6, 2026
• First Posted (Actual): May 13, 2026

Study Record Updates

• Last Update Posted (Actual): May 13, 2026
• Last Update Submitted That Met QC Criteria: May 6, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Solid tumor; mRNA vaccine; IL-22; Cancer metastasis
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Neoplastic Processes; Pathological Conditions, Signs and Symptoms; Neoplasm Metastasis
• Other Study ID Numbers: 2026-520

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No