Safety and Tolerability of AI-Designed Pan-Cancer Neoantigen mRNA Vaccine (PAN-NeoVax) in Advanced Solid Tumors

NCT07560943 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: 2026(826)
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

This is a phase I, open-label, single-arm, single-center, dose-escalation study to evaluate the safety, tolerability, and preliminary anti-tumor efficacy of PAN-NeoVax, an artificial intelligence (AI)-designed pan-cancer neoantigen mRNA vaccine delivered via lipid nanoparticles (LNP), administered by intratumoral injection in patients with advanced or metastatic solid tumors who have failed second-line therapy. The study employs a classical "3+3" dose-escalation design with three dose levels (25 μg, 50 μg, and 100 μg mRNA). Each subject will receive 5 doses of basic immunization (first 4 doses at weekly intervals, fifth dose at 1 month after the fourth dose).

Conditions

Cancer

Keywords

Cancer; mRNA; Biotherapy

Outcome Measures

Primary Outcomes (1)

• Number of Participants with DLT

  From the first dose to 3 weeks post-dose.

Secondary Outcomes (6)

• Objective Response Rate

  From the time when the patients were enrolled in the study until one month after the last dose was injected.The time window was typically 2 months.

• Disease Control Rate

  From the time when the patients were enrolled in the study until one month after the last dose was injected.The time window was typically 2 months.

• Time to First Response

  From the time when the patients were enrolled in the study until one month after the last dose was injected.The time window was typically 2 months.

• Duration of Response

  From the time when the patients were enrolled in the study until one month after the last dose was injected.The time window was typically 2 months.

• Progression - Free Survival(PFS)

  From the time when the patients were enrolled in the study until three months after the last dose was injected. The time window was typically 6 months.

Study Arms (3)

PAN-NeoVax Dose Level 1 (25 μg)

EXPERIMENTAL

Biological: PAN-NeoVax (25 μg)

PAN-NeoVax Dose Level 2 (50 μg)

EXPERIMENTAL

Biological: PAN-NeoVax (50 μg)

PAN-NeoVax Dose Level 3 (100 μg)

EXPERIMENTAL

Biological: PAN-NeoVax (100 μg)

Interventions

PAN-NeoVax (25 μg) BIOLOGICAL

25 μg PAN-NeoVax administered by intratumoral injection; 5 doses of basic immunization (first 4 doses at weekly intervals, fifth dose at 1 month after the fourth dose).

PAN-NeoVax Dose Level 1 (25 μg)

PAN-NeoVax (50 μg) BIOLOGICAL

50 μg PAN-NeoVax administered by intratumoral injection; 5 doses of basic immunization (first 4 doses at weekly intervals, fifth dose at 1 month after the fourth dose).

PAN-NeoVax Dose Level 2 (50 μg)

PAN-NeoVax (100 μg) BIOLOGICAL

100 μg PAN-NeoVax administered by intratumoral injection; 5 doses of basic immunization (first 4 doses at weekly intervals, fifth dose at 1 month after the fourth dose).

PAN-NeoVax Dose Level 3 (100 μg)

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female patients aged ≥18 and ≤70 years
• Histopathologically confirmed advanced recurrent/metastatic malignant solid tumors that have failed second-line therapy with no standard treatment options available (priority enrollment of advanced head and neck squamous cell carcinoma and malignant melanoma patients)
• ECOG Performance Status score: 0-1
• Estimated life expectancy ≥3 months
• At least 28 days since prior chemotherapy, radiotherapy, or surgery
• At least 6 weeks since prior use of nitrosoureas or mitomycin C
• Adequate organ function 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×ULN
• ALT or AST ≤2.5×ULN (≤5×ULN if liver metastases present)
• Creatinine clearance ≥60 mL/min (Cockcroft-Gault formula)
• Left ventricular ejection fraction (LVEF) ≥50%
• Signed written informed consent and willingness to comply with scheduled visits, treatment plans, laboratory tests, and other study requirements

You may not qualify if:

• Participation in another clinical drug trial within 4 weeks
• Tumor located adjacent to major blood vessels or trachea
• Poorly controlled cardiac conditions: NYHA class \>2 heart failure, unstable angina, myocardial infarction within 1 year, or clinically significant supraventricular or ventricular arrhythmias requiring treatment or intervention
• Pregnant or breastfeeding women
• Active pulmonary tuberculosis, bacterial or fungal infection (≥Grade 2 per NCI-CTCAE v5.0); HIV infection, active HBV or HCV infection
• History of psychotropic substance abuse that cannot be discontinued, or mental disorders
• Active autoimmune disease or history of autoimmune disease (exceptions: vitiligo; childhood asthma in complete remission requiring no adult intervention; asthma requiring bronchodilator intervention is excluded)
• Currently receiving immunosuppressive therapy
• History of drug abuse or known medical, psychological, or social conditions (e.g., alcoholism, drug addiction)
• Known allergy, hypersensitivity, or intolerance to PAN-NeoVax or any excipient; history of severe allergic reactions to any drug, food, or vaccine (including anaphylactic shock, allergic laryngeal edema, allergic dyspnea, allergic purpura, thrombocytopenic purpura, Arthus reaction)
• Female subjects with pregnancy plans or male subjects whose partners have pregnancy plans from screening through 12 months after the last dose
• Any serious concomitant disease that, in the investigator's judgment, would jeopardize patient safety or ability to complete the study

Sponsors & Collaborators

• West China Hospital: lead

Study Sites (1)

West China Hospital

Chengdu, Sichuan, 610000, China

Location

MeSH Terms

Conditions

Neoplasms

Central Study Contacts

Xingchen Peng

CONTACT

18980606753; pxx2014@163.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: April 24, 2026
• First Posted: May 1, 2026
• Study Start: April 28, 2026
• Primary Completion (Estimated): April 28, 2027
• Study Completion (Estimated): December 28, 2027
• Last Updated: May 1, 2026

Record last verified: 2026-04

Locations

CN (1)