AI-designed pan-cancer neoantigen mRNA vaccine (PAN-NeoVax) for adults with advanced solid tumors

A Phase I Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of an Artificial Intelligence-Designed Tumor mRNA Vaccine in Patients With Advanced or Metastatic Solid Tumors

This study will test whether an AI-designed pan-cancer neoantigen mRNA vaccine (PAN-NeoVax), injected directly into tumors, is safe and tolerable for adults with advanced solid tumors who have failed second-line therapy.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 9 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: West China Hospital (Academic / other)
• Drugs / interventions: chemotherapy
• Locations: 2 sites (Chengdu, Sichuan and 1 other locations)
• Trial ID: NCT07560943 on ClinicalTrials.gov

What this trial studies

This phase 1, open-label, single-arm, single-center dose-escalation trial gives PAN-NeoVax intratumorally to adults with advanced or metastatic solid tumors after second-line therapy failure. The trial uses a classical 3+3 design with three dose levels (25 µg, 50 µg, 100 µg) and each participant receives five doses (four weekly doses followed by a booster one month after the fourth dose). PAN-NeoVax is an AI-designed neoantigen mRNA formulated in lipid nanoparticles to stimulate tumor-directed immune responses. Primary endpoints focus on safety and tolerability with collection of preliminary anti-tumor activity signals.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18–70 with histologically confirmed advanced or metastatic solid tumors who have failed second-line therapy, have ECOG 0–1, and meet the trial's organ-function and timing requirements.

Not a fit: Patients who have effective standard treatment options remaining, poor performance status, significant organ dysfunction, or life expectancy under three months are unlikely to benefit from this trial.

Why it matters

Potential benefit: If successful, PAN-NeoVax could trigger tumor-specific immune responses and provide a new option for patients who have exhausted standard therapies.

How similar studies have performed: Other neoantigen mRNA vaccines have shown encouraging immune responses and early signals in melanoma, but an AI-designed pan-cancer vaccine delivered intratumorally is a novel approach that is not yet proven.

Eligibility criteria

Inclusion Criteria:

1. Male or female patients aged ≥18 and ≤70 years
2. 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)
3. ECOG Performance Status score: 0-1
4. Estimated life expectancy ≥3 months
5. At least 28 days since prior chemotherapy, radiotherapy, or surgery
6. At least 6 weeks since prior use of nitrosoureas or mitomycin C
7. 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%
8. Signed written informed consent and willingness to comply with scheduled visits, treatment plans, laboratory tests, and other study requirements

Exclusion Criteria:

1. Participation in another clinical drug trial within 4 weeks
2. Tumor located adjacent to major blood vessels or trachea
3. 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
4. Pregnant or breastfeeding women
5. Active pulmonary tuberculosis, bacterial or fungal infection (≥Grade 2 per NCI-CTCAE v5.0); HIV infection, active HBV or HCV infection
6. History of psychotropic substance abuse that cannot be discontinued, or mental disorders
7. 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)
8. Currently receiving immunosuppressive therapy
9. History of drug abuse or known medical, psychological, or social conditions (e.g., alcoholism, drug addiction)
10. 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)
11. Female subjects with pregnancy plans or male subjects whose partners have pregnancy plans from screening through 12 months after the last dose
12. Any serious concomitant disease that, in the investigator's judgment, would jeopardize patient safety or ability to complete the study

Where this trial is running

Chengdu, Sichuan and 1 other locations

• West China Hospital — Chengdu, Sichuan, China (Not_yet_recruiting)
• The West China Hospital — Chengdu, Sichuan, China (Recruiting)

Study contacts

• Study coordinator: Xingchen Peng

Conditions Cancer; mRNA; Biotherapy