NCT06927297

Phase I Study of the Safety, Tolerability, and Efficacy of an Engineered Mitochondrial Vaccine in Advanced Solid Tumors

A Clinical Trial to Evaluate the Safety, Tolerability, and Preliminary Antitumor Activity of an Engineered Mitochondrial Vaccine in Treatment of Advanced Solid Tumors

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: West China Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This study is a dose-escalation, prospective clinical trial to assess the safety, tolerability, and preliminary therapeutic efficacy of engineered mitochondria expressing specific tumor antigen in patients with advanced solid tumors.

Detailed description

Advanced malignant solid tumors have limited treatment options and poor prognosis, which is a major health challenge worldwide. As a promising immunotherapy strategy, cancer vaccines use tumor-specific antigens or tumor-associated antigens to activate body-specific immune responses and effectively inhibit tumor growth and metastasis. Current research suggests that mitochondria are a form of archaeozoic proteus that is integrated into modern eukaryotes by symbiosis with anaerobic pre-eukaryotic (host) cells. Mitochondria are key mediators of innate and adaptive immune processes, and many mitochondrial components and metabolites (e.g., mitochondrial DNA, proteins, etc.) are released in response to cellular injury or stress as damage-associated molecular patterns (DAMPs) to promote inflammatory responses. These DAMPs elicit an innate immune response by activating pattern recognition receptors (PRRs) such as TLRs and NOD-like receptors (NLRs). A mitochondrial engineering platform for the generation of antigen-rich mitochondria as a cancer vaccine has been successfully established. This study includes four dose escalation cohorts and aims to evaluate the safety, tolerability, and preliminary anti-tumor activity of the engineered mitochondrial vaccine in patients with advanced solid tumors.

Conditions

• Advanced Solid Tumor Malignancies
• Advanced Solid Malignancies

Interventions

Type	Name	Description
BIOLOGICAL	Engineered mitochondrial vaccine	Participants were assigned to one of four dose-escalation groups (25 μg, 50 μg, 100 μg, or 200 μg per dose) and received subcutaneous injections of the engineered mitochondrial vaccine in a prime-boost schedule. The regimen consisted of 3 doses of primary immunization, 1 dose of boost immunization and subsequent personalized treatment.

Timeline

• Start date: 2025-05-09
• Primary completion: 2026-12-31
• Completion: 2028-04-30
• First posted: 2025-04-15
• Last updated: 2026-03-17

Locations

1 site across 1 country: China