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

March 15, 2026 updated by: Xingchen Peng, West China Hospital

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

12

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Xingchen Peng, Ph.D
  • Phone Number: +8618980606753
  • Email: pxx2014@163.com

Study Locations

  • China
    • Sichuan
      • Chengdu, Sichuan, China, 610041
        • Recruiting
        • West China Hospital, Sichuan University
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Patients aged 18 to 75 years at the time of acquisition informed consent form.
  2. Patients with advanced malignant solid tumors such as non-small cell lung cancer or colorectal cancer, confirmed by histology or cytology, who are unable to receive or tolerate standard treatments, or who refuse standard treatments, exhibit positive expression of target antigens in the tumor tissue.
  3. The presence of at least one measurable or evaluable lesion according to RECIST v1.1 criteria.
  4. Eastern Cooperative Oncology Group (ECOG) performance status score: 0-2.
  5. Predicted survival time ≥3 months.

  6. The main organs are functioning well and the following requirements are met within 7 days before receiving treatment:

    ① Hemoglobin (HGB) ≥80 g/L (no blood transfusion within 14 days); Absolute neutrophil count (ANC) >1.5×109/L; White blood cell count ≥3.0×109/L; Platelet count (PLT) ≥80×109/L;

    ② Total bilirubin ≤1.5× upper limit of normal value (ULN); Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≤2.5×ULN; If there was liver metastasis, ALT or AST≤5×ULN;

    ③ Creatinine (SCr) ≤1.5×ULN or creatinine clearance (CRCI) estimated by Cockcroft-Gault formula ≥60 mL/min;

    ④ Prothrombin time (PT), international normalized ratio (INR) ≤1.5×ULN (unless anticoagulation with warfarin);

    ⑤ Cardiac function: left ventricular ejection fraction ≥50%. QTcF interval ≤450 ms.

  7. Men of childbearing potential and women of childbearing age voluntarily use effective contraceptive methods (e.g., condoms, intrauterine devices, spermicides) from the time of signing the informed form until 6 months after the completion of vaccination, and contraceptive use is not allowed. Female cancer patients who have a negative pregnancy test and agree not to breastfeed during the study and for at least 18 months after receipt of the trial vaccine;
  8. The washout period of previous anti-tumor therapy should be at least 4 weeks, and the washout period of molecular targeted drugs should be at least 5 half-lives. Palliative radiotherapy needs to have been completed for at least 2 weeks; Chest radiation therapy needed to have been completed for at least 3 months, and major surgery needed to have been completed with at least 4 weeks of recovery.

Exclusion Criteria:

  1. The patient has a history of other tumors in the past, except for the history of malignant tumors that have been cured and have not recurred within 5 years before screening, such as basal cell carcinoma of the skin, squamous cell carcinoma of the skin, superficial bladder cancer, cervical cancer in situ, and intramucosal cancer of the gastrointestinal tract, which the investigator considers to be eligible for enrollment.
  2. Have any uncontrolled clinical diseases (e.g., diseases of the respiratory system, circulatory system, digestive system, nervous system, hematologic system, urogenital system, endocrine system) or psychiatric or other major medical condition that the investigator considers to interfere with the provision of informed consent, to interfere with the interpretation of the trial results, to pose a risk to the study participants, or to otherwise interfere with the achievement of the study objectives.
  3. Have any active autoimmune disease or a history of autoimmune disease. Participants with asthma for which medical intervention with bronchodilators was required could not be included.
  4. Allergy to the trial drug (including any excipients). Previous history of severe allergy to any drug, food or vaccination, such as anaphylactic shock, allergic laryngeal edema, allergic dyspnea, allergic purpura, thrombocytopenic purpura, local allergic necrosis reaction (Arthus reaction), etc.
  5. There are contraindications to subcutaneous injection.
  6. Received prior antitumor therapeutic vaccine or cellular immunotherapy.
  7. Participated in other drug or device clinical trials 4 weeks before screening.
  8. Study participants on systemic therapy with corticosteroids (>10 mg/ day of prednisone or equivalent doses of other glucocorticoids) or other immunosuppressive agents within 14 days before the first dose of vaccine. Inhaled or topical steroids and adrenal hormone replacement at a therapeutic dose of prednisone of 10 mg or less per day were allowed in the absence of active autoimmune disease.
  9. Before the first dose of the study drug. Any toxic effects from previous antineoplastic therapy have not recovered to NCI CTCAE grade 5.0 ≤1 (any degree of alopecia, other than grade 2 previous platinum-based treatment-related neuropathy).
  10. Has active infection including tuberculosis, hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection.
  11. Have a history of substance abuse or known medical, psychological, or social conditions such as alcohol or drug abuse.
  12. Have received any vaccine within 30 days before receiving the study vaccine or plan to receive any vaccine other than the study vaccine during the study.
  13. The presence of any other factor that was deemed by the investigator to preclude study participant entry into the trial or that study participant had any medical condition that could interfere with the assessment of the safety or efficacy of the study treatment.
  14. Study participants who were unwilling or unable to comply with study requirements.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort group of engineered mitochondria vaccine for dose-escalation
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 using a prime-boost regimen.
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Adverse events
Time Frame: 1 year
1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Objective Response Rate (ORR)
Time Frame: 6 months
The ratio of participants assessed with complete response (CR) or partial response (PR) as a best overall response.
6 months
Disease Control Rate (DCR)
Time Frame: 6 months
The ratio of subjects assessed with CR or PR or stable disease (SD) as a best overall response.
6 months
PFS
Time Frame: 6 months
PFS is defined as the duration until disease progression or death in participants from the first dose of immunization.
6 months
OS
Time Frame: 1 year
OS is defined as the duration until death in participants from the first dose of immunization.
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

West China Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 9, 2025

Primary Completion (Estimated)

December 31, 2026

Study Completion (Estimated)

April 30, 2028

Study Registration Dates

First Submitted

April 4, 2025

First Submitted That Met QC Criteria

April 13, 2025

First Posted (Actual)

April 15, 2025

Study Record Updates

Last Update Posted (Actual)

March 17, 2026

Last Update Submitted That Met QC Criteria

March 15, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2025-41

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

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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