- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg NCT07572864
Neoadjuvant ICI and Mitochondrial Vaccine for Resectable HNSCC
6. maj 2026 opdateret af: Xingchen Peng, West China Hospital
Efficacy and Safety of Immune Checkpoint Inhibitors in Combination With Engineered Mitochondrial Vaccine as Neoadjuvant and Adjuvant Therapy for Resectable Head and Neck Squamous Cell Carcinoma: A Single-Arm, Single-Center Clinical Study.
Head and neck squamous cell carcinoma (HNSCC) presents a significant clinical challenge, as over 60% of patients are diagnosed at a locally advanced stage with a high risk of recurrence.
Although the landmark KEYNOTE-689 trial established neoadjuvant immune checkpoint inhibitor (ICI) therapy as a new standard of care, the pathological complete response (pCR) rate remains unsatisfactory at only 3.0%, highlighting an urgent need for optimized combination strategies.
This prospective, single-arm, single-center clinical study aims to evaluate the safety, tolerability, and preliminary efficacy of a novel neoadjuvant and adjuvant regimen combining an engineered mitochondrial vaccine (IMP3-Mito) with ICIs for patients with resectable, IMP3-positive locally advanced HNSCC.
The rationale is based on a "Prime-and-Release" synergistic mechanism: the engineered mitochondrial vaccine serves as a potent "natural adjuvant" to activate dendritic cells and prime tumor-specific T-cell responses against the IMP3 antigen, while the ICI subsequently releases the immune brakes within the tumor microenvironment.
By integrating these two modalities, the study seeks to achieve deeper pathological responses and improve long-term survival, while simultaneously providing clinical evidence for the transformative potential of the mitochondrial engineering platform in overcoming the limitations of conventional tumor vaccines.
Studieoversigt
Status
Rekruttering
Betingelser
Intervention / Behandling
Undersøgelsestype
Interventionel
Tilmelding (Anslået)
9
Fase
- Fase 2
Kontakter og lokationer
Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.
Studiekontakt
- Navn: Xingchen Peng, Professor
- Telefonnummer: +8618980606753
- E-mail: pxx2014@163.com
Undersøgelse Kontakt Backup
- Navn: Yiyan Pei, Doctor
- E-mail: dr.peiyy@gmail.com
Studiesteder
-
-
Sichuan
-
Chengdu, Sichuan, Kina, 610041
- Rekruttering
- West China Hospital, Sichuan University
-
Kontakt:
- Xingchen Peng, Professor
- Telefonnummer: 18980606753
- E-mail: pxx2014@163.com
-
Chengdu, Sichuan, Kina, 610041
- Ikke rekrutterer endnu
- West China Hospital, Sichuan University
-
Kontakt:
- Xingchen Peng
- Telefonnummer: 18980606753
- E-mail: pxx2014@163.com
-
-
Deltagelseskriterier
Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.
Berettigelseskriterier
Aldre berettiget til at studere
- Voksen
- Ældre voksen
Tager imod sunde frivillige
Ingen
Beskrivelse
Inclusion Criteria:
- Aged ≥ 18 years, both genders eligible.
Pathologically confirmed head and neck squamous cell carcinoma (HNSCC) meeting the following criteria:
- Clinical stage II-IVB according to the AJCC 8th Edition (nasopharyngeal carcinoma is excluded);
- Positive expression of IMP3 protein;
- Clinically resectable as determined by a Multidisciplinary Team (MDT);
- Subjects must be willing and able to undergo radical surgery.
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 1.
Adequate organ and bone marrow function, defined as:
- Hematology: Absolute neutrophil count (ANC) ≥ 1.5 × 10^9/L; Platelet count (PLT) ≥ 80 × 10^9/L; Hemoglobin (HGB) ≥ 8 g/dL;
- Liver Function: Aspartate aminotransferase (AST), Alanine aminotransferase (ALT), and Alkaline phosphatase (ALP) ≤ 2.5 × Upper Limit of Normal (ULN); Total bilirubin (TBIL) ≤ 1.5 × ULN;
- Albumin (ALB) ≥ 2.8 g/dL;
- Renal Function: Serum creatinine (Cr) ≤ 1.5 × ULN or Creatinine Clearance (CCR) > 60 mL/min;
- Coagulation: International Normalized Ratio (INR) ≤ 1.5; Activated Partial Thromboplastin Time (APTT) ≤ 1.5 × ULN.
- Subjects must voluntarily participate in the study, sign the Informed Consent Form (ICF), and be able to comply with the scheduled visits and protocol procedures.
Exclusion Criteria:
- History of other malignant tumors within the past 5 years, except for cured basal cell carcinoma of the skin, squamous cell carcinoma of the skin, superficial bladder cancer, cervical cancer in situ, gastrointestinal intramucosal carcinoma, or other malignancies that the investigator deems eligible.
- Any active autoimmune disease or history of autoimmune disease, including but not limited to immune-related neurological diseases, multiple sclerosis, autoimmune (demyelinating) neuropathy, Guillain-Barre syndrome, myasthenia gravis, systemic lupus erythematosus (SLE), connective tissue disease, scleroderma, inflammatory bowel disease (Crohn's disease and ulcerative colitis), autoimmune hepatitis, toxic epidermal necrolysis (TEN), or Stevens-Johnson syndrome (except for Type I diabetes mellitus on a stable dose of insulin).
Contraindications related to subcutaneous injection (specific to vaccination):
- Active inflammation, trauma, or skin breakdown at the intended injection site;
- Severe bleeding or coagulation tendency, or significantly reduced platelets/clotting factors assessed by the investigator as high risk for bleeding;
- Any abnormal or permanent body art (e.g., tattoos) at the intended injection site that, in the investigator's opinion, would interfere with the observation of local skin reactions.
- Known allergy to the study drugs or any excipients. History of severe allergies to any drugs, food, or vaccines (e.g., anaphylactic shock, laryngeal edema, dyspnea, Henoch-Schonlein purpura, thrombocytopenic purpura, or Arthus reaction).
Prior receipt of any of the following treatments:
- Prior treatment with PD-1, PD-L1, PD-L2, CTLA-4, EGFR antibodies, or EGFR-TKIs;
- Prior vaccination with any anti-tumor vaccines;
- Receipt of any live/active vaccines against infectious diseases (e.g., influenza, varicella) within 4 weeks prior to the first dose or planned during the study period.
- Requirement for systemic steroid therapy (prednisone equivalent dose > 10 mg/day) within 14 days prior to enrollment.
- Major surgery or severe trauma within 4 weeks prior to the first dose.
- Toxicity from prior anti-tumor therapy not recovered to ≤ CTCAE (Version 5.0) Grade 1 (except for alopecia, or sequelae of neurotoxicity related to prior platinum therapy) or levels specified in the inclusion/exclusion criteria.
- Severe medical conditions, including: Grade II or higher cardiac dysfunction (NYHA criteria); ischemic heart disease (e.g., myocardial infarction or angina); clinically significant supraventricular or ventricular arrhythmias; poorly controlled diabetes (fasting blood glucose ≥ 10 mmol/L); poorly controlled hypertension (SBP > 150 mmHg and/or DBP > 100 mmHg); LVEF < 50% on echocardiography; QTc interval > 450 msec (males) or > 470 msec (females); or other ECG abnormalities deemed by the investigator to pose extra risk.
- History of interstitial lung disease (ILD), non-infectious pneumonitis, or high suspicion of ILD; or any condition that might interfere with the detection or management of drug-related pulmonary toxicity (except for asymptomatic drug-induced or radiation-induced pneumonitis); active tuberculosis (TB) or history of uncontrolled TB.
- Hyperthyroidism or organic thyroid disease. Hypothyroidism on a stable dose of thyroid replacement therapy or hypothyroidism that can be controlled by replacement therapy (as confirmed by the investigator and/or endocrinology) is eligible.
- Active infection, unexplained fever occurring within 48 hours prior to the first dose, or use of systemic antibiotics within 1 week prior to signing the Informed Consent Form (ICF).
- Active Hepatitis B (HBV DNA ≥ 2000 IU/mL or 10^4 copies/mL), Hepatitis C (HCV antibody positive and HCV RNA above the limit of detection), or known history of HIV infection or AIDS.
- History of neurological or psychiatric disorders, such as epilepsy or dementia.
- History of drug abuse or alcohol abuse within the past 3 months.
- Pregnant or lactating women; subjects (or their partners) planning for pregnancy or engaging in unprotected sexual intercourse from screening until 3 months after the end of the study.
- Receipt of any other investigational drug within 4 weeks prior to the first dose, or concurrent enrollment in another clinical study, except for observational (non-interventional) studies or the follow-up phase of an interventional study.
- Other factors judged by the investigator that may interfere with the completion of the study treatment or follow-up.
Studieplan
Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
|
Eksperimentel: IMP3-Mito Vaccine plus ICI Combination Group
Participants with resectable IMP3-positive LA-HNSCC will receive neoadjuvant engineered mitochondrial vaccine (IMP3-Mito) in combination with immune checkpoint inhibitors, followed by surgery and subsequent adjuvant combination therapy.
|
A vaccine targeting the IMP3 antigen, utilizing an engineered mitochondrial platform.
Administered via subcutaneous injection.
PD-1 inhibitor administered via intravenous infusion to synergistic with the mitochondrial vaccine.
|
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Major Pathologic Response
Tidsramme: At the time of surgery (approximately 6 weeks after enrollment).
|
Major Pathologic Response (MPR) was defined as fewer than 10% viable tumor cells.
|
At the time of surgery (approximately 6 weeks after enrollment).
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Number of Participants Who Experienced an Adverse Event (AE)
Tidsramme: From the first dose until the completion of the 12-month post-operative follow-up (approximately 14 months in total).
|
An AE was defined as any unfavorable and unintended change in the structure, function, or chemistry of the body temporally associated with the use of the product, whether or not considered related to the use of the product.
Any worsening (i.e., any clinically significant adverse change in frequency and/or intensity) of a pre-existing condition which was temporally associated with the use of the product was also an AE.
The number of participants who experienced an AE was reported for each arm.
|
From the first dose until the completion of the 12-month post-operative follow-up (approximately 14 months in total).
|
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Pathologic Complete Response
Tidsramme: At the time of surgery (approximately 6 weeks after enrollment).
|
Pathologic Complete Response (pCR) was defined as the absence of viable tumor cells.
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At the time of surgery (approximately 6 weeks after enrollment).
|
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Objective Response Rate
Tidsramme: From the start of neoadjuvant therapy to the end of neoadjuvant therapy, the duration is approximately 2 months
|
Objective Response Rate (ORR) was defined as the percentage of participants with a best overall response of CR or PR using RECIST Criteria.
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From the start of neoadjuvant therapy to the end of neoadjuvant therapy, the duration is approximately 2 months
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Samarbejdspartnere og efterforskere
Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.
Sponsor
Publikationer og nyttige links
Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.
Generelle publikationer
- Luo J, Mo F, Zhang Z, Hong W, Lan T, Cheng Y, Fang C, Bi Z, Qin F, Yang J, Zhang Z, Li X, Que H, Wang J, Chen S, Wu Y, Yang L, Li J, Wang W, Chen C, Wei X. Engineered mitochondria exert potent antitumor immunity as a cancer vaccine platform. Cell Mol Immunol. 2024 Nov;21(11):1251-1265. doi: 10.1038/s41423-024-01203-4. Epub 2024 Aug 20.
- Fang C, Mo F, Liu L, Du J, Luo M, Men K, Na F, Wang W, Yang H, Wei X. Oxidized mitochondrial DNA sensing by STING signaling promotes the antitumor effect of an irradiated immunogenic cancer cell vaccine. Cell Mol Immunol. 2021 Sep;18(9):2211-2223. doi: 10.1038/s41423-020-0456-1. Epub 2020 May 12.
- Tse SW, McKinney K, Walker W, Nguyen M, Iacovelli J, Small C, Hopson K, Zaks T, Huang E. mRNA-encoded, constitutively active STINGV155M is a potent genetic adjuvant of antigen-specific CD8+ T cell response. Mol Ther. 2021 Jul 7;29(7):2227-2238. doi: 10.1016/j.ymthe.2021.03.002. Epub 2021 Mar 5.
- Pierini S, Fang C, Rafail S, Facciponte JG, Huang J, De Sanctis F, Morgan MA, Uribe-Herranz M, Tanyi JL, Facciabene A. A Tumor Mitochondria Vaccine Protects against Experimental Renal Cell Carcinoma. J Immunol. 2015 Oct 15;195(8):4020-7. doi: 10.4049/jimmunol.1500281. Epub 2015 Sep 16.
- Fan L, Wu D, Goremykin V, Xiao J, Xu Y, Garg S, Zhang C, Martin WF, Zhu R. Phylogenetic analyses with systematic taxon sampling show that mitochondria branch within Alphaproteobacteria. Nat Ecol Evol. 2020 Sep;4(9):1213-1219. doi: 10.1038/s41559-020-1239-x. Epub 2020 Jul 13.
- Gulley JL, Borre M, Vogelzang NJ, Ng S, Agarwal N, Parker CC, Pook DW, Rathenborg P, Flaig TW, Carles J, Saad F, Shore ND, Chen L, Heery CR, Gerritsen WR, Priou F, Langkilde NC, Novikov A, Kantoff PW. Phase III Trial of PROSTVAC in Asymptomatic or Minimally Symptomatic Metastatic Castration-Resistant Prostate Cancer. J Clin Oncol. 2019 May 1;37(13):1051-1061. doi: 10.1200/JCO.18.02031. Epub 2019 Feb 28.
Datoer for undersøgelser
Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.
Studer store datoer
Studiestart (Anslået)
28. april 2026
Primær færdiggørelse (Anslået)
1. maj 2027
Studieafslutning (Anslået)
1. maj 2028
Datoer for studieregistrering
Først indsendt
24. april 2026
Først indsendt, der opfyldte QC-kriterier
30. april 2026
Først opslået (Faktiske)
7. maj 2026
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
11. maj 2026
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
6. maj 2026
Sidst verificeret
1. maj 2026
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
- Neoplasmer efter sted
- Neoplasmer
- Neoplasmer efter histologisk type
- Neoplasmer i hoved og hals
- Neoplasmer, kirtel og epitel
- Karcinom
- Karcinom, pladecelle
- Planocellulært karcinom i hoved og hals
- Antineoplastiske midler, immunologiske
- Antineoplastiske midler
- Molekylære mekanismer for farmakologisk virkning
- Farmakologiske handlinger
- Kemiske handlinger og anvendelser
- Terapeutiske anvendelser
- Immune Checkpoint-hæmmere
Andre undersøgelses-id-numre
- 2026(876)
Plan for individuelle deltagerdata (IPD)
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