Study to Identify Biomarkers of Oral Cavity Cancer Response to Neoadjuvant Immunotherapy Prior to Definitive Surgery

NCT07343596 | Recruiting DMC FDA Device

• 5 other identifiers: 2025-1536A533300SMPH/HUMAN ONCOLOGY/HUMAN ONCOProtocol Version 12/30/25UW25009
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical trial is to study whether researchers can create a patient-specific tumor system, called a culture vessel, in a timely manner and determine if it can predict how someone will respond to a specific therapy. Participants will:

• Undergo a research biopsy
• Take pembrolizumab per standard of care prior to surgery

Conditions

Oral Cavity Cancer

Outcome Measures

Primary Outcomes (3)

• Changes in Gene Expression

  Identification of pathways that are activated in samples showing substantial response versus those that do not. Gene expression changes will be evaluated through mRNA sequencing and bioinformatic analysis.

  up to 2 months

• Phenotypic Changes in the Immune Microenvironment

  Pathways critical for immune response will be assessed using microscopy and a custom analysis pipeline to assess changes in immune cell localization, staining and morphology that will be compared to a control condition and reported as increased or decreased compared to control. Secreted factors will be assessed in culture media using multiplex bead-based ELISA and quantification of secreted factors will be reported as a concentration (pg/ml or ng/ml) and compared to determine increase or decrease compared to control condition.

  up to 2 months

• Genotypic Changes in the Immune Microenvironment

  Genotypic changes will be assessed using bulk or single cell sequencing approaches. Differential gene expression between control and treatment conditions will be reported and compared between conditions. Gene enrichment pathway analysis will also be completed and compared between control and treatment conditions. For single cell sequencing analysis identified cell clusters will be compared between treatment conditions.

  up to 2 months

Secondary Outcomes (2)

• Tumor response

  8 weeks

• Feasibility of implementation of a patient-specific culture vessel

  8 weeks

Study Arms (1)

Standard of Care: Pembrolizumab

EXPERIMENTAL

Participants receive pembrolizumab by a needle (IV) in the arm once per standard of care (SOC)

Biological: Pembrolizumab; Device: Patient-Specific Culture Vessel

Interventions

Pembrolizumab BIOLOGICAL

IV injection of pembrolizumab

Standard of Care: Pembrolizumab

Patient-Specific Culture Vessel DEVICE

LumeNEXT platform, which employs a 3D matrix and allows molding of blood and lymphatic vessels

Standard of Care: Pembrolizumab

Eligibility Criteria

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

You may qualify if:

• Written informed consent and HIPAA authorization for release of personal health information. NOTE: HIPAA authorization may be included in the informed consent or obtained separately
• Age 18 years and older at the time of registration consent
• Eastern Cooperative Oncology Group (ECOG) Performance Status of 0-2 within 30 days prior to enrollment
• Suspected clinical American Joint Committee on Cancer (AJCC) 8th edition stage II-IVB (T2-T4b N0-N3) oral cavity cancer (oral tongue, floor of mouth, buccal, gingival, retromolar trigone, lip, hard palate) amenable to surgical resection. Patients will be consented prior to research biopsy.
• Primary tumor of at least 2 cm which is amenable to a 250 mm3 (e.g.10 mm x 5 mm x 5 mm) research biopsy, equivalent to one-two forceps biopsies.
• Patients may not have received prior chemotherapy or immunotherapy for the oral cavity malignancy. Patients must have completed other cancer therapies unrelated to their oral cavity cancer greater than 30 days prior to enrollment.
• Participants of childbearing potential must agree to contraception during and for 100 days after study therapy.
• Females of childbearing potential must have a negative serum pregnancy test within 7 days prior to treatment. NOTE: Females are considered of childbearing potential unless they are surgically sterile (have undergone a hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or they are naturally postmenopausal for \> 12 consecutive months.
• Demonstrate adequate organ function

You may not qualify if:

• Subjects with a diagnosed auto-immune disease (exceptions: subjects with controlled diabetes mellitus type I, thyroid disease, rheumatoid arthritis, vitiligo and alopecia areata not requiring treatment with immunosuppressants are eligible)
• Pregnant or breastfeeding
• Known additional invasive malignancy that is active and/or progressive requiring treatment; exceptions include basal cell or squamous cell skin cancer, in situ cervical or bladder cancer, or other cancer for which the subject has been disease free for at least three years prior to enrollment. This excludes the index oral cavity cancer.
• Has received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent or with an agent directed to another co-inhibitory T-cell receptor.
• Has received prior radiotherapy treatment or systemic anti-cancer therapy including investigational agents for the HNC under study prior to randomization/allocation.
• Has received a live vaccine within 30 days prior to the first dose of study drug. Examples of live vaccines include, but are not limited to, the following: measles, mumps, rubella, varicella/zoster (chicken pox), yellow fever, rabies, Bacillus Calmette-Guérin, and typhoid vaccine. Seasonal influenza vaccines for injection are generally killed virus vaccines and are allowed; however, intranasal influenza vaccines (eg, FluMist®) are live attenuated vaccines and are not allowed.
• Has a history of (non-infectious) pneumonitis that required steroids or has current pneumonitis.
• Has had previous allogeneic tissue/solid organ transplant.
• Subjects who require systemic treatment doses of corticosteroids (prednisone equivalent greater than or equal to 10 mg daily), or other immunosuppressive drugs
• Has radiographically detectable (even if asymptomatic and/or previously treated) central nervous system metastases and/or carcinomatous meningitis as assessed by local site investigator and radiology review
• Has Grade 3-4 bleeding due to the underlying malignancy
• Subjects with known human immunodeficiency virus (HIV) infection, active or chronic hepatitis B or hepatitis C infection based on medical history. No testing for HIV, Hepatitis B, or Hepatitis C is required for enrollment. Subjects cannot be positive for HBV DNA, HCV RNA, hepatitis B surface antigen, or anti-hepatitis B core antibody. Patients with Hepatitis B surface antigen negative and anti-hepatis B core antibody positive with undetectable HBV DNA by polymerase chain reaction may enroll.
• Has known psychiatric or substance abuse disorders that would interfere with cooperating with the requirements of the study.
• Subjects who cannot provide independent, legal, informed consent

Sponsors & Collaborators

• University of Wisconsin, Madison: lead

Study Sites (1)

University of Wisconsin - Madison

Madison, Wisconsin, 53705, United States

RECRUITING

MeSH Terms

Conditions

Mouth Neoplasms

Interventions

pembrolizumab

Condition Hierarchy (Ancestors)

Head and Neck Neoplasms; Neoplasms by Site; Neoplasms; Mouth Diseases; Stomatognathic Diseases

Study Officials

• Adam Burr, MD, PhD

  University of Wisconsin, Madison

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Adam Burr, MD, PhD

CONTACT

608-915-0100; aburrr@humonc.wisc.edu

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 6, 2026
• First Posted: January 15, 2026
• Study Start: April 15, 2026
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: April 22, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share

Locations

US (1)