Oral Cavity CancerNCT07343596
Study to Identify Biomarkers of Oral Cavity Cancer Response to Neoadjuvant Immunotherapy Prior to Definitive Surgery
A study testing Pembrolizumab and Patient-Specific Culture Vessel in people with Oral Cavity Cancer, run by University of Wisconsin, Madison at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Oral Cavity Cancer
- What is being tested?
- Pembrolizumab and Patient-Specific Culture Vessel
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Apr 2026to Dec 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 must be considered appropriate candidates for standard-of-care neoadjuvant pembrolizumab by the treating physician.
Who cannot join
- 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. Pregnancy testing is not a research-required procedure. Any pregnancy testing will be performed as part of standard clinical care, as determined by the treating team.
- 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.
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Wisconsin - Madison
Madison, Wisconsin, 53705
Adam Burr, MD, PhD, 608-915-0100, aburr@humonc.wisc.edu
About this study
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
Read the detailed description
Immunotherapy has improved survival for selected patients with recurrent/metastatic head and neck cancer. The Keynote-689 study results published in the New Eng J Med demonstrating improved event free survival for head and neck cancer patients receiving pembrolizumab prior to surgery prompted the June 12, 2025 FDA approval of pembrolizumab as a standard of care option. However, selecting those patients most likely to benefit from this approach is challenging due to a lack of predictive biomarkers.
In this trial, the team will investigate biomarkers using single cell RNA sequencing and a patient-specific culture vessel. These models will not be used to make clinical decisions in this study. Rather, to explore whether a patient-specific culture vessel readout may be feasible to incorporate within a clinical trial workflow. If this proves feasible within an 8-week timeframe, and promising biomarkers of response are identified, this culture vessel may be worthy to study further in subsequent clinical trials.
The investigators chose 2 months as a time point because it is quicker than physicians can currently determine patient overall response (typically 4-6 months) to immunotherapy and still long enough to allow the model to be developed and tested appropriately.
Primary objective: To identify biomarkers predicting treatment response to neoadjuvant immunotherapy in oral cavity cancer
Secondary objectives:
- To correlate tumor response to immunotherapy in the patient-specific culture vessel to clinical tumor response to immunotherapy in vivo (the patient) after treatment with pembrolizumab immunotherapy.
- To determine the feasibility of utilizing the patient-specific culture vessel within a clinical trial workflow.
- Conditions
- Oral Cavity Cancer
- Being tested
- Pembrolizumab, Patient-Specific Culture Vessel
- Medical subject headings
- Mouth Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PembrolizumabBiological
IV injection of pembrolizumab
Patient-Specific Culture VesselDevice
LumeNEXT platform, which employs a 3D matrix and allows molding of blood and lymphatic vessels
| Study group | Role | What they receive |
|---|---|---|
| Standard of Care: Pembrolizumab | Experimental | Participants receive pembrolizumab by a needle (IV) in the arm once per standard of care (SOC) Receives: Biological: Pembrolizumab; Device: Patient-Specific Culture Vessel |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: up to 2 months
Secondary outcomes (2)
Tumor response
Tumor response in the pathologic specimen is assessed by the area of treatment effect. This is defined as the area of tumor necrosis with associated keratinaceous debris. It will be reported as the ratio of the area of tumor effect divided by the total area of tumor.
Measured: 8 weeks
Feasibility of implementation of a patient-specific culture vessel
Feasibility is defined as availability of patient-specific culture vessel data.
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2026
- Study start15 Apr 2026
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Oral Cavity CancerStudies of the same condition, open and under way.
- More trials run by University of Wisconsin, MadisonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Wisconsin, Madison
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Adam Burr, MD, PhD, Principal investigator, University of Wisconsin, Madison
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07343596
- Sponsor's protocol ID
- 2025-1536
- Other IDs
- A533300, SMPH/HUMAN ONCOLOGY/HUMAN ONCO, Protocol Version 7/28/26, UW25009
- Status last verified
- Sep 2026
- First submitted
- 6 Jan 2026
- Last updated
- 16 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study to Identify Biomarkers of Oral Cavity Cancer Response to Neoadjuvant Immunotherapy Prior to Definitive Surgery
- ClinicalTrials.gov ID
- NCT07343596
- Acronym / sponsor ID
- 2025-1536
- Conditions
- Oral Cavity Cancer
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- University of Wisconsin, MadisonUniversity, hospital or other
- Study start
- 15 Apr 2026 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 15 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
Immunotherapy has improved survival for selected patients with recurrent/metastatic head and neck cancer. The Keynote-689 study results published in the New Eng J Med demonstrating improved event free survival for head and neck cancer patients receiving pembrolizumab prior to surgery prompted the June 12, 2025 FDA approval of pembrolizumab as a standard of care option. However, selecting those patients most likely to benefit from this approach is challenging due to a lack of predictive biomarkers.
In this trial, the team will investigate biomarkers using single cell RNA sequencing and a patient-specific culture vessel. These models will not be used to make clinical decisions in this study. Rather, to explore whether a patient-specific culture vessel readout may be feasible to incorporate within a clinical trial workflow. If this proves feasible within an 8-week timeframe, and promising biomarkers of response are identified, this culture vessel may be worthy to study further in subsequent clinical trials.
The investigators chose 2 months as a time point because it is quicker than physicians can currently determine patient overall response (typically 4-6 months) to immunotherapy and still long enough to allow the model to be developed and tested appropriately.
Primary objective: To identify biomarkers predicting treatment response to neoadjuvant immunotherapy in oral cavity cancer
Secondary objectives:
- To correlate tumor response to immunotherapy in the patient-specific culture vessel to clinical tumor response to immunotherapy in vivo (the patient) after treatment with pembrolizumab immunotherapy.
- To determine the feasibility of utilizing the patient-specific culture vessel within a clinical trial workflow.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Standard of Care: Pembrolizumab | Experimental | Biological: Pembrolizumab, Device: Patient-Specific Culture VesselParticipants receive pembrolizumab by a needle (IV) in the arm once per standard of care (SOC) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (14 listed)
- 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. Pregnancy testing is not a research-required procedure. Any pregnancy testing will be performed as part of standard clinical care, as determined by the treating team.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PembrolizumabIV injection of pembrolizumab | Biological | Standard of Care: Pembrolizumab |
| Patient-Specific Culture VesselLumeNEXT platform, which employs a 3D matrix and allows molding of blood and lymphatic vessels | Device | Standard of Care: Pembrolizumab |
Primary outcome
Changes in Gene Expression
Time frame: up to 2 months
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.
Phenotypic Changes in the Immune Microenvironment
Time frame: up to 2 months
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.
Genotypic Changes in the Immune Microenvironment
Time frame: up to 2 months
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.
2 secondary outcomes
Tumor response
Time frame: 8 weeks
Feasibility of implementation of a patient-specific culture vessel
Time frame: 8 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2026.
- Individual participant dataSharing planned: Yes
Related trials for Oral Cavity Cancer
RecruitingPhase 1/2
Study of INBRX-106 and INBRX-106 in Combination With Pembrolizumab (Keytruda®) in Subjects With Locally Advanced or Metastatic Solid Tumors (Hexavalent OX40 Agonist)
Solid Tumor, Non-Small Cell Lung Cancer
RecruitingPhase 2
Proton Spatially Fractionated Radiotherapy or Photon Stereotactic Body Radiotherapy in Combination With Pembrolizumab Before Surgery for the Treatment of Locally Advanced and Radiation-Naive Locoregionally Recurrent Oral Cavity Cancer, OSPRI Trial
Locally Advanced Oral Cavity Squamous Cell Carcinoma, Locally Recurrent Oral Cavity Squamous Cell Carcinoma
Active, not recruitingPhase 1/2
Study of Oral MRT-2359 in Selected Cancer Patients
NSCLC, SCLC
RecruitingPhase 1/2
Docetaxel and SX-682 in Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma, Salivary Gland Carcinoma, and Advanced Prostate Cancer
Head and Neck Cancer, Head and Neck Squamous Cell Carcinoma
RecruitingPhase 2
A Phase 2 Study of VS-7375 in Patients With KRAS G12D-Mutated Non-Small Cell Lung Cancer
Non-Small Cell Lung Cancer
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Adam Burr, MD, PhD principal investigator
University of Wisconsin, Madison
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Pembrolizumab and Patient-Specific Culture Vessel in people with Oral Cavity Cancer, run by University of Wisconsin, Madison at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Adam Burr, MD, PhD (608-915-0100, aburrr@humonc.wisc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes in Gene Expression (time frame: up to 2 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2026.