Clinical trial NCT04883645: Neoadjuvant Imiquimod Immunotherapy for Oral Cancer
Official title: Pilot Clinical Trial of Neoadjuvant Toll-like Receptor 7 Agonist (Imiquimod) Immunotherapy in Early-Stage Oral Squamous Cell Carcinoma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: EARLY_PHASE1
Conditions: Oral Squamous Cell Carcinoma
Lead sponsor: Medical University of South Carolina
Sponsor class: OTHER
Collaborators: name: National Cancer Institute (NCI)
First posted: 2021-05-12
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this research study is to find out what effects, good and/or bad, topical application of the drug Aldara will have on patients and on their oral cancer. Aldara is a drug that activates toll-like receptor (TLR) in oral cancer cells causing self-destruction of tumor cells. It also activates immune cells to attack and eliminate cancer cells. Aldara is currently approved by the Food and Drug Administration (FDA) for treatment of skin cancer and melanoma. Its use in this study is 'off-label' (use of a drug approved by FDA for skin cancer to treat oral cancer in this study).

The preliminary efficacy of topical imiquimod in a neoadjuvant setting in patients with early-stage oral squamous cell carcinoma will be determined by a reduction in tumor cellularity in post-treatment tissue compared to pre-treatment tissue. Safety and tolerability will be evaluated by CTCAE v5 criteria. The effect of imiquimod on the tumor immune microenvironment will be assessed by performing quantitative multiplex immunofluorescence.

Detailed description: The researchers propose an exploratory clinical trial to evaluate the efficacy of topical imiquimod, a TLR-7 agonist, in patients with early-stage oral squamous cell carcinoma. The analysis of pre- and post-treatment tumor specimen collected from patients treated on this study will be used for quantitative immunoflourescence analysis to assess the immunomodulatory activity of imiquimod in human tumor samples. The researchers hypothesize that TLR-7 stimulation will reduce the size of the tumor in patients with early-stage oral squamous cell carcinoma. The researchers anticipate that activation of immune cells will correlate with response to therapy.

Eligibility criteria: Inclusion Criteria:

* Previously untreated and biopsy confirmed oral squamous cell carcinoma (OSCC)
* Clinical (TNM) stage I or II
* Age \>= 18 years
* Eastern Cooperative Oncology Group (ECOG) =\< 2

Exclusion Criteria:

* Patients associated with prior therapy requiring treatment with systemic immunosuppressive treatments with the exception of vitiligo, childhood asthma that has resolved, residual endocrinopathies requiring replacement therapy, or psoriasis that does not require systemic treatment
* Treatment with any other investigational agents
* Requirement for immunosuppressive intraoral topical or systemic corticosteroids prior to the study
* Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
* HIV positive patients on combination antiretroviral therapy
* Have evidence of any other significant oral mucosal condition, clinical disorder, physical examination finding, or laboratory finding that, as judged by the investigator, makes it undesirable for the patient to participate in the study
* Pregnant women are excluded from this study because imiquimod may have adverse effect on the fetus (FDA pregnancy risk category C). Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with imiquimod, breastfeeding should be discontinued if the mother is receiving study treatment
* Male patients unwilling or unable to comply with pregnancy prevention measures
* Subjects not receiving initial surgical treatment at Medical University of South Carolina (MUSC)

Primary outcomes:
measure: Number of Participants With 50% Reduction in Tumor Cell Count and Major Pathologic Response
timeFrame: Following 28 days of treatment
Secondary outcomes:
measure: Safety of Imiquimod Therapy
timeFrame: 2 months
Other outcomes:
measure: Changes in Immune Cell Count
timeFrame: Following 28 days of treatment
measure: RFS Follow-up
timeFrame: 1 year
Locations:
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
Study officials:
affiliation: Medical University of South Carolina
name: Angela Yoon, DDS
role: PRINCIPAL_INVESTIGATOR
References: