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Mitomycin in Treating Patients with P16 Positive Oropharyngeal or P16 Negative Head and Neck Cancer That is Resistant to Platin, Fluorouracil, Cetuximab, and Taxane

Trial Status: Active

This phase II trial studies how well mitomycin works in treating patients with tumor protein (p)16 positive oropharyngeal or p16 negative head and neck squamous cell cancer that is resistant to platin, fluorouracil, cetuximab, and taxane. Drugs used in chemotherapy, such as mitomycin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Inclusion Criteria

  • Histologically or cytologically confirmed incurable HNSCC of the oral cavity, oropharynx, larynx, hypopharynx, and/or level 1-3 neck node with non-cutaneous squamous cell carcinoma (SCC) and unknown primary; “incurable” is defined as metastatic disease or a local or regional recurrence in a previously irradiated site that is unresectable (or patient declines resection)
  • Progression following platin, 5-FU, cetuximab and taxane given for incurable disease
  • Measurable disease defined as lesions that can be accurately measured in at least one dimension (longest diameter to be recorded) as >= 10 mm with computed tomography (CT) scan, as >= 20 mm by chest x-ray, or >= 10 mm with calipers by clinical exam per Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
  • Tissue available (either initial diagnostic or recurrent tissue specimen) for p16 testing (if p16 status is already known, this criterion may be waived)
  • Eastern Cooperative Oncology Group (ECOG) performance status =< 3
  • Absolute neutrophil count >= 1,000/mcl
  • Platelets >= 75,000/mcl
  • Total bilirubin =< 1.5 mg/dL
  • Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 2.5 x upper limit of normal (ULN)
  • Alkaline phosphatase =< 2.5 x ULN, unless bone metastasis is present in the absence of liver metastasis
  • Creatinine below ULN (males 0.7-1.30 mg/dl; females 0.6-1.10 mg/dl) OR creatinine clearance >= 60 mL/min/1.73 m^2 for patients with creatinine levels above institutional normal
  • Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control, abstinence) prior to study entry, for the duration of study participation, and for 1 month after completing treatment; should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately
  • Patients who have known human immunodeficiency virus (HIV) positivity must be on a 3-drug antiviral regimen that does not include zidovudine, and must have a cluster of differentiation (CD)4 count > 100/mm^3 and virus load < 5000 copies/ml, and are placed on a regimen to prevent pneumocystis pneumonia (PCP) reactivation during treatment
  • Ability to understand and willingness to sign an Institutional Review Board (IRB) approved written informed consent document (or that of legally authorized representative, if applicable)

Exclusion Criteria

  • Other active malignancy with the exception of basal cell or squamous cell carcinoma of the skin which were treated with local resection only, carcinoma in situ of the cervix, or synchronous head and neck (H&N) primaries
  • Currently receiving any other investigational agents
  • 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
  • Pregnant and/or breastfeeding; patient must have a negative pregnancy test within 7 days of start of study treatment
  • Known active central nervous system (CNS) metastases; subjects with previously treated brain metastases may participate provided they are stable (without evidence of progression by imaging for at least four weeks prior to the first dose of trial treatment and any neurologic symptoms have returned to baseline), have no evidence of new or enlarging brain metastases, and are not using steroids for at least 28 days prior to treatment
  • A history of allergic reactions attributed to compounds of similar chemical or biologic composition to mitomycin C or other agents used in the study


Saint Louis
Siteman Cancer Center at Washington University
Status: ACTIVE
Contact: Peter J. Oppelt
Phone: 636-916-9922


I. To determine the tumor response rate of patients with incurable head and neck squamous cell carcinoma (HNSCC) that progressed following platin, 5-FU (fluorouracil), cetuximab and taxane who are treated with mitomycin C (mitomycin), stratified for p16 positive (p16+) oropharyngeal squamous cell carcinoma (OPSCC) and p16 negative (p16-) HNSCC.

II. Post October 2020: To determine the tumor response rate of patients with incurable p16+ OPSCC HNSCC that progressed following platin, and immunotherapy who are treated with mitomycin C.


I. To determine progression-free survival (PFS) of these patients, stratified for p16+ OPSCC and p16- HNSCC.

II. To evaluate the instances of grades 3 and 4 adverse events (using Common Terminology Criteria for Adverse Events [CTCAE] version 3.0) on this trial.

III. To determine the overall survival (OS) of these patients, stratified for p16+ OPSCC and p16- HNSCC.

IV. To characterize the quality of life of patients on this trial.


I. To evaluate tumor tissue and blood for immunohistochemical analyses relating to response to mitomycin C.


Patients receive mitomycin intravenously (IV) on day 1. Cycles repeat every 5 weeks in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up at 28 days and then every 3 months.

Trial Phase Phase II

Trial Type Treatment

Lead Organization
Siteman Cancer Center at Washington University

Principal Investigator
Peter J. Oppelt

  • Primary ID 201503060
  • Secondary IDs NCI-2015-00406
  • ID NCT02369458