Stage I Oral Cavity Squamous Cell Carcinoma, Stage II Oral Cavity Squamous Cell Carcinoma
Conditions
Brief summary
This randomized phase II clinical trial studies how well photodynamic therapy with HPPH works compared to standard of care surgery in treating patients with oral cavity cancer. Photodynamic therapy can destroy or control disease by using a combination of drug, such as HPPH, and light and may be as effective as surgery in treating patients with oral cavity cancer.
Detailed description
PRIMARY OBJECTIVES: I. To test the non-inferiority of photodynamic therapy (PDT) to standard of care surgery by comparing the rate of tumor response after PDT to those observed after surgery, at 24 months post treatment. SECONDARY OBJECTIVES: I. To determine quality of life (QoL) at 6, 12, 18 and 24 months post PDT or surgery. II. To assess the toxicity using the Cancer Therapy Evaluation Program (CTEP) National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE Version 4.0). TERTIARY OBJECTIVES: I. Immune markers (T cells). II. To investigate the correlation of the diffuse reflectance optical spectroscopy with tumor response to PDT. OUTLINE: Patients are randomized to 1 of 2 treatment arms. ARM I: Patients undergo standard of care surgery on day 1. ARM II: Patients receive HPPH intravenously (IV) over 1 hour on day 0 and undergo PDT on day 1. After completion of study treatment, patients are followed up every 3-4 months in year 1 and every 3-6 months in year 2.
Interventions
DRUGHPPH
Given IV
OTHERLaboratory Biomarker Analysis
Correlative studies
DRUGPhotodynamic Therapy
Undergo PDT
OTHERQuality-of-Life Assessment
Ancillary studies
PROCEDURETherapeutic Conventional Surgery
Undergo standard of care surgery
Sponsors
National Cancer Institute (NCI)
Roswell Park Cancer Institute
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Have an Eastern Cooperative Oncology Group (ECOG) performance status of =\< 2 * Participants with previously untreated T1/T2 N0 squamous cell carcinoma of the oral cavity with or without extension to the oropharynx * Histologically confirmed squamous cell carcinoma of the target tumor(s) * Tumor thickness is 4 mm or less (measured clinically and/or by computed tomography \[CT\] or magnetic resonance imaging \[MRI\] scan) * CT or MRI of the neck to confirm staging * Tumor accessible for unrestricted illumination for photodynamic therapy (PDT) (accessibility as determined by the physician) * Life expectancy of at least 12 months in the judgment of the physician * Participants of child-bearing potential must agree to use adequate contraceptive methods (e.g., hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation; should a woman become pregnant or suspect she is pregnant while she is participating in this study, she should inform her treating physician immediately * Participant or legal representative must understand the investigational nature of this study and sign an Institutional Review Board approved written informed consent form prior to receiving any study related procedure
Exclusion criteria
* Those who have had chemotherapy or radiotherapy or targeted agents within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier * Those with known brain metastases should be excluded from this clinical trial because of their poor prognosis and because they often develop progressive neurologic dysfunction that would confound the evaluation of neurologic and other adverse events * Those with porphyria or with known hypersensitivity to porphyrins or porphyrin-like compounds * White blood cells (WBC) \< 4,000 * Total serum bilirubin \> 2 mg/dL * Serum creatinine \> 2 mg/dL * Alkaline phosphatase (hepatic) or serum glutamic-oxaloacetic transaminase (SGOT) \> 3 times the upper normal limit * Diagnostic biopsy reveals perineural invasion (PNI) and/or lymphovascular invasion (LVI) * 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 * Nodal disease as detected by clinical exam or CT * Pregnant or nursing females * Unwilling or unable to follow protocol requirements * Any condition which in the investigator's opinion deems the participant an unsuitable candidate to receive study drug * Received an investigational agent within 30 days prior to enrollment * Trismus or compromised airway * Previous treatment in the target tumor area
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Rate of Tumor Response as Evaluated by Response Evaluation Criteria in Solid Tumors Version 1.1 | At 24 months | Objective tumor response will be tabulated overall. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Quality of Life as Measured by the University of Washington Quality of Life Questionnaire Version 4 | Baseline up to 24 months | In the analysis of QoL simple data analyses will initially take place including individual participant-level profile plots and overall mean plots used to examining the mean structure. Formal statistical examination of longitudinal patterns will be done through the use of a mixed model. Once the model is fit, specific linear contrasts based on the estimated model parameters will be constructed and used to test hypotheses concerning between time points and between group comparisons |
| Adverse Events as Graded by CTCAE Version 4.0 Are Reported | Up to 30 days after administration of HPPH | The frequency of toxicities will be tabulated by grade. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Arm I (Surgery) Patients undergo standard of care surgery on day 1.
Quality-of-Life Assessment: Ancillary studies
Therapeutic Conventional Surgery: Undergo standard of care surgery | 1 |
| Arm II (HPPH, PDT) Patients receive HPPH IV over 1 hour on day 0 and undergo PDT on day 1.
HPPH: Given IV
Laboratory Biomarker Analysis: Correlative studies
Photodynamic Therapy: Undergo PDT
Quality-of-Life Assessment: Ancillary studies | 1 |
| Total | 2 |
Baseline characteristics
| Characteristic | Arm I (Surgery) | Arm II (HPPH, PDT) | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 1 Participants | 0 Participants | 1 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 1 Participants | 1 Participants |
| Age, Continuous | 86 years | 55 years | 71 years |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 1 Participants | 1 Participants | 2 Participants |
| Sex: Female, Male Female | 1 Participants | 0 Participants | 1 Participants |
| Sex: Female, Male Male | 0 Participants | 1 Participants | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 1 | 0 / 1 |
| other Total, other adverse events | 1 / 1 | 1 / 1 |
| serious Total, serious adverse events | 0 / 1 | 1 / 1 |
Outcome results
Primary
Rate of Tumor Response as Evaluated by Response Evaluation Criteria in Solid Tumors Version 1.1
Objective tumor response will be tabulated overall.
Time frame: At 24 months
Population: Due to PI departure and early termination with few patients, only the counts of events have been calculated and no testing was done
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Arm I (Surgery) | Rate of Tumor Response as Evaluated by Response Evaluation Criteria in Solid Tumors Version 1.1 | No evidence of disease | 1 Participants |
| Arm I (Surgery) | Rate of Tumor Response as Evaluated by Response Evaluation Criteria in Solid Tumors Version 1.1 | Other | 0 Participants |
| Arm II (HPPH, PDT) | Rate of Tumor Response as Evaluated by Response Evaluation Criteria in Solid Tumors Version 1.1 | No evidence of disease | 0 Participants |
| Arm II (HPPH, PDT) | Rate of Tumor Response as Evaluated by Response Evaluation Criteria in Solid Tumors Version 1.1 | Other | 1 Participants |
Secondary
Adverse Events as Graded by CTCAE Version 4.0 Are Reported
The frequency of toxicities will be tabulated by grade.
Time frame: Up to 30 days after administration of HPPH
Population: Due to PI departure with few patients, only the counts of events have been calculated and no tabulation was done..
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Arm I (Surgery) | Adverse Events as Graded by CTCAE Version 4.0 Are Reported | 1 Participants |
| Arm II (HPPH, PDT) | Adverse Events as Graded by CTCAE Version 4.0 Are Reported | 1 Participants |
Secondary
Change in Quality of Life as Measured by the University of Washington Quality of Life Questionnaire Version 4
In the analysis of QoL simple data analyses will initially take place including individual participant-level profile plots and overall mean plots used to examining the mean structure. Formal statistical examination of longitudinal patterns will be done through the use of a mixed model. Once the model is fit, specific linear contrasts based on the estimated model parameters will be constructed and used to test hypotheses concerning between time points and between group comparisons
Time frame: Baseline up to 24 months
Population: Due to PI departure with few patients, only the counts of events have been calculated and no testing was done..
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Arm I (Surgery) | Change in Quality of Life as Measured by the University of Washington Quality of Life Questionnaire Version 4 | Outstanding Overall QoL | 1 Participants |
| Arm I (Surgery) | Change in Quality of Life as Measured by the University of Washington Quality of Life Questionnaire Version 4 | Very good to Outstanding Overall QoL | 0 Participants |
| Arm II (HPPH, PDT) | Change in Quality of Life as Measured by the University of Washington Quality of Life Questionnaire Version 4 | Outstanding Overall QoL | 0 Participants |
| Arm II (HPPH, PDT) | Change in Quality of Life as Measured by the University of Washington Quality of Life Questionnaire Version 4 | Very good to Outstanding Overall QoL | 1 Participants |