Photodynamic Therapy With HPPH Compared to Standard of Care Surgery in Treating Patients With Oral Cavity Cancer

Phase 2

Terminated

Conditions: Stage I Oral Cavity Squamous Cell Carcinoma
Stage II Oral Cavity Squamous Cell Carcinoma

Interventions: Drug: HPPH
Procedure: Therapeutic Conventional Surgery
Other: Laboratory Biomarker Analysis
Drug: Photodynamic Therapy
Other: Quality-of-Life Assessment

Registration Number: NCT03090412

Lead Sponsor: Roswell Park Cancer Institute

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.

Recruitment & Eligibility

Status: TERMINATED

Sex: All

Target Recruitment: 2

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

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Arm I (surgery)	Therapeutic Conventional Surgery	Patients undergo standard of care surgery on day 1.
Arm II (HPPH, PDT)	Quality-of-Life Assessment	Patients receive HPPH IV over 1 hour on day 0 and undergo PDT on day 1.
Arm I (surgery)	Quality-of-Life Assessment	Patients undergo standard of care surgery on day 1.
Arm II (HPPH, PDT)	HPPH	Patients receive HPPH IV over 1 hour on day 0 and undergo PDT on day 1.
Arm II (HPPH, PDT)	Laboratory Biomarker Analysis	Patients receive HPPH IV over 1 hour on day 0 and undergo PDT on day 1.
Arm II (HPPH, PDT)	Photodynamic Therapy	Patients receive HPPH IV over 1 hour on day 0 and undergo PDT on day 1.

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.