This is a single center, open-label, interventional pilot study to assess the feasibility, safety, and preliminary efficacy of intralesional 5-FU injections for the treatment of oral leukoplakia (OL).
PRIMARY OBJECTIVE: I. Evaluate the feasibility of intralesional 5-fluorouracil (5-FU) injection for the treatment of oral leukoplakia SECONDARY OBJECTIVE: I. Evaluate the safety profile and side effects of the study drug. OUTLINE: Participants diagnosed with OL will receive multiple intralesional injections of 5-FU solution over a maximum period of 6 weeks, followed by a follow-up visit after 3 months.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Given intralesionally by injection
University of California, San Francisco
San Francisco, California, United States
Proportion of participants s who complete 5-FU injections
The number of participants who completed the full course of 3 intralesional injections will be compared to the overall total of participants. If the the mucosa becomes ulcerated or participants are unable/unwilling to continue with therapy, 5FU injections will be stopped.
Time frame: Up to 6 weeks
Number of participants with treatment-related adverse events
The number of participants with documented treatment-related adverse events classified by the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 will be reported
Time frame: Up to 3 months
Change in area of clinically visible oral leukoplakia lesions
The total change in area (measured to nearest millimeter (mm) of clinically visible oral leukoplakia lesion will be defined by the product of the two largest perpendicular measurements. The two largest perpendicular measurements of the treated oral leukoplakia lesion will be measured and multiplied to calculate the lesion area at baseline and again at 3 months.
Time frame: Up to 3 months
Proportion of participants with a change in histologic grade
A board-certified oral and maxillofacial pathologist will review biopsy specimens obtained from the treated oral leukoplakia before and after treatment with 5FU and determine change in histologic grade from baseline.
Time frame: Up to 3 months
Change in scores on the oral mucosal diseases quality of life questionnaire (COMD-QLQ)
The oral mucosal diseases quality of life questionnaire (COMD-QLQ) is a validated instrument that will assesses the effect that oral leukoplakia has on participants' daily life activities before and after 5-FU intralesional therapy. The survey consists of 24 questions with five response options per item. The response for each item is coded from 0 to 4 with "not at all=0" and "extremely=4" (at time of scoring, for 3 questions, the Likert scale is reversed). The summary of the overall score ranges from 0 to 104, with a higher score indicating a poorer patient-assessed quality of life score.
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Time frame: Up to 3 months