Intralesional 5-fluorouracil Injection for the Treatment of Oral Leukoplakia

Phase 1

Withdrawn

• Conditions: Oral Leukoplakia
• Interventions: Drug: 5-fluorouracil

• Registration Number: NCT05505539
• Lead Sponsor: University of California, San Francisco

Brief Summary

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).

Detailed Description

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 Timeline

• Study First Submitted: 2022-06-06
• Study First Submitted QC: 2022-08-15
• Study First Posted: 2022-08-17
• Status Verified: 2022-09
• Last Update Submitted: 2022-09-20
• Last Update Posted: 2022-09-22
• Study Start: 2022-09-30
• Primary Completion: 2023-08-31
• Study Completion: 2023-12-31

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

• Adults, 18 years of age or older
• Any gender, race, or ethnicity
• Clinical diagnosis of oral leukoplakia and biopsy proven dysplasia (any grade)
• Oral leukoplakia at least 1 cm in largest diameter
• Ability to understand and willingness to sign a written informed consent document
• Willingness to provide blood and tissue from diagnostic biopsies
• Any smoking history is permitted

Exclusion Criteria

• Pregnant or lactating women
• Men and women unwilling to use contraception while on study
• History of malignancy that required cytotoxic chemotherapy within the previous 3 months
• Use of 5-FU (systemic or topical) within 3 months prior to study enrollment
• History of allergic reaction or severe hypersensitivity to 5-FU and/or lidocaine
• Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
5-fluorouracil	5-fluorouracil	Participants will receive up to 3 intralesional 5-fluorouracil injections (0.25 mL of a 50mg/mL solution) once every 2 weeks into an oral leukoplakia lesion.

Primary Outcome Measures

Name	Time	Method
Proportion of participants s who complete 5-FU injections	Up to 6 weeks	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.
Number of participants with treatment-related adverse events	Up to 3 months	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

Secondary Outcome Measures

Name	Time	Method
Proportion of participants with a change in histologic grade	Up to 3 months	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.
Change in area of clinically visible oral leukoplakia lesions	Up to 3 months	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.
Change in scores on the oral mucosal diseases quality of life questionnaire (COMD-QLQ)	Up to 3 months	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.

Trial Locations

Locations (1)

University of California, San Francisco; 🇺🇸 San Francisco, California, United States