Intralesional 5-fluorouracil Injection for the Treatment of Oral Leukoplakia (OL_5FU)

Intralesional 5-fluorouracil Injection for the Treatment of Oral Leukoplakia: a Pilot Study

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).

Study Overview

• Status: Withdrawn
• Conditions: Oral Leukoplakia
• Intervention / Treatment: Drug: 5-fluorouracil

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 Type: Interventional
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • San Francisco, California, United States, 94158
      • University of California, San Francisco

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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.	Drug: 5-fluorouracil; Given intralesionally by injection; Other Names: 5FU

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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	Up to 3 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	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.	Up to 3 months

Collaborators and Investigators

• Sponsor: University of California, San Francisco
• Investigators: Principal Investigator: Kyle Jones, DDS, PhD, University of California, San Francisco

Study record dates

Study Major Dates

• Study Start (Anticipated): September 30, 2022
• Primary Completion (Anticipated): August 31, 2023
• Study Completion (Anticipated): December 31, 2023

Study Registration Dates

• First Submitted: June 6, 2022
• First Submitted That Met QC Criteria: August 15, 2022
• First Posted (Actual): August 17, 2022

Study Record Updates

• Last Update Posted (Actual): September 22, 2022
• Last Update Submitted That Met QC Criteria: September 20, 2022
• Last Verified: September 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Site; Head and Neck Neoplasms; Stomatognathic Diseases; Mouth Diseases; Pathological Conditions, Anatomical; Precancerous Conditions; Mouth Neoplasms; Leukoplakia; Leukoplakia, Oral; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Antimetabolites, Antineoplastic; Antimetabolites; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Fluorouracil
• Other Study ID Numbers: OL_5FU; 22-36039 (Other Identifier: University of California, San Francisco)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No