Intralesional 5-fluorouracil Injection for the Treatment of Oral Leukoplakia

NCT05505539 | Withdrawn Phase 1 FDA Drug

• 2 other identifiers: OL_5FU22-36039
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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).

Conditions

Oral Leukoplakia

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (3)

• Change in area of clinically visible oral leukoplakia lesions

  Up to 3 months

• Proportion of participants with a change in histologic grade

  Up to 3 months

• Change in scores on the oral mucosal diseases quality of life questionnaire (COMD-QLQ)

  Up to 3 months

Study Arms (1)

5-fluorouracil

EXPERIMENTAL

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

Interventions

5-fluorouracil DRUG

Given intralesionally by injection

Also known as: 5FU

5-fluorouracil

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of California, San Francisco: lead

Study Sites (1)

University of California, San Francisco

San Francisco, California, 94158, United States

Location

MeSH Terms

Conditions

Leukoplakia, Oral

Interventions

Fluorouracil

Condition Hierarchy (Ancestors)

Mouth Neoplasms; Head and Neck Neoplasms; Neoplasms by Site; Neoplasms; Leukoplakia; Precancerous Conditions; Mouth Diseases; Stomatognathic Diseases; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Uracil; Pyrimidinones; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Kyle Jones, DDS, PhD

  University of California, San Francisco

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 6, 2022
• First Posted: August 17, 2022
• Study Start: September 30, 2022
• Primary Completion: August 31, 2023
• Study Completion: December 31, 2023
• Last Updated: September 22, 2022

Record last verified: 2022-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)