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

Primary Purpose

Oral Leukoplakia

Status
Withdrawn
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
5-fluorouracil
Sponsored by
University of California, San Francisco
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Oral Leukoplakia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of California, San Francisco

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

5-fluorouracil

Arm Description

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.

Outcomes

Primary Outcome Measures

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.
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

Secondary Outcome Measures

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.
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.
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.

Full Information

First Posted
June 6, 2022
Last Updated
September 20, 2022
Sponsor
University of California, San Francisco
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1. Study Identification

Unique Protocol Identification Number
NCT05505539
Brief Title
Intralesional 5-fluorouracil Injection for the Treatment of Oral Leukoplakia
Acronym
OL_5FU
Official Title
Intralesional 5-fluorouracil Injection for the Treatment of Oral Leukoplakia: a Pilot Study
Study Type
Interventional

2. Study Status

Record Verification Date
September 2022
Overall Recruitment Status
Withdrawn
Why Stopped
Principal Investigator no longer with university.
Study Start Date
September 30, 2022 (Anticipated)
Primary Completion Date
August 31, 2023 (Anticipated)
Study Completion Date
December 31, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of California, San Francisco

4. Oversight

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
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Oral Leukoplakia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)

8. Arms, Groups, and Interventions

Arm Title
5-fluorouracil
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
5-fluorouracil
Other Intervention Name(s)
5FU
Intervention Description
Given intralesionally by injection
Primary Outcome Measure Information:
Title
Proportion of participants s who complete 5-FU injections
Description
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
Title
Number of participants with treatment-related adverse events
Description
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
Secondary Outcome Measure Information:
Title
Change in area of clinically visible oral leukoplakia lesions
Description
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
Title
Proportion of participants with a change in histologic grade
Description
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
Title
Change in scores on the oral mucosal diseases quality of life questionnaire (COMD-QLQ)
Description
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.
Time Frame
Up to 3 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kyle Jones, DDS, PhD
Organizational Affiliation
University of California, San Francisco
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of California, San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94158
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Intralesional 5-fluorouracil Injection for the Treatment of Oral Leukoplakia

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