Oral LeukoplakiaPhase 1OL_5FUNCT05505539
Intralesional 5-fluorouracil Injection for the Treatment of Oral Leukoplakia
A Phase 1 study testing 5-fluorouracil in people with Oral Leukoplakia, run by University of California, San Francisco at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Oral Leukoplakia
- What is being tested?
- 5-fluorouracil
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Sep 2022to 31 Dec 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California, San Francisco
San Francisco, California, 94158
About this study
Official title: 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).
Read the detailed description
PRIMARY OBJECTIVE:
- Evaluate the feasibility of intralesional 5-fluorouracil (5-FU) injection for the treatment of oral leukoplakia
SECONDARY OBJECTIVE:
- 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.
- Conditions
- Oral Leukoplakia
- Being tested
- 5-fluorouracil
- Medical subject headings
- Leukoplakia, Oral
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
5-fluorouracilDrug
Also called 5FU
Given intralesionally by injection
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: 5-fluorouracil |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Measured: Up to 3 months
Secondary outcomes (3)
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.
Measured: 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.
Measured: 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.
Measured: Up to 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2022
- Study start30 Sep 2022 (estimated)
- Primary completion31 Aug 2023 (estimated)
- Study completion31 Dec 2023 (estimated)
Why it stopped: Principal Investigator no longer with university.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Oral LeukoplakiaStudies of the same condition, open and under way.
- More trials run by University of California, San FranciscoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Kyle Jones, DDS, PhD, Principal investigator, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05505539
- Sponsor's protocol ID
- OL_5FU
- Other IDs
- 22-36039
- Acronym
- OL_5FU
- Status last verified
- Sep 2022
- First submitted
- 6 Jun 2022
- Last updated
- 22 Sep 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intralesional 5-fluorouracil Injection for the Treatment of Oral Leukoplakia: a Pilot Study
- ClinicalTrials.gov ID
- NCT05505539
- Acronym / sponsor ID
- OL_5FU / OL_5FU
- Conditions
- Oral Leukoplakia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Principal Investigator no longer with university.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- Study start
- 30 Sep 2022 (estimated)
- Primary completion
- 31 Aug 2023 (estimated)
- Study completion
- 31 Dec 2023 (estimated)
- First posted
- 17 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- Evaluate the feasibility of intralesional 5-fluorouracil (5-FU) injection for the treatment of oral leukoplakia
SECONDARY OBJECTIVE:
- 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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 5-fluorouracil | Experimental | Drug: 5-fluorouracilParticipants 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 5-fluorouracilAlso: 5FUGiven intralesionally by injection | Drug | 5-fluorouracil |
Primary outcome
Proportion of participants s who complete 5-FU injections
Time frame: 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
Time frame: 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
3 secondary outcomes
Change in area of clinically visible oral leukoplakia lesions
Time frame: Up to 3 months
Proportion of participants with a change in histologic grade
Time frame: Up to 3 months
Change in scores on the oral mucosal diseases quality of life questionnaire (COMD-QLQ)
Time frame: Up to 3 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Sep 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kyle Jones, DDS, PhD principal investigator
University of California, San Francisco
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing 5-fluorouracil in people with Oral Leukoplakia, run by University of California, San Francisco at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 22 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of participants s who complete 5-FU injections (time frame: Up to 6 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Sep 2022.