ErythroleukoplakiaPhase 2NCT03603223
Pembrolizumab in Treating Participants With Leukoplakia
A Phase 2 study testing Pembrolizumab in people with Erythroleukoplakia and Leukoplakia, run by Jonsson Comprehensive Cancer Center at 3 sites in United States.
Trial summary
This phase II pilot trial studies how well pembrolizumab works in treating leukoplakia. Monoclonal antibodies, such as pembrolizumab, may interfere with the ability of tumor cells to grow and spread. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Erythroleukoplakia, Leukoplakia, Verrucous Oral Leukoplakia
- What is being tested?
- Pembrolizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 3 sitesUnited States
- Study dates
- 3 May 2019to 1 Aug 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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
- Be willing and able to provide written informed consent/assent for the trial.
- Subjects must have leukoplakia, erythroleukoplakia or proliferative verrucous leukoplakia (PVL) with lesions measurable in 2 dimensions, not amenable to surgical resection or radiation or who have refused surgery or radiation. Patients must have at least 1 lesion that can be followed on treatment. (Patients who have undergone complete excision of lesions and are clinically without evidence of disease will not be eligible for study.)
- Evidence of moderate or severe dysplasia or carcinoma in situ.
- Baseline biopsy specimen available for biomarker analysis or willingness to undergo fresh baseline biopsy.
- Willingness to consent to photographs of lesions.
- Willingness to undergo biopsy at 6 months.
Show all 20Show fewer
- Have a performance status of 0 or 1 on the Eastern Cooperative Oncology Group (ECOG) performance scale.
- Absolute neutrophil count (ANC) >= 1,500 /mcL within 10 days of treatment initiation.
- Platelets >= 100,000/mcL within 10 days of treatment initiation.
- Hemoglobin >= 9 g/dL or >= 5.6 mmol/L without transfusion or erythropoietin (EPO) dependency (within 7 days of assessment).
- Serum creatinine =< 1.5 X upper limit of normal (ULN) OR measured or calculated creatinine clearance (CrCl) >= 60 mL/min for subject with creatinine levels > 1.5 X institutional ULN within 10 days of treatment initiation. (Glomerular filtration rate [GFR] can also be used in place of creatinine or CrCl).
- Creatinine clearance should be calculated per institutional standard.
- Serum total bilirubin =< 1.5 X ULN OR direct bilirubin =< ULN for subjects with total bilirubin levels > 1.5 ULN within 10 days of treatment initiation.
- Aspartate aminotransferase (AST) serum glutamic oxaloacetic transaminase (SGOT) and alanine aminotransferase (ALT) serum glutamate pyruvate transaminase (SGPT) =< 2.5 X ULN OR =< 5 X ULN for subjects with liver metastases within 10 days of treatment initiation.
- Albumin >= 2.5 mg/dL within 10 days of treatment initiation.
- International normalized ratio (INR) or prothrombin time (PT) =< 1.5 X ULN unless subject is receiving anticoagulant therapy as long as PT or partial thromboplastin time (PTT) is within therapeutic range of intended use of anticoagulants. (Within 10 days of treatment initiation.)
- Activated partial thromboplastin time (aPTT) =< 1.5 X ULN unless subject is receiving anticoagulant therapy as long as PT or PTT is within therapeutic range of intended use of anticoagulants. (Within 10 days of treatment initiation.)
- Female subject of childbearing potential should have a negative urine or serum pregnancy within 10 days prior to receiving the first dose of study medication. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required.
- Female subjects of childbearing potential should be willing to use 2 methods of birth control or be surgically sterile, or abstain from heterosexual activity for the course of the study through 120 days after the last dose of study medication. Subjects of childbearing potential are those who have not been surgically sterilized or have not been free from menses for > 1 year.
- Male subjects should agree to use an adequate method of contraception starting with the first dose of study therapy through 120 days after the last dose of study therapy.
Who cannot join
- Patients with leukoplakia, erythroleukoplakia or PVL who have only mild dysplasia or hyperplasia are excluded.
- Is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy or used an investigational device within 4 weeks of the first dose of treatment.
- Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy > prednisone 10 mg daily or equivalent, or any other form of immunosuppressive therapy within 7 days prior to the first dose of trial treatment.
- Has a known history of active TB (Bacillus tuberculosis).
- Hypersensitivity to pembrolizumab or any of its excipients.
- Has had a prior anti-cancer monoclonal antibody (mAb) within 4 weeks prior to study day 1 or who has not recovered (i.e., =< grade 1 or at baseline) from adverse events due to agents administered more than 4 weeks earlier.
Show all 22Show fewer
- Has had prior chemotherapy, targeted small molecule therapy, or radiation therapy within 2 weeks prior to study day 1 or who has not recovered (i.e., =< grade 1 or at baseline) from adverse events due to a previously administered agent.
- Note: Subjects with =< grade 2 neuropathy are an exception to this criterion and may qualify for the study.
- Note: If subject received major surgery, they must have recovered adequately from the toxicity and/or complications from the intervention prior to starting therapy.
- Has a known additional malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer.
- Has known active central nervous system (CNS) metastases and/or carcinomatous meningitis. Subjects with previously treated brain metastases may participate provided they are stable (without evidence of progression by imaging for at least four weeks prior to the first dose of trial treatment and any neurologic symptoms have returned to baseline), have no evidence of new or enlarging brain metastases, and are not using steroids for at least 7 days prior to trial treatment. This exception does not include carcinomatous meningitis which is excluded regardless of clinical stability.
- Has active autoimmune disease that has required systemic treatment in the past 2 years (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment.
- Has known history of, or any evidence of active, non-infectious pneumonitis.
- Has an active infection requiring systemic therapy.
- Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the subject?s participation for the full duration of the trial, or is not in the best interest of the subject to participate, in the opinion of the treating investigator.
- Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
- Is pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial, starting with the pre-screening or screening visit through 120 days after the last dose of trial treatment.
- Has received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent.
- Has a known history of human immunodeficiency virus (HIV) (HIV 1/2 antibodies).
- Has known active hepatitis B (e.g., hepatitis B virus surface antigen [HBsAg] reactive) or hepatitis C virus (e.g., HCV ribonucleic acid [RNA] [qualitative] is detected).
- Has received a live vaccine within 30 days of planned start of study therapy.
- Note: Seasonal influenza vaccines for injection are generally inactivated flu vaccines and are allowed; however intranasal influenza vaccines (e.g., Flu-Mist) are live attenuated vaccines, and are not allowed.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Not yet recruiting
UC San Diego Moores Cancer Center
La Jolla, California, 92093
Ezra E. Cohen, 858-543-6161, ecohen@ucsd.edu
Ezra E. Cohen
- Not yet recruiting
USC / Norris Comprehensive Cancer Center
Los Angeles, California, 90033
Jorge J. Nieva, 323-865-0421, jorge.nieva@med.usc.edu
Jorge J. Nieva
- Recruiting
UCLA / Jonsson Comprehensive Cancer Center
Los Angeles, California, 90095
Deborah J. Wong, 310-794-4955, dewong@mednet.ucla.edu
Deborah J. Wong
About this study
Official title: A Phase II Open Label, Single Arm Study to Evaluate the Efficacy of Pembrolizumab for Leukoplakia
This phase II pilot trial studies how well pembrolizumab works in treating leukoplakia. Monoclonal antibodies, such as pembrolizumab, may interfere with the ability of tumor cells to grow and spread.
Read the detailed description
PRIMARY OBJECTIVES:
- Clinical response rate at 6 months? percent of patients with complete response and partial response at 6 months.
SECONDARY OBJECTIVES:
- Histologic response rate at 6 months. II. Change in clinical impression based on photographs of the lesion. III. Clinical response rate at 9 months and 12 months. IV. Toxicity.
EXPLORATORY OBJECTIVES:
- PD-L1 expression in leukoplakia lesions and biomarker analysis.
OUTLINE:
Participants receive pembrolizumab intravenously (IV) over 30 minutes on day 1. Courses repeat every 3 weeks for 6 months in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, participants are followed up at 30 days and every 3 months for 2 years.
- Conditions
- Erythroleukoplakia, Leukoplakia, Verrucous Oral Leukoplakia
- Being tested
- Pembrolizumab
- Medical subject headings
- Leukoplakia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PembrolizumabBiological
Also called Keytruda, Lambrolizumab, MK-3475, SCH 900475
Given IV
| Study group | Role | What they receive |
|---|---|---|
| Treatment (pembrolizumab) | Experimental | Participants receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks for 6 months in the absence of disease progression or unacceptable toxicity. Receives: Biological: Pembrolizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical response rate
If there is a signal of efficacy based on this analysis, further exploratory analyses using Cox-proportional hazards regression, will be performed to identify variables which correlate to improved 6-month clinical response rate will be performed. Variables will include demographics, age, gender, pack-years smoking history, size of the lesions, prior history of leukoplakia or erythroleukoplakia, and human papillomavirus (HPV) status. While the study is not powered to detect a statistically difference between these groups, such an analysis may help identify a population more likely to benefit from treatment.
Measured: At 6 months
Secondary outcomes (1)
Clinical response rate
Response rate data will be reported in tabular form.
Measured: At 9 and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jul 2018
- Study start3 May 2019
- Primary completion1 Aug 2027 (estimated)
- Study completion1 Aug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ErythroleukoplakiaStudies of the same condition, open and under way.
- More trials run by Jonsson Comprehensive Cancer CenterThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 26 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Jonsson Comprehensive Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Merck Sharp & Dohme LLC
- Responsible party
- Sponsor
- Investigators
- Deborah Wong, Principal investigator, UCLA / Jonsson Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03603223
- Sponsor's protocol ID
- 16-001548
- Other IDs
- NCI-2018-01227, Pembrolizumab Leukoplakia, 16-001548
- Status last verified
- Aug 2026
- First submitted
- 19 Jun 2018
- Last updated
- 20 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Open Label, Single Arm Study to Evaluate the Efficacy of Pembrolizumab for Leukoplakia
- ClinicalTrials.gov ID
- NCT03603223
- Acronym / sponsor ID
- 16-001548
- Conditions
- Erythroleukoplakia, Leukoplakia, Verrucous Oral Leukoplakia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 26 participants (estimated)
- Lead sponsor
- Jonsson Comprehensive Cancer CenterUniversity, hospital or other
- Collaborators
- Merck Sharp & Dohme LLC
- Study start
- 3 May 2019 (actual)
- Primary completion
- 1 Aug 2027 (estimated)
- Study completion
- 1 Aug 2028 (estimated)
- First posted
- 27 Jul 2018 (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 phase II pilot trial studies how well pembrolizumab works in treating leukoplakia. Monoclonal antibodies, such as pembrolizumab, may interfere with the ability of tumor cells to grow and spread.
Detailed description
PRIMARY OBJECTIVES:
- Clinical response rate at 6 months? percent of patients with complete response and partial response at 6 months.
SECONDARY OBJECTIVES:
- Histologic response rate at 6 months. II. Change in clinical impression based on photographs of the lesion. III. Clinical response rate at 9 months and 12 months. IV. Toxicity.
EXPLORATORY OBJECTIVES:
- PD-L1 expression in leukoplakia lesions and biomarker analysis.
OUTLINE:
Participants receive pembrolizumab intravenously (IV) over 30 minutes on day 1. Courses repeat every 3 weeks for 6 months in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, participants are followed up at 30 days and every 3 months for 2 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (pembrolizumab) | Experimental | Biological: PembrolizumabParticipants receive pembrolizumab IV over 30 minutes on day 1. Courses repeat every 3 weeks for 6 months in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- Be willing and able to provide written informed consent/assent for the trial.
- Subjects must have leukoplakia, erythroleukoplakia or proliferative verrucous leukoplakia (PVL) with lesions measurable in 2 dimensions, not amenable to surgical resection or radiation or who have refused surgery or radiation. Patients must have at least 1 lesion that can be followed on treatment. (Patients who have undergone complete excision of lesions and are clinically without evidence of disease will not be eligible for study.)
- Evidence of moderate or severe dysplasia or carcinoma in situ.
- Baseline biopsy specimen available for biomarker analysis or willingness to undergo fresh baseline biopsy.
- Willingness to consent to photographs of lesions.
Key exclusion criteria (22 listed)
- Patients with leukoplakia, erythroleukoplakia or PVL who have only mild dysplasia or hyperplasia are excluded.
- Is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy or used an investigational device within 4 weeks of the first dose of treatment.
- Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy > prednisone 10 mg daily or equivalent, or any other form of immunosuppressive therapy within 7 days prior to the first dose of trial treatment.
- Has a known history of active TB (Bacillus tuberculosis).
- Hypersensitivity to pembrolizumab or any of its excipients.
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 |
|---|---|---|
| PembrolizumabAlso: Keytruda, Lambrolizumab, MK-3475, SCH 900475Given IV | Biological | Treatment (pembrolizumab) |
Primary outcome
Clinical response rate
Time frame: At 6 months
If there is a signal of efficacy based on this analysis, further exploratory analyses using Cox-proportional hazards regression, will be performed to identify variables which correlate to improved 6-month clinical response rate will be performed. Variables will include demographics, age, gender, pack-years smoking history, size of the lesions, prior history of leukoplakia or erythroleukoplakia, and human papillomavirus (HPV) status. While the study is not powered to detect a statistically difference between these groups, such an analysis may help identify a population more likely to benefit from treatment.
1 secondary outcome
Clinical response rate
Time frame: At 9 and 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2026.
Related trials for Erythroleukoplakia
Not yet recruitingObservational study
Evaluation of Artificial Intelligence in Diagnosis and Risk Assessment of Oral Potentially Malignant Disorders
Oral Lichen Planus, Oral Leukoplakia
RecruitingObservational study
Evaluation of Oral Potentially Malignant Disorders (OPMDs) with STRATICYTE™
Mouth Diseases
RecruitingObservational study
Early Prediction of Oral Cancer by S100A7 Immunohistochemistry Signature-based Assessment
Oral Neoplasm
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.
Deborah Wong principal investigator
UCLA / Jonsson Comprehensive Cancer Center
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pembrolizumab in people with Erythroleukoplakia and Leukoplakia, run by Jonsson Comprehensive Cancer Center at 3 sites 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 20 inclusion and 22 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: looking for participants now, as of the sponsor's last update on 20 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Thu P Ly (310-794-2464, tply@mednet.ucla.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinical response rate (time frame: At 6 months). 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 20 Aug 2026.