Actively Recruiting
A Phase II Open Label, Single Arm Study to Evaluate the Efficacy of Pembrolizumab for Leukoplakia
Led by Jonsson Comprehensive Cancer Center ยท Updated on 2025-07-30
26
Participants Needed
3
Research Sites
52 weeks
Total Duration
On this page
Sponsors
J
Jonsson Comprehensive Cancer Center
Lead Sponsor
M
Merck Sharp & Dohme LLC
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating pembrolizumab, a monoclonal antibody, in treating leukoplakia, including erythroleukoplakia and proliferative verrucous leukoplakia (PVL). This phase II pilot trial aims to study how well pembrolizumab works in reducing the size and severity of these lesions, which may interfere with tumor cell growth and spread. The study also explores biomarkers like PD-L1 expression to better understand the disease and treatment effects. Participants receive pembrolizumab intravenously over 30 minutes on day 1, with treatments repeating every 3 weeks for up to 6 months, provided there is no disease progression or unacceptable side effects. After completing treatment, participants undergo follow-up visits at 30 days and then every 3 months for 2 years to monitor their condition and response to therapy. During the study, participants will have biopsies and photographs of lesions taken at baseline and at 6 months to assess changes. Researchers will evaluate clinical and histologic responses at 6, 9, and 12 months, as well as monitor any treatment-related toxicity. Routine blood tests and performance status assessments help ensure participant safety throughout the trial. The primary outcome is the clinical response rate at 6 months, with additional measures tracked during follow-up.
CONDITIONS
Brief Title
Pembrolizumab in Treating Participants With Leukoplakia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Willing and able to provide written informed consent or assent for the trial
- Have leukoplakia, erythroleukoplakia, or proliferative verrucous leukoplakia with measurable lesions not suitable for surgery or radiation, or have refused these treatments
- Have at least one lesion that can be followed during treatment
- Evidence of moderate or severe dysplasia or carcinoma in situ
- Baseline biopsy specimen available or willingness to undergo a new biopsy
- Willingness to consent to lesion photographs
- Willingness to undergo biopsy at 6 months
- Performance status of 0 or 1 on the ECOG scale
- Absolute neutrophil count (ANC) of 1,500/mcL or higher within 10 days before treatment
- Platelet count of 100,000/mcL or higher within 10 days before treatment
- Hemoglobin 9 g/dL or higher without transfusion or EPO dependency within 7 days before treatment
- Kidney function with serum creatinine less than or equal to 1.5 times upper limit of normal or creatinine clearance 60 mL/min or higher within 10 days before treatment
- Total bilirubin less than or equal to 1.5 times upper limit of normal within 10 days before treatment
- Liver enzymes (AST and ALT) less than or equal to 2.5 times upper limit of normal or less than or equal to 5 times upper limit if liver metastases present within 10 days before treatment
- Albumin level 2.5 mg/dL or higher within 10 days before treatment
- INR or prothrombin time less than or equal to 1.5 times upper limit of normal unless on anticoagulants with therapeutic levels within 10 days before treatment
- aPTT less than or equal to 1.5 times upper limit of normal unless on anticoagulants with therapeutic levels within 10 days before treatment
- Female participants of childbearing potential must have a negative pregnancy test within 10 days before first dose
- Female participants of childbearing potential must use two methods of birth control or be surgically sterile or abstain from heterosexual activity during the study and for 120 days after last dose
- Male participants must use adequate contraception during the study and for 120 days after last dose
You will not qualify if you...
- Have only mild dysplasia or hyperplasia without more severe disease
- Currently receiving or recently received investigational therapy or device within 4 weeks before treatment
- Diagnosis of immunodeficiency or receiving systemic steroids over prednisone 10 mg daily or other immunosuppressive therapy within 7 days before treatment
- Known active tuberculosis
- Allergy or hypersensitivity to pembrolizumab or its ingredients
- Received anti-cancer monoclonal antibodies within 4 weeks before treatment or have not recovered from prior related side effects
- Received chemotherapy, targeted small molecules, or radiation within 2 weeks before treatment or not recovered from side effects (except for grade 2 or less neuropathy)
- Known active malignancy requiring treatment except certain skin cancers or in situ cervical cancer
- Known active brain metastases or carcinomatous meningitis
- Active autoimmune disease needing systemic treatment in past 2 years
- History or evidence of active non-infectious pneumonitis
- Active infection needing systemic therapy
- Any condition or lab abnormality that could affect participation or study results
- Psychiatric or substance abuse disorders interfering with cooperation
- Pregnant, breastfeeding, or planning to conceive or father children during the study period
- Prior treatment with anti-PD-1, anti-PD-L1, or anti-PD-L2 agents
- Known HIV infection
- Active hepatitis B or hepatitis C infection
- Received live vaccine within 30 days before planned treatment start (seasonal flu shots allowed except nasal spray)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 6 months
Participants receive pembrolizumab intravenously every 3 weeks for 6 months unless disease progression or unacceptable toxicity occurs.
Approximately 8 visits every 3 weeks (in-person)
Duration - 2 years and 1 month
After treatment completion, participants are followed up to monitor clinical response and safety for 2 years.
1 visit at 30 days post-treatment and quarterly visits every 3 months for 2 years (in-person)
Trial Site Locations
Total: 3 locations
1
UC San Diego Moores Cancer Center
La Jolla, California, United States, 92093
Not Yet Recruiting
2
USC / Norris Comprehensive Cancer Center
Los Angeles, California, United States, 90033
Not Yet Recruiting
3
UCLA / Jonsson Comprehensive Cancer Center
Los Angeles, California, United States, 90095
Actively Recruiting
Research Team
T
Thu P Ly
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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