Pembrolizumab in Treating Participants With Leukoplakia

NCT03603223 | Recruiting Phase 2 DMC FDA Drug

• 3 other identifiers: 16-001548NCI-2018-01227Pembrolizumab Leukoplakia
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 3

Brief 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.

Conditions

Erythroleukoplakia; Leukoplakia; Verrucous Oral Leukoplakia

Outcome Measures

Primary Outcomes (1)

• 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.

  At 6 months

Secondary Outcomes (1)

• Clinical response rate

  At 9 and 12 months

Other Outcomes (2)

• PD-L1 positivity

  Up to 2 years

• Correlation of response with PD-L1 positivity

  Up to 2 years

Study Arms (1)

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.

Biological: Pembrolizumab

Interventions

Pembrolizumab BIOLOGICAL

Given IV

Also known as: Keytruda, Lambrolizumab, MK-3475, SCH 900475

Treatment (pembrolizumab)

Eligibility Criteria

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

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Jonsson Comprehensive Cancer Center: lead
• Merck Sharp & Dohme LLC: collaborator

Study Sites (3)

UC San Diego Moores Cancer Center

La Jolla, California, 92093, United States

NOT YET RECRUITING

USC / Norris Comprehensive Cancer Center

Los Angeles, California, 90033, United States

NOT YET RECRUITING

UCLA / Jonsson Comprehensive Cancer Center

Los Angeles, California, 90095, United States

RECRUITING

MeSH Terms

Conditions

Leukoplakia

Interventions

pembrolizumab

Condition Hierarchy (Ancestors)

Precancerous Conditions; Neoplasms; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms

Study Officials

• Deborah Wong

  UCLA / Jonsson Comprehensive Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Thu P Ly

CONTACT

310-794-2464; tply@mednet.ucla.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 19, 2018
• First Posted: July 27, 2018
• Study Start: May 3, 2019
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): August 1, 2027
• Last Updated: July 30, 2025

Record last verified: 2025-07

Locations

US (3)