What side effects are associated with this type of intervention?

The most common treatments for leukoplakia, particularly those involving monoclonal antibodies like pembrolizumab, can lead to side effects such as fatigue, nausea, and infusion reactions. More severe immune-mediated adverse events may include pneumonitis, colitis, hepatitis, endocrinopathies, and various skin reactions.

What prior FDA approvals exist?

Currently, there are no specific FDA-approved treatments for leukoplakia that involve monoclonal antibodies or similar immunotherapies. However, pembrolizumab, a monoclonal antibody that modulates the immune system to interfere with tumor cell growth and spread, is being studied in clinical trials for its potential efficacy in treating leukoplakia. Pembrolizumab has been approved for various other cancers, indicating its broader application in oncology.

What other types of research exist?

As of now, there are no specific novel alternatives to Pembrolizumab mentioned for the treatment of leukoplakia in the provided context. However, patients should consult their healthcare providers about ongoing clinical trials and emerging therapies in immunotherapy that might be applicable.

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Monoclonal Antibodies

Pembrolizumab for Leukoplakia

Q: What is the mechanism of action of this treatment?

The most common treatments for Leukoplakia, particularly those involving monoclonal antibodies like Pembrolizumab, work by modulating the immune system. Pembrolizumab targets the PD-1 pathway, a checkpoint that regulates immune responses. By inhibiting this pathway, Pembrolizumab enhances the immune system's ability to attack abnormal cells, including pre-cancerous cells found in Leukoplakia. This mechanism is crucial for Leukoplakia patients as it helps prevent the progression of these lesions into cancer, offering a targeted and potentially more effective treatment option.

Phase 2

Recruiting

Led By Deborah Wong

Research Sponsored by Jonsson Comprehensive Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Have a performance status of 0 or 1 on the Eastern Cooperative Oncology Group (ECOG) performance scale

Baseline biopsy specimen available for biomarker analysis or willingness to undergo fresh baseline biopsy

Must not have

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

Patients with leukoplakia, erythroleukoplakia or PVL who have only mild dysplasia or hyperplasia are excluded

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 2 years

Awards & highlights

No Placebo-Only Group

Summary

This trial studies how well pembrolizumab works in treating leukoplakia. Pembrolizumab helps the immune system fight cancer by blocking a protein that prevents immune cells from attacking cancer cells. The trial targets patients with leukoplakia, aiming to see if this medication can effectively treat this condition. Pembrolizumab is an immune checkpoint inhibitor that has been used in the treatment of various cancers, including non-small cell lung cancer, melanoma, and head and neck cancers.

Who is the study for?

This trial is for adults with moderate to severe dysplasia or carcinoma in situ within oral leukoplakia lesions that can't be removed surgically or treated with radiation, or for those who refuse such treatments. Participants must have measurable lesions, provide consent for photographs and biopsies, and meet specific health criteria including blood counts and organ function tests. Women of childbearing potential must use contraception.

What is being tested?

The study is testing the effectiveness of pembrolizumab, a monoclonal antibody designed to block tumor growth by interfering with certain proteins on cells. This phase II pilot trial aims to see how well it works against leukoplakia by potentially preventing the abnormal cells from growing and spreading.

What are the potential side effects?

Pembrolizumab may cause immune-related side effects like inflammation in various organs (e.g., lungs), skin reactions, hormone gland problems (like thyroid dysfunction), infusion-related reactions, fatigue, appetite changes, nausea, itching or rash.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am fully active or restricted in physically strenuous activity but can do light work.

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I can provide a biopsy sample or am willing to have a new biopsy for the study.

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My biopsy shows moderate or severe abnormal cell growth.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I haven't had cancer treatment with a monoclonal antibody in the last 4 weeks or have recovered from its side effects.

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I do not have mild dysplasia or hyperplasia with my leukoplakia or erythroleukoplakia.

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I am currently being treated for an infection.

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I have or had lung inflammation not caused by an infection.

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I haven't needed systemic treatment for an autoimmune disease in the last 2 years.

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I have not received a live vaccine within the last 30 days, except for the flu shot.

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I have been diagnosed with HIV.

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I have an active tuberculosis infection.

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I have been treated with drugs targeting PD-1, PD-L1, or PD-L2.

Select...

I am not on high-dose steroids or other drugs that weaken my immune system.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Clinical response rate

Other study objectives

Correlation of response with PD-L1 positivity

PD-L1 positivity

Side effects data

From 2024 Phase 3 trial • 804 Patients • NCT03040999

64%

Radiation skin injury

63%

Stomatitis

58%

Anaemia

56%

Nausea

48%

Dry mouth

45%

Constipation

45%

Weight decreased

44%

Dysphagia

42%

Neutrophil count decreased

33%

Dysgeusia

33%

Vomiting

32%

Fatigue

31%

White blood cell count decreased

28%

Hypomagnesaemia

26%

Decreased appetite

25%

Hypothyroidism

25%

Hypokalaemia

24%

Lymphocyte count decreased

24%

Platelet count decreased

23%

Oropharyngeal pain

23%

Blood creatinine increased

22%

Diarrhoea

22%

Odynophagia

20%

Hypoacusis

20%

Alanine aminotransferase increased

20%

Hyponatraemia

19%

Tinnitus

19%

Oral candidiasis

19%

Asthenia

16%

Pyrexia

16%

Cough

15%

Aspartate aminotransferase increased

15%

Rash

14%

Insomnia

13%

Acute kidney injury

13%

Pharyngeal inflammation

13%

Pruritus

12%

Dysphonia

12%

Gamma-glutamyltransferase increased

11%

Pneumonia

11%

Dehydration

10%

Hyperthyroidism

10%

Hypoalbuminaemia

10%

Hypocalcaemia

10%

Headache

10%

Productive cough

Neck pain

Peripheral sensory neuropathy

Gastrooesophageal reflux disease

Hiccups

Hyperglycaemia

Hyperuricaemia

Dizziness

Hypophosphataemia

Urinary tract infection

Ear pain

Localised oedema

Hyperkalaemia

Erythema

Oral pain

Abdominal pain upper

Arthralgia

Anxiety

Febrile neutropenia

Dyspepsia

Saliva altered

Back pain

Oedema peripheral

Hypertension

Dyspnoea

Nasopharyngitis

Alopecia

Dry skin

Sepsis

Pneumonia aspiration

Trismus

Pneumonitis

Laryngeal oedema

Malnutrition

Pharyngeal haemorrhage

Cellulitis

Septic shock

Systemic infection

Clostridium difficile colitis

Cardiac arrest

Death

Bronchitis

Hepatitis

Immune-mediated hepatitis

Oesophagitis

General physical health deterioration

Hypophagia

Tumour haemorrhage

Cerebrovascular accident

Syncope

Acute respiratory failure

Aspiration

Colitis

Mouth haemorrhage

Hypersensitivity

Acute myocardial infarction

Abscess neck

Device related infection

Stoma site infection

Vascular device infection

Wound infection

Hypercalcaemia

Pulmonary embolism

Respiratory failure

100%

80%

60%

40%

20%

Study treatment Arm

Pembrolizumab + CRT Followed by Pembrolizumab

Placebo + CRT Followed by Placebo

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (pembrolizumab)Experimental Treatment1 Intervention

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pembrolizumab

2017

Completed Phase 3

~3150

0 / 13Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Leukoplakia, particularly those involving monoclonal antibodies like Pembrolizumab, work by modulating the immune system. Pembrolizumab targets the PD-1 pathway, a checkpoint that regulates immune responses. By inhibiting this pathway, Pembrolizumab enhances the immune system's ability to attack abnormal cells, including pre-cancerous cells found in Leukoplakia. This mechanism is crucial for Leukoplakia patients as it helps prevent the progression of these lesions into cancer, offering a targeted and potentially more effective treatment option.

Review Article: Immune Landscape and Immunotherapy Options in Cervical Carcinoma.Novel immune-modulating drugs for advanced head and neck cancer.Immunotherapy of head and neck cancer: current and future considerations.