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Study of Pembrolizumab (MK-3475) for High Risk Oral Intra-Epithelial Neoplasias

This study is currently recruiting participants.
Verified December 2017 by M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier:
First Posted: August 29, 2016
Last Update Posted: December 6, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Merck Sharp & Dohme Corp.
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

The goal of this clinical research study is to compare pembrolizumab to standard of care observation (no treatment) in controlling oral pre-malignant lesions and IENs. The safety and tolerability of pembrolizumab will also be studied.

This is an investigational study. Pembrolizumab is FDA approved and commercially available for the treatment of certain types of melanoma and non-small cell lung cancer (NSCLC). It is currently being used for research purposes in head and neck cancer. The study doctor can explain how the study drug is designed to work.

Up to 250 people will be enrolled in this study. All will take part at MD Anderson.

Condition Intervention Phase
Oral Pre-malignant Lesion(s) Intra-epithelial Neoplasias History of Invasive Oral Cancer Drug: Pembrolizumab Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Personalized, Randomized, Phase 2 Study of Pembrolizumab (MK-3475) for High Risk Oral Intra-Epithelial Neoplasias

Resource links provided by NLM:

Further study details as provided by M.D. Anderson Cancer Center:

Primary Outcome Measures:
  • Oral Cancer-Free Survival of Patients with High Risk Oral Intra-Epithelial Neoplasias (IEN) Treated with Pembrolizumab Versus Observation [ Time Frame: 7 years ]
    Oral cancer-free survival defined as time from randomization to the development of histologically confirmed oral cancer or death of any cause, whichever occurs first.

Estimated Enrollment: 250
Actual Study Start Date: June 14, 2017
Estimated Study Completion Date: June 2024
Estimated Primary Completion Date: June 2024 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LOH Pembrolizumab Group

Loss of Heterozygosity (LOH) positive group.

Pembrolizumab by vein on Day 1 of Cycles 1-4.

Drug: Pembrolizumab
200 mg by vein on Day 1 of a 21 day cycle for 4 cycles.
Other Names:
  • Keytruda
  • MK-3475
  • SCH-900475
No Intervention: LOH Observational Group

Loss of Heterozygosity (LOH) positive group.

Participants only observed during course of study.

  Show Detailed Description


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes

Inclusion Criteria:

  1. Histological evidence of oral intra-epithelial neoplasia within 12 months prior to enrollment. Subjects with a history or clinical diagnosis suggestive of oral intra-epithelial neoplasia, or patients with a history of invasive oral cancer are eligible, but must have a confirmed histological diagnosis of oral intra-epithelial neoplasia before randomization. Histological evidence of oral intraepithelial neoplasia on an invasive oral cancer resection specimen is acceptable. A visible, measurable, clinical lesion (such as leukoplakia and/or erythroplakia) is not required. Only individuals with high risk profiles will be considered eligible for randomization. High risk profiles are defined as patients without a prior oral cancer and have LOH at 3p14 and/or 9p21 plus at least at one additional chromosomal site (4q,8p,11p,13q, or 17p) or patients with a prior oral cancer history and have LOH at 3p14 and/or 9p21. All high risk patients must also meet the additional eligibility criteria (2-9).
  2. Be willing and able to provide written informed consent.
  3. Be >/= 18 years of age on day of signing informed consent for the trial.
  4. Be willing to provide tissue from a newly obtained oral biopsy.
  5. Have a performance status of 0-2 on the ECOG Performance Scale.
  6. Demonstrate adequate organ function as defined: Hematological: Absolute Neutrophil Count >/=1,500/mcL, Platelets >/= 75,000/mcL. Hepatic: Serum total bilirubin </= 1.5 X ULN or Direct Bilirubin </= ULN for subjects with total bilirubin levels > 1.5 ULN. AST (SGOT) and ALT (SGPT) </=2.5 X ULN
  7. Female subject of childbearing potential should have a negative urine or serum pregnancy test < 72 hours 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.
  8. 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 study therapy 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 > 1 year.
  9. 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 the study therapy.

Exclusion Criteria:

  1. Is currently participating and receiving study therapy with potential anti-neoplastic activity, or has participated in a study of an investigational agent and received study therapy with potential anti-neoplastic activity within 4 weeks of the first dose of treatment.
  2. Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to the first dose of trial treatment.
  3. Has a known history of active TB (Bacillus Tuberculosis).
  4. Hypersensitivity to pembrolizumab or any of its excipients.
  5. 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 2 at baseline) from adverse events due to agents administered more than 4 weeks earlier.
  6. 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 2 or at baseline) from adverse events due to a previously administered agent. Note: If the subject received major surgery, they must have recovered adequately from the toxicity and/or complications from the intervention prior to starting therapy.
  7. Has a known additional malignancy that is progressing or requires active treatment other than adjuvant hormonal therapy. Exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin or in situ cervical cancer.
  8. 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.
  9. Has a known history of, or any evidence of active, non-infectious pneumonitis.
  10. Has an active infection requiring systemic therapy.
  11. 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.
  12. Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
  13. Is pregnant, or breastfeeding, or expecting to conceive or father children within the projected duration of treatment with pembrolizumab, starting with the pre-screening or screening visit through 120 days after the last dose of trial treatment.
  14. Has received prior therapy with anti-PD-1, anti-PD-L1, or anti-PD-L2 agent.
  15. Has a history of Human Immunodeficiency Virus (HIV) (HIV 1/2 antibodies).
  16. Has known active Hepatitis B (e.g., HBsAg reactive) or Hepatitis C (e.g., HCV RNA [qualitative] is detected).
  17. 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 are live attenuated vaccines, and are not allowed.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02882282

Contact: Renata Ferrarotto, MD 713-792-6363 CR_Study_Registration@mdanderson.org

United States, Texas
University of Texas MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Contact: Clinical Research Operations       CR_Study_Registration@mdanderson.org   
Sponsors and Collaborators
M.D. Anderson Cancer Center
Merck Sharp & Dohme Corp.
Principal Investigator: Renata Ferrarotto, MD M.D. Anderson Cancer Center
  More Information

Additional Information:
Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT02882282     History of Changes
Other Study ID Numbers: 2016-0193
NCI-2017-00479 ( Registry Identifier: NCI CTRP )
First Submitted: August 24, 2016
First Posted: August 29, 2016
Last Update Posted: December 6, 2017
Last Verified: December 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by M.D. Anderson Cancer Center:
Diseases of oral cavity salivary glands and jaws
High risk oral intra-epithelial neoplasias
Oral pre-malignant lesion(s)
History of invasive oral cancer
Observation only

Additional relevant MeSH terms:
Mouth Neoplasms
Precancerous Conditions
Carcinoma in Situ
Head and Neck Neoplasms
Neoplasms by Site
Mouth Diseases
Stomatognathic Diseases
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Antineoplastic Agents