RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. The use of pioglitazone may be effective in preventing head and neck cancer.

PURPOSE: This phase II trial is studying the effectiveness of pioglitazone in preventing head and neck cancer in patients who have oral leukoplakia.

Condition	Treatment or Intervention	Phase
lip and oral cavity cancer Oropharyngeal Cancer Oral Leukoplakia	Drug: pioglitazone  Procedure: cancer prevention intervention  Procedure: chemoprevention of cancer	Phase II

Further Study Details: 

OBJECTIVES: Primary

• Determine whether pioglitazone reverses leukoplakia in patients with hyperplastic or dysplastic oral cavity or oropharyngeal leukoplakia.

Secondary

• Determine the safety and tolerability of this drug in these patients.

OUTLINE: This is an open-label study.

Patients receive oral pioglitazone once daily for 12 weeks in the absence of disease progression, unacceptable toxicity, or the development of carcinoma.

Patients are followed at 4, 8, 12, and 16 weeks.

PROJECTED ACCRUAL: A total of 13-33 patients will be accrued for this study within 2 years.

Ages Eligible for Study:  18 Years and above,  Genders Eligible for Study:  Both

Criteria

DISEASE CHARACTERISTICS:

• Diagnosis of oral cavity or oropharyngeal leukoplakia meeting 1 of the following criteria:
• Biopsy-proven hyperplasia in high-risk anatomic areas (e.g., floor of the mouth, mobile tongue, oropharynx, or in any erythroplakia lesion)
• Mild, moderate, or severe dysplasia at any site of the oral cavity or oropharynx within the lesion
• Measurable lesion that is clinically characterized by leukoplakia, erythroplakia, or erythroleukoplakia
• Index lesion must be located in an anatomic site accessible by punch biopsy
• Able to be assessed by bi-directional measurements

PATIENT CHARACTERISTICS: Age

• 18 and over

Performance status

• ECOG 0-2

Life expectancy

• More than 3 months

Hematopoietic

• Hemoglobin ≥ lower limit of normal for males and post-menopausal females OR
• Hemoglobin ≥ 11 g/dL for premenopausal females
• WBC > 3,000/mm^3
• Platelet count > 125,000/mm^3

Hepatic

• Bilirubin < 1.5 times upper limit of normal (ULN)
• AST and ALT < 1.5 times ULN

Renal

• BUN < 1.5 times ULN
• Creatinine < 1.5 times ULN

Other

• Not pregnant or nursing
• Negative pregnancy test
• Fertile patients must use effective barrier contraception
• No contraindication to thiazolidinediones
• No allergy to pioglitazone or other thiazolidinediones
• No serious oral infection
• No invasive carcinoma within the past 60 months except nonmelanoma skin cancer or carcinoma in situ of the cervix
• No concurrent malignancy

PRIOR CONCURRENT THERAPY: Biologic therapy

• More than 3 months since prior biologic or immunologic therapy

Chemotherapy

• Not specified

Endocrine therapy

• No concurrent insulin for diabetes

Radiotherapy

• No prior radiotherapy to the oral cavity

Surgery

• Not specified

Other

• More than 3 months since prior chemopreventative agents
• More than 3 months since prior experimental therapy
• More than 3 months since prior megadose vitamins or alternative therapy
• No prior thiazolidinediones
• No prior participation in this study
• No concurrent pharmacologic treatment for diabetes
• Concurrent chronic use of non-steroidal anti-inflammatory drugs allowed

Minnesota
      University of Minnesota Cancer Center, Minneapolis,  Minnesota,  55455,  United States; Recruiting

Study chairs or principal investigators

Frank G. Ondrey, MD, PhD,  Principal Investigator,  University of Minnesota Cancer Center   

Study ID Numbers:  CDR0000393562; UMN-0109M07254; UMN-2001LS068; NCT00099021
Record last reviewed:  February 2005
Last Updated:  March 3, 2005
Record first received:  December 8, 2004
ClinicalTrials.gov Identifier:  NCT00099021
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2005-04-08