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A Phase IIa Cancer Prevention Trial of the PPAR Gamma Agonist Pioglitazone in Oral Leukoplakia

Phase 2
18 Years
Not Enrolling
Head and Neck Cancer, Oral Leukoplakia

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Trial Information

A Phase IIa Cancer Prevention Trial of the PPAR Gamma Agonist Pioglitazone in Oral Leukoplakia


I. Determine whether pioglitazone (pioglitazone hydrochloride) reverses leukoplakia in
patients with hyperplastic or dysplastic oral cavity or oropharyngeal leukoplakia.


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

OUTLINE: This is an open-label study.

Patients receive pioglitazone hydrochloride orally (PO) once daily (QD) for 12 weeks in the
absence of disease progression, unacceptable toxicity, or the development of carcinoma.

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

Inclusion Criteria


- ECOG 0-2

- Diagnosis of oral cavity or oropharyngeal leukoplakia meeting 1 of the following

- 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

- Able to be assessed by bi-directional measurements

- Life expectancy: More than 3 months

- Hemoglobin >= lower limit of normal for males and post-menopausal females OR

- Hemoglobin >= 11 g/dL for premenopausal females

- WBC > 3,000/mm^3

- Hepatic: Bilirubin < 1.5 times upper limit of normal (ULN); AST and ALT < 1.5 times

- Renal: BUN < 1.5 times ULN; Creatinine < 1.5 times ULN

- 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

- More than 3 months since prior biologic or immunologic therapy

- No concurrent insulin for diabetes

- No prior radiotherapy to the oral cavity

- 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

- Platelet count > 125,000/mm^3

- Index lesion must be located in an anatomic site accessible by punch biopsy

Type of Study:


Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Prevention

Outcome Measure:

Patients' Overall Response

Outcome Description:

Overall Response= reviewing both the clinical and histological responses and assigning the worst category. Complete Response (CR) = Clinical CR and Histologic CR, or Histologic CR Partial Response (PR) = Clinical CR or PR and Histologic PR or Stable Disease (SD) Stable Disease (SD) = Clinical SD and Histologic PR or SD Progressive Disease (PD) = Clinical PD and/or Histologic PD

Outcome Time Frame:

Week 16 (4 weeks post dose)

Safety Issue:


Principal Investigator

Frank Ondrey

Investigator Role:

Principal Investigator

Investigator Affiliation:

University of Minnesota Medical Center-Fairview


United States: Institutional Review Board

Study ID:




Start Date:

June 2003

Completion Date:

Related Keywords:

  • Head and Neck Cancer
  • Oral Leukoplakia
  • Head and Neck Neoplasms
  • Leukoplakia
  • Leukoplakia, Oral



University of Minnesota Medical Center-Fairview Minneapolis, Minnesota  55455