Pioglitazone Hydrochloride in Preventing Head and Neck Cancer in Patients With Oral Leukoplakia


This phase II trial studies how well pioglitazone hydrochloride works in preventing head and neck cancer in patients who have oral leukoplakia. Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. The use of pioglitazone hydrochloride may be effective in preventing head and neck cancer.

Full Title of Study: “A Phase IIa Cancer Prevention Trial of the PPAR Gamma Agonist Pioglitazone in Oral Leukoplakia”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Prevention
    • Masking: None (Open Label)
  • Study Primary Completion Date: September 2008

Detailed Description

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


  • Drug: pioglitazone hydrochloride
    • Given PO

Arms, Groups and Cohorts

  • Experimental: Prevention (pioglitazone hydrochloride)
    • Patients receive pioglitazone hydrochloride PO QD for 12 weeks in the absence of disease progression, unacceptable toxicity, or the development of carcinoma.

Clinical Trial Outcome Measures

Primary Measures

  • Patients’ Overall Response
    • Time Frame: Week 16 (4 weeks post dose)
    • 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

Secondary Measures

  • Patients’ Clinical Response
    • Time Frame: Week 16 (4 weeks post dose)
    • Determined by measurement of lesions- Complete Response (CR)= disappearance of all lesions, Partial Response (PR)= >or= 50% decrease in sum of lesions, Stable Disease (SD) = does not meet CR,PR or Progressive Disease (PD), and PD= >or= 25% increase in sum of lesions
  • Patients’ Histological (Tissue) Response
    • Time Frame: Week 16 (4 weeks post dose)
    • Determined by biopsy results before and 4 weeks after treatment: Complete Response (CR) =complete reversal of dysplasia or hyperplasia, Partial Response (PR) = >or=50% decrease in sum of lesions, no increase in 1 or more lesions and no new lesion occurs, Stable Disease (SD0 = not CR, PR or Progressive Disease (PD), PD = >or= 25% increase in sum of lesions or new lesion or progression to invasive carcinoma.

Participating in This Clinical Trial


  • ECOG 0-2 – 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 – 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 ULN – 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

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • National Cancer Institute (NCI)
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Frank Ondrey, Principal Investigator, University of Minnesota Medical Center-Fairview

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