Alternate Title
Basic Trial Information
Objectives
Entry Criteria
Expected Enrollment
Outcomes
Outline
Trial Contact Information
Related Information
Registry Information

Alternate Title

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

Basic Trial Information

Phase Type Status Age Sponsor Protocol IDs Phase II Prevention Closed 18 and over NCI UMN-0109M07254 UMN-2001LS068, N01-CN-15000, NCT00099021

Special Category: NCI Web site featured trial

Objectives

Primary

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

Secondary

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

Entry 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

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

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/mm³
• Platelet count > 125,000/mm³

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

Expected Enrollment

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

Outcomes

Effects on reversal of leukoplakia

Safety and tolerability

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.

Trial Contact Information

Trial Lead Organizations

Masonic Cancer Center at University of Minnesota

Frank Ondrey, MD, PhD, Principal investigator		Ph: 612-625-3200; 888-226-2376 Email: ondre002@tc.umn.edu

Related Information

Featured trial article

Registry Information
Official Title		A Phase IIA Cancer Prevention Trial of PPAR Gamma Agonist Pioglitazone in Oral Leukoplakia
Trial Start Date		2003-06-04
Trial Completion Date		2007-08-31
Registered in ClinicalTrials.gov		NCT00099021
Date Submitted to PDQ		2004-09-27
Information Last Verified		2006-12-03
NCI Grant/Contract Number		CA77598, CN15000