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

NCT ID: NCT00099021

Last Updated: 2022-02-11

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2003-06-30
• Study Completion Date: 2008-06-27

Brief Summary

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.

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.

Conditions

Head and Neck Cancer; Oral Leukoplakia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

pioglitazone hydrochloride

Intervention Type: DRUG

Given PO

Interventions

pioglitazone hydrochloride

Given PO

Intervention Type: DRUG

Other Intervention Names

Actos; pioglitazone

Eligibility Criteria

Inclusion Criteria

Criteria:

• 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Cancer Institute (NCI)

NIH

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Frank Ondrey

Role: PRINCIPAL_INVESTIGATOR

University of Minnesota Medical Center-Fairview

Locations

University of Minnesota Medical Center-Fairview

Minneapolis, Minnesota, United States

Countries

United States

References

Gates JC, Abouyared M, Shnayder Y, Farwell DG, Day A, Alawi F, Moore M, Holcomb AJ, Birkeland A, Epstein J. Clinical Management Update of Oral Leukoplakia: A Review From the American Head and Neck Society Cancer Prevention Service. Head Neck. 2025 Feb;47(2):733-741. doi: 10.1002/hed.28013. Epub 2024 Nov 25.

Reference Type: DERIVED

PMID: 39584361 (View on PubMed)

Other Identifiers

NCI-2009-00862

Identifier Type: REGISTRY

Identifier Source: secondary_id

CDR0000393562

Identifier Type: -

Identifier Source: secondary_id

2001LS068

Identifier Type: -

Identifier Source: secondary_id

0109 M 07254

Identifier Type: OTHER

Identifier Source: secondary_id

N01-CN-15000

Identifier Type: OTHER

Identifier Source: secondary_id

N01CN15000

Identifier Type: OTHER

Identifier Source: secondary_id

NCI-2009-00862

Identifier Type: -

Identifier Source: org_study_id