Clinical trial · Interventional
Pioglitazone Hydrochloride in Preventing Head and Neck Cancer in Patients With Oral Leukoplakia
A Phase IIa Cancer Prevention Trial of the PPAR Gamma Agonist Pioglitazone in Oral Leukoplakia
NCT00099021CI-TRIAL-00056481completedPhase 2Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Head and Neck Cancer | Malignant Head and Neck Neoplasm | ALIAS | 0.90 |
| Oral Leukoplakia | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| pioglitazone hydrochloride | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Prevention (pioglitazone hydrochloride)
- description
- Patients receive pioglitazone hydrochloride PO QD for 12 weeks in the absence of disease progression, unacceptable toxicity, or the development of carcinoma.
- interventionNames
- Drug: pioglitazone hydrochloride
Primary outcomes (1)
- measure
- Patients' Overall Response
- timeFrame
- Week 16 (4 weeks post dose)
- 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
Secondary outcomes (2)
- measure
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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
References
Publications (2)
- DERIVEDGates 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. PMID 39584361
- DERIVEDPuri A, Wuertz B, Rhodus NL, Ondrey FG. Safety of oral mucosal punch biopsy and other oral biospecimen collections in clinical research. Oral Surg Oral Med Oral Pathol Oral Radiol. 2025 Mar;139(3):344-351. doi: 10.1016/j.oooo.2024.10.084. Epub 2024 Oct 30. PMID 39532605