Trial · NCT00951379
Pioglitazone for Oral Premalignant Lesions
NCT00951379, Phase 2, Terminated, National Cancer Institute (NCI); results first posted 2016-04-06
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Pioglitazone for Oral Premalignant Lesions. Trials, record NCT00951379. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00951379
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Pioglitazone for Oral Premalignant Lesions
- NCT number
- NCT00951379
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 52 participants, actual
- Lead sponsor
- National Cancer Institute (NCI)
- Conditions
- Oral Leukoplakia
- Interventions
- Pioglitazone hydrochloride, placebo, laboratory biomarker analysis
- First posted
- 2009-08-04
- Last updated
- 2016-04-06
- Results first posted
- 2016-04-06
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Response <Clinical and Histologic Response Defined as 50% or Greater Reduction in the Sum of the Measured Products of Perpendicular Dimensions of the Target Lesion(s) or Improvement in the Degree of Dysplasia or Hyperplasia> | Response assessed at Week 24 ±1 Week | Overall Dichotomized Clinical and Histologic Response defined as 50% or greater reduction in sum of the measured products of perpendicular dimensions of the target lesion(s) or improvement in the degree of dysplasia or hyperplasia where complete (CR) or partial response (PR) in either clinical or histologic outcome assessed according to criteria given recorded as Response or No Response and analyzed as a dichotomous variable. Clinical Response = CR: Disappearance all evidence target \& non-target lesions; PR: \>/=50% reduction in sum products of diameters all target lesion(s). Non-target lesions may not increase \>/=25% in size \& no new lesion. Histologic Response = CR: Complete reversal of dysplasia or hyperplasia to normal epithelium in all biopsied lesions. PR: Improvement of degree of dysplasia or hyperplasia in all biopsied lesion(s) from advanced to early, or from early to normal epithelium in some lesions while other biopsied lesions remain stable. |
| Primary | Dichotomized Histologic Response (HR): Participant Complete or Partial Response Defined as =/>50% Reduction in the Sum of the Measured Products of Perpendicular Dimensions of the Target Lesion(s) or Improvement in the Degree of Dysplasia or Hyperplasia | Response assessed at Week 24 ±1 Week | HR according to criteria \& recorded as Response or No Response, analyzed as dichotomous variable. CR: Complete reversal dysplasia/hyperplasia to normal epithelium in all biopsied lesions. PR: Improvement of degree dysplasia/hyperplasia in all biopsied lesion from advanced to early, or from early to normal epithelium in some lesions while other biopsied lesions remain stable. No Change (NC): No change in degree dysplasia/hyperplasia in all biopsied lesions, anything not CR, PR or PD. Progressive Disease (PD): Any increase in severity histology grade any biopsied lesion. Early premalignant lesion: lesion defined high risk, indicated by presence of one: hyperplasia at high-risk sites (dorsal, lateral or ventral tongue, or floor of mouth) ONLY, or mild dysplasia. Advanced premalignant lesion: lesion with presence of one: moderate dysplasia or severe dysplasia (excluding CIS), erythroplakia with hyperplasia or of any severity of dysplasia. |
| Primary | Dichotomized Clinical Response: Participant Complete or Partial Response Defined as =/>50% Reduction in the Sum of the Measured Products of Perpendicular Dimensions of the Target Lesion(s) or Improvement in the Degree of Dysplasia or Hyperplasia | Response assessed at Week 24 ±1 Week | Clinical Response assessed according to criteria \& recorded as Response or No Response, analyzed as dichotomous variable. Complete Response (CR): Disappearance of all evidence of target AND non-target lesions. Partial Response (PR): greater than or equal to 50% reduction in the sum of the products of diameters of all target lesion(s). Non-target lesions may not increase greater than or equal 25% in size and no new lesion may appear. No Change (NC): No change in the size of the lesion(s) and no new lesions appearing, i.e. anything that is not CR, PR or PD. Progressive Disease (PD): Any increase greater than or equal to 25% in the product of the diameters of any measurable lesions or in the estimated size of non-measurable lesions or the appearance of an unequivocal new lesion. |
| Secondary | Number of Participants With >5.0 mg/L in Level of C-reactive Protein in Plasma | Baseline to end of study, 24 weeks | Degree of change of C-reactive protein (CRP) in plasma serum via blood tests. The longitudinal regression models for analysis of the change in CRP in plasma used, with suitable transformation if necessary to satisfy the model assumptions, with treatment, stage and biomarker value at screening visit as covariates. |
| Secondary | Number of Participants With Level of C-reactive Protein in Plasma Decrease From >5.0 mg/L to <= 5.0 mg/L From Baseline to End of Study | Baseline to end of study, 24 weeks | The longitudinal regression models for analysis of the change in CRP in plasma will be used, with suitable transformation if necessary to satisfy the model assumptions, with treatment, stage and biomarker value at screening visit as covariates. |
| Secondary | Number of Participant With Clinical Response by Baseline Characteristics: Tobacco Use | Up to 26 weeks | Tobacco use summarized by treatment stratified by stage and by group. In addition, the effects of tobacco and alcohol use on the primary endpoint of clinical response assessed. |
| Secondary | Number of Participant With Clinical Response by Baseline Characteristics: Alcohol Use | Up to 26 weeks | Alcohol use will be summarized by treatment stratified by stage and by group. In addition, the effects of tobacco and alcohol use on the primary endpoint of clinical and pathological response assessed using statistical regression models in an exploratory fashion. Heavy drinkers are participants who drank every day; Light drinkers are participants who drank on some days; Non-drinkers are former drinkers or those who never drank alcohol. |
| Secondary | Number of Participants Affected by Adverse Events Assessed Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 (v4.0) | Up to 26 weeks | All adverse events (including serious) and clinical laboratory toxicity summarized affected organ system. Reporting based on the NCI CTCAE v4.0 by treatment, details included in later Adverse Event Module of results. |
| Secondary | Biomarker Measurements at Scheduled Visits: Tissue Levels of Cyclin D1 | Baseline to end of study, 24 weeks | Tissue levels of Cyclin D1 as indirect measures of pharmacological effect assessed by immunohistochemistry (IHC). Tissue levels of biomarkers assessed from the biopsy obtained at the Screening Clinic Visit and Week 24 ± 1 Week, and plasma levels of biomarkers assessed from blood collected at the Baseline Clinic Visit and Week 24 ± 1 Week. Data reported as percentage of cells staining positive, according to nuclear or cytoplasmic compartments. |
| Secondary | Biomarker Measurements at Scheduled Visits: Tissue Levels of Ki-67 | Baseline to end of study, 24 weeks | Tissue levels of Ki-67 for proliferation assessed by immunohistochemistry (IHC). Tissue levels of biomarkers assessed from the biopsy obtained at the Screening Clinic Visit and Week 24 ± 1 Week, and plasma levels of biomarkers assessed from blood collected at the Baseline Clinic Visit and Week 24 ± 1 Week. Data reported as percentage of cells staining positive, according to nuclear or cytoplasmic compartments. |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Overall Response <Clinical and Histologic Response Defined as 50% or Greater Reduction in the Sum of the Measured Products of Perpendicular Dimensions of the Target Lesion(s) or Improvement in the Degree of Dysplasia or Hyperplasia> | Response assessed at Week 24 ±1 Week | All participants who received at least one dose of treatment. One participant in the Pioglitazone arm never received study drug. | participants | NUMBER |
| SECONDARY | Number of Participants With >5.0 mg/L in Level of C-reactive Protein in Plasma | Baseline to end of study, 24 weeks | Pioglitazone's 26 had 24 evaluable specimens at baseline \& 20 evaluable at end of study with 2 of those not available for CRP\>5 baseline measure: Placebo's 25 had 25 evaluable specimens at baseline \& 21 at end of study. Analysis based on specimen viability/availability, no exclusions made for other reasons, participant needed 1/+ dose of treatment. | participants | NUMBER |
| SECONDARY | Number of Participants With Level of C-reactive Protein in Plasma Decrease From >5.0 mg/L to <= 5.0 mg/L From Baseline to End of Study | Baseline to end of study, 24 weeks | Pioglitazone's 26 had 20 evaluable specimens at baseline \& 20 at end of study with 2 of those not available for the CRP\>5 baseline measure \& Placebo's 25 had 25 evaluable at baseline \& 21 evaluable at end of study. Analysis based on specimen viability/availability, no exclusions made for other reasons, participants needed 1/+ dose of treatment. | participants | NUMBER |
| SECONDARY | Number of Participant With Clinical Response by Baseline Characteristics: Tobacco Use | Up to 26 weeks | Analysis includes all participants who received at least one dose of treatment. One participant in the Pioglitazone arm never received study drug. | participants | NUMBER |
| SECONDARY | Number of Participant With Clinical Response by Baseline Characteristics: Alcohol Use | Up to 26 weeks | All participants who received at least one dose of treatment. One participant in the Pioglitazone arm never received study drug. | participants | NUMBER |
| SECONDARY | Number of Participants Affected by Adverse Events Assessed Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 (v4.0) | Up to 26 weeks | Population includes all enrolled participants. | participants | NUMBER |
| SECONDARY | Biomarker Measurements at Scheduled Visits: Tissue Levels of Cyclin D1 | Baseline to end of study, 24 weeks | Two participants in the Pioglitazone arm were not analyzed for Cyclin D1 end of study score. Analysis is based on specimen viability/availability. No exclusions are made for any other reason. | percentage of staining cells positive | MEAN |
| SECONDARY | Biomarker Measurements at Scheduled Visits: Tissue Levels of Ki-67 | Baseline to end of study, 24 weeks | Four participants in the Pioglitazone arm were not analyzed for Ki-67 end of study score. Analysis is based on specimen viability/availability. No exclusions are made for any other reason. | percentage of staining cells positive | MEAN |
| SECONDARY | Biomarker Measurements at Scheduled Visits: Tissue Levels of p21 | Baseline to end of study, 24 weeks | Two participants in the Pioglitazone arm were not analyzed for p21 end of study score. Analysis is based on specimen viability/availability. No exclusions are made for any other reason. | percentage of staining cells positive | MEAN |
| SECONDARY | Biomarker Measurements at Scheduled Visits: Tissue Levels of B-cell Lymphoma 2 (Bcl2) | Baseline to end of study, 24 weeks | Four participants of 25 overall in the Pioglitazone arm were not analyzed for Bcl2 end of study score and one participant in Placebo was not analyzed for Bcl2 baseline score. Analysis is based on specimen viability/availability. No exclusions are made for any other reason. | percentage of staining cells positive | MEAN |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group98
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
Overall Response <Clinical and Histologic Response Defined as 50% or Greater Reduction in the Sum of the Measured Products of Perpendicular Dimensions of the Target Lesion(s) or Improvement in the Degree of Dysplasia or Hyperplasia> | Arm I (Pioglitazone Hydrochloride) | Response | 12 | participants | |
Overall Response <Clinical and Histologic Response Defined as 50% or Greater Reduction in the Sum of the Measured Products of Perpendicular Dimensions of the Target Lesion(s) or Improvement in the Degree of Dysplasia or Hyperplasia> | Arm II (Placebo) | Response | 8 | participants | |
Overall Response <Clinical and Histologic Response Defined as 50% or Greater Reduction in the Sum of the Measured Products of Perpendicular Dimensions of the Target Lesion(s) or Improvement in the Degree of Dysplasia or Hyperplasia> | Arm I (Pioglitazone Hydrochloride) | No Response | 14 | participants | |
Overall Response <Clinical and Histologic Response Defined as 50% or Greater Reduction in the Sum of the Measured Products of Perpendicular Dimensions of the Target Lesion(s) or Improvement in the Degree of Dysplasia or Hyperplasia> | Arm II (Placebo) | No Response | 17 | participants | |
| Number of Participants With >5.0 mg/L in Level of C-reactive Protein in Plasma | Pioglitazone: CRP>5 at Baseline | Yes | 6 | participants | |
| Number of Participants With >5.0 mg/L in Level of C-reactive Protein in Plasma | Pioglitazone: CRP>5 End of Study | Yes | 1 | participants | |
| Number of Participants With >5.0 mg/L in Level of C-reactive Protein in Plasma | Placebo: CRP> 5 Baseline | Yes | 4 | participants | |
| Number of Participants With >5.0 mg/L in Level of C-reactive Protein in Plasma | Placebo: CRP>5 End of Study | Yes | 4 | participants | |
| Number of Participants With >5.0 mg/L in Level of C-reactive Protein in Plasma | Pioglitazone: CRP>5 at Baseline | No | 18 | participants | |
| Number of Participants With >5.0 mg/L in Level of C-reactive Protein in Plasma | Pioglitazone: CRP>5 End of Study | No | 19 | participants |
1 to 10 of 98Source ClinicalTrials.gov
See all 98 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Arm I (Pioglitazone Hydrochloride) | 27 | |
| Overall Study | STARTED | Arm II (Placebo) | 25 | |
| Overall Study | COMPLETED | Arm I (Pioglitazone Hydrochloride) | 21 | |
| Overall Study | COMPLETED | Arm II (Placebo) | 23 | |
| Overall Study | NOT COMPLETED | Arm I (Pioglitazone Hydrochloride) | 6 | |
| Overall Study | NOT COMPLETED | Arm II (Placebo) | 2 |
Source ClinicalTrials.gov
Adverse events228
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | anemia | Blood and lymphatic system disorders | Arm I (Pioglitazone Hydrochloride) | 1 | 27 | 2 |
| other | anemia | Blood and lymphatic system disorders | Arm II (Placebo) | 0 | 25 | 0 |
| other | palpitations | Cardiac disorders | Arm I (Pioglitazone Hydrochloride) | 2 | 27 | 2 |
| other | palpitations | Cardiac disorders | Arm II (Placebo) | 0 | 25 | 0 |
| other | hearing issues | Ear and labyrinth disorders | Arm I (Pioglitazone Hydrochloride) | 0 | 27 | 0 |
| other | hearing issues | Ear and labyrinth disorders | Arm II (Placebo) | 1 | 25 | 1 |
| other | hearing loss, earwax | Ear and labyrinth disorders | Arm I (Pioglitazone Hydrochloride) | 0 | 27 | 0 |
| other | hearing loss, earwax | Ear and labyrinth disorders | Arm II (Placebo) | 1 | 25 | 1 |
| other | bilateral burning eye | Eye disorders | Arm I (Pioglitazone Hydrochloride) | 0 | 27 | 0 |
| other | bilateral burning eye | Eye disorders | Arm II (Placebo) | 1 | 25 | 1 |
1 to 10 of 228Source ClinicalTrials.gov
See all 228 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Arm I (Pioglitazone Hydrochloride) | ||
| Total, all-cause mortality | Arm II (Placebo) | ||
| Total, other adverse events | Arm I (Pioglitazone Hydrochloride) | 22 | 27 |
| Total, other adverse events | Arm II (Placebo) | 24 | 25 |
| Total, serious adverse events | Arm I (Pioglitazone Hydrochloride) | 3 | 27 |
| Total, serious adverse events | Arm II (Placebo) | 1 | 25 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 39584361 | DERIVED |
Source PubMed
Grants3
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years | Source |
|---|---|---|---|---|---|---|
| P30CA016672 | The University of Texas MD Anderson Cancer Center Support Grant (CCSG) | KOONG, ALBERT | UNIVERSITY OF TX MD ANDERSON CAN CTR | NATIONAL CANCER INSTITUTE | 1985 to 2026 | NIH RePORTER |
| N01CN035153 | PHASE 1 AND PHASE 2 CLINICAL TRIALS OF CANCER CHEMOPREVENTIVE AGENTS --261035153 | BAILEY, HOWARD H. | UNIVERSITY OF WISCONSIN - MAD | DIVISION OF CANCER PREVENTION AND CONTROL | 2004 to 2006 | NIH RePORTER |
| N01CN035159 | PHASE I and II CLINICAL TRIALS OF CHEMOPREVENTIVE AGENTS-261035159-261035159 | LIPPMAN, SCOTT M. | UNIVERSITY OF TEXAS M D ANDER | DIVISION OF CANCER PREVENTION AND CONTROL | 2004 to 2006 | NIH RePORTER |
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Arm I (Pioglitazone Hydrochloride) vs Arm II (Placebo): serious adverse events |
Source Trial comparisons
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
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