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

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ClinicalTrials.gov Identifier: NCT00099021
Recruitment Status : Completed
First Posted : December 9, 2004
Results First Posted : November 25, 2009
Last Update Posted : April 30, 2019
Sponsor:
Information provided by (Responsible Party):
National Cancer Institute (NCI)

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Prevention
Conditions Head and Neck Cancer
Oral Leukoplakia
Intervention Drug: pioglitazone hydrochloride
Enrollment 21
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Pioglitazone Patients
Hide Arm/Group Description Patients with measurable leukoplakia who received all study medication (oral pioglitazone once daily for 12 weeks) and completed the trial.
Period Title: Overall Study
Started 21
Completed 21
Not Completed 0
Arm/Group Title Pioglitazone Patients
Hide Arm/Group Description Patients with measurable leukoplakia who received all study medication (oral pioglitazone once daily for 12 weeks) and completed the trial.
Overall Number of Baseline Participants 21
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants
<=18 years
0
   0.0%
Between 18 and 65 years
19
  90.5%
>=65 years
2
   9.5%
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 21 participants
56.1
(27 to 75)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants
Female
12
  57.1%
Male
9
  42.9%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 21 participants
21
1.Primary Outcome
Title Patients' Overall Response
Hide 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

Time Frame Week 16 (4 weeks post dose)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Pioglitazone Patients
Hide Arm/Group Description:
Patients with measurable leukoplakia who received all study medication (oral pioglitazone once daily for 12 weeks) and completed the trial.
Overall Number of Participants Analyzed 21
Measure Type: Number
Unit of Measure: Participants
Complete Response 0
Partial Response 15
Stable Disease 2
Progressive Disease 4
Partial + Complete Response 15
2.Secondary Outcome
Title Patients' Clinical Response
Hide Description 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
Time Frame Week 16 (4 weeks post dose)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Pioglitazone Patients
Hide Arm/Group Description:
Patients with measurable leukoplakia who received all study medication (oral pioglitazone once daily for 12 weeks) and completed the trial.
Overall Number of Participants Analyzed 21
Measure Type: Number
Unit of Measure: Participants
Complete Response 3
Partial Response 14
Stable Disease 4
Progressive Disease 0
Partial + Complete 17
3.Secondary Outcome
Title Patients' Histological (Tissue) Response
Hide Description 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.
Time Frame Week 16 (4 weeks post dose)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Pioglitazone Patients
Hide Arm/Group Description:
Patients with measurable leukoplakia who received all study medication (oral pioglitazone once daily for 12 weeks) and completed the trial.
Overall Number of Participants Analyzed 21
Measure Type: Number
Unit of Measure: Participants
Complete Response 1
Partial Response 2
Stable Disease 14
Progressive Disease 4
Partial + Complete Response 3
4.Other Pre-specified Outcome
Title Interleukin 6, 8 and Vascular Endothelial Growth Factors Elaboration in the Oral Cavity and Serum
Hide Description Quantitative studies of serum and saliva components for a pre and post treatment possible biomarker.
Time Frame Pre (Day 0) and Post (Week 12) Treatment
Outcome Measure Data Not Reported
5.Other Pre-specified Outcome
Title Quantitative Oil Red O, AP2 (FABP4) and FABP5 Staining
Hide Description Immune histochemistry / tissue staining for a possible biomarker.
Time Frame Pre (Day 0) and Post (Week 12) Treatment
Outcome Measure Data Not Reported
6.Other Pre-specified Outcome
Title Involucrin and Transglutaminase Staining
Hide Description Immune histochemistry / tissue staining for a possible biomarker.
Time Frame Pre (Day 0) and Post (Week 12) Treatment
Outcome Measure Data Not Reported
7.Other Pre-specified Outcome
Title Cyclin D1 and p21 Immune Histochemistry
Hide Description Immune histochemistry / tissue staining for a possible biomarker.
Time Frame Pre (Day 0) and Post (Week 12) Treatment
Outcome Measure Data Not Reported
8.Other Pre-specified Outcome
Title Cyclooxygenase-2 Staining
Hide Description Immune histochemistry / tissue staining for a possible biomarker.
Time Frame Pre (Day 0) and Post (Week 12) Treatment
Outcome Measure Data Not Reported
9.Other Pre-specified Outcome
Title Piogliotazone Gamma Immune Histochemistry
Hide Description Immune histochemistry / tissue staining for a possible biomarker.
Time Frame Pre (Day 0) and Post (Week 12) Treatment
Outcome Measure Data Not Reported
10.Other Pre-specified Outcome
Title Ki 67 Labeling Index
Hide Description Immune histochemistry / tissue staining for a possible biomarker.
Time Frame Pre (Day 0) and Post (Week 12) Treatment
Outcome Measure Data Not Reported
11.Other Pre-specified Outcome
Title Apotosis (Cell Death)
Hide Description Immune histochemistry / tissue staining for a possible biomarker.
Time Frame Pre (Day 0) and Post (Week 12) Treatment
Outcome Measure Data Not Reported
12.Other Pre-specified Outcome
Title Nf Kappa B p65
Hide Description Immune histochemistry / tissue staining for a possible biomarker.
Time Frame Pre (Day 0) and Post (Week 12) Treatment
Outcome Measure Data Not Reported
Time Frame From first dose of Pioglitazone through Week 16 (end of study).
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Pioglitazone Patients
Hide Arm/Group Description Patients with measurable leukoplakia who received all study medication (oral pioglitazone once daily for 12 weeks) and completed the trial.
All-Cause Mortality
Pioglitazone Patients
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Pioglitazone Patients
Affected / at Risk (%) # Events
Total   0/21 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Pioglitazone Patients
Affected / at Risk (%) # Events
Total   18/21 (85.71%)    
Blood and lymphatic system disorders   
Anemia  1  1/21 (4.76%)  1
Bleeding gums, occasional  1  1/21 (4.76%)  1
Peripheral edema * 1  6/21 (28.57%)  7
Puffy eyes * 1  1/21 (4.76%)  1
Cardiac disorders   
Facial edema  1  1/21 (4.76%)  1
Pounding pulse * 1  1/21 (4.76%)  1
Transient elevated blood pressure * 1  1/21 (4.76%)  1
Ear and labyrinth disorders   
Ear pain, right side  1  1/21 (4.76%)  1
Ringing in ears * 1  1/21 (4.76%)  1
Eye disorders   
Stye-inside corner, right eye * 1  1/21 (4.76%)  1
Gastrointestinal disorders   
Abdominal bloating  1  1/21 (4.76%)  1
Black stools  1  1/21 (4.76%)  1
Diarrhea  1  3/21 (14.29%)  3
Dry mouth  1  1/21 (4.76%)  1
Increased heartburn * 1  1/21 (4.76%)  1
Nausea * 1  4/21 (19.05%)  5
Oral canker sores * 1  1/21 (4.76%)  2
Oral cavity/gum pain * 1  2/21 (9.52%)  2
Sore throat  1  1/21 (4.76%)  1
General disorders   
Chest pain  1  1/21 (4.76%)  1
Fatigue  1  3/21 (14.29%)  5
Headache  1  5/21 (23.81%)  5
Insomnia * 1  2/21 (9.52%)  2
Weight loss * 1  1/21 (4.76%)  1
Infections and infestations   
Bronchitis  1  1/21 (4.76%)  1
Left eye crusty, burning, reddened * 1  1/21 (4.76%)  1
Rhinitis * 1  1/21 (4.76%)  1
Strep throat * 1  1/21 (4.76%)  1
Upper respiratory infection * 1  4/21 (19.05%)  4
Urinary tract infection * 1  2/21 (9.52%)  2
Metabolism and nutrition disorders   
AST elevated  1  1/21 (4.76%)  1
Decreased uric acid  1  1/21 (4.76%)  1
Hematuria  1  1/21 (4.76%)  1
Hypercholesterolemia  1  1/21 (4.76%)  1
Hypernatremia  1  1/21 (4.76%)  1
Hyponatremia * 1  1/21 (4.76%)  1
Musculoskeletal and connective tissue disorders   
Back pain  1  2/21 (9.52%)  2
Joint pain * 1  4/21 (19.05%)  5
Muscle stiffness, aches, myalgia * 1  3/21 (14.29%)  5
Weakness * 1  1/21 (4.76%)  1
Nervous system disorders   
Lightheadness, dizzy * 1  3/21 (14.29%)  3
Pain, mild stinging sensation * 1  1/21 (4.76%)  3
Shakey, tremor * 1  1/21 (4.76%)  1
Psychiatric disorders   
Anxiety  1  1/21 (4.76%)  1
Depression  1  1/21 (4.76%)  1
Renal and urinary disorders   
WBCs in urine, asymptomatic * 1  1/21 (4.76%)  1
Reproductive system and breast disorders   
Post menopausal spotting * 1  1/21 (4.76%)  1
Respiratory, thoracic and mediastinal disorders   
Asthma symptoms  1  1/21 (4.76%)  2
Cough  1  2/21 (9.52%)  2
Skin and subcutaneous tissue disorders   
Diaphoresis  1  1/21 (4.76%)  1
Eczema  1  1/21 (4.76%)  3
Groin pain right hernia site (wound)  1  1/21 (4.76%)  1
Nail splitting * 1  1/21 (4.76%)  1
Ulcer at old gallbladder incision site * 1  1/21 (4.76%)  1
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE

Study was closed prior to full accrual because results were very good and funding was discontinued (R-01 application was filed.)

Immunohistochemistry (Outcomes #4 through #12) were not done due to laboratory limitations of time.

Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Frank G. Ondrey, M.D.
Organization: Masonic Cancer Center, University of Minnesota
Phone: 612-625-3200
EMail: ondre002@umn.edu
Layout table for additonal information
Responsible Party: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT00099021     History of Changes
Other Study ID Numbers: NCI-2009-00862
NCI-2009-00862 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
CDR0000393562
2001LS068
0109 M 07254 ( Other Identifier: University of Minnesota Medical Center-Fairview )
N01-CN-15000 ( Other Identifier: DCP )
N01CN15000 ( Other Identifier: US NIH Grant/Contract Award Number )
First Submitted: December 8, 2004
First Posted: December 9, 2004
Results First Submitted: October 13, 2009
Results First Posted: November 25, 2009
Last Update Posted: April 30, 2019