Trial Outcomes & Findings for Pioglitazone Hydrochloride in Preventing Head and Neck Cancer in Patients With Oral Leukoplakia (NCT NCT00099021)

Last Updated: 2022-02-11

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

21 participants

Primary outcome timeframe

Week 16 (4 weeks post dose)

Results posted on

2022-02-11

Participant Flow

Participant milestones

Participant milestones
Measure	Pioglitazone Patients Patients with measurable leukoplakia who received all study medication (oral pioglitazone once daily for 12 weeks) and completed the trial.
Overall Study STARTED	21
Overall Study COMPLETED	21
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Pioglitazone Patients n=21 Participants Patients with measurable leukoplakia who received all study medication (oral pioglitazone once daily for 12 weeks) and completed the trial.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	19 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants
Age, Continuous	56.1 years n=5 Participants
Sex: Female, Male Female	12 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants
Region of Enrollment United States	21 participants n=5 Participants

PRIMARY outcome

Timeframe: Week 16 (4 weeks post dose)

Outcome measures

Outcome measures
Measure	Pioglitazone Patients n=21 Participants Patients with measurable leukoplakia who received all study medication (oral pioglitazone once daily for 12 weeks) and completed the trial.
Patients' Overall Response Complete Response	0 Participants
Patients' Overall Response Partial Response	15 Participants
Patients' Overall Response Stable Disease	2 Participants
Patients' Overall Response Progressive Disease	4 Participants
Patients' Overall Response Partial + Complete Response	15 Participants

SECONDARY outcome

Timeframe: Week 16 (4 weeks post dose)

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

Outcome measures

Outcome measures
Measure	Pioglitazone Patients n=21 Participants Patients with measurable leukoplakia who received all study medication (oral pioglitazone once daily for 12 weeks) and completed the trial.
Patients' Clinical Response Complete Response	3 Participants
Patients' Clinical Response Partial Response	14 Participants
Patients' Clinical Response Stable Disease	4 Participants
Patients' Clinical Response Progressive Disease	0 Participants
Patients' Clinical Response Partial + Complete	17 Participants

SECONDARY outcome

Timeframe: Week 16 (4 weeks post dose)

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.