MedPath

Eflornithine and Sulindac in Preventing Colorectal Cancer in Patients With Colon Polyps

Phase 3
Completed
Conditions
Precancerous Condition
Interventions
Other: placebo
Other: laboratory biomarker analysis
Registration Number
NCT00118365
Lead Sponsor
National Cancer Institute (NCI)
Brief Summary

This randomized phase III trial is studying eflornithine and sulindac to see how well they work compared to a placebo in preventing colorectal cancer in patients with colon polyps. Chemoprevention is the use of certain drugs to keep cancer from forming, growing, or coming back. The use of eflornithine and sulindac may prevent colorectal cancer. It is not yet known whether eflornithine and sulindac are more effective than a placebo in preventing colorectal cancer

Detailed Description

PRIMARY OBJECTIVES:

I. Compare the rate of new adenomatous polyp formation in patients with a history of adenomatous polyps of the colon treated with eflornithine and sulindac vs placebo.

II. Correlate the effects of eflornithine and sulindac on polyamine and prostaglandin content in the flat mucosa with the rate of new adenoma formation in these patients.

III. Compare the rate of side effects in patients treated with these regimens.

OUTLINE: This is a randomized, double-blind, placebo-controlled, multicenter study. Patients are stratified according to participating center and aspirin use (yes vs no).

Patients receive oral double placebo once daily for 4 weeks. Patients who are more than 70% compliant by pill measurement or self reporting are randomized to 1 of 2 treatment arms.

Arm I: Patients receive oral double placebo once daily.

Arm II: Patients receive oral eflornithine (DFMO) and oral sulindac once daily.

In both arms, treatment continues for 36 months in the absence of unacceptable toxicity or the development of an invasive malignancy.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
375
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Arm II (placebo)laboratory biomarker analysisPatients receive oral double placebo once daily. In both arms, treatment continues for 36 months in the absence of unacceptable toxicity or the development of an invasive malignancy.
Arm II (placebo)placeboPatients receive oral double placebo once daily. In both arms, treatment continues for 36 months in the absence of unacceptable toxicity or the development of an invasive malignancy.
Arm I (eflornithine and sulindac)laboratory biomarker analysisPatients receive oral eflornithine (DFMO) and oral sulindac once daily. In both arms, treatment continues for 36 months in the absence of unacceptable toxicity or the development of an invasive malignancy.
Arm I (eflornithine and sulindac)eflornithinePatients receive oral eflornithine (DFMO) and oral sulindac once daily. In both arms, treatment continues for 36 months in the absence of unacceptable toxicity or the development of an invasive malignancy.
Arm I (eflornithine and sulindac)sulindacPatients receive oral eflornithine (DFMO) and oral sulindac once daily. In both arms, treatment continues for 36 months in the absence of unacceptable toxicity or the development of an invasive malignancy.
Primary Outcome Measures
NameTimeMethod
Detection of Any Adenoma at the End of the StudyUp to 36 months

Detection of any adenoma at the end of the study. This analysis is based on the participants who had the end-of-study colonscopy procedure done.

Secondary Outcome Measures
NameTimeMethod
Detection of Any Adenoma at the End of the Study Stratified by Putrescine Response and TreatmentUp to 36 months

Putrescine responder = Putrescine values at 36-month are decreased by \>=30% from baseline Putrescine nonresponder = Putrescine values at 36-month are increased, or decreased by \< 30% from baseline The analysis cohort is based on the participants whose data are available and complete.

Biomarker in Adenoma: CEAAt the end of the study

carcino-embryonic antigen (CEA) is adenocarcinoma tissue marker that is expressed during adenoma formation.

Biomarker in Adenoma: Sialyl-TN (B72.3)At the end of the study

sialyl-Tn (B72.3) is adenocarcinoma tissue marker that is expressed during adenoma formation.

Detection of Any Adenoma at the End of the Study Stratified by Baseline Prostaglandin E2 (PGE2) and TreatmentUp to 36 months

This analysis is based on the participants who had the end-of-study colonscopy procedure done and their baseline PGE2 values are available. The low PGE2 is defined as the values that are below the median PGE2 value in the analysis cohort. The high PGE2 is defined as the values that are above the median PGE2 value in the analysis cohort.

Detection of Any Adenoma at the End of the Study Stratified by Baseline Spermidine-to-spermine Ratio and TreatmentUp 36 months

The low is defined as the ratios that are below the median spermidine-to-spermine ratio in the analysis cohort. The high is defined as the ratios that are above the median spermidine-to-spermine ratio in the analysis cohort.

In the finalized datasaet, the total number of adnoma detected in the placebo group is 55. The descrepancy in the total number of adnoma detected in placebo group between Outcome Measure 1 and this oucome is due to the revolution of the datatset.

The analysis cohort is based on the participants whose data are available and complete.

Baseline Putrescine by ODC GenotypeBaseline

ODC genotype is the genotype of single nucleotide polymorphisms (SNP) in the ornithine decarboxylase (ODC) promoter The analysis cohort is based on the participants whose data are available and complete.

At the End of the Study - Putrescine Response by ODC GenotypeAt the end of the study

Putrescine responder was defined as (tissue putrescine value at baseline - tissue putrescine value at the end of the study)/(tissue putrescine value at baseline) ≥ the threshold. Putrescine non-responder was defined as (tissue putrescine value at baseline - tissue putrescine value at the end of the study)/(tissue putrescine value at baseline) \< the threshold. The thresholds range from 0.25 to 0.45 with an increment of 0.5. The below data are shown for the threshold of 0.30.

ODC genotype is the genotype of single nucleotide polymorphisms (SNP) in the ornithine decarboxylase (ODC) promoter The analysis cohort is based on the participants whose data are available and complete.

Detection of Any Adenoma at the End of the Study Stratified by Baseline Putrescine and TreatmentUp 36 months

The low is defined as the values that are below the median putrescine level in the analysis cohort. The high is defined as the values that are above the median putrescine level in the analysis cohort.

Baseline Spermine by ODC GenotypeBaseline

ODC genotype is the genotype of single nucleotide polymorphisms (SNP) in the ornithine decarboxylase (ODC) promoter The analysis cohort is based on the participants whose data are available and complete.

At the End of the Study - Spermidine Response by ODC GenotypeAt the end of the study

Spermidine responder was defined as (tissue spermidine value at baseline - tissue spermidine value at the end of the study)/(tissue spermidine value at baseline) ≥ the threshold. Spermidine non-responder was defined as (tissue spermidine value at baseline - tissue spermidine value at the end of the study)/(tissue spermidine value at baseline) \< the threshold. The thresholds range from 0.25 to 0.45 with an increment of 0.5. The below data are shown for the threshold of 0.30.

ODC genotype is the genotype of single nucleotide polymorphisms (SNP) in the ornithine decarboxylase (ODC) promoter The analysis cohort is based on the participants whose data are available and complete.

Number of Participants Have Adenoma Recurrence in Each ODC1 Genotytpe by Treatment GroupUp to 36 months

ODC genotype is the genotype of single nucleotide polymorphisms (SNP) in the ornithine decarboxylase (ODC) promoter The analysis cohort is based on the participants whose data are available and complete.

Detection of Any Adenoma at the End of the Study Stratified by Prostaglandin E2 (PGE2) Response and TreatmentUp to 36 months

PGE2 Responder = PGE2 values at 36-month are decreased by \>=30% in PGE2 values from baseline PGE2 nonresponder = PGE2 values at 36-month are increased, or decreased by \< 30% from baseline The analysis cohort is based on the participants whose data are available and complete.

Baseline Spermidine by ODC GenotypeBaseline

ODC genotype is the genotype of single nucleotide polymorphisms (SNP) in the ornithine decarboxylase (ODC) promoter The analysis cohort is based on the participants whose data are available and complete.

Biomarker in Adenoma: ApoptosisAt the end of the study

Apoptosis expression was assessed using cytoplasmic staining. The definitions for the category level for the Apoptosis are: 1. focal (less than 10% cells that are positively stained); 2. less than 50% cells are positively stained; 3. more than 50% cells are positively stained.

Biomarker in Adenoma: Bcl-2At the end of the study, up to 3 years

bcl-2 is the anti-apoptotic protein BCL2

Detection of Any Adenoma at the End of the Study Stratified by Spermidine-to-spermine Ratio Response and TreatmentUp to 36 months

Spermidine-to-spermine ratio responder = ratios at 36-month are decreased by \>=30% from baseline Spermidine-to-spermine ratio nonresponder = ratios at 36-month are increased, or decreased by \< 30% from baseline The analysis cohort is based on the participants whose data are available and complete.

Adverse Events With a Grade of 3 and AboveUp to 36 months

Participants reported at least 1 adverse event with a grade of 3 and above, regardless if the event is defined as serious per protocol or other.

Per protocol, not all grade 3 events are considered as serious events.

At the End of the Study - Spermine Response by ODC GenotypeAt the end of the study

Spermine responder was defined as (tissue spermine value at baseline - tissue spermine value at the end of the study)/(tissue spermine value at baseline) ≥ the threshold. Spermine non-responder was defined as (tissue spermine value at baseline - tissue spermine value at the end of the study)/(tissue spermine value at baseline) \< the threshold. The thresholds range from 0.25 to 0.45 with an increment of 0.5. The below data are shown for the threshold of 0.30.

ODC genotype is the genotype of single nucleotide polymorphisms (SNP) in the ornithine decarboxylase (ODC) promoter The analysis cohort is based on the participants whose data are available and complete.

Biomarker in Adenoma - Ki-67At the end of the study

Estimated mean percent of cells staining postivie for the Ki-67 based on the GEE approach with adjustment for covariates

Biomarker in Adenoma - p53At the end of the study

Estimated mean percent of cells staining postivie for p53 based on GEE approach with adjument for covariates.

Tumor protein p53, also known as p53, cellular tumor antigen p53, phosphoprotein p53, or tumor suppressor p53, is a protein that in humans is encoded by the TP53 gene.

Trial Locations

Locations (1)

University of California Medical Center At Irvine-Orange Campus

🇺🇸

Orange, California, United States

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