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A Clinical Trial of COX and EGFR Inhibition in Familial Polyposis Patients (FAPEST)

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ClinicalTrials.gov Identifier: NCT01187901
Recruitment Status : Completed
First Posted : August 24, 2010
Results First Posted : June 17, 2016
Last Update Posted : June 17, 2016
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Jewel Samadder, University of Utah

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Prevention
Condition Adenomatous Polyposis Coli
Interventions Drug: Erlotinib
Drug: Sulindac
Drug: Placebo A
Drug: Placebo B
Enrollment 92

Recruitment Details  
Pre-assignment Details  
Arm/Group Title Sulindac-erlotinib Placebo
Hide Arm/Group Description Sulindac 150 mg twice daily in combination with erlotinib 75 mg per day for 6 months Placebo capsules matching erlotinib active comparator (Placebo A) once daily and placebo capsules matching sulindac active comparator (Placebo B) twice daily for 6 months
Period Title: Overall Study
Started 46 46
Completed 37 36
Not Completed 9 10
Reason Not Completed
Protocol Violation             1             0
Lost to Follow-up             1             2
Unrelated Health Reasons             1             2
Suspected Allergic Reaction             1             1
Adverse Event             3             0
No Endpoint due to Early Study Halt             2             3
Pregnancy             0             2
Arm/Group Title Sulindac-erlotinib Placebo Total
Hide Arm/Group Description Sulindac 150 mg twice daily in combination with erlotinib 75 mg per day for 6 months Placebo capsules matching erlotinib active comparator (Placebo A) once daily and placebo capsules matching sulindac active comparator (Placebo B) twice daily for 6 months Total of all reporting groups
Overall Number of Baseline Participants 46 46 92
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 46 participants 46 participants 92 participants
42  (14) 41  (14) 41  (14)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 46 participants 46 participants 92 participants
Female
28
  60.9%
28
  60.9%
56
  60.9%
Male
18
  39.1%
18
  39.1%
36
  39.1%
Familial Adenomatous Polyposis (FAP) Status  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 46 participants 46 participants 92 participants
Classic FAP 32 32 64
Attenuated FAP 14 14 28
1.Primary Outcome
Title Change in Duodenal Polyp Burden From Baseline to 6 Months
Hide Description A comparison between the Sulindac-erlotinib and Placebo arms of the change in polyp burden from a 10-centimeter segment of the duodenum, measured as the sum of the diameters of the polyps, in millimeters (mm), from the duodenal segment (6-month polyp burden minus baseline polyp burden).
Time Frame Baseline and 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Sulindac-erlotinib Placebo
Hide Arm/Group Description:
Sulindac 150 mg twice daily in combination with erlotinib 75 mg per day for 6 months
Placebo capsules matching erlotinib active comparator (Placebo A) once daily and placebo capsules matching sulindac active comparator (Placebo B) twice daily for 6 months
Overall Number of Participants Analyzed 46 46
Median (Inter-Quartile Range)
Unit of Measure: mm
-8.5
(-9.5 to -7.0)
8.0
(5.0 to 9.5)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Sulindac-erlotinib
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
2.Secondary Outcome
Title Change in Duodenal Polyp Burden From Baseline to 6 Months in Classic Familial Adenomatous Polyposis (FAP) Participants
Hide Description A comparison between the Sulindac-erlotinib and Placebo arm Classic FAP subgroups of the change in polyp burden from a 10-centimeter segment of the duodenum, measured as the sum of the diameters of the polyps, in millimeters (mm), from the duodenal segment (6-month polyp burden minus baseline polyp burden).
Time Frame Baseline and 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Classic FAP participants are defined as those presenting with more than 100 colonic adenomas and either (1) multiple family members with a classic FAP phenotype or (2) an adenomatous polyposis coli (APC) mutation in a region of the gene known to correlate with Classic FAP, or (3) both.
Arm/Group Title Sulindac-erlotinib Placebo
Hide Arm/Group Description:
Sulindac 150 mg twice daily in combination with erlotinib 75 mg per day for 6 months
Placebo capsules matching erlotinib active comparator (Placebo A) once daily and placebo capsules matching sulindac active comparator (Placebo B) twice daily for 6 months
Overall Number of Participants Analyzed 32 32
Median (Inter-Quartile Range)
Unit of Measure: mm
-8.5
(-9.5 to -7.3)
8.5
(4.5 to 11.3)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Sulindac-erlotinib, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
3.Secondary Outcome
Title Change in Duodenal Polyp Burden From Baseline to 6 Months in Attenuated FAP Participants
Hide Description A comparison between the Sulindac-erlotinib and Placebo arm Attenuated FAP subgroups of the change in polyp burden from a 10-centimeter segment of the duodenum, measured as the sum of the diameters of the polyps, in millimeters (mm), from the duodenal segment (6-month polyp burden minus baseline polyp burden).
Time Frame Baseline and 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Attenuated FAP participants are defined with the presence of a mutation in a portion of the adenomatous polyposis coli (APC) gene known to correlate with attenuated FAP and presentation of a milder phenotype in terms of polyp density in the participant and the family. All participants with attenuated FAP had a confirmed mutation in the APC gene.
Arm/Group Title Sulindac-erlotinib Placebo
Hide Arm/Group Description:
Sulindac 150 mg twice daily in combination with erlotinib 75 mg per day for 6 months
Placebo capsules matching erlotinib active comparator (Placebo A) once daily and placebo capsules matching sulindac active comparator (Placebo B) twice daily for 6 months
Overall Number of Participants Analyzed 14 14
Median (Inter-Quartile Range)
Unit of Measure: mm
-8.0
(-9.5 to -5.5)
7.0
(5.0 to 9.5)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Sulindac-erlotinib, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
4.Secondary Outcome
Title Change in Number of Duodenal Polyps From Baseline to 6 Months
Hide Description A comparison between the Sulindac-erlotinib and Placebo arms of the change in number of polyps in a 10-centimeter segment of the duodenum (6-month polyp count minus baseline polyp count).
Time Frame Baseline and 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Sulindac-erlotinib Placebo
Hide Arm/Group Description:
Sulindac 150 mg twice daily in combination with erlotinib 75 mg per day for 6 months
Placebo capsules matching erlotinib active comparator (Placebo A) once daily and placebo capsules matching sulindac active comparator (Placebo B) twice daily for 6 months
Overall Number of Participants Analyzed 46 46
Median (Inter-Quartile Range)
Unit of Measure: polyps
-2.8
(-4.0 to -1.5)
4.3
(3.1 to 5.5)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Sulindac-erlotinib, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
5.Secondary Outcome
Title Change in Number of Duodenal Polyps From Baseline to 6 Months in Classic FAP Participants
Hide Description A comparison between the Sulindac-erlotinib and Placebo arm Classic FAP subgroups of the change in number of polyps in a 10-centimeter segment of the duodenum (6-month polyp count minus baseline polyp count)
Time Frame Baseline and 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Classic FAP participants are defined as those presenting with more than 100 colonic adenomas and either (1) multiple family members with a classic FAP phenotype or (2) an adenomatous polyposis coli (APC) mutation in a region of the gene known to correlate with Classic FAP, or (3) both
Arm/Group Title Sulindac-erlotinib Placebo
Hide Arm/Group Description:
Sulindac 150 mg twice daily in combination with erlotinib 75 mg per day for 6 months
Placebo capsules matching erlotinib active comparator (Placebo A) once daily and placebo capsules matching sulindac active comparator (Placebo B) twice daily for 6 months
Overall Number of Participants Analyzed 32 32
Median (Inter-Quartile Range)
Unit of Measure: polyps
-2.1
(-4.0 to -0.5)
4.0
(2.5 to 5.6)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Sulindac-erlotinib, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
6.Secondary Outcome
Title Change in Number of Duodenal Polyps From Baseline to 6 Months in Attenuated FAP Participants
Hide Description A comparison between the Sulindac-erlotinib and Placebo arm Attenuated FAP subgroups of the change in number of polyps in a 10-centimeter segment of the duodenum (6-month polyp count minus baseline polyp count)
Time Frame Baseline and 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Attenuated FAP participants are defined with the presence of a mutation in a portion of the adenomatous polyposis coli (APC) gene known to correlate with attenuated FAP and presentation of a milder phenotype in terms of polyp density in the participant and the family. All participants with attenuated FAP had a confirmed mutation in the APC gene.
Arm/Group Title Sulindac-erlotinib Placebo
Hide Arm/Group Description:
Sulindac 150 mg twice daily in combination with erlotinib 75 mg per day for 6 months
Placebo capsules matching erlotinib active comparator (Placebo A) once daily and placebo capsules matching sulindac active comparator (Placebo B) twice daily for 6 months
Overall Number of Participants Analyzed 14 14
Median (Inter-Quartile Range)
Unit of Measure: polyps
-4.3
(-6.0 to -2.5)
4.9
(3.6 to 6.0)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Sulindac-erlotinib, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Time Frame 6 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Sulindac-erlotinib Placebo
Hide Arm/Group Description Sulindac 150 mg twice daily in combination with erlotinib 75 mg per day for 6 months Placebo capsules matching erlotinib active comparator (Placebo A) once daily and placebo capsules matching sulindac active comparator (Placebo B) twice daily for 6 months
All-Cause Mortality
Sulindac-erlotinib Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Sulindac-erlotinib Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/46 (0.00%)   0/46 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Sulindac-erlotinib Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   43/46 (93.48%)   33/46 (71.74%) 
Eye disorders     
Dry Eye  1  9/46 (19.57%)  1/46 (2.17%) 
Gastrointestinal disorders     
Oral Mucositis  1  18/46 (39.13%)  5/46 (10.87%) 
Diarrhea  1  12/46 (26.09%)  6/46 (13.04%) 
Nausea  1  11/46 (23.91%)  6/46 (13.04%) 
Abdominal Pain  1  4/46 (8.70%)  8/46 (17.39%) 
Dyspepsia  1  4/46 (8.70%)  2/46 (4.35%) 
Lower Gastrointestinal Hemorrhage  1  3/46 (6.52%)  0/46 (0.00%) 
General disorders     
Fatigue  1  5/46 (10.87%)  5/46 (10.87%) 
Investigations     
Aminotransferase Increase  1  3/46 (6.52%)  4/46 (8.70%) 
Musculoskeletal and connective tissue disorders     
Pain in Extremity  1  4/46 (8.70%)  10/46 (21.74%) 
Nervous system disorders     
Headache  1  4/46 (8.70%)  8/46 (17.39%) 
Skin and subcutaneous tissue disorders     
Rash Acneiform  1  40/46 (86.96%)  9/46 (19.57%) 
Dry Skin  1  7/46 (15.22%)  7/46 (15.22%) 
Alopecia  1  5/46 (10.87%)  1/46 (2.17%) 
Vascular disorders     
Hypertension  1  3/46 (6.52%)  0/46 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (4.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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
Name/Title: N. Jewel Samadder
Organization: University of Utah
Phone: 801-213-4211
Responsible Party: Jewel Samadder, University of Utah
ClinicalTrials.gov Identifier: NCT01187901     History of Changes
Other Study ID Numbers: 00039278
P01CA073992 ( U.S. NIH Grant/Contract )
First Submitted: August 3, 2010
First Posted: August 24, 2010
Results First Submitted: May 10, 2016
Results First Posted: June 17, 2016
Last Update Posted: June 17, 2016