Trial In Pediatric Patients With Familial Adenomatous Polyposis (FAP)

Phase 3

Terminated

Brief Summary: To test whether celecoxib can be used to prevent colon polyp formation in children with familial adenomatous polyposis (FAP).

Detailed Description: Per DMC recommendation, the study was terminated early (31Oct2013) due to low enrollment and low endpoint accumulation rate. No safety concerns were involved in the decision to terminate the study.

Recruitment & Eligibility

Inclusion Criteria

Age 10-17 years
Confirmed deleterious FAP genotype based on central genetic testing or personal history ot >2 colorectal adenomas and a parent with the diagnosis of FAP and either A, B or C below A: Non-attenuated FAP genotype B: Attenuated FAP genotype and a personal history of colorectal adenomas and a first degree relative with FAP C: No genotype identified with a personal history of > 2 adenomas and have a parent with FAP
Less than 30 polyps, which need to be removed to render the colon polyp-free before study drug can be given

Exclusion Criteria

Diagnosis of attenuated FAP based on central genetic testing in the absence of a personal history of >2 colorectal adenomas and a first degree relative (parent or sibling) with FAP.
Sensitivity to COX-2 inhibitors

Arm && Interventions

Group	Intervention	Description
Celecoxib	Celecoxib	celecoxib, 16 mg/kg/day, for 5 years
Placebo	Placebo	Masked, placebo comparator

Primary Outcome Measures

Name	Time	Method
Time to Disease Progression	5 years	Time to disease progression was defined as the time from randomization to the earliest occurrence of one or more of the following events: 1. Appearance of ≥20 polyps (\>2 mm in size) at any colonoscopy during the study (Polyps); or 2. Diagnosis of colorectal malignancy (ColMal).

Secondary Outcome Measures

Name	Time	Method
Time to Treatment Failure	5 years	Time to treatment failure was defined as time from randomization to the earliest occurrence of one or more of the following: * Appearance of ≥20 polyps (\>2 mm in size) at any colonoscopy during the study (Polyps), or * Diagnosis of colorectal malignancy (ColMal), or * Treatment related dropout (DO). The treatment related dropout was defined as insufficient clinical response, progression of disease, death, adverse event, treatment-related laboratory abnormality, subject no longer willing to participate in study, and other reasons that might be related to treatment as determined by treating physicians in a blind fashion before database release.
Total Number of Colorectal Polyps	Years 1 - 5	Total number of colorectal polyps \>2 mm in size, that were detected over Years 1 - 5 cumulatively. Weighted total number of colorectal polyps over Years 1 - 5 cumulatively was defined as the total number of colorectal polyps \>2 mm in size, that were detected over Years 1 - 5, divided by the number of colonoscopies that the participant had during the study.
Colorectal Polyp Burden	Years 1 - 5	The polyp burden was defined as the sum of the largest diameters of all polyps (\>2 mm in size) over Years 1 - 5 cumulatively. Weighted colorectal polyp burden over Years 1 - 5 cumulatively was defined as the polyp burden over Years 1 - 5 divided by the number of colonoscopies that the participant had during the study.