Trial In Pediatric Patients With Familial Adenomatous Polyposis (FAP)

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

Detailed Description
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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.

Official Title
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A Phase III Placebo-Controlled Trial Of Celecoxib In Genotype Positive Subjects With Familial Adenomatous Polyposis

Conditions
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Adenomatous Polyposis Coli

Intervention / Treatment
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* Drug: Celecoxib
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 10 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Celecoxib<br>celecoxib, 16 mg/kg/day, for 5 years | Drug: Celecoxib<br>* celecoxib, 16 mg/kg/day, for 5 years<br>* Other names: celebrex, SC-58635;|
| Placebo Comparator: Placebo<br>Masked, placebo comparator | Drug: Placebo<br>* Masked, placebo comparator<br>|

What is the study measuring?
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Primary Outcome Measures

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

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to Treatment Failure | 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. | 5 years | 
| Total Number of Colorectal Polyps | 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. | Years 1 - 5 | 
| Colorectal Polyp Burden | 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. | Years 1 - 5 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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FAP