Familial adenomatous polyposis (FAP) is a rare genetic condition that predisposes patients to develop hundreds to thousands of potentially precancerous polyps (adenomas) in the colon and rectum, usually beginning in adolescence. The standard of care has long been considered to be prophylactic colectomy with ileorectal anastomosis or ileo–pouch anastomosis at diagnosis. The least invasive method for maintaining a minimal polyp burden is through colonoscopic polypectomy. However, polypectomy is effective only for a short period of time in patients with FAP due to the continuous development of new polyps in large numbers. The addition of a drug that can stabilize or regress adenomas and that would maintain the polyp burden within the realm of endoscopic management could potentially delay or even prevent the need for surgery and would be of substantial benefit to patients with FAP. The potential for celecoxib in cancer prevention was first confirmed clinically in patients with FAP in whom celecoxib 400 mg twice a day was effective in reducing the number of intestinal polyps.
The pediatric FAP phase III protocol is designed to compare efficacy of celecoxib versus placebo in polyp prevention over a 5-year treatment period in subjects between the ages of 10 to 17 years inclusive with a confirmed diagnosis of FAP based on genetic predisposition testing.
Subject Eligibility
Study Design
Approximately 200 eligible subjects with FAP from multiple international centers will be randomized in a 1:1 ratio to receive either celecoxib or matching placebo for up to 5 years.
Requirements
Annual colonoscopy and polypectomy at the University of Michigan. Annual physical examination and blood work at the University of Michigan. Monitoring phone calls every 3 months.
Primary Investigator: D. Kim Turgeon, MD
Co-investigator: Elena Stoffel, MD, MPH
Study Coordinator: Victoria M. Raymond, e-mail: vraymond@med.umich.edu phone: (734) 647-0822
Familial adenomatous polyposis (FAP), MYH-associated polyposis, and attenuated familial adenomatous polyposis (AFAP) are rare inherited disorders characterized by the development of multiple polyps in the colon that may eventually lead to cancer. In patients with these conditions, hundreds to thousands of polyps can form in the large intestine.
This study is being conducted to determine whether taking a study drug can reduce the growth and/or progression of polyps in patients with FAP, MYH-associated polyposis, and FAP. Specifically, the study will test whether the combination of sulindac and CPP-1X (eflornithine, DFMO) is safe and more effective at reducing polyp burden than either drug by itself.
Approximately 150 eligible subjects with FAP from multiple international centers will be randomized in a 1:1:1 ratio to receive either CPP-1X plus sulindac, CPP-1X plus a placebo, or sulindac plus a placebo for up to 2 years.
Biannnual colonoscopy and upper endoscopy, physical examination and blood work at the University of Michigan. Monitoring phone calls every 3 months.
Primary Investigator: Elena Stoffel, MD, MPH
Co-investigator: D. Kim Turgeon, MD
Study Coordinator: Erika Koeppe, e-mail: eskoeppe@med.umich.edu phone: (734) 998-1274