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A Randomized Phase III Trial of Low Dose Sulindac Combined With Eflornithine in Patients With Familial Adenomatous Polyposis (FAP)

Phase 3
18 Years
65 Years
Not Enrolling
Familial Adenomatous Polyposis

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Trial Information

A Randomized Phase III Trial of Low Dose Sulindac Combined With Eflornithine in Patients With Familial Adenomatous Polyposis (FAP)

Inclusion Criteria:

- Diagnosis of phenotypic Familial Adenomatous Polyposis (FAP) of the colorectum based
on meeting the criteria in one of two groups: Group 1-Greater than 100 adenomatous
colorectal polyps prior to age 40. Group 2-Greater than 10 adenomatous polyps and age
<40 or greater than 25 polyps and age >40; combined with a dominant family history or
genotype: More than 100 polyps in a first-degree relative; More than 25 polyps in 2
relatives in 2 generations, including a first-degree family member; Genetic diagnosis
in a relative; Genetic diagnosis by in vitro synthesized truncated protein or similar

- No colorectal surgery or prior colon surgery for polyposis at least 1 year prior
(total abdominal colectomy with ileal-rectal anastomosis, or total proctocolectomy
with ilea pouch-anal reconstruction.

- Baseline endoscopy

1. If no prior colorectal surgery, at least 3 polyps in a cluster each ≥ 2 mm in
diameter; or

2. If rectum is in situ and to be assessed, baseline rectal segment endoscopy
documenting 3 or more rectal polyps each at least 2 mm in diameter in a defined
cluster and/or at least 6 polyps, ≥ 2 mm in diameter, in the distal 10 cm of

3. If ileal pouch neo-rectum is in place, 3 or more pouch polyps in a cluster ≥ 2
mm in diameter, or at least 6 polyps, ≥ 2 mm in diameter, in the distal 10 cm
of pouch.

4. Clinical/pathological grading of duodenal polyps will utilize the Spigelman

- Hematopoietic: no significant hematologic dysfunction; WBC ≥3,000/mm3; platelet count
≥100,000/mm3; hemoglobin ≥10g/dL; no known or prior clinical coagulopathy.

- Hepatic: bilirubin ≤ 1.5 times ULN; AST and ALT ≤ 1.5 times ULN; Alkaline phosphatase
≤ 1.5 times ULN.

- Renal: No significant renal dysfunction; creatinine ≤ 1.5 times ULN.

- Hearing: no clinically significant hearing loss that affects everyday life.

- Not pregnant or nursing.

- Negative serum pregnancy test if female of child-bearing potential.

- Absence of gross blood in stool.

- Fertile patients must use effective contraception.

- Stool occult blood either negative or minimal (1+).

- No prior hypersensitivity to cyclooxygenase-2 inhibitors, sulfonamides, NSAIDs, or
salicylates; no NSAID associated symptoms of gastritis.

- No discrete gastric or duodenal ulcer greater than 5 mm within the past year except
Helicobacter pylori-related peptic ulcer disease treated successfully with
antibiotics (as documented by an endoscopy.

- No invasive malignancy within the past 5 years except stage I or II colon or rectal
cancer or resected nonmelanomatous skin cancer.

- No other significant medical or psychiatric problems that would preclude study

- No chronic adrenocorticosteroids.

- No prior pelvic irradiation.

- At least 3 months since prior investigational agents.

- Patients may not be receiving or plan to receive corticosteroids.

- Concomitant NSAID use outside this study may not exceed 4 days per month.

- Use of 81 mg daily aspirin or 650 mg aspirin not more than once a week.

- No concurrent warfarin, fluconazole, or lithium.

- Must be willing and able to sign informed consent.

Exclusion Criteria:

- High Risk for cardiovascular disease including clinical diabetes mellitus (Type I or
II) requiring glycemic medications; Prior personal history of cardiovascular disease
or, two or more of the following - hypertension or use of anti-hypertensive
medications, hyperlipidemia or use of lipid-lowering medications or current smoker.

- Hearing loss that affects everyday life and or for which a hearing aid is required.

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment

Outcome Measure:

Efficacy of Eflornithine plus Sulindac compared to Eflornithine alone and Sulindac alone determined by change in the number of polyps 2 mm or greater in a defined focal area of the rectum or pouch at baseline and after completion of the study treatment.

Outcome Time Frame:

6 months from the start of treatment.

Safety Issue:


Principal Investigator

Alfred M. Cohen, M.D.

Investigator Role:

Study Director

Investigator Affiliation:

Cancer Prevention Pharmaceuticals, Inc.


United States: Food and Drug Administration

Study ID:




Start Date:

March 2011

Completion Date:

June 2013

Related Keywords:

  • Familial Adenomatous Polyposis
  • colon polyps
  • Adenomatous Polyposis Coli